رادار التجارب AI | ||
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حالة التجربة السريرية NCT07290244 لـ Open Angle Glaucoma (OAG)، NAION( Non-arteritic Anterior Ischemic Optic Neuropathy) هي يقبل مشاركين. اطلعوا على جميع التفاصيل في عرض البطاقة الخاص برادار التجارب السريرية وأدوات اكتشاف الذكاء الاصطناعي. أو يمكنكم طرح أي سؤال هنا. | ||
دراسة واحدة تطابق معايير الفلتر
عرض البطاقة
Evaluating ER-100 for Safety in People With Glaucoma or Non-Arteritic Anterior Ischemic Optic Neuropathy (Optic Nerve Conditions) المرحلة الأولى ١٨ جرعة واحدة
تفاصيل التجربة السريرية متاحة بشكل أساسي باللغة الإنجليزية. ومع ذلك، يمكن لـ رادار التجارب AI مساعدتك؛ ما عليك سوى النقر على «وصف الدراسة» لعرض ومناقشة معلومات الدراسة باللغة التي اخترتها.
التجربة السريرية NCT07290244 مصممة لدراسة علاج لـOpen Angle Glaucoma (OAG)، NAION( Non-arteritic Anterior Ischemic Optic Neuropathy). إنها دراسة تدخُّلية من المرحلة الأولى وهي يقبل مشاركين. بدأت في ١٣ رمضان ١٤٤٧ هـ مع خطة لتجنيد ١٨ مشاركًا. تقودها Life Biosciences Inc.، ومن المتوقع اكتمالها بحلول ١٩ ذو القعدة ١٤٥٣ هـ. تم تحديث البيانات الأخيرة من ClinicalTrials.gov في ٢٠ رمضان ١٤٤٧ هـ.
الملخص
The goal of this clinical trial is to evaluate the safety and tolerability of a single dose of ER-100 in adults with optic nerve conditions, specifically Open Angle Glaucoma (OAG) and Non-Arteritic Anterior Ischemic Optic Neuropathy (NAION). The main questions it aims to answer are:
- Is ER-100 safe when given as a single dose to people with OAG or NAION
- What side effects may occur, if any, after taking ER-100?
P...
عرض المزيدوصف مفصل
This first-in-human (FIH), Phase 1 clinical trial is designed to evaluate the safety and tolerability of ER-100, an investigational epigenetic therapy candidate intended for age-related optic neuropathies, such as Open Angle Glaucoma (OAG) and Non Arteritic Anterior Ischemic Optic Neuropathy(NAION) Over time, factors such as age, disease, injury, and lifestyle can leave marks on cells that affect how genes function. ...عرض المزيد
العنوان الرسمي
A Phase 1 Single Dose Study to Evaluate the Safety and Tolerability of ER-100 in Optic Neuropathies [Open Angle Glaucoma (OAG) and Non-arteritic Anterior Ischemic Optic Neuropathy (NAION)]
الحالات الطبية
Open Angle Glaucoma (OAG)NAION( Non-arteritic Anterior Ischemic Optic Neuropathy)معرّفات دراسة أخرى
- LB100-001
NCT معرّف
تاريخ البدء (فعلي)
2026-03-02
آخر تحديث مُنشور
2026-03-09
تاريخ الاكتمال (المقدر)
2032-03
عدد المشاركين المخطط لهم
١٨
نوع الدراسة
تدخُّلية
المرحلة
المرحلة الأولى
الحالة
يقبل مشاركين
الكلمات الرئيسية
Optic Neuropathy
Retinal Diseases
Aging/Cellular Aging
Open-Angle Glaucoma
Non-Arteritic Anterior Ischemic Optic Neuropathy
Partial Epigenetic Reprogramming
Epigenetic Therapy
Retinal Diseases
Aging/Cellular Aging
Open-Angle Glaucoma
Non-Arteritic Anterior Ischemic Optic Neuropathy
Partial Epigenetic Reprogramming
Epigenetic Therapy
الغرض الأساسي
العلاج
طريقة توزيع المشاركين
غير عشوائي
نموذج التدخل
تصميم تسلسلي
التعمية
لا شيء (تجربة مفتوحة)
مجموعات/التدخلات
| مجموعة المشاركين/الذراع | التدخل/العلاج |
|---|---|
تجريبيةOAG - Low Dose ER-100 (2 x 10^11 vg/eye) Participants with Open Angle Glaucoma will receive a low dose of ER-100 administered to one eye. ER-100 is delivered via a modified adeno-associated virus (AAV) vector and activated by systemic doxycycline taken for 8 weeks (56 days). This dose level begins with a sentinel participant followed by additional participants after DSMB review. | ER-100 epigenetic therapy ER-100 is an investigational AAV-based epigenetic therapy administered via intravitreal injection to one eye. It uses a modified adeno-associated virus (AAV) vector to deliver instructions for producing three transcription factors-OCT4, SOX2, and KLF4 (collectively referred to as OSK)-intended to reverse age-related epigenetic changes in retinal cells. Systemic doxycycline is administered for 8 weeks (56 days) to act...عرض المزيد |
تجريبيةOAG - High Dose ER-100 (6 x 10^11 vg/eye) Participants with Open Angle Glaucoma will receive a higher dose of ER-100 administered to one eye. ER-100 is delivered via a modified AAV vector and activated by systemic doxycycline for 8 weeks. This dose level also begins with a sentinel participant and proceeds following DSMB review. | ER-100 epigenetic therapy ER-100 is an investigational AAV-based epigenetic therapy administered via intravitreal injection to one eye. It uses a modified adeno-associated virus (AAV) vector to deliver instructions for producing three transcription factors-OCT4, SOX2, and KLF4 (collectively referred to as OSK)-intended to reverse age-related epigenetic changes in retinal cells. Systemic doxycycline is administered for 8 weeks (56 days) to act...عرض المزيد |
تجريبيةNAION - Selected Dose ER-100 Participants with Non-Arteritic Anterior Ischemic Optic Neuropathy will receive ER-100 at a dose selected based on safety and tolerability data from the OAG cohort. ER-100 is administered to one eye and activated by systemic doxycycline for 8 weeks. Initial enrollment is limited to three participants, with potential expansion to six following DSMB review. | ER-100 epigenetic therapy ER-100 is an investigational AAV-based epigenetic therapy administered via intravitreal injection to one eye. It uses a modified adeno-associated virus (AAV) vector to deliver instructions for producing three transcription factors-OCT4, SOX2, and KLF4 (collectively referred to as OSK)-intended to reverse age-related epigenetic changes in retinal cells. Systemic doxycycline is administered for 8 weeks (56 days) to act...عرض المزيد |
النتيجة الرئيسية
النتيجة الثانوية
| مقياس النتيجة | وصف القياس | الإطار الزمني |
|---|---|---|
Incidence of Treatment-Emergent Adverse Events (TEAEs) During and Post-Doxycycline Activation Period | This outcome tracks any new or worsening health problems that occur while participants are receiving and completed oral doxycycline.
These health problems are called treatment-emergent adverse events (TEAEs). Researchers record how often these events happen as well as their nature and seriousness.
This helps determine whether ER-100 causes any short-term side effects during this phase. | Baseline to Day 56, Day 112 |
Incidence of Dose-Limiting Toxicities During and Post-Doxycycline Activation Period | This outcome measures how often participants experience dose-limiting toxicities, which are side effects serious enough to prevent increasing the dose, while receiving and completed oral doxycycline.
These toxicities are defined by the study protocol and may differ for participants with open-angle glaucoma (OAG) and non-arteritic anterior ischemic optic neuropathy (NAION).
This helps identify the highest dose that can be given safely during treatment. | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Liver Function Tests - LFTs) During and Post-Doxycycline Activation Period | Liver function tests (LFTs) measure specific enzymes and proteins in a blood sample to check how well the liver is working. These include alanine aminotransferase (ALT), aspartate aminotransferase (AST), alkaline phosphatase (ALP), gamma-glutamyl transferase (GGT), and lactate dehydrogenase (LDH). When liver cells are injured, these enzymes leak into the bloodstream, and higher levels on a blood test can signal a problem. Compared to baseline, higher levels can mean the liver is under stress or injured. Unit of Measure: Units per liter (U/L) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Blood Protein Levels) During and Post-Doxycycline Activation Period | Blood proteins such as albumin and total protein help show nutritional status and how well the liver is making proteins. Compared to baseline, lower levels can mean poor nutrition or liver problems. Unit of Measure: Grams per deciliter (g/dL) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Calcium, Glucose, Bilirubin, Creatinine, Blood Urea Nitrogen) During and Post-Doxycycline Activation Period | These tests show how the body manages minerals, sugar, and waste. Calcium helps with bone and muscle function. Glucose shows blood sugar control. Bilirubin shows liver health. Creatinine and blood urea nitrogen (BUN) show kidney function. Compared to baseline, higher or lower levels can mean problems with the liver, kidneys, or metabolism. Unit of Measure: Milligrams per deciliter (mg/dL) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Electrolytes: Sodium, Potassium, Chloride) During and Post-Doxycycline Activation Period | Electrolytes help keep the body's fluids and nerves working properly. Changes can mean dehydration or kidney problems. This outcome looks at how much sodium (Na), potassium (K), and chloride (Cl) levels change from baseline. Unit of Measure: Millimoles per liter (mmol/L) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Electrolytes: Bicarbonate and Magnesium) During and Post-Doxycycline Activation Period | Bicarbonate helps control the body's acid balance. Magnesium is important for muscles and nerves. Changes can mean metabolic or kidney problems. This outcome looks at how much these levels change from baseline. Unit of Measure: Milliequivalents per liter (mEq/L) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Hemoglobin) During and Post-Doxycycline Activation Period | Hemoglobin is a protein in red blood cells that carries oxygen. Lower levels can mean anemia, which is when the blood cannot carry enough oxygen. This outcome looks at how much hemoglobin changes from baseline. Unit of Measure: Grams per deciliter (g/dL) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Hematocrit) During and Post-Doxycycline Activation Period | Hematocrit shows the percentage of blood made up of red blood cells. Compared to baseline, lower levels can mean anemia, which is when the blood cannot carry enough oxygen. Unit of Measure: Percent (%) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (White Blood Cell and Platelet Counts) During and Post-Doxycycline Activation Period | White blood cells help fight infection. Platelets help blood clot. Compared to baseline, changes can mean infection, immune problems, or bleeding risk. Unit of Measure: Billion cells per liter (x10\^9/L) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Red Blood Cell Count) During and Post-Doxycycline Activation Period | Red blood cells carry oxygen throughout the body. Compared to baseline, lower levels can mean anemia, which is when the blood cannot carry enough oxygen. Unit of Measure: Trillion cells per liter (x10\^12/L) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Erythrocyte Sedimentation Rate) During and Post-Doxycycline Activation Period | This test shows inflammation in the body. Compared to baseline, higher values can mean infection or other inflammatory conditions. Unit of Measure: Millimeters per hour (mm/hr) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (C-Reactive Protein) During and Post-Doxycycline Activation Period | C-reactive protein shows inflammation in the body. Compared to baseline, higher values can mean infection or other inflammatory conditions. Unit of Measure: Milligrams per liter (mg/L) | Baseline to Day 56, Day 112 |
Change in Safety Laboratory Tests (Urine Test Results) During and Post-Doxycycline Activation Period | Urine tests check for blood, protein, sugar, and other substances. Compared to baseline, changes can mean kidney or urinary tract problems. Unit of Measure: Presence or absence | Baseline to Day 56, Day 112 |
Change from Baseline in the Intraocular Pressure (IOP) in the Treated Eye During and Post-Doxycycline Activation Period - Safety | This outcome measures changes in pressure inside the treated eye while participants are receiving and completed oral doxycycline.
Eye pressure is measured in millimeters of mercury (mmHg).
On Day 1, pressure is measured using an instrument named Goldmann Applanation Tonometry (GAT), which is the gold standard. This instrument will gently touch the eye with a probe after applying numbing drops.
After Day 1, pressure is measured using another, non-contact method, which uses a puff of air.
If pressure increases significantly, GAT may be repeated to confirm.
Depending on their values, higher or lower pressures compared to the participant's baseline may indicate safety concerns related to the treatment. | Baseline to Day 56, Day 112 |
Change from Baseline in the Best Corrected Visual Acuity (BCVA) Letter Score in the Treated Eye During and Post-Doxycycline Activation Period - Safety | This outcome measures the change in visual clarity (corrected with glasses or contact lenses for refractive errors like nearsightedness, farsightedness, or astigmatism) in the treated eye while participants are receiving and completed oral doxycycline.
BCVA is assessed using a special chart called the Early Treatment Diabetic Retinopathy Study (ETDRS) chart.
The number of letters read correctly is recorded as a letter score, ranging from 0 to 100. A drop in score from the baseline may indicate a safety issue affecting vision, while stable or improved scores suggest no adverse impact.
Trial frames with the lens prescription determined at screening will be used for these assessments. | Baseline to Day 56, Day 112 |
Change from Baseline in Humphrey Visual Field (HVF) Test Results During and Post-Doxycycline Activation Period - Safety | This outcome measures the change in central and peripheral (side) field of vision while participants are receiving and completed oral doxycycline.
The field of vision is assessed using the Humphrey Visual Field (HVF) test, which checks how well a person can see lights that flash in different areas of their visual field while they focus on a central point.
Results are reported as a Mean Deviation (MD) score in decibels (dB). For participants with open-angle glaucoma (OAG), the test uses the SITA Standard 24-2 protocol. For participants with non-arteritic anterior ischemic optic neuropathy (NAION), a larger light stimulus (size V) is used.
Compared to baseline, a more negative MD score means more vision loss, and a significant worsening in MD may suggest a safety concern. | Baseline to Day 56, Day 112 |
Change from Baseline in Pattern Electroretinogram (pERG) During and Post-Doxycycline Activation Period - Safety | This outcome measures the change in retinal function while participants are receiving and completed oral doxycycline.
The pERG is a test that checks how well the retina responds to visual patterns like black and white stripes. It helps evaluate the health of retinal ganglion cells, which are important for sending visual signals to the brain.
The test provides two types of results:
* Amplitude (measured in microvolts, µV): This shows the strength of the retina's response.
* Peak time (measured in milliseconds, ms): This shows how quickly the retina responds.
Lower amplitudes or slower response times than those recorded at baseline may indicate worsening retinal function. A significant decline in these results may suggest a safety concern related to disease progression or if there are signs that the treatment is causing effects. | Baseline to Day 56, Day 112 |
Change from Baseline in Quantitative Contrast Sensitivity Function (qCSF) During and Post-Doxycycline Activation Period - Safety | This outcome measures the change in contrast sensitivity while participants are receiving and completed oral doxycycline.
The quantitative Contrast Sensitivity Function (qCSF), also called quick CSF, is a computerized test that evaluates how well a person can detect differences in contrast, for example, the ability to discern subtle differences in shading or light and dark between an object and its background.
Lower scores than those recorded at baseline may indicate worsening visual function, and a significant decline in one or more parameters may suggest a safety concern. Changes in these scores are monitored to detect potential trea | Baseline to Day 56, Day 112 |
Change from Baseline in Optical Coherence Tomography (OCT): Ganglion Cell Layer (GCL) Thickness During and Post-Doxycycline Activation Period - Safety | This outcome measures the change in the thickness of the ganglion cell layer (GCL) while participants are receiving and completed oral doxycycline.
Optical Coherence Tomography (OCT) is a non-invasive imaging test that uses light waves to take detailed cross-sectional pictures of the retina. OCT will be conducted using an instrument called the Heidelberg Spectralis system.
The GCL contains the cell bodies of retinal ganglion cells, which are critical for transmitting visual information from the retina to the brain. GCL thickness is measured in micrometers (µm).
A significant decrease in GCL thickness from baseline may suggest a safety concern related to disease progression or if there are signs that the treatment is causing effects. | Baseline to Day 56, Day 112 |
Change from Baseline in Optical Coherence Tomography (OCT): Retinal Nerve Fiber Layer (RNFL) Thickness During and Post-Doxycycline Activation Period - Safety | This outcome measures the change in the thickness of the retinal nerve fiber layer (RNFL) while participants are receiving and completed oral doxycycline.
Optical Coherence Tomography (OCT) is a non-invasive imaging test that uses light waves to take detailed cross-sectional pictures of the retina. OCT will be conducted using the Heidelberg Spectralis system.
The RNFL contains nerve fibers that help carry visual signals from the eye to the brain. RNFL thickness is measured in micrometers (µm).
A significant decrease in RNFL thickness from baseline may suggest a safety concern related to disease progression or if there are signs that the treatment is causing effects. | Baseline to Day 56, Day 112 |
Change from Baseline in Slit Lamp Exam Results During and Post-Doxycycline Activation Period - Safety | This outcome measures changes in the health of the front structures of the eye while participants are receiving and completed oral doxycycline.
A slit lamp exam is a routine eye test that uses a bright light and microscope to closely examine the front parts of the eye, including the cornea, iris, lens, and the white part of the eye. The exam helps detect signs of irritation, inflammation, infection, or other possible damage.
Changes from baseline observed during the slit lamp exam might be signs that the treatment is causing effects or that the disease is getting worse. | Baseline to Day 56, Day 112 |
| مقياس النتيجة | وصف القياس | الإطار الزمني |
|---|---|---|
Change from Baseline in the Intraocular Pressure (IOP) in the Treated Eye During and Post-Doxycycline Activation Period - Efficacy | This outcome measures the change in pressure inside the treated eye while participants are receiving and completed oral doxycycline.
Eye pressure is measured in millimeters of mercury (mmHg).
On Day 1, pressure is measured using an instrument named Goldmann Applanation Tonometry (GAT), which is the gold standard. This instrument will gently touch the eye with a probe after applying numbing drops.
After Day 1, pressure is measured using another, non-contact method, which uses a puff of air.
If pressure increases significantly, GAT may be repeated to confirm.
Lower pressures compared to baseline may indicate disease improvement. | Baseline to Day 56, Day 112 |
Change from Baseline in the Intraocular Pressure (IOP) in the Treated Eye - Long-term Follow-up Period - Safety | This outcome measures changes in pressure inside the treated eye after participants stop taking oral doxycycline during the long-term follow-up period.
Eye pressure is measured in millimeters of mercury (mmHg).
On Day 1, pressure is measured using Goldmann Applanation Tonometry (GAT), which gently touches the eye with a probe after applying numbing drops.
After Day 1, pressure is measured using non-contact tonometry, which uses a puff of air.
If pressure increases significantly, GAT may be repeated to confirm.
Depending on their values, higher or significantly lower pressures compared to the participant's baseline may indicate progression or need for further safety monitoring. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Change from Baseline in the Best Corrected Visual Acuity (BCVA) Letter Score in the Treated Eye During and Post-Doxycycline Activation Period - Efficacy | This outcome measures the change in visual clarity (corrected with glasses or contact lenses for refractive errors like nearsightedness, farsightedness, or astigmatism) in the treated eye while participants are receiving and completed oral doxycycline.
This outcome measures whether visual clarity improves in the treated eye while participants are receiving oral doxycycline.
BCVA is assessed using a special chart called the Early Treatment Diabetic Retinopathy Study (ETDRS) chart.
The number of letters read correctly is recorded as a letter score, ranging from 0 to 100. An increase in score from the baseline may suggest the treatment is helping improve how well someone sees.
Trial frames with the lens prescription determined at screening will be used for these assessments. | Baseline to Day 56, Day 112 |
Change from Baseline in the Best Corrected Visual Acuity (BCVA) Letter Score in the Treated Eye - Long-term Follow-up Period - Safety | This outcome measures the change in visual clarity (corrected with glasses or contact lenses for refractive errors like nearsightedness, farsightedness, or astigmatism) in the treated eye after participants stop taking oral doxycycline during the long-term follow-up period.
BCVA is assessed using a special chart called Early Treatment Diabetic Retinopathy Study (ETDRS) chart.
The number of letters read correctly is recorded as a letter score, ranging from 0 to 100. A drop in score from baseline may indicate a safety issue affecting vision, while stable or improved scores suggest no adverse impact.
Trial frames with the lens prescription determined at screening will be used for these assessments. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Change from Baseline in Humphrey Visual Field (HVF) Test Results During and Post-Doxycycline Activation Period - Efficacy | This outcome measures the change in central and peripheral (side) field of vision while participants are receiving and completed oral doxycycline.
The field of vision is assessed using the Humphrey Visual Field (HVF) test, which checks how well a person can see lights that flash in different areas of their visual field while they focus on a central point.
Results are reported as a Mean Deviation (MD) score in decibels (dB). For participants with open-angle glaucoma (OAG), the test uses the SITA Standard 24-2 protocol. For participants with non-arteritic anterior ischemic optic neuropathy (NAION), a larger light stimulus (size V) is used.
Compared to baseline, a more negative MD score means more vision loss, while scores closer to zero suggest better or more normal vision. Improvements in MD from baseline may suggest a positive effect of the treatment. | Baseline to Day 56, Day 112 |
Change from Baseline in Humphrey Visual Field (HVF) Test Results - Long-term Follow-up Period - Safety | This outcome measures the change in peripheral (side) field of vision after participants stop taking oral doxycycline during the long-term follow-up period.
Peripheral field of vision vision is assessed using the Humphrey Visual Field (HVF) test, which checks how well a person can see lights that flash in different areas of their visual field while they focus on a central point.
Results are reported as a Mean Deviation (MD) score in decibels (dB). For participants with open-angle glaucoma (OAG), the test uses the SITA Standard 24-2 protocol.For participants with non-arteritic anterior ischemic optic neuropathy (NAION), a larger light stimulus (size V) is used.
A more negative MD score comparing to baseline means more vision loss, and a significant worsening in MD may suggest a safety concern. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Change from Baseline in Quantitative Contrast Sensitivity Function (qCSF) During and Post-Doxycycline Activation Period - Efficacy | This outcome measures the change in contrast sensitivity while participants are receiving and completed oral doxycycline.
The quantitative Contrast Sensitivity Function (qCSF), also called quick CSF, is a computerized test that evaluates how well a person can detect differences in contrast - for example, the ability to discern subtle differences in shading or light and dark between an object and its background.
Higher scores suggest better visual function, while lower scores may indicate difficulty seeing in low-contrast or low-light conditions. Changes in these scores from baseline may reflect signs that the treatment is causing effects. | Baseline to Day 56, Day 112 |
Change from Baseline in Quantitative Contrast Sensitivity Function (qCSF) - Long-term Follow-up Period - Safety | This outcome measures the change in contrast sensitivity after participants stop taking oral doxycycline during the long-term follow-up period.
The quantitative Contrast Sensitivity Function (qCSF), also called quick CSF, is a computerized test that evaluates how well a person can detect differences in contrast - for example, for example, the ability to discern subtle differences in shading or light and dark between an object and its background.
Lower scores than the baseline values may indicate worsening visual function, and a significant decline in one or more parameters may suggest a safety concern. Changes in these scores from baseline are monitored to see if there are signs that the treatment is causing effects or that the disease is getting worse. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Change from Baseline in Pattern Electroretinogram (pERG) During and Post-Doxycycline Activation Period - Efficacy | This outcome measures the change in retinal function while participants are receiving and completed oral doxycycline.
The pERG is a test that checks how well the retina responds to visual patterns like black and white stripes. It helps evaluate the health of retinal ganglion cells, which are important for sending visual signals to the brain.
The test provides two types of results:
* Amplitude (measured in microvolts, µV): This shows the strength of the retina's response.
* Peak time (measured in milliseconds, ms): This shows how quickly the retina responds.
Higher numbers mean the retina is responding well. Lower numbers or slower response times may suggest that the retina is not working as well as it should. Changes in these results from baseline values may show whether the condition is improving or getting worse during treatment. | Baseline to Day 56, Day 112 |
Change from Baseline in Pattern Electroretinogram (pERG) - Long-term Follow-up Period - Safety | This outcome measures the change in retinal function after participants stop taking oral doxycycline during the long-term follow-up period.
The pERG is a test that checks how well the retina responds to visual patterns like black and white stripes. It helps evaluate the health of retinal ganglion cells, which are important for sending visual signals to the brain.
The test provides two types of results:
* Amplitude (measured in microvolts, µV): This shows the strength of the retina's response.
* Peak time (measured in milliseconds, ms): This shows how quickly the retina responds.
Lower amplitudes or slower response times than baseline values may indicate worsening retinal function. A significant decline in these results may suggest a safety concern related to signs that the treatment is causing effects or that the disease is getting worse. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Change from Baseline in Optical Coherence Tomography (OCT): Ganglion Cell Layer (GCL) Thickness During and Post-Doxycycline Activation Period - Efficacy | This outcome measures the change in the thickness of the ganglion cell layer (GCL) while participants are receiving and completed oral doxycycline.
Optical Coherence Tomography (OCT) is a non-invasive imaging test that uses light waves to take detailed cross-sectional pictures of the retina. OCT will be conducted using the Heidelberg Spectralis system.
The GCL contains the cell bodies of retinal ganglion cells, which are critical for transmitting visual information from the retina to the brain. GCL thickness is measured in micrometers (µm).
Changes in GCL thickness from baseline values may reflect there are signs that the treatment is causing effects or that the disease is getting worse. | Baseline to Day 56, Day 112 |
Change from Baseline in Optical Coherence Tomography (OCT): Ganglion Cell Layer (GCL) Thickness - Long-term Follow-up Period - Safety | This outcome measures the change in the thickness of the ganglion cell layer (GCL) after participants stop taking oral doxycycline during the long-term follow-up period.
Optical Coherence Tomography (OCT) is a non-invasive imaging test that uses light waves to take detailed cross-sectional pictures of the retina. OCT will be conducted using the Heidelberg Spectralis system.
The GCL contains the cell bodies of retinal ganglion cells, which are critical for transmitting visual information from the retina to the brain. GCL thickness is measured in micrometers (µm).
A significant decrease in GCL thickness from baseline values may suggest a safety concern related to signs that the treatment is causing effects or that the disease is getting worse. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Change from Baseline in Optical Coherence Tomography (OCT): Retinal Nerve Fiber Layer (RNFL) Thickness During and Post-Doxycycline Activation Period - Efficacy | This outcome measures the change in the thickness of the retinal nerve fiber layer (RNFL) while participants are receiving and completed oral doxycycline.
Optical Coherence Tomography (OCT) is a non-invasive imaging test that uses light waves to take detailed cross-sectional pictures of the retina. OCT will be conducted using the Heidelberg Spectralis system.
The RNFL contains nerve fibers that help carry visual signals from the eye to the brain. RNFL thickness is measured in micrometers (µm).
Changes in RNFL thickness from baseline values may reflect signs that the treatment is causing effects or that the disease is getting worse. | Baseline to Day 56, Day 112 |
Change from Baseline in Optical Coherence Tomography (OCT): Retinal Nerve Fiber Layer (RNFL) Thickness - Long-term Follow-up Period - Safety | This outcome measures the change in the thickness of the retinal nerve fiber layer (RNFL) after participants stop taking oral doxycycline during the long-term follow-up period.
Optical Coherence Tomography (OCT) is a non-invasive imaging test that uses light waves to take detailed cross-sectional pictures of the retina. OCT will be conducted using the Heidelberg Spectralis system.
The RNFL contains nerve fibers that help carry visual signals from the eye to the brain. RNFL thickness is measured in micrometers (µm).
A significant decrease in RNFL thickness from baseline values may suggest a safety concern related to signs that the treatment is causing effects or that the disease is getting worse. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Change in Neutralizing Antibodies (NAbs) to AAV2 During and Post-Doxycycline Activation Period | This outcome measures changes in the body's immune response to the AAV2 virus while participants are receiving and completed oral doxycycline.
Specifically, it looks at neutralizing antibodies, which are proteins made by the immune system that can block the virus from working.
Blood samples are tested to see how much antibody is present. The results are reported as a number called a "titer," which shows how strong the response is.
* A higher titer means the body is making more antibodies that can block the virus.
* A lower titer or no detectable antibodies means the virus is less likely to be blocked.
These results help researchers understand how the body reacts to the treatment and whether the immune system might affect how well the therapy works. | Baseline to Day 56, Day 112 |
Change in Cellular Immune Response to ER-100 Peptides via ELISpot During and Post-Doxycycline Activation Period | This outcome measures how the immune system's T cells respond to parts of the ER-100 treatment while participants are receiving and completed oral doxycycline.
A lab test called ELISpot is used to count how many immune cells release a signal when they recognize parts of the treatment.
Each signal shows up as a spot in the test - more spots mean a stronger immune response, and fewer spots mean a weaker response.
This helps researchers understand how active the immune system is and whether it might affect how well the treatment works or how the body tolerates it. | Baseline to Day 56, Day 112 |
Change in ER-100 Viral Shedding via qPCR to During and Post-Doxycycline Activation Period | This outcome measures whether any part of the ER-100 treatment is released from the body while participants are receiving and completed oral doxycycline.
Samples are collected from tears, nasal swab, saliva, blood, urine, and feces. A lab test called qPCR is used to detect tiny amounts of ER-100 DNA in these fluids.
If ER-100 DNA is found, it means the treatment is leaving the body through these fluids - this is called viral shedding.
If no ER-100 DNA is found, it means the treatment is staying inside the body. More shedding may suggest the body is clearing the treatment faster, while less or no shedding may mean the treatment is staying localized or being retained longer.
This helps researchers understand how the treatment moves through the body and whether it could be passed to others. | Baseline to Day 56, Day 112 |
Change in Biodistribution of ER-100 Vector DNA in Aqueous Humor During Doxycycline Activation Period | This outcome measures whether any part of the ER-100 treatment is found in the fluid inside the eye (called aqueous humor) while participants are receiving oral doxycycline.
This test is only done in participants with open-angle glaucoma (OAG).
A lab test called qPCR is used to detect tiny amounts of ER-100 DNA in the eye fluid.
* If ER-100 DNA is found, it means the treatment has reached the inside of the eye.
* If no ER-100 DNA is found, it means the treatment has not entered that part of the eye.
This helps researchers understand where the treatment travels in the body and whether it stays in the intended area. | Baseline to Day 56 |
Change from Baseline in Slit Lamp Exam Results - Long-term Follow-up Period - Safety | This outcome measures changes in the health of the front structures of the eye after participants stop taking oral doxycycline during the long-term follow-up period.
A slit lamp exam is a routine eye test that uses a bright light and microscope to closely examine the front parts of the eye, including the cornea, iris, lens, and the white part of the eye. The exam helps detect signs of irritation, inflammation, infection, or other damage.
Changes from baseline observed during the slit lamp exam might be signs that the treatment is causing effects or that the disease is getting worse. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Incidence of Treatment-Emergent Adverse Events (TEAEs) - Long-term Follow-up Period | This outcome tracks any new or worsening health problems that occur after participants stop taking oral doxycycline during the long-term follow-up period.
These health problems are called treatment-emergent adverse events (TEAEs). Researchers record how often these events happen, what kind they are (such as headaches, infections, or changes in lab tests), and how serious they are.
This helps determine whether the ER-100 treatment causes any long-term side effects or health concerns, even months or years after the initial treatment. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
Change in Safety Laboratory Test Results - Long-term Follow-up Period | This outcome measures changes in routine lab test results after participants stop taking oral doxycycline, during the long-term follow-up period.
These tests check things like liver and kidney function, blood counts, and other indicators of general health.
If lab values change over time, it may show how the body is responding to the ER-100 treatment in the long term.
This helps researchers monitor for any delayed side effects or health concerns. | Baseline to Month 6, Year 1, Year 2, Year 3, Year 4, Year 5 |
مساعد المشاركة
معايير الأهلية
الأعمار المؤهلة للدراسة
بالغ, كبار السن
العمر الأدنى للدراسة
40 Years
الجنس المؤهل
الكل
- Have clear eye structures and be able to have your pupils safely dilated so the doctor can examine the back of your eye.
- Able to understand the study and sign a consent form.
- Be between 40 and 85 years old.
- Willing and able to follow the study schedule, including all visits and tests, and speak a language for which the study materials are available.
- If a participant can become pregnant, must agree to use a condom and one highly effective form of birth control during sex for at least 4 months after receiving the study drug (ER-100).
For participants with open-angle glaucoma (OAG):
- Diagnosis of open-angle glaucoma in the study eye.
- Eye pressure must be less than 30 mmHg, measured with a standard test.
- Visual field test must show moderate to advanced vision loss (MD score between -6 and -20 dB).
- Not expected to need glaucoma surgery in the study eye within 2 months after receiving ER-100.
- Have reasonably good vision in the study eye (at least 20/80 on a standard eye chart).
For participants with NAION (non-arteritic anterior ischemic optic neuropathy):
- Had a sudden, painless loss of vision in one eye within 14 days before receiving ER-100, confirmed by a specialist. Having had NAION in the other eye is okay.
- The affected eye must show swelling of the optic nerve.
- Visual field test must show vision loss consistent with optic nerve damage (MD worse than -3.0 dB).
- If only one eye is affected, there must be a difference in pupil response between the two eyes.
- Have vision in the affected eye between 20/40 and 2/500 on a standard eye chart.
- History of optic neuritis (inflammation of the optic nerve) or repeated episodes of eye inflammation (uveitis) not caused by injury or surgery.
- Allergic reactions to tetracycline antibiotics or steroid medications.
- Moderate to severe cataracts, macular problems, or corneal issues that could interfere with eye testing.
- Unable to keep your eyes focused on a target during testing.
- Had cataract surgery or other eye surgery (including laser procedures) within 3 months before receiving the study drug.
- Had cancer (except for basal cell skin cancer) within the past 5 years.
- Have Type 1 diabetes, or poorly controlled Type 2 diabetes (A1c greater than 7 despite treatment).
- Have memory or thinking problems that prevent you from understanding the study or completing the required tests.
- Pregnant or breastfeeding.
- Have a weakened immune system, including a history of organ transplant, or test positive for HIV, hepatitis B or C, or tuberculosis.
- Have any other condition that, in the opinion of the study doctor, could increase your risk from the study drug or procedures, affect the study results, or make it hard for you to complete the study.
- Have macular disease, advanced diabetic eye disease, or other eye conditions that limit vision in the study eye.
- Eye pressure at screening is 30 mmHg or higher.
- Taking certain medications (warfarin, dilantin, carbamazepine, or barbiturates) within 14 days before starting the study or during the first 8 weeks.
- Have any other eye or vision problem that, in the opinion of the study doctor, could affect safety or interfere with vision testing.
- Have previously received any gene therapy using adeno-associated virus (AAV).
Additional Exclusion Criteria for Participants with Open-Angle Glaucoma (OAG):
- Diagnosed with glaucoma before age 40.
Additional Exclusion Criteria for Participants with NAION:
- Show signs of giant cell arteritis (a type of blood vessel inflammation), based on abnormal blood tests.
- Had NAION start in both eyes at the same time.
Life Biosciences Inc.جهة اتصال مركزية للدراسة
جهة اتصال: Life Biosciences, (857) 400-9245, [email protected]
2 مواقع الدراسة في 1 بلدان
California
Global Research Management, Inc., Glendale, California, 91204, United States
Logi El-Harazi, جهة اتصال, 818-246-2560, [email protected]
Sevada Minassians, جهة اتصال, 818-246-2560, [email protected]
يقبل مشاركين
South Carolina
Charleston Neuroscience Institute, Charleston, South Carolina, 29414, United States
Alise Chatelain, جهة اتصال, 843-972-3857, [email protected]
Terry Blewer, جهة اتصال, 843-936-1882, [email protected]
يقبل مشاركين