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메이라지티엑스Long-Term Follow-Up Gene Therapy Study for Achromatopsia CNGB3 and CNGA3 34 유전자 치료 장기 추적
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임상시험 NCT03278873은(는) 색맹에 대해 알아보는 관찰연구입니다. 현재 상태는 조기 종료이며, 연구는 2017년 6월 29일에 시작되어 34명의 참여자를 모집하고 있습니다. 메이라지티엑스이(가) 진행하며, 2024년 4월 4일까지 완료될 예정입니다. ClinicalTrials.gov의 가장 최근 정보는 2025년 6월 11일에 갱신되었습니다.
간단한 개요
This is a longer-term follow-up study of patients with achromatopsia associated with defects in CNGA3 who participated in a clinical trial in which they received AAV-CNGA3 retinal gene therapy, or of patients with achromatopsia associated with defects in CNGB3 who participated in a clinical trial in which they received AAV-CNGB3 retinal gene therapy.
상세한 설명
The follow-up study is designed to collect data on the longer-term safety and efficacy of AAV-CNGA3 retinal gene therapy and AAV-CNGB3 retinal gene therapy.
공식 제목
Long-term Follow-up Study of Participants Following an Open Label, Multi-centre, Phase I/II Dose Escalation Trial of a Recombinant Adeno-associated Virus Vector (AAV2/8-hCARp.hCNGB3 and AAV2/8-hG1.7p.coCNGA3) for Gene Therapy of Adults and Children With Achromatopsia Owing to Defects in CNGB3 or CNGA3
질환명
색맹기타 연구 식별자
- MGT007
- 2016-003856-59 (EudraCT 번호)
NCT 번호
실제 연구 시작일
2017-06-29
최신 업데이트 게시
2025-06-11
예상 연구 완료일
2024-04-04
계획된 등록 인원
34
연구종류
관찰연구
상태
조기 종료
키워드
Achromatopsia
CNGA3
CNGB3
CNGA3
CNGB3
시험군 / 개입
| 참가자 그룹/시험군 | 개입/치료 |
|---|---|
Low dose of AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single low dose of AAV-CNGA3 or AAV-CNGB3 | Prior exposure to AAV-CNGA3 or AAV-CNGB3 Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively. |
Intermediate dose of AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single intermediate dose of AAV-CNGA3 or AAV-CNGB3 | Prior exposure to AAV-CNGA3 or AAV-CNGB3 Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively. |
Other dose of AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single other dose (between the intermediate and high dose) of AAV-CNGA3 or AAV-CNGB3 | Prior exposure to AAV-CNGA3 or AAV-CNGB3 Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively. |
High dose of AAV-CNGA3 or AAV-CNGB3 Subretinal administration of a single high dose of AAV-CNGA3 or AAV-CNGB3 | Prior exposure to AAV-CNGA3 or AAV-CNGB3 Participants previously received AAV-CNGA3 or AAV-CNGB3 in an open-label, Phase 1/2 dose escalation trial for adults and children with achromatopsia owing to defects in CNGA3 or CNGB3, respectively. |
주요결과변수
이차결과변수
| 결과변수 | 측정값 설명 | 시간 범위 |
|---|---|---|
Incidence of Adverse Events Related to the Treatment | The primary outcome measure is the longer-term safety of treatment with AAV-CNGA3 or AAV-CNGB3, assessed by the absence of IMP-related adverse events. | 5 Years |
| 결과변수 | 측정값 설명 | 시간 범위 |
|---|---|---|
Improvements in Visual Function as Assessed by Visual Acuity at Month 12 | Change from baseline to Month 12 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in the treated eye. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. | 12 months |
Improvements in Visual Function as Assessed by Visual Acuity at Month 60 | Change from baseline to Month 60 in best corrected visual acuity (BCVA) using Early Treatment Diabetic Retinopathy Study (ETDRS) chart letter score in the treated eye. The direction of improvement from baseline is an increase in the number of ETDRS letters read over time. | 60 months |
Improvements in Retinal Function as Assessed by Static Perimetry at Month 12 | Change from baseline to Month 12 in contrast sensitivity in the treated eye. The direction of improvement is an increase in sensitivity. | 12 months |
Improvements in Retinal Function as Assessed by Static Perimetry at Month 60 | Change from baseline to Month 60 in contrast sensitivity in the treated eye. The direction of improvement is an increase in sensitivity. | 60 months |
Quality of Life at Month 12 Measured by QoL Questionnaires in Children and Adolescents | Change from baseline to Month 12 in EuroQol-5D-Y Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement, and a negative change from baseline reflects worsening. | 12 months |
Quality of Life at Month 60 Measured by QoL Questionnaires in Children and Adolescents | Change from baseline to Month 60 in EuroQol-5D-Y Visual Analogue Scale (EQ-VAS) in children and adolescents. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement, and a negative change from baseline reflects worsening. | 60 months |
Quality of Life at Month 12 Measured by QoL Questionnaires in Adults | Change from baseline to Month 12 in EuroQol-5D-5L Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement, and a negative change from baseline reflects worsening. | 12 months |
Quality of Life at Month 60 Measured by QoL Questionnaires in Adults | Change from baseline to Month 60 in EuroQol-5D-5L Visual Analogue Scale (EQ-VAS) in adults. EQ-VAS uses a scale from 0 to 100, where 0 represents the worst imaginable health state and 100 represents the best imaginable health state. A positive change from baseline reflects improvement, and a negative change from baseline reflects worsening. | 60 months |
적격성 기준
Inclusion in the study will be limited to individuals who:
- Are able to give informed consent or assent, with or without the guidance of their parent(s)/guardian(s), where appropriate
- Received AAV2/8-hCARp.hCNGB3 or AAV2/8-hG1.7p.coCNGA3 by intraocular administration in the prior open-label, Phase I/II, dose escalation study (EudraCT 2016-002290-35 or EudraCT 2018-003431-29)
- Are willing to adhere to the protocol and long-term follow-up
Individuals will be excluded who:
Are unwilling or unable to meet the requirements of the study
메이라지티엑스연락처 정보가 없습니다.
2 2개국에 임상시험 장소
Michigan
Kellogg Eye Center, Ann Arbor, Michigan, 48105, United States
Moorfields Eye Hospital NHS Foundation Trust, London, United Kingdom