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El ensayo clínico NCT03406611 para Homocistinuria está activo, no reclutando. Consulte la vista de tarjeta del Radar de Ensayos Clínicos y las herramientas de descubrimiento de IA para conocer todos los detalles. O haga cualquier pregunta aquí.
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Pegtibatinase As an Enzyme Therapy for Patients with Homocystinuria Caused by Cystathionine Beta-Synthase Deficiency (COMPOSE) Fase I, Fase II 32 Dietético Novedoso

Activo, no reclutando
Los detalles del ensayo clínico están disponibles principalmente en inglés. ¡Sin embargo, IA Trial Radar puede ayudar! Simplemente haga clic en 'Explicar el estudio' para ver y discutir la información del estudio en el idioma que haya seleccionado.
El ensayo clínico NCT03406611 está diseñado para estudiar el tratamiento de Homocistinuria. Es un estudio intervencionista de Fase I Fase II. Su estado actual es: activo, no reclutando. El estudio se inició el 22 de enero de 2019, con el objetivo de reclutar a 32 participantes. Dirigido por Travere Therapeutics, se espera que finalice el 1 de diciembre de 2026. Los datos se actualizaron por última vez en ClinicalTrials.gov el 22 de noviembre de 2024.
Resumen
Homocystinuria caused by Cystathionine Beta-Synthase (CBS) Deficiency is a rare autosomal-recessive metabolic condition characterized by an excess of homocysteine (Hcy) in the plasma, tissues and urine. It is due to reduced or absent activity of the CBS enzyme, and is also known as classical homocystinuria. The symptoms associated with homocystinuria are variable in severity and time of onset across patients. Some af...Mostrar más
Título oficial

A Double Blind, Randomized, Placebo-controlled, Phase 1/2 Study to Assess the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Effects on Clinical Outcomes of Pegtibatinase (TVT-058), Administered Subcutaneously in Patients with Cystathionine Beta-Synthase Deficient Homocystinuria (COMPOSE)

Condiciones médicas
Homocistinuria
Otros ID del estudio
  • CBS-HCY-CT-01
Número del NCT
NCT03406611
Inicio del estudio (real)
2019-01-22
Última actualización
2024-11-22
Fecha de finalización (estimada)
2026-12
Inscripción (prevista)
32
Tipo de estudio
Intervencionista
FASE
Fase I
Fase II
Estado general
Activo, no reclutando
Objetivo principal
Tratamiento
Método de asignación
Aleatorizado
Modelo de intervención
Paralelo
Enmascaramiento
Cuádruple ciego
Brazos / Intervenciones
Grupo de participantesIntervención/Tratamiento
Comparador activoPegtibatinase
Pegtibatinase
Pegtibatinase sterile solution for subcutaneous injection
Comparador placeboPlacebo
PLACEBO
Normal saline for subcutaneous injection
Resultado primario
Medida de resultadoDescripción de la medidaPeriodo de tiempo
Incidence of AEs
Incidence of AEs (by type, severity and relationship to study drug)
Through double-blind study completion, approximately 10 months per patient
Anti-pegtibatinase antibodies
Presence and levels of anti-pegtibatinase antibodies in plasma as measured by antibody titers
Through double-blind study completion, approximately 10 months per patient
Anti-PEG antibodies
Presence and levels of anti-PEG antibodies in plasma as measured by antibody titers
Through double-blind study completion, approximately 10 months per patient
Resultado secundario
Medida de resultadoDescripción de la medidaPeriodo de tiempo
Changes in pegtibatinase levels
Changes in pegtibatinase levels following single and repeat administration at specified timepoints
Through double-blind study completion, approximately 10 months per patient
Changes in Met cycle metabolites levels - tHcy
Changes in total homocysteine levels in micromoles
Through double-blind study completion, approximately 10 months per patient
Changes in Met cycle metabolites levels - total Cys
Changes in total cysteine levels in micromoles
Through double-blind study completion, approximately 10 months per patient
Changes in Met cycle metabolites levels - Met
Changes in methionine levels in micromoles
Through double-blind study completion, approximately 10 months per patient
Changes in Met cycle metabolites levels - Cth
Changes in cystathionine levels in micromoles
Through double-blind study completion, approximately 10 months per patient
Changes in Met cycle metabolites levels - Phe
Changes in phenylalanine levels in micromoles
Through double-blind study completion, approximately 10 months per patient
Descriptive ophthalmology examination findings
Comprehensive ophthalmological examination (for each eye: visual acuity \[myopia, hyperopia, exotropia\], slit lamp examination \[ectopic lentis, cataracts, corneal abrasion, and uveitis\], retinal examination \[retinal degeneration, retinal detachment, retinitis pigmentosa, uveitis)\]). Assessment of presence and severity of findings.
Through double-blind study completion, approximately 10 months per patient
Bone densitometry using dual-energy X-ray absorptionmetry (DEXA) scans
Through double-blind study completion, approximately 10 months per patient
Cognitive assessments using the National Institutes of Health Toolbox Cognition Battery score
Through double-blind study completion, approximately 10 months per patient
Patient Reported Outcome (PRO): Quality of Life in Neurological Disorders [Neuro-QoL]
The Quality of Life in Neurological Disorders \[Neuro-QoL\] includes Anxiety Short Form, Depression Short Form, Satisfaction with Social Roles Short Form, Cognition Function Short Form for 18+ years of age; Anxiety Short Form, Depression Short Form, Social Relations - Interaction with Peers Short Form, and Cognitive Function Short Form for Ages 12 to 17 years old
Through double-blind study completion, approximately 10 months per patient
Patient Reported Outcome (PRO): Quality of Life by 36-Item Short Form Survey [SF-36]
Through double-blind study completion, approximately 10 months per patient
Patient Reported Outcome (PRO): Quality of Life by EuroQol 5-Dimentional Instrument [EQ 5D]
Through double-blind study completion, approximately 10 months per patient
Asistente de participación
Criterios de elegibilidad

Criterios de edad
Niño, Adulto, Adulto mayor
Edad mínima
12 Years
Criterios de sexo
Todos
  • Confirmed diagnosis of homocystinuria based on genetic confirmation and plasma tHcy ≥50 micromoles and documentation of previous tHcy level ≥80 micromoles
  • Willing and able to provide written, signed informed consent and to comply with all study related procedures.
  • Subjects born biologically as female who are of child-bearing potential must have a negative pregnancy test at screening and be willing to have additional pregnancy tests during the study. Subjects born biologically as male who identify as female and are not of childbearing potential are not required to undergo pregnancy tests
  • Sexually active subjects who have childbearing potential or those who have partners of childbearing potential must be willing to use acceptable methods of contraception while on the study and for 4 weeks after the end of study
  • Willing to maintain a stable diet with no significant modifications while on study and complete a daily diet diary.

  • Previous exposure to pegtibatinase and/or previous participation in a clinical trial that included administration of pegtibatinase
  • Use of any investigational product or investigational medical device within 30 days prior to Screening, or while on study
  • Use or planned use of any injectable drugs containing PEG (other than pegtibatinase or COVID-19 vaccines), including medroxyprogesterone (eg, Depo-Provera) injection, within 3 months prior to Screening and during study participation
  • Known hypersensitivity to PEG-containing product or any components of pegtibatinase
  • A positive test for HIV antibody, hepatitis B surface antigen, or hepatitis C antibody
  • A history of organ transplantation, chronic immunosuppressive therapy, or substance abuse
  • Pregnant or breastfeeding at Screening or planning to become pregnant (self or partner) or to breastfeed at any time during the study
  • Concurrent disease or condition or planned major surgery that would interfere with study participation or safety in the opinion of the investigator.
  • Any condition that, in the view of the investigator, places the subject at high risk of poor treatment compliance or of not completing the study
  • Major surgery planned during the study period
Travere Therapeutics, Inc. logoTravere Therapeutics
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8 Centros del estudio en 1 países

California

Travere Investigational Site - Virtual Site, Culver City, California, 90230, United States

Colorado

Travere Investigational Site, Aurora, Colorado, 80045, United States

Florida

Travere Investigational Site, Miami, Florida, 33136, United States

Indiana

Travere Investigational Site, Indianapolis, Indiana, 46202, United States

Maine

Travere Investigational Site, Portland, Maine, 04102, United States

Massachusetts

Travere Investigational Site, Boston, Massachusetts, 02115, United States

New York

Travere Investigational Site, New York, New York, 10029, United States

Pennsylvania

Travere Investigational Site, Philadelphia, Pennsylvania, 19104, United States