Study to Evaluate ACDN-01 in ABCA4-related Stargardt Retinopathy (STELLAR)
Stargardt Disease · Cone Rod Dystrophy · Juvenile Macular Degeneration · Stargardt Disease 1
De qué trata este estudio
El registro no identifica actualmente este estudio como reclutando. Consulta su estado antes de planificar una evaluación.
- Tipo de estudio
- De intervención
- Fase
- Fase 1 / Fase 2
- Rango de edad
- 12 años - No especificado
- Sexo admitido
- Todos los sexos
- Voluntarios sanos
- No aceptados
- Participantes
- 15 Estimado
Estos son puntos de partida, no una decisión de elegibilidad. El equipo debe revisar todos los requisitos.
Las explicaciones y la navegación están en español. Los textos proporcionados por el registro, incluidos títulos, resúmenes y criterios, se conservan en su idioma original. Puedes pedir orientación en español a WCR.
Acerca de la investigación
This study is an open-label, single ascending dose clinical trial in participants who have ABCA4-related retinopathies. This is the first-in-human clinical trial in which ACDN-01 will be evaluated for safety, tolerability, and preliminary efficacy following a single subretinal injection of ACDN-01.
Leer la descripción completa
This is an open-label, single ascending dose study of ACDN-01 in adult participants with ABCA4-related retinopathy. The study is designed to evaluate the safety, tolerability, and evidence of biological effect of SAD levels (low, medium, and high) of ACDN-01 when delivered subretinally.
Participants will be followed on study for 2 years for the primary safety and preliminary efficacy endpoints, after which they will continue in the study in a 3-year long-term follow-up period, for a total study duration of 5 years.
¿Quién podría participar?
La lista siguiente contiene el texto del patrocinador. Puede incluir condiciones, tratamientos previos, resultados de pruebas y otros requisitos. Aparecer en tu búsqueda no significa que califiques.
Key Inclusion Criteria:
- Presence of mutations in the ABCA4 gene
- ABCA4 retinopathy phenotype (Stargardt disease type 1 or cone-rod dystrophy)
- Area of atrophy located in the macula of the study eye
- BCVA of 20/50 (0.4 logMAR) or worse
Key Exclusion Criteria:
- The presence of pathogenic or likely pathogenic mutations in other genes known to cause cone-rod dystrophy or Stargardt maculopathy
- Retinal disease other than ABCA4-related retinopathy
- Presence of a medical condition (systemic or ophthalmic), psychiatric condition, including substance abuse disorder, or physical examination or laboratory finding that may in the opinion of the principal investigator and sponsor preclude adherence to the scheduled study visits, safe participation in the study, or affect the results of the study.
Tratamiento, grupos y participación
El registro puede describir tratamientos y duración sin incluir un calendario completo. Pide al equipo las visitas, procedimientos, riesgos, costos y alternativas. Mantén tu atención habitual salvo que tu médico te indique un cambio.
- Asignación
- Sin asignación al azar
- Enmascaramiento
- Sin enmascaramiento
Low dose of ACDN-01
One time low dose of ACDN-01.
Drug: ACDN-01
Mid-dose of ACDN-01
One time mid-dose of ACDN-01.
Drug: ACDN-01
High dose of ACDN-01
One time high dose of ACDN-01.
Drug: ACDN-01
ACDN-01 DRUG
ACDN-01 is an AAV-based vector carrying a DNA construct encoding for an ABCA4 RNA exon editor. One time administration is via subretinal injection.
Qué evalúan los investigadores
Estas medidas indican qué se estudia. No prueban que un tratamiento funcione.
Medidas principales
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
Plazo: 12 months
To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy.
Medidas secundarias
Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
Plazo: 12 months
To determine maximum tolerated dose (MTD) for subsequent clinical evaluation.
To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
Plazo: 24 months
To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in the area of macular atrophy in mm2 as measured on FAF over 24 months.
To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).
Plazo: 24 months
To evaluate for evidence of preliminary efficacy based on change from baseline in the study eye in change in thickness in retinal layers as measured on OCT, over 24 months.
Otras medidas
To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity.
Plazo: 24 months
Best Corrected Visual Acuity is measured by number of ETDRS letters read in each eye compared to baseline.
To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.
Plazo: 24 months
Patient-reported outcome measures will be measured by overall and subscale scores on the Michigan Retinal -Degeneration Questionnaire (MRDQ).
To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
Plazo: 24 months
Contrast sensitivity is measured using qCSF tests and will measure number of letters read and change in AUC (area under curve).
To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
Plazo: 24 months
Contrast sensitivity is measured using Pelli-Robson tests and will measure number of letters read and change in AUC (area under curve).
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Plazo: 24 months
Retinal sensitivity will be measured by change in the location of detectable points.
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Plazo: 24 months
Retinal sensitivity will be measured by change in the intensity (dB) of detectable points.
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Plazo: 24 months
Retinal sensitivity will be measured by change in the number of detectable points.
Revisa las tablas completas y sus limitaciones en el registro oficial.
Abrir resultados oficiales ↗Centros de investigación (11)
El estado general puede diferir del estado de un centro. Confirma la disponibilidad antes de trasladarte.
11 centros
University of San FranciscoSan Francisco, California, United StatesEstado desconocido
Vitreo Retinal AssociatesGainesville, Florida, United StatesEstado desconocido
Bascom Palmer Eye InstituteMiami, Florida, United StatesEstado desconocido
Wilmer Eye Institute at John HopkinsBaltimore, Maryland, United StatesEstado desconocido
Massachusetts Eye and EarBoston, Massachusetts, United StatesEstado desconocido
University of Michigan Kellogg Eye CenterAnn Arbor, Michigan, United StatesEstado desconocido
Cincinnati Eye InstituteCincinnati, Ohio, United StatesEstado desconocido
Casey Eye Institute OHSUPortland, Oregon, United StatesEstado desconocido
Children's Hospital of PhiladelphiaPhiladelphia, Pennsylvania, United StatesEstado desconocido
Retina Foundation of the SouthwestDallas, Texas, United StatesEstado desconocido
Retina Consultants of TexasHouston, Texas, United StatesEstado desconocido
Equipo y fechas
- Patrocinador principal
- Ascidian Therapeutics, Inc
- Inicio del estudio
- 11 jun 2024 Real
- Finalización primaria
- 1 ago 2030 Estimado
- Finalización del estudio
- 1 dic 2030 Estimado
- Primera publicación
- 21 jun 2024 Real
- Última actualización
- 10 nov 2026 Real
Contactos oficiales del estudio
No hay un contacto central indicado. Revisa los centros y el registro oficial.
Título oficial del estudio
ACDN-01-001: Open-Label, Single Ascending Dose Study to Evaluate the Safety, Tolerability, and Preliminary Efficacy of Subretinal ACDN-01 in Participants With ABCA4-related Retinopathy
Consulta a Wellness Clinical Research
Tu mensaje va a WCR, incluso si otra organización realiza este estudio. WCR puede orientarte, pero podría no realizar este estudio, tener acceso a su equipo ni poder inscribirte.
Fuente: API v2 de ClinicalTrials.gov. Patrocinadores e investigadores envían la información. La NLM no aprueba la seguridad ni la ciencia de todos los estudios registrados. Aviso del registro.
Consultado: 12 nov 2026 · Última actualización del registro: 10 nov 2026