Estudio para evaluar ACDN-01 en retinopatía relacionada con ABCA4 (STELLAR)
ACDN-01-001: Estudio abierto de dosis única ascendente para evaluar la seguridad, tolerabilidad y eficacia preliminar del ACDN-01 subretiniano en participantes con retinopatía relacionada con ABCA4
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.
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Inscripción (Estimado)
Fase
- Fase 2
- Fase 1
Contactos y Ubicaciones
Ubicaciones de estudio
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California
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San Francisco, California, Estados Unidos, 94158
- University of San Francisco
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Florida
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Gainesville, Florida, Estados Unidos, 32607
- Vitreo Retinal Associates
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Miami, Florida, Estados Unidos, 33136
- Bascom Palmer Eye Institute
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Maryland
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Baltimore, Maryland, Estados Unidos, 21218
- Wilmer Eye Institute at John Hopkins
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Massachusetts
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Boston, Massachusetts, Estados Unidos, 02114
- Massachusetts Eye and Ear
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Michigan
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Ann Arbor, Michigan, Estados Unidos, 48105
- University of Michigan Kellogg Eye Center
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Ohio
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Cincinnati, Ohio, Estados Unidos, 45245
- Cincinnati Eye Institute
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Oregon
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Portland, Oregon, Estados Unidos, 97239
- Casey Eye Institute OHSU
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Pennsylvania
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Philadelphia, Pennsylvania, Estados Unidos, 19104
- Children's Hospital of Philadelphia
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Texas
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Dallas, Texas, Estados Unidos, 75382
- Retina Foundation of the Southwest
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Houston, Texas, Estados Unidos, 77401
- Retina Consultants of Texas
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
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.
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: No aleatorizado
- Modelo Intervencionista: Asignación Secuencial
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
|
Experimental: Low dose of ACDN-01
One time low dose of ACDN-01. |
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. |
|
Experimental: Mid-dose of ACDN-01
One time mid-dose of ACDN-01. |
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. |
|
Experimental: High dose of ACDN-01
One time high dose of ACDN-01. |
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 mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
Periodo de tiempo: 12 months
|
To evaluate the safety and tolerability of a single dose of ACDN-01 when administered to participants with ABCA4-related retinopathy. |
12 months
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
Periodo de tiempo: 12 months
|
To determine maximum tolerated dose (MTD) for subsequent clinical evaluation. |
12 months
|
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To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
Periodo de tiempo: 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. |
24 months
|
|
To evaluate for evidence of preliminary efficacy based on optical coherence tomography (OCT).
Periodo de tiempo: 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. |
24 months
|
Otras medidas de resultado
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
|
To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity.
Periodo de tiempo: 24 months
|
Best Corrected Visual Acuity is measured by number of ETDRS letters read in each eye compared to baseline. |
24 months
|
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To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.
Periodo de tiempo: 24 months
|
Patient-reported outcome measures will be measured by overall and subscale scores on the Michigan Retinal -Degeneration Questionnaire (MRDQ). |
24 months
|
|
To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
Periodo de tiempo: 24 months
|
Contrast sensitivity is measured using qCSF tests and will measure number of letters read and change in AUC (area under curve). |
24 months
|
|
To assess for evidence of preliminary efficacy of ACDN-01 based on contrast sensitivity.
Periodo de tiempo: 24 months
|
Contrast sensitivity is measured using Pelli-Robson tests and will measure number of letters read and change in AUC (area under curve). |
24 months
|
|
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Periodo de tiempo: 24 months
|
Retinal sensitivity will be measured by change in the location of detectable points. |
24 months
|
|
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Periodo de tiempo: 24 months
|
Retinal sensitivity will be measured by change in the intensity (dB) of detectable points. |
24 months
|
|
To assess for evidence of preliminary efficacy of ACDN-01 based on microperimetry.
Periodo de tiempo: 24 months
|
Retinal sensitivity will be measured by change in the number of detectable points. |
24 months
|
Colaboradores e Investigadores
Patrocinador
Investigadores
- Director de estudio: Murray Abramson, MD, PhD, Ascidian Therapeutics
Publicaciones y enlaces útiles
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio (Actual)
Finalización primaria (Estimado)
Finalización del estudio (Estimado)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Actual)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
- Enfermedades Genéticas Congénitas
- Enfermedades de los ojos
- Enfermedades De Los Ojos Hereditarias
- Enfermedades de la retina
- Distrofias retinales
- Degeneración retinal
- Degeneración macular
- Enfermedades y anomalías congénitas, hereditarias y neonatales
- Distrofias de conos y bastones
- Enfermedad de Stargardt
Otros números de identificación del estudio
- ACDN-01-001
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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