Studio per valutare ACDN-01 nella retinopatia correlata ad ABCA4 (STELLAR)
ACDN-01-001: studio in aperto con dose singola crescente per valutare la sicurezza, la tollerabilità e l'efficacia preliminare dell'ACDN-01 sottoretinico in partecipanti con retinopatia correlata ad 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.
Panoramica dello studio
Stato
Condizioni
Intervento / Trattamento
Descrizione dettagliata
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 di studio
Iscrizione (Stimato)
Fase
- Fase 2
- Fase 1
Contatti e Sedi
Luoghi di studio
-
-
California
-
San Francisco, California, Stati Uniti, 94158
- University of San Francisco
-
-
Florida
-
Gainesville, Florida, Stati Uniti, 32607
- Vitreo Retinal Associates
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Miami, Florida, Stati Uniti, 33136
- Bascom Palmer Eye Institute
-
-
Maryland
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Baltimore, Maryland, Stati Uniti, 21218
- Wilmer Eye Institute at John Hopkins
-
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Massachusetts
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Boston, Massachusetts, Stati Uniti, 02114
- Massachusetts Eye and Ear
-
-
Michigan
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Ann Arbor, Michigan, Stati Uniti, 48105
- University of Michigan Kellogg Eye Center
-
-
Ohio
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Cincinnati, Ohio, Stati Uniti, 45245
- Cincinnati Eye Institute
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Oregon
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Portland, Oregon, Stati Uniti, 97239
- Casey Eye Institute OHSU
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Pennsylvania
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Philadelphia, Pennsylvania, Stati Uniti, 19104
- Children's Hospital of Philadelphia
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Texas
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Dallas, Texas, Stati Uniti, 75382
- Retina Foundation of the Southwest
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Houston, Texas, Stati Uniti, 77401
- Retina Consultants of Texas
-
-
Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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.
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: Non randomizzato
- Modello interventistico: Assegnazione sequenziale
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
|---|---|
|
Sperimentale: 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. |
|
Sperimentale: 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. |
|
Sperimentale: 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. |
Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Safety and tolerability of ACDN-01 as measured by the number and severity of adverse events and serious adverse events.
Lasso di tempo: 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
|
Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
Maximum tolerated dose for subsequent clinical evaluation will be determined by review of all available safety and tolerability data.
Lasso di tempo: 12 months
|
To determine maximum tolerated dose (MTD) for subsequent clinical evaluation. |
12 months
|
|
To evaluate for evidence of preliminary efficacy based on fundus autofluorescence (FAF).
Lasso di tempo: 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).
Lasso di tempo: 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
|
Altre misure di risultato
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
|---|---|---|
|
To assess for evidence of preliminary efficacy of ACDN-01 based on best corrected visual acuity.
Lasso di tempo: 24 months
|
Best Corrected Visual Acuity is measured by number of ETDRS letters read in each eye compared to baseline. |
24 months
|
|
To assess for evidence of preliminary efficacy of ACDN-01 based on patient reported outcome measures.
Lasso di tempo: 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.
Lasso di tempo: 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.
Lasso di tempo: 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.
Lasso di tempo: 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.
Lasso di tempo: 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.
Lasso di tempo: 24 months
|
Retinal sensitivity will be measured by change in the number of detectable points. |
24 months
|
Collaboratori e investigatori
Sponsor
Investigatori
- Direttore dello studio: Murray Abramson, MD, PhD, Ascidian Therapeutics
Pubblicazioni e link utili
Studiare le date dei record
Studia le date principali
Inizio studio (Effettivo)
Completamento primario (Stimato)
Completamento dello studio (Stimato)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Effettivo)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Parole chiave
Termini MeSH pertinenti aggiuntivi
Altri numeri di identificazione dello studio
- ACDN-01-001
Piano per i dati dei singoli partecipanti (IPD)
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Informazioni su farmaci e dispositivi, documenti di studio
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