Phase 2
N=15
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
Choroideremia · CHM (Choroideremia) Gene Mutations
Bottom Line
View on ClinicalTrials.gov: NCT02341807 ↗Enrolled (actual)
15
Serious AEs
33.3%
Results posted
Nov 2023
Primary outcome: Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) — 5; 5; 5 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 2
- Interventions
- AAV2-hCHM (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- Male
- Sponsor
- Spark Therapeutics, Inc.
- Primary completion
- Oct 2022
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Treatment-Emergent Adverse Events (TEAEs) |
5; 5; 5 | — |
| SECONDARY Number of Participants With Anti-AAV2 Viral Capsid Antibody Titers That Rose Above Baseline At Least Once After Dosing |
0; 1; 3 | — |
| SECONDARY Number of Participants With Cellular Immune Response to AAV2 Through Interferon Gamma Enzyme-linked Immunosorbent Spot (ELISpot) Assay |
0; 0; 0 | — |
| SECONDARY Number of Participants With Cellular Immune Response to Rab Escore Protein-1 (REP-1) Through Interferon Gamma ELISPOT Assay |
2; 0; 0 | — |
Summary
This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.
Eligibility Criteria
Inclusion Criteria
- Male at least 18 years of age diagnosed with CHM gene mutation
- Central visual field (VF) <30° in any of the 24 meridians (using Goldmann perimetry III4e isopter) in the eye to be injected
- Any evidence of functioning outer retinal cells within the central 10°
Exclusion Criteria
- Previous history of ocular inflammatory disease (uveitis)
- Prior intraocular surgery within six months
- Participation in a previous gene therapy research trial within one year of enrollment or participation in any other ocular gene therapy trial
- Participation in a clinical study with an investigational drug in the past six months
- Grossly asymmetrical disease, or other eye morbidity, which may render the contralateral eye ineffective as a control
- Visual acuity <20/200 on standard Early Treatment of Diabetic Retinopathy Study (ETDRS) testing in the eye to be injected
- Presence of disease which may preclude the participant from participation in this trial
- Use of medications known to be neuroprotective or retino-toxic that could potentially interfere with the disease process and/or cause ocular adverse events; individuals who discontinue use of these compounds for 6 months may become eligible
- Identification by the investigator as being unable or unwilling to perform/be compliant with study procedures.
Data sourced from ClinicalTrials.gov (NCT02341807). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.