Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations
Status: | Active, not recruiting |
---|---|
Healthy: | No |
Age Range: | 18 - Any |
Updated: | 3/14/2019 |
Start Date: | January 2015 |
End Date: | October 2019 |
A Phase 1/2 Safety Study in Subjects With CHM (Choroideremia) Gene Mutations Using an Adeno-Associated Virus Serotype 2 Vector to Deliver the Normal Human CHM Gene [AAV2-hCHM] to the Retina
This clinical study evaluates the safety and tolerability of AAV2-hCHM in subjects with
Choroideremia gene mutations. Two dose groups will be evaluated.
Choroideremia gene mutations. Two dose groups will be evaluated.
The primary objective is to evaluate the safety and tolerability of subretinal administration
of AAV2-hCHM, in an inter-subject group dose escalation in individuals with choroideremia,
based on a comprehensive clinical monitoring plan. The secondary objectives are to define the
dose of AAV2-hCHM required to achieve stable, or improved, visual function/functional vision
and to assess development of immune responses to AAV2 and REP-1.
of AAV2-hCHM, in an inter-subject group dose escalation in individuals with choroideremia,
based on a comprehensive clinical monitoring plan. The secondary objectives are to define the
dose of AAV2-hCHM required to achieve stable, or improved, visual function/functional vision
and to assess development of immune responses to AAV2 and REP-1.
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 ETDRS testing in the eye to be injected
- Presence of disease which may preclude the subject 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.
We found this trial at
3
sites
Univ of Pennsylvania Penn has a long and proud tradition of intellectual rigor and pursuit...
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Children's Hospital of Philadelphia Since its start in 1855 as the nation's first hospital devoted...
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Massachusetts Eye & Ear Infirmary Whether you see our physicians at Mass. Eye and Ear's...
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