Choroideremia Clinical Trial
Official title:
An Open Label Dose Escalation Phase 1 Clinical Trial of Retinal Gene Therapy for Choroideraemia Using an Adeno-associated Viral Vector (AAV2) Encoding Rab-escort Protein 1 (REP1)
- Primary objective: To assess the safety and tolerability of the AAV.REP1 vector,
administered at two different doses to the retina in 12 patients with a diagnosis of
choroideremia.
- Secondary Objective: To identify any therapeutic benefit as evidenced by a slowing down of
the retinal degeneration assessed by functional and anatomical methods in the treated eye
compared to the control eye 24 months after gene delivery.
Detailed description may be found in the following scientific publication:
Retinal gene therapy in patients with choroideremia: initial findings from a phase 1/2
clinical trial, The Lancet, Volume 383, Issue 9923, Pages 1129 - 1137 (29 March 2014).
Links: www.thelancet.com/journals/lancet/article/PIIS0140-6736(13)62117-0/abstract ;
http://dx.doi.org/doi:10.1016/S0140-6736(13)62117-0
;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Completed |
NCT02553135 -
Choroideremia Gene Therapy Clinical Trial
|
Phase 2 | |
| Completed |
NCT03507686 -
A Safety Study of Retinal Gene Therapy for Choroideremia With Administration of BIIB111
|
Phase 2 | |
| Recruiting |
NCT05258032 -
Structural and Functional Characterization of Rare Ocular Diseases
|
||
| Recruiting |
NCT05282953 -
A Phase I/II Dose-escalating Study of the Safety, Tolerability and Efficacy of KIO-301 Administered Intravitreally to Patients With Retinitis Pigmentosa and Choroideremia (ABACUS)
|
Phase 1/Phase 2 | |
| Withdrawn |
NCT05045703 -
The Dark-Adapted Retinal Function Response in Choroideremia (DARC) Study
|
N/A | |
| Recruiting |
NCT01866371 -
High Resolution Retinal Imaging
|
||
| Completed |
NCT01603576 -
Pilot Study of a Suprachoroidal Retinal Prosthesis
|
N/A | |
| Enrolling by invitation |
NCT03584165 -
Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa
|
Phase 3 | |
| Completed |
NCT03359551 -
Natural History of the Progression of Choroideremia Study
|
||
| Recruiting |
NCT02435940 -
Inherited Retinal Degenerative Disease Registry
|
||
| Active, not recruiting |
NCT04483440 -
Dose Escalation Study of Intravitreal 4D-110 in Patients With Choroideremia
|
Phase 1 | |
| Completed |
NCT02671539 -
THOR - Tübingen Choroideremia Gene Therapy Trial
|
Phase 2 | |
| Completed |
NCT02670980 -
Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy
|
N/A | |
| Completed |
NCT02341807 -
Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations
|
Phase 1/Phase 2 | |
| Completed |
NCT03406416 -
Study of a Suprachoroidal Retinal Prosthesis
|
N/A | |
| Enrolling by invitation |
NCT05158049 -
Longitudinal Study of a Bionic Eye
|
||
| Terminated |
NCT02994368 -
"Natural History" Study of Choroideremia
|
||
| Terminated |
NCT01654562 -
The Short-term Effects of Simvastatin on the Vision of Males Affected by Choroideremia
|
Phase 1/Phase 2 | |
| Active, not recruiting |
NCT00427180 -
IRIS PILOT - Extended Pilot Study With a Retinal Implant System
|
N/A | |
| Completed |
NCT04750785 -
A Study to Assess Choroideremia (CHM) Health Outcomes
|