Clinical Trials Logo
  1. Home
  2. Choroideremia
  3. NCT02341807

Safety and Dose-escalation Study of AAV2-hCHM in Participants With CHM (Choroideremia) Gene Mutations

Choroideremia Clinical Trial

Official title:
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

Clinical Trial Summary

This clinical study evaluates the safety and tolerability of AAV2-hCHM in participants with Choroideremia gene mutations.

Clinical Trial Description

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 adeno-associated virus vector, serotype 2 (AAV2) and Rab escort protein 1 (REP-1). ;

Study Design

Related Conditions & MeSH terms

Clinical Trial Details
NCT number NCT02341807
Study type Interventional
Source Spark Therapeutics
Contact
Status Completed
Phase Phase 1/Phase 2
Start date January 15, 2015
Completion date October 12, 2022
View Details
See also
  Status Clinical Trial Phase
Completed NCT02553135 - Choroideremia Gene Therapy Clinical Trial Phase 2
Completed NCT01461213 - Gene Therapy for Blindness Caused by Choroideremia Phase 1/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Withdrawn NCT05045703 - The Dark-Adapted Retinal Function Response in Choroideremia (DARC) Study N/A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 NCT02670980 - Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy N/A
Completed NCT02671539 - THOR - Tübingen Choroideremia Gene Therapy Trial 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