Indication
Choroideremia
6 clinical trials
5 products
Product
BIIB111Clinical trial
An Open-Label Safety Study of Retinal Gene Therapy for Choroideremia With Bilateral, Sequential Administration of Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)Status: Completed, Estimated PCD: 2022-06-29
Product
AAV2-hCHMClinical trial
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 RetinaStatus: Completed, Estimated PCD: 2022-10-12
Product
KIO-301Clinical trial
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)Status: Recruiting, Estimated PCD: 2024-12-01
Product
4D-110Clinical trial
Phase 1 Open-Label, Dose-Escalation Study of the Safety, Tolerability and Preliminary Efficacy of Intravitreal 4D-110 in Patients With ChoroideremiaStatus: Active (not recruiting), Estimated PCD: 2024-06-01
Clinical trial
A Randomised, Open Label, Outcomes-Assessor Masked, Prospective, Parallel Controlled Group, Phase 3 Clinical Trial of Retinal Gene Therapy for Choroideremia Using an Adeno-Associated Viral Vector (AAV2) Encoding Rab Escort Protein 1 (REP1)Status: Completed, Estimated PCD: 2020-12-01
Clinical trial
A Long-term Follow-up Study to Evaluate the Safety and Efficacy of Retinal Gene Therapy in Subjects With Choroideremia Previously Treated With Adeno-Associated Viral Vector Encoding Rab Escort Protein-1 (AAV2-REP1) and in Subjects With X-Linked Retinitis Pigmentosa Previously Treated With Adeno-Associated Viral Vector Encoding RPGR (AAV8-RPGR) in an Antecedent StudyStatus: , Estimated PCD: 2026-06-04
Product
BIIB112