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Clinical Trial Summary

The objective of the study is to evaluate the efficacy and safety of a single sub-retinal injection of AAV2-REP1 in subjects with Choroideremia.


Clinical Trial Description

This is a phase 3 clinical trial of a gene therapy vector made from adeno-associated virus (AAV) called AAV2-REP1 for the treatment of Choroideremia. Participating subjects will be required to attend a screening visit during which their suitability for the study will be assessed, and eligible subjects will be randomized to either AAV2-REP1 treatment or the control group. ;


Study Design


Related Conditions & MeSH terms


NCT number NCT03496012
Study type Interventional
Source Nightstar Therapeutics
Contact Nightstar Therapeutics
Phone +44 (0)207 062 2777
Email researchenquiries@nightstartx.com
Status Recruiting
Phase Phase 3
Start date December 11, 2017
Completion date March 31, 2020

See also
  Status Clinical Trial Phase
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Completed NCT01461213 - Gene Therapy for Blindness Caused by Choroideremia Phase 1/Phase 2
Recruiting NCT03507686 - A Safety Study of Retinal Gene Therapy for Choroideremia Phase 2
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 AAV2-REP1 for the Treatment of Choroideremia (SOLSTICE)
Recruiting NCT03359551 - Natural History of the Progression of Choroideremia Study
Recruiting NCT02435940 - Inherited Retinal Degenerative Disease Registry N/A
Active, not recruiting NCT02670980 - Compensation for Blindness With the Intelligent Retinal Implant System (IRIS V2) in Patients With Retinal Dystrophy N/A
Active, not recruiting NCT02671539 - THOR - Tübingen Choroideremia Gene Therapy Trial Phase 2
Active, not recruiting NCT02341807 - Safety and Dose Escalation Study of AAV2-hCHM in Subjects With CHM (Choroideremia) Gene Mutations Phase 1/Phase 2
Enrolling by invitation NCT03406416 - Study of a Suprachoroidal Retinal Prosthesis N/A
Active, not recruiting 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
Recruiting NCT02407678 - REP1 Gene Replacement Therapy for Choroideremia Phase 2
Completed NCT02077361 - An Open Label Clinical Trial of Retinal Gene Therapy for Choroideremia Phase 1/Phase 2
Completed NCT01864486 - Restoring Vision With the Intelligent Retinal Implant System (IRIS V1)in Patients With Retinal Dystrophy N/A