A Safety Study of Retinal Gene Therapy for Choroideremia (GEMINI)
|The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.|
|ClinicalTrials.gov Identifier: NCT03507686|
Recruitment Status : Active, not recruiting
First Posted : April 25, 2018
Last Update Posted : February 24, 2020
|Condition or disease||Intervention/treatment||Phase|
|Choroideremia||Drug: AAV2-REP1||Phase 2|
This is a phase 2 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 undergo a surgical procedure under general anaesthesia consisting of vitrectomy, retinal detachment and sub-retinal administration of AAV2-REP1.
Subjects will be treated bilaterally, with the interval between eyes determined by discussion between the investigator and Nightstar. Participating subjects will be required to attend follow up visits for 12 months following the second eye surgery.
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||60 participants|
|Intervention Model:||Single Group Assignment|
|Masking:||None (Open Label)|
|Official Title:||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)|
|Actual Study Start Date :||November 6, 2017|
|Estimated Primary Completion Date :||February 28, 2022|
|Estimated Study Completion Date :||February 28, 2022|
Bilateral sub-retinal administration of AAV2-REP1.
Sub-retinal injection of AAV2-REP1 after vitrectomy.
Other Name: Gene Therapy
- Adverse events as a measure of safety and tolerability [ Time Frame: up to 2 years ]Evaluation of safety as measured by adverse events.
- Best Corrected Visual Acuity (BCVA) [ Time Frame: up to 2 years ]ETDRS visual acuity chart
- Fundus autofluorescence (AF) [ Time Frame: up to 2 years ]Change in AF (mm2)
- Optical coherence tomography (OCT) [ Time Frame: up to 2 years ]Ellipsoid Zone
- Microperimetry [ Time Frame: up to 2 years ]Change in Sensitivity (dB)
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03507686
|United States, Florida|
|Miami, Florida, United States, 33136|
|United States, Massachusetts|
|Boston, Massachusetts, United States, 02114|
|United States, Ohio|
|Cincinnati, Ohio, United States, 45242|
|United States, Oregon|
|Portland, Oregon, United States, 97232|