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 Study

Who is this study for? Patients with Choroideremia, X-Linked Retinitis Pigmentosa
Status: Enrolling_by_invitation
Location: See all (25) locations...
Intervention Type: Genetic
Study Type: Interventional
Study Phase: Phase 3
SUMMARY

The objective of the study is to evaluate the long-term safety and efficacy of a sub-retinal injection of BIIB111 in participants with Choroideremia (CHM) who have been previously treated with BIIB111 and who have exited an antecedent study; these treated participants will be compared with untreated control participants who have exited the STAR (NCT03496012) study and BIIB112 in participants with X-linked retinitis pigmentosa (XLRP) who have been previously treated with BIIB112 and who have exited an antecedent study.

Eligibility
Participation Requirements
Sex: Male
Minimum Age: 18
Healthy Volunteers: f
View:

• CHM Participants

• a. Have participated in and exited from an interventional study that investigated the safety and efficacy of a sub-retinal injection of BIIB111 for CHM.

• XLRP Participants

• a. Have received a sub-retinal injection of BIIB112 for XLRP and have exited an antecedent study.

Locations
United States
California
UCLA - Jules Stein Eye Institute
Los Angeles
Florida
Vitreo Retinal Associates PA - The Millennium Center
Gainesville
University of Miami
Miami
Massachusetts
MEEI Massachusets Eye and Ear Infirmary
Boston
Maryland
Johns Hopkins Hospital
Baltimore
New York
Columbia University Medical Center
New York
Ohio
Cincinnati Eye Institute - Blue Ash
Cincinnati
Oregon
OHSU - Casey Eye Institute
Portland
Texas
Retina Foundation of the Southwest
Dallas
Wisconsin
University of Wisconsin School of Medicine
Madison
Other Locations
Brazil
Instituto Genetica Ocular
São Paulo
Canada
The Northern Alberta Clinical Trials and Research Centre
Edmonton
McGill University Health Centre
Montreal
The University of British Columbia - Eye Care Centre
Vancouver
Denmark
Rigshospitalet-Glostrup, Oejenafdelingen
Glostrup Municipality
Finland
Helsinki University Central Hospital (HUCH)
Helsinki
France
CHU Montpellier - Saint Eloi
Montpellier
Centre Hospitalier National d Ophtalmologie (CHNO) des Quinze-Vingts
Paris
Germany
Universitäts-Augenklinik Bonn
Bonn
Universitats Klinikum Tubingen - Institute for Ophthalmic Research
Tübingen
Netherlands
Radboudumc
Nijmegen
United Kingdom
Moorfields Eye Hospital
London
Manchester Royal Eye Hopsital
Manchester
John Radcliffe Hospital
Oxford
Southampton General Hospital
Southampton
Time Frame
Start Date: 2018-06-04
Completion Date: 2026-06-04
Participants
Target number of participants: 330
Treatments
Experimental: BIIB111
Participants previously treated with sub-retinal injection of BIIB111 in antecedent studies 273CH301 (NCT03496012) and 273CH203 (NCT03507686) will be enrolled. Participants previously treated with this same sub-retinal injection (rAAV2-REP1) in antecedent studies 20150371 (NCT02553135), Pro00028599 (NCT02077361), THOR-TUE-01 (NCT02671539), CHM09/01 (NCT01461213) and REGEN2015 (NCT02407678) will also be invited for enrollment. This is a follow-up study, investigational product was administered in the previous study.
Experimental: BIIB112
Participants previously treated with sub-retinal injection of BIIB112 in the antecedent study 274RP101 (NCT03116113) will be enrolled. This is a follow-up study, investigational product was administered in the previous study.
No_intervention: Untreated
Untreated participants who served as controls in the antecedent study 273CH301 (NCT03496012), investigating treatment with BIIB111, will be enrolled. This is a follow-up study, participants will not be administered study medication nor receive a sham surgery.
Sponsors
Leads: NightstaRx Ltd, a Biogen Company

This content was sourced from clinicaltrials.gov