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Completed Phase 3 Interventional

Long-term Safety and Efficacy Follow-up of BIIB111 for the Treatment of Choroideremia and BIIB112 for the Treatment of X-Linked Retinitis Pigmentosa

ClinicalTrials.gov ID: NCT03584165

Public ClinicalTrials.gov record NCT03584165. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.

ClinicalTrials.gov public records Last synced Sep 5, 2026, 7:27 PM EDT

Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.

Official title

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

Brief summary

Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.

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.

Study identification

NCT ID
NCT03584165
Recruitment status
Completed
Study type
Interventional
Phase
Phase 3
Enrollment
277 participants

Conditions and interventions

Interventions

Genetic

Eligibility (public fields only)

Age range
18 Years and older
Sex
Male
Healthy volunteers
Healthy volunteers not accepted

This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.

Study timeline

Start date
Jun 3, 2018
Primary completion
Jun 21, 2026
Completion
Jun 21, 2026
Last update posted
Jul 5, 2026

2018 – 2026

United States locations

U.S. sites
10
U.S. states
9
U.S. cities
10
Facility City State ZIP Site status
UCLA - Jules Stein Eye Institute Los Angeles California 90095-7065
Vitreo Retinal Associates PA - The Millennium Center Gainesville Florida 32607
University of Miami Miami Florida 33136
Johns Hopkins Hospital Baltimore Maryland 21287-0005
MEEI Massachusets Eye and Ear Infirmary Boston Massachusetts 02114-3002
Columbia University Medical Center New York New York 10032
Cincinnati Eye Institute - Blue Ash Cincinnati Ohio 45242-5664
OHSU - Casey Eye Institute Portland Oregon 97239
Retina Foundation of the Southwest Dallas Texas 75231-5080
University of Wisconsin School of Medicine Madison Wisconsin 53705-3644

Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.

Non-U.S. locations

This page focuses on the U.S. directory. The official record also lists 15 non-U.S. sites.

About this trial record page

What this page shows
Public field values for ClinicalTrials.gov record NCT03584165, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
What this page does not do
No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
Where the data comes from
Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
Last refresh
Last update posted Jul 5, 2026 · Synced Sep 5, 2026

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Open the official record

The complete protocol, eligibility criteria, and contact information for NCT03584165 live on ClinicalTrials.gov.

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