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NCT03584165CompletedPhase 3

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

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

Source: ClinicalTrials.gov · Last updated 6 Jul 2026

Brief summary (registry, in English)

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.

Status (registry)
Completed
Phase (registry)
Phase 3
Study type
Interventional
Enrollment
277 (actual)
First submitted
29 Jun 2018
Start date
4 Jun 2018
Primary completion date
22 Jun 2026
Lead sponsor
NightstaRx Ltd, a Biogen Company
NCT number
NCT03584165
EU CT number
2023-507994-16-00
EudraCT number
2017-003104-42

What you commit to

  • Study type: Interventional · Phase 3
  • Participants enrolled: 277
  • From 4 Jun 2018 to the primary completion date, 22 Jun 2026
  • 24 sites in 9 countries
  • Visits and tests: ask the trial team, the registry does not list them

Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.

Who cannot take part

Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.

Other

  • Participants are not eligible for study participation if they meet the following exclusion criterion.
  • In the opinion of the Investigator and/or the Sponsor, it is not in the participant's best interest to participate in the study.
    • NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Who the study is looking for

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

Age in the registry: 18 years to 64 years · Sex: Male · Does not accept healthy volunteers

Full criteria on ClinicalTrials.gov

Sites (24)

Sites in registry order. Save your city in your profile to sort them by distance.

SiteCityCountrySite status
Instituto Genetica Ocular
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São PauloBrazilUnknown status
The Northern Alberta Clinical Trials and Research Centre
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EdmontonCanadaUnknown status
McGill University Health Centre
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MontrealCanadaUnknown status
The University of British Columbia - Eye Care Centre
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VancouverCanadaUnknown status
Universitaetsklinikum Tuebingen AöR
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TuebingenGermanyCompleted
Rigshospitalet-Glostrup, Oejenafdelingen
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Glostrup MunicipalityDenmarkUnknown status
Helsinki University Central Hospital (HUCH)
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HelsinkiFinlandUnknown status
Centre Hospitalier Universitaire De Montpellier
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Montpellier Cedex 5FranceCompleted
CHNO des Quinze Vingts Paris
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ParisFranceCompleted
Moorfields Eye Hospital
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LondonUnited KingdomUnknown status
Manchester Royal Eye Hopsital
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ManchesterUnited KingdomUnknown status
John Radcliffe Hospital
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OxfordUnited KingdomUnknown status
Southampton General Hospital
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SouthamptonUnited KingdomUnknown status
Radboudumc
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NijmegenNetherlandsUnknown status
Johns Hopkins Hospital
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BaltimoreUnited StatesUnknown status
MEEI Massachusets Eye and Ear Infirmary
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BostonUnited StatesUnknown status
Cincinnati Eye Institute - Blue Ash
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CincinnatiUnited StatesUnknown status
Retina Foundation of the Southwest
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DallasUnited StatesUnknown status
Vitreo Retinal Associates PA - The Millennium Center
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GainesvilleUnited StatesUnknown status
UCLA Jules Stein Eye Institute
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Los AngelesUnited StatesUnknown status
University of Wisconsin School of Medicine
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MadisonUnited StatesUnknown status
University of Miami
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MiamiUnited StatesUnknown status
Columbia University Medical Center
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New YorkUnited StatesUnknown status
OHSU Casey Eye Institute
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PortlandUnited StatesUnknown status

This page is information, not medical advice. Discuss any trial with your doctor.