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

Part of paid clinical trials in Los Angeles, California.

Sponsor
NightstaRx Ltd, a Biogen Company
Study ID
NCT03584165
Phase
PHASE3
Status
Enrolling By Invitation

Conditions

  • Choroideremia
  • X-Linked Retinitis Pigmentosa

Eligibility Criteria

Sex
MALE
Age
18 Years - N/A
Healthy Volunteers
Not accepted

Interventions

  • BIIB111 — GENETIC
    Administered as specified in the treatment arm.
  • BIIB112 — GENETIC
    Administered as specified in the treatment arm.

Study Details

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.

Key Dates

Start date
Jun 4, 2018
Status verified
Jun 2025
Primary completion
Jun 4, 2026
Completion
Jun 4, 2026

Study Design

Enrollment
330 participants (estimated)
Allocation
NON_RANDOMIZED
Intervention model
PARALLEL
Primary purpose
OTHER

Arms

  • 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.

Primary Outcome Measure

Percentage of Participants with Adverse Events (AEs) [ Time Frame: Up to 5 years ]

Locations (10)

FacilityCityStateZIPSite coordinators
UCLA - Jules Stein Eye InstituteLos AngelesCalifornia90095-7065-
Vitreo Retinal Associates PA - The Millennium CenterGainesvilleFlorida32607-
University of MiamiMiamiFlorida33136-
Johns Hopkins HospitalBaltimoreMaryland21287-0005-
MEEI Massachusets Eye and Ear InfirmaryBostonMassachusetts02114-3002-
Columbia University Medical CenterNew YorkNew York10032-
Cincinnati Eye Institute - Blue AshCincinnatiOhio45242-5664-
OHSU - Casey Eye InstitutePortlandOregon97239-
Retina Foundation of the SouthwestDallasTexas75231-5080-
University of Wisconsin School of MedicineMadisonWisconsin53705-3644-

Find similar trials in Los Angeles, CA

By research site
UCLA - Jules Stein Eye Institute· Los Angeles, CAVitreo Retinal Associates PA - The Millennium Center· Gainesville, FLUniversity of Miami· Miami, FLJohns Hopkins Hospital· Baltimore, MDMEEI Massachusets Eye and Ear Infirmary· Boston, MAColumbia University Medical Center· New York, NY

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