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Trial and Study Record

AB-1003 Gene Therapy Study in Adults With LGMD2I/R9

Phase 1/2 placebo-controlled gene-therapy study in adults with genetically confirmed FKRP-related LGMD2I/R9.

TrialTherapy DevelopmentTrial ReadinessFamilies

Key Takeaway

The current registry record lists nine actual participants and active follow-up without recruitment. It does not establish access, eligibility, safety, or benefit outside the protocol.

Why this record matters

  • The registry describes randomized, placebo-controlled sequential dose cohorts and a single intravenous infusion.
  • Enrollment is nine actual participants, with estimated primary completion in December 2028 and estimated overall completion in December 2032.

Eligibility and participation notes

  • The record describes adults aged 18 to 65 with a clinical LGMD2I/R9 diagnosis, confirmed FKRP variants, and protocol-defined walking ability.
  • Selected exclusions include defined cardiac, renal, liver, respiratory, prior gene-therapy, investigational-treatment, and AAV9-antibody criteria; the complete current record controls.
  • These criteria describe a study that is not recruiting and are not an individual eligibility assessment.

Limits and cautions

  • ClinicalTrials.gov reports information submitted by the responsible party and can change; current follow-up does not mean new enrollment is available.
  • The record has no posted results and lists no expanded-access program.
  • Listed study locations identify protocol sites, not places currently accepting participants.

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