Key Takeaway
This Phase 1 record describes six participants in an FKRP gene-transfer study with long-term follow-up. It is listed as active, not recruiting; it does not establish clinical benefit or offer enrollment.
Trial and Study Record
ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9
Phase 1 FKRP gene-transfer trial record for ATA-100, designed to assess safety, pharmacodynamics, and immunogenicity with long-term follow-up.
TrialTherapy DevelopmentTrial ReadinessFamilies
Why this record matters
- Directly relevant to the current therapeutic pipeline because it evaluates an FKRP-specific gene-transfer strategy rather than nonspecific supportive care.
- Safety and tolerability are the primary outcome; functional and biomarker measures are secondary outcomes.
- The record lists primary completion on 2025-02-01 and estimated study completion on 2029-09-30, with follow-up up to five years after treatment.
Eligibility and participation notes
- The criteria required ambulant patients aged at least 16 years with genetically confirmed LGMDR9; the structured age fields specify 16 to 99 years.
- Required moderate diaphragmatic muscle impairment.
- Excluded patients with detectable serum neutralizing antibodies against AAV9 or cardiomyopathy.
Limits and cautions
- Phase 1 design and very small enrollment limit what can be inferred about benefit.
- Exclusion of cardiomyopathy means the study population does not represent every person living with FKRP-related disease.
- The sponsor describes a broader phase 1b/2b program; that does not change the Phase 1 label of this registry record. No results are posted in this record.
- Listed countries describe study sites, not confirmed places to enroll. The eligibility notes are a selection of the source criteria.
Primary Sources
Direct links for verification
Primary registry or publication source used for the structured study summary on this page.
Sponsor announcement dated 2025-04-03 describes a broader phase 1b/2b program. The ClinicalTrials.gov record summarized here is listed as Phase 1.
Related Content
Related routes inside the site
Study Record
BBP-418 (Ribitol) FORTIFY Trial in LGMD2I/LGMDR9
Phase 3 randomized ribitol trial in FKRP-related LGMD2I/LGMDR9, with trial details and separately dated sponsor-reported regulatory updates.
Study Record
Study of BBP-418 in Patients With LGMD2I
Phase 2 open-label ribitol study in FKRP-related LGMD2I/LGMDR9 that provides the main early clinical development context for the BBP-418 program.
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.