A Phase 1 Multicenter Study to Evaluate the Safety and Tolerability of Intravenous GNT0006, Adeno-associated Viral Vector Carrying the FKRP Gene, in Patients With FKRP-related Limb-girdle Muscular Dystrophy (LGMDR9, Formerly LGMD2I)

Trial ID: NCT05224505

Interventional - Phase 1 - Active, not recruiting

Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up

Conditions

• LGMDR9

Interventions

• ATA-100 (AAV9 encoding FKRP gene)

Eligibility

Inclusion criteria

• 1\. Female and male ambulant patients
• 2\. Patients ≥ 16 years old
• 3\. Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
• 4\. Moderate diaphragmatic muscle impairment

Exclusion criteria

• 1\. Detectable serum neutralizing antibodies against AAV9
• 2\. Cardiomyopathy

Sponsor

Atamyo Therapeutics

Source: ClinicalTrials.gov

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