ATA-100 (Formerly GNT0006) Gene Therapy Trial in Patients With LGMDR9

Atamyo Therapeutics

Status and phase: Active, not recruiting; Phase 1

Conditions: LGMDR9

Treatments: Biological: ATA-100 (AAV9 encoding FKRP gene)

Study type: Interventional

Funder types: Industry

Identifiers

NCT05224505

2021-004276-33 (EudraCT Number)

ATA-001-FKRP

Details and patient eligibility

About

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

Full description

Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene.

This study is an open-label dose escalation phase with long-term follow-up (LTFU) period.

Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients.

All subjects will be followed for up to 5 years after active IMP (ATA-100) administration.

Enrollment: 6 patients

Sex: All

Ages: 16 to 99 years old

Volunteers: No Healthy Volunteers

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

Trial design

Primary purpose: Treatment

Allocation: Non-Randomized

Interventional model: Sequential Assignment

Masking: Quadruple Blind

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6 participants in 2 patient groups

Cohort 1

Experimental group

Description:

Cohort 1: single intravenous injection 9.0E+12 vg/Kg

Treatment:

Biological: ATA-100 (AAV9 encoding FKRP gene)

Cohort 2

Experimental group

Description:

Cohort 2: single intravenous injection 2.7E+13 vg/Kg

Treatment:

Biological: ATA-100 (AAV9 encoding FKRP gene)