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NCT05973630 | NOT YET RECRUITING | LGMD2C

ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5
Sponsor:

Atamyo Therapeutics

Brief Summary:

The purpose of ATA-003-GSAR study is to evaluate the safety and tolerability of a single intravenous infusion of ATA-200 in pediatric patients with limb girdle muscular dystrophy type 2c/R5 (LGMD R5). Patients will be treated sequentially in 2 dose-cohorts.

Condition or disease

lagand2k

Intervention/treatment

ATA-200

Phase

PHASE1

PHASE2

Detailed Description:

This is a multicenter Phase 1b assessing the safety and tolerability of 2 doses of ATA-200 for the treatment of LGMDR5. The dose escalation phase will enroll ambulant patients with LGMDR5. Two dose cohorts (C1) and (C2) will be enrolled sequentially and enrollment will be staggered with at least 4-week interval between 2 patient treatments. An initial cohort C1 of three (3) patients will receive a potentially effective dose corresponding to the minimum effective dose (MED) in preclinical studies. Enrollment of three (3) patients in the 2nd higher dose cohort C2 (with a 7-fold safety margin relative to the highest safe dose in the GLP toxicology study) will be initiated following review of the one-month safety data post-administration in cohort C1 by an independent Data Safety Monitoring Board (DSMB). Time point of primary interest for safety evaluation and dose selection for future studies is at 6 months. All subjects will be followed up for an additional 4.5 years after completion of the evaluation period.

Study Type : INTERVENTIONAL
Estimated Enrollment : 6 participants
Masking : NONE
Primary Purpose : TREATMENT
Official Title : A Phase 1-2, Open-label, Dose Escalation Study to Evaluate the Safety of 2 Doses of Intravenous ATA-200, an Adeno-associated Viral Vector Carrying the Human SGCG Gene, in Patients with Gamma-sarcoglycanopathy (LGMDR5)
Actual Study Start Date : 2024-12-01
Estimated Primary Completion Date : 2026-06-30
Estimated Study Completion Date : 2031-12-31

Information not available for Arms and Intervention/treatment

Ages Eligible for Study: 6 Years to 13 Years
Sexes Eligible for Study: ALL
Accepts Healthy Volunteers:
Criteria
Inclusion Criteria
  • * Confirmed diagnosis of LGMDR5 before age of 10, based on clinical presentation and genotyping
  • * Ambulant male or female patients aged 6 to less than 12 years of age at screening
  • * Able to perform the 10-meter walk test (10MWT) in less than 15 sec and to rise from chair with or without arm support
Exclusion Criteria
  • * Detectable neutralizing antibodies against AAV8
  • * Cardiomyopathy with left ventricular ejection fraction (LVEF) \< 50%
  • * Respiratory assistance
  • * Concomitant medical condition that might interfere with LGMDR5 evolution
  • * Acute illness within 4 weeks of anticipated IMP administration
  • * Current participation in another clinical trial with investigational medicinal product
  • * Previous participation in gene and cell therapy trials
  • * Any condition that would contraindicate immunosuppressant treatment
  • * Presence of any permanent items (e.g., metal braces) precluding undergoing MRI
  • * Any vaccination 1 month prior to planned IMP administration
  • * Serology consistent with HIV exposure or active hepatitis B or C infection
  • * Grade 2 or higher lab abnormalities for liver function tests, creatinine, hemogram and coagulation
ATA-200 Dose-escalation Gene Therapy Trial in Patients with LGMDR5

Location Details

NCT05973630

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How to Participate

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Locations

Not yet recruiting

United States, Florida

Child Health Research Institute

Gainsville, florida, United States, 32610

Not yet recruiting

France,

Hospital Keychain

Paris, France,

Not yet recruiting

Italy,

Maggiore Policlinico Hospital

Milano, Italy,