ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5

• ClinicalTrials.gov Identifier: NCT05973630
• Recruitment Status: Not yet recruiting
• First Posted: August 3, 2023
• Last Update Posted: March 7, 2024
• Sponsor: Atamyo Therapeutics
• Information provided by (Responsible Party): Atamyo Therapeutics

Tracking Information

• First Submitted Date: July 24, 2023
• First Posted Date: August 3, 2023
• Last Update Posted Date: March 7, 2024
• Estimated Study Start Date: May 1, 2024
• Estimated Primary Completion Date: December 31, 2025 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: August 1, 2023)

• Incidence of adverse events [ Time Frame: 0-6 months ]
  Collection of adverse events at each visit
• Incidence of treatment-emergent adverse events, [ Time Frame: 0-6 months ]
  Collection of adverse events at each visit
• Incidence of serious adverse events [ Time Frame: 0-6 months ]
  Collection of adverse events at each visits

• Original Primary Outcome Measures: Same as current
• Current Secondary Outcome Measures: Not Provided
• Original Secondary Outcome Measures: Not Provided
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: ATA-200 Dose-escalation Gene Therapy Trial in Patients With LGMDR5
• 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)
• 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.

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
• Study Phase: Phase 1; Phase 2

Study Design

Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description:

Dose escalation study

Masking: None (Open Label)
Primary Purpose: Treatment

• Condition: LGMD2C

Intervention

Biological: ATA-200

single intravenous infusion

Study Arms

• Experimental: Cohort 1
  ATA-200 Dose level 1: 1.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
  Intervention: Biological: ATA-200
• Experimental: Cohort 2
  ATA-200 Dose level 2: 3.0E+14 vg/Kg, solution for injection, single IV infusion over 2h
  Intervention: Biological: ATA-200

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Not yet recruiting
• Estimated Enrollment (submitted: August 1, 2023): 6
• Original Estimated Enrollment: Same as current
• Estimated Study Completion Date: June 30, 2030
• Estimated Primary Completion Date: December 31, 2025 (Final data collection date for primary outcome measure)

Eligibility 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

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 6 Years to 12 Years (Child)
• Accepts Healthy Volunteers: No

Contacts

Contact: Damien Bouvier; +33972662469; d.bouvier@atamyo.com

Contact: Sophie Olivier; s.olivier@atamyo.com

• Listed Location Countries: France, Italy

Administrative Information

• NCT Number: NCT05973630
• Other Study ID Numbers: ATA-003-GSAR
• Has Data Monitoring Committee: Yes

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

• IPD Sharing Statement: Not Provided
• Current Responsible Party: Atamyo Therapeutics
• Original Responsible Party: Same as current
• Current Study Sponsor: Atamyo Therapeutics
• Original Study Sponsor: Same as current
• Collaborators: Not Provided
• Investigators: Not Provided
• PRS Account: Atamyo Therapeutics
• Verification Date: March 2024