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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT05973630
Recruitment Status : Not yet recruiting
First Posted : August 3, 2023
Last Update Posted : March 7, 2024
Sponsor:
Information provided by (Responsible Party):
Atamyo Therapeutics

Study Description
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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 Intervention/treatment Phase
LGMD2C Biological: ATA-200 Phase 1 Phase 2

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 Design
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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 6 participants
Allocation: Non-Randomized
Intervention Model: Sequential Assignment
Intervention Model Description: Dose escalation study
Masking: None (Open Label)
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)
Estimated Study Start Date : May 1, 2024
Estimated Primary Completion Date : December 31, 2025
Estimated Study Completion Date : June 30, 2030

Resource links provided by the National Library of Medicine


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


Outcome Measures
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Primary Outcome Measures :
  1. Incidence of adverse events [ Time Frame: 0-6 months ]
    Collection of adverse events at each visit

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

  3. Incidence of serious adverse events [ Time Frame: 0-6 months ]
    Collection of adverse events at each visits


Eligibility Criteria
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Ages Eligible for Study:   6 Years to 12 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
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
Contacts and Locations
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Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT05973630


Contacts
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Contact: Damien Bouvier +33972662469 d.bouvier@atamyo.com
Contact: Sophie Olivier s.olivier@atamyo.com

Locations
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France
Hopital Trousseau
Paris, France
Contact: Marina Colella, MD, PhD    + 33 (0)1 71 73 80 42    m.colella@institut-myologie.org   
Italy
Ospedale Maggiore Policlinico
Milano, Italy
Contact: Giacomo Pietro Comi, MD    +39 0255033802    giacomo.comi@unimi.it   
Sponsors and Collaborators
Atamyo Therapeutics
More Information
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Responsible Party: Atamyo Therapeutics
ClinicalTrials.gov Identifier: NCT05973630    
Other Study ID Numbers: ATA-003-GSAR
First Posted: August 3, 2023    Key Record Dates
Last Update Posted: March 7, 2024
Last Verified: March 2024

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