Clincosm LogoSign in Get a demo

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

Sponsor
Atamyo Therapeutics (Industry)
Overall Status
Not yet recruiting
CT.gov ID
NCT05973630
Collaborator
(none)
6
Enrollment
2
Arms
77.9
Anticipated Duration (Months)

Study Details

Study Description

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
  • 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

Study Type:
Interventional
Anticipated Enrollment :
6 participants
Allocation:
Non-Randomized
Intervention Model:
Sequential Assignment
Intervention Model Description:
Dose escalation studyDose 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 Gamma-sarcoglycan Gene, in Patients With Gamma-sarcoglycanopathy (Limb-girdle Muscular Dystrophy LGMDR5)
Anticipated Study Start Date :
Jan 1, 2024
Anticipated Primary Completion Date :
Dec 31, 2025
Anticipated Study Completion Date :
Jun 30, 2030

Arms and Interventions

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

Primary Outcome Measures

  1. Incidence of adverse events [0-6 months]

    Collection of adverse events at each visit

  2. Incidence of treatment-emergent adverse events, [0-6 months]

    Collection of adverse events at each visit

  3. Incidence of serious adverse events [0-6 months]

    Collection of adverse events at each visits

Eligibility Criteria

Criteria

Ages Eligible for Study:
6 Years to 12 Years
Sexes Eligible for Study:
All
Accepts Healthy Volunteers:
No
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

Locations

No locations specified.

Sponsors and Collaborators

  • Atamyo Therapeutics

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.
Responsible Party:
Atamyo Therapeutics
ClinicalTrials.gov Identifier:
NCT05973630
Other Study ID Numbers:
  • ATA-003-GSAR
First Posted:
Aug 3, 2023
Last Update Posted:
Aug 3, 2023
Last Verified:
Jul 1, 2023
Studies a U.S. FDA-regulated Drug Product:
No
Studies a U.S. FDA-regulated Device Product:
No

Study Results

No Results Posted as of Aug 3, 2023