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

Description

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.

Conditions

LGMD2C

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Study Overview

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Study Details

Study overview

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.

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)

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

Condition
LGMD2C
Intervention / Treatment

-

Contacts and Locations

Gainsville

Child Health Research Institute, Gainsville, Florida, United States, 32610

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

For general information about clinical research, read Learn About Studies.

Eligibility 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
  • * 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

Ages Eligible for Study

6 Years to 13 Years

Sexes Eligible for Study

ALL

Accepts Healthy Volunteers

No

Collaborators and Investigators

Atamyo Therapeutics,

Study Record Dates

2032-01-31