A Phase I/II, 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, formerly LGMD2C). - ATA-003-GSAR

Atamyo Therapeutics0 sites6 target enrollmentAugust 8, 2023

Conditionslimb-girdle muscular dystrophy type R5 (previously named LGMD2C)MedDRA version: 20.0Level: PTClassification code: 10028356Term: Muscular dystrophy Class: 100000004850 Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Overview

Phase: Phase 1
Intervention: Not specified
Conditions: limb-girdle muscular dystrophy type R5 (previously named LGMD2C)
Sponsor: Atamyo Therapeutics
Enrollment: 6
Status: Active, not recruiting
Last Updated: last year

Overview Investigators Eligibility Criteria Outcomes Similar Trials

Overview

Brief Summary

No summary available.

Registry

who.int

Start Date

August 8, 2023

End Date

TBD

Last Updated

last year

Study Type

Interventional clinical trial of medicinal product

Sex

All

Investigators

Sponsor

Atamyo Therapeutics

Eligibility Criteria

Inclusion Criteria

•Ambulant male or female patients at least 6 and less than 12 years of age at screening, Confirmed LGMDR5 diagnosis before age of 10, based on clinical presentation and genotyping identifying the SGCG gene mutations, Able to Perform the 10\-meter walk test (10MWT) within 15 sec without help, such as cane, or bilateral help, such as elbow crutches or orthotic devices below the knees and to rise from a standard\-height chair with or without arm support, Signed written informed consent before any study related procedure is performed, Patient medical status sufficiently stable and ability of patient and parents/legal guardian, in the opinion of the investigator, to adhere to the study visits schedule and other protocol requirements., Seronegative patients for neutralizing antibodies against AAV8

Exclusion Criteria

•Previous participation in gene and cell therapy trials, Inability to cooperate with muscle testing or to perform respiratory function tests, Presence or history of concomitant muscular or other medical condition that might interfere with LGMDR5 evolution or that would confound scientific rigor or interpretation of results, e.g., current infectious episode (pulmonary, ENT, etc.), abnormal laboratory test if clinically significant, Current or history of significant heart, lung, hepato\-biliary or renal disease or impairment that jeopardize the safety of the subject according to the investigator, Acute illness within 4 weeks of the anticipated IMP administration which may interfere with study assessments, Current participation in a clinical trial of another investigational medicinal product, Any condition that would contraindicate treatment with immunosuppressant therapy, Presence of any permanent items (e.g., metal braces) precluding undergoing MRI, Any vaccination 1 month prior to the planned IMP administration, Serology consistent with HIV exposure or active hepatitis B or C infection, Grade 2 or higher lab abnormalities for LFT (except isolated AST increase), bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT, and a PTT, according to current version of CTCAE. Isolated AST increase associated with CK levels \> 800 U/L, ALT \< 2 x ULN, and GLDH within normal range, is not exclusionary., Known hypersensitivity to IMP excipients, to eculizumab, murine proteins, or any excipients in eculizumab formulation, and to contrast media, Cardiomyopathy based on physical and cardiological examination and echocardiography with Left Ventricular Ejection Fraction (LVEF) below 50%, Any respiratory assistance, including non\-invasive daytime or nocturnal ventilation