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ATA-100 gene therapy trial in patients with limb-girdle muscular dystrophy
EU CTIS ID: 2023-506677-36-00
What this study is testing
To assess the safety and tolerability of intravenous administration of ATA-100 in ambulant patients with LGMDR9 at two different dosage levels and to select the recommended dose for future studies
- Phase I and Phase II (Integrated)- First administration to humans
A plain-language read of the study's public EU CTIS listing. The study team confirms the details.
Who can take part
You may be able to join if
- Ambulant male or female patients at least 16 years old
- Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
- Able to: i) Perform the 10-meter walk test (10MWT) within 30 sec with unilateral help, such as cane, or bilateral help, such as elbow crutches or orthotic devices below the knees ii) Rise from a standard-height chair with or without arm support
- Diaphragmatic muscle impairment defined as forced vital capacity (FVC) between 40 and 80% (inclusive) of the expected value
- Effective contraception
- Signed written informed consent before any study related procedure is performed
You likely can't join if
- Detectable serum neutralizing antibodies against AAV9
- Current participation in a clinical trial of another investigational medicinal product
- Previous participation in gene and cell therapy trials
- 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
See the full eligibility criteria
- Ambulant male or female patients at least 16 years old
- Documented LGMDR9 diagnosis based on clinical presentation and genotyping confirming the FKRP gene mutations
- Able to: i) Perform the 10-meter walk test (10MWT) within 30 sec with unilateral help, such as cane, or bilateral help, such as elbow crutches or orthotic devices below the knees ii) Rise from a standard-height chair with or without arm support
- Diaphragmatic muscle impairment defined as forced vital capacity (FVC) between 40 and 80% (inclusive) of the expected value
- Effective contraception
- 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 visit schedule and other protocol requirements.
- Detectable serum neutralizing antibodies against AAV9
- Current participation in a clinical trial of another investigational medicinal product
- Previous participation in gene and cell therapy trials
- 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, bilirubin, creatinine, hemoglobin, WBC count, platelet count, PT, and a PTT, according to current version of CTCAE.
- Known hypersensitivity to IMP excipients, to eculizumab, murine proteins, or any excipients in eculizumab formulation
- 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
- 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 LGMDR9 evolution or that would confound scientific rigor or interpretation of results, e.g., current infectious episode (pulmonary, ENT,...), abnormal laboratory test if clinically significant
- Acute illness within 4 weeks of the anticipated IMP administration which may interfere with study assessments
- Recent immunosuppressive treatment within 3 months prior to screening
- 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
The study team makes the final eligibility decision.
Where it's taking place
- United Kingdom
Questions & answers
Do participants get paid in this trial?
This listing doesn't specify compensation. Many trials still reimburse travel or offer a stipend, so it's worth asking the study team when you connect.
Is it free to join, and do I need insurance?
Searching and applying through BridgeMD is free. In clinical trials the study-related treatment and visits are generally provided at no cost to you. You usually don't need insurance to take part - confirm specifics with the study team.
How long does this study last?
The listing doesn't state an exact length. The study team walks you through the schedule and number of visits before you decide to enroll.
Who can join this trial?
This study is enrolling all sexes, 18-64 years, 65+ years, 0-17 years. The study team makes the final eligibility decision.
Where is this trial taking place?
Study sites include United Kingdom. Enter your location above to see the nearest site and check your eligibility.
Explore other conditions
BridgeMD is an information and trial-matching tool - not medical advice, and not the study sponsor. Details come from EU CTIS; the study team decides eligibility.