Phase 1 dose escalation study to assess tolerability and safety of ATA-100 with 5-year follow-up
Multicenter, Phase 1 study evaluating safety, pharmacodynamic, and immunogenicity of ATA-100, an Adeno-Associated Virus (AAV) vector carrying the human FKRP transgene. This study is an open-label dose escalation phase with long-term follow-up (LTFU) period. Two dose cohorts will be enrolled sequentially and enrollment. An initial cohort of three (3) patients will receive a potentially effective dose, followed by a 2nd higher dose cohort of 3 patients. All subjects will be followed for up to 5 years after active IMP (ATA-100) administration.
Study Type
INTERVENTIONAL
Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
6
Single intravenous infusion on Day 0
Rigshospitalet, University of Copenhagen Blegdamsvej 9
Copenhagen, Denmark
Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital
Paris, France
Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing
Newcastle upon Tyne, United Kingdom
Safety and tolerability
Incidence of Adverse Events and significant laboratory changes
Time frame: Baseline through 12 months
Combined endpoint (global testing)
Change in North Star Assessment for musculary Dystrophy (NSAD) and Forced Vital capacity (FVC %) from baseline
Time frame: Baseline through 12 months
Timed Up and Go (TUG) test
Secondary endpoint
Time frame: Baseline through 12 months
Change from baseline in velocity as measured by 10MWT
10-meter walk test
Time frame: Baseline through 12 months
2-minute walk distance test
Secondary endpoint
Time frame: Baseline through 12 months
Cardiac MRI
To measure cardiac function (left ejection fraction)
Time frame: Baseline through 12 months
Muscle MRI
To measure change from baseline in fat repartition fraction in thigh and leg skeletal muscles
Time frame: Baseline through 12 months
Muscle Biopsy
Quantification of FKRP positive muscle fibers
Time frame: Baseline through 12 months
Muscle Biopsy
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Percentage of glycosylation
Time frame: Baseline through 12 months
Patient reported outcome and quality of life assessment
Quality of Life in genetic Neuromuscular Disease (QoL-gNMD), with a range from 0 to 78, the higher the score the worse the quality of life
Time frame: Baseline through 12 months
Patient reported outcome and quality of life assessment
ACTIVLIM, scale measuring level of limitation in performing daily activities (total score ranging from 0 to 44, with the lower score the highest limitation)
Time frame: Baseline through 12 months