Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2021-004276-33 | EudraCT Number |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Cohort 1 | Experimental | Cohort 1: single intravenous injection 9.0E+12 vg/Kg |
|
| Cohort 2 | Experimental | Cohort 2: single intravenous injection 2.7E+13 vg/Kg |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ATA-100 (AAV9 encoding FKRP gene) | Biological | Single intravenous infusion on Day 0 |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety and tolerability | Incidence of Adverse Events and significant laboratory changes | Baseline through 12 months |
| Measure | Description | Time Frame |
|---|---|---|
| Combined endpoint (global testing) | Change in North Star Assessment for musculary Dystrophy (NSAD) and Forced Vital capacity (FVC %) from baseline | Baseline through 12 months |
| Timed Up and Go (TUG) test |
Not provided
Inclusion Criteria:
Exclusion Criteria:
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Rigshospitalet, University of Copenhagen Blegdamsvej 9 | Copenhagen | 2100 | Denmark | |||
| Institute of Myology Pitié-Salpêtrière Hospital 47 Bd de l'Hôpital |
Not provided
| ID | Term |
|---|---|
| C564612 | Muscular Dystrophy, Limb-Girdle, Type 2I |
Not provided
Not provided
Not provided
Single IV administration of ATA-100, open label, 2 sequential cohorts
Not provided
Not provided
Not provided
Secondary endpoint
| Baseline through 12 months |
| Change from baseline in velocity as measured by 10MWT | 10-meter walk test | Baseline through 12 months |
| 2-minute walk distance test | Secondary endpoint | Baseline through 12 months |
| Cardiac MRI | To measure cardiac function (left ejection fraction) | Baseline through 12 months |
| Muscle MRI | To measure change from baseline in fat repartition fraction in thigh and leg skeletal muscles | Baseline through 12 months |
| Muscle Biopsy | Quantification of FKRP positive muscle fibers | Baseline through 12 months |
| Muscle Biopsy | Percentage of glycosylation | 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 | 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) | Baseline through 12 months |
| Paris |
| 75013 |
| France |
| Royal Victoria Infirmary Queen Victoria Road Level 6 Leazes Wing | Newcastle upon Tyne | NE1 4LP | United Kingdom |