Duchenne Muscolar Dystrophy MedDRA version: 9.1 Level: PT Classification code 10013801
Conditions
Interventions
Pharmaceutical Form: Capsule, hard
INN or Proposed INN: Tacrolimus
Concentration unit: mg/kg milligram(s)/kilogram
Concentration type: equal
Concentration number: 1-
Product Name: hMABs
Pharmaceutica
Sponsors
FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Age between 6 and 14 years at time of study entry. 2. Completion of clinical trial DMD01 (“Outcome measures validation study for children affected by Duchenne Muscular Dystrophy”). 3. Availability of HLA identical donor MABS previously collected in good number and quality as specified in a separate clinical protocol (DMD02). 4. Written informed consent of caregivers of DMD patients and patient’s assent. Are the trial subjects under 18? yes Number of subjects for this age range: F.1.2 Adults (18-64 years) no F.1.2.1 Number of subjects for this age range F.1.3 Elderly (>=65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Positive hepatitis B surface antigen, hepatitis C antibody test, or human immunodeficiency virus (HIV) test. 2. Presence of immune deficiency, neoplastic or autoimmune disease (based on clinical history). 3. Bleeding disorder. 4. Any known allergies to products likely to be used in the study. 5. Prior or ongoing medical condition (e.g. concomitant illness, psychiatric condition, behavioural disorder, drug abuse), medical history, physical findings, or laboratory abnormality that, in the investigator’s opinion, could adversely affect the safety of the subject, makes it unlikely that the course of treatment or follow-up would be completed, or could impair the assessment of study results. 6. Ongoing participation in any other therapeutic clinical trial (use of steroids will be considered standard care and therefore permitted). 7. If abnormal heart US: LVEF (ventricular left ventricular ejection fraction) 100?). 11. Presence of significant impairment of renal or hepatic function defined as serum creatinine =1.5 ? upper limit of normal (ULN), serum bilirubin =1.5 ? ULN.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Safety: To assess the incidence of adverse events in DMD patients treated with intra-arterial injections of allo-MABS from an HLA-identical family donor during immunosuppressive treatment with FK506 (tacrolimus). Efficacy: To determine the effect of multiple intra-arterial injections of allo-MABS from an HLA-identical family donor in modifying muscle strength in DMD patients during immunosuppressive treatment with FK506 (tacrolimus).;Secondary Objective: Safety To assess the long term incidence of adverse events (any grade) in DMD patients treated with intra-arterial injections of allo-MABS during immunosuppressive treatment with FK506 (tacrolimus). Efficacy. To determine the ability of allo-MABS from an HLA-identical family donor injected intra-arterially during immunosuppressive therapy with FK506 (tacrolimus) to give rise to dystrophin positive muscle fibers, ameliorate muscle architecture. To evaluate engraftment of allo-MABS in treated DMD patients during and after immunosuppressive therapy with FK506 (tacrolimus).;Primary end point(s): Safety. Acceptable incidence and severity of local and systemic adverse events (tolerated grade I-II) in DMD patients treated with intra-arterial injections of allo-MABS during immunosuppressive treatment with FK506 (tacrolimus) up to 1 year from the first infusion. Efficacy. Improvement or stabilization of muscle strength in DMD patients treated with intra-arterial injections of allo-MABS during immunosuppressive treatment with FK506 (tacrolimus) evaluated 1 year from the first infusion. | — |
Countries
Italy
Outcome results
None listed