Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - ND

• Conditions: Duchenne Muscolar Dystrophy; MedDRA version: 9.1Level: PTClassification code 10013801

• Registration Number: EUCTR2011-000176-33-IT
• Lead Sponsor: FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Study Start: 2011-02-23
• Last Update Posted: 19 March 2012

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: Male
• Target Recruitment: Not specified

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

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) < 45% or ECG finding significant for underlying cardiac impairment. 8. Pulmonary function tests assessed by spirometry (if cooperative) of FEV1 and FVC <50% of the predicted values. If non cooperative, pulse oximetry < 95 % in room air. 9. Change of medication related to DMD within last 3 months with the exception of adjustment based on weight gain of current medications. 10. Presence of severe scoliosis (curve >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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified