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Note: This record shows only 22 elements of the WHO Trial Registration Data Set. To view changes that have been made to the source record, or for additional information about this trial, click on the URL below to go to the source record in the primary register.
Register: EUCTR
Last refreshed on: 19 March 2012
Main ID:  EUCTR2011-000176-33-IT
Date of registration: 23/02/2011
Prospective Registration: Yes
Primary sponsor: FONDAZIONE CENTRO S. RAFFAELE DEL MONTE TABOR
Public title: Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - ND
Scientific title: Cell Therapy Of Duchenne Muscular Dystrophy by intra-arterial delivery of HLA-identical allogeneic mesoangioblasts - ND
Date of first enrolment: 14/02/2011
Target sample size:
Recruitment status: Authorised-recruitment may be ongoing or finished
URL:  https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2011-000176-33
Study type:  Interventional clinical trial of medicinal product
Study design:  Controlled: no Randomised: no Open: no Single blind: no Double blind: no Parallel group: no Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: Placebo: Other:  
Phase: 
Countries of recruitment
Italy
Contacts
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Key inclusion & exclusion 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:
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.


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes
Health Condition(s) or Problem(s) studied
Duchenne Muscolar Dystrophy
MedDRA version: 9.1 Level: PT Classification code 10013801
Intervention(s)

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
Pharmaceutical Form: Suspension for injection
Other descriptive name: HLA-identical allogeneic mesoangioblasts
Concentration unit: % percent
Concentration type: equal
Concentration number: 1-

Product Name: hMABs
Pharmaceutical Form: Suspension for injection
Other descriptive name: HLA-identical allogeneic mesoangioblasts
Concentration unit: % percent
Concentration type: equal
Concentration number: 1-

Product Name: hMABs
Pharmaceutical Form: Suspension for injection
Other descriptive name: HLA-identical allogeneic mesoangioblasts
Concentration unit: % percent
Concentration type: equal
Concentration number: 1-

Primary Outcome(s)
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).
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.
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).
Secondary Outcome(s)
Secondary ID(s)
DMD03
Source(s) of Monetary Support
Secondary Sponsor(s)
Ethics review
Results
Results available:
Date Posted:
Date Completed:
URL:
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