Mesoangioblast-based gene therapy for Duchenne Muscular Dystrophy: a phase I/IIa study / DMD04

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: Not specified

Target Recruitment: 5

Inclusion Criteria

1.Age between 12 and 16 years at time of study entry, provided that participants matching the eligibility criteria can be identified. Otherwise patients of progressively younger age may be recruited up to a minimum age of 8 years.
2.Non-ambulant at the time of recruitment.
3.Confirmed diagnosis of DMD with documented exon 51 skippable mutations in dystrophin gene.
4.Progression of muscle degeneration = to 50% reduction of muscle mass as determined by quantitative MRI (grade 2: Kinali et al. 2011).
5.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: 5
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range 0
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

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, making 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 or treatment with exon skipping oligonucleotides. Use of steroids is considered standard care, as well as concomitant medications (e.g. B blockers, ACEI, ARBs, vitamin D, bisphosphonates) and therefore permitted.
7.FS (fraction shortening) < 28% or ECG finding significant for underlying cardiac impairment.
8.Pulmonary function tests assessed by spirometry (if cooperative) of FEV1 and FVC <30% of the predicted values. If unable, 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 >50°).
11.Presence of significant impairment of renal or hepatic function.