Restrictive respiratory syndrome with frequent pulmonary infections and cardiomyopathy. wasting of skeletal muscle, severe local inflammation and, at least initially, muscle regeneration. MedDRA version: 17.0 Level: PT Classification code 10064571 Term: Gene mutation System Organ Class: 10010331 - Congenital, familial and genetic disorders
Conditions
Interventions
Trade Name: Isosorbide Dinitrate 20 mg
Product Name: Isosorbide Dinitrate
Pharmaceutical Form: Tablet
INN or Proposed INN: ISOSORBIDE DINITRATE
CAS Number: 87-33-2
Pharmaceutical form of the placebo:
Sponsors
PARENT PROJECT ONLUS
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. 1. Patient written informed assent and parents/guardians written informed consent: provision of consent to participate in the study as shown by the signature on the Volunteer Consent Form after verbal and written information 2. Confirmed diagnosis of Duchenne muscular dystrophy (muscle biopsy, genetic and biochemical analysis) 3. Age =10 years and =65 years) no F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: 1. Any acute co-morbid condition interfering with the well-being of the patient within 7 days of enrolment (including bacterial infections, viral infectious processes, food poisoning, temperature > 37° C). Patients will be excluded from enrolment until the infection had been appropriately treated and resolved; 2. any gastrointestinal conditions which, in the opinion of the Investigator, may interfere with the absorption of the drug or render the subjects unable to take oral medication (gastric ulcer, peptic ulcer, stomach acid, frequent diarrhoea, gastrointestinal surgery); 3. a left ventricular ejection fraction (EF) of < 30%; 4. forced vital capacity (FVC) < 40% of predicted; 5. history of recurrent headache; 6. history or presence of allergy or intolerance to the study drugs or their components or drugs of their class, or a history of drug or other allergy that, in the opinion of the responsible physician, contraindicates their participation; 7. inability to take tablets, as assessed by the Investigator by the end of the screening period; 8. previous or ongoing medical conditions, medical history, physical findings or laboratory abnormalities that could affect safety, make it unlikely that treatment and follow up will be correctly completed or impair the assessment of study results, in the judgment of the Investigator; 9. weight of less than 13 kilograms; 10. participation in any other investigational drug or therapy study within the previous 6 months.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To assess the efficacy of the combination of ibuprofen and isosorbide dinitrate (ISDN) in delaying the worsening of the muscular motor function in patients with Duchenne muscular dystrophy assessed by the Performance of Upper Limb (PUL) scale. ;Secondary Objective: To assess the safety and tolerability of the combination of ibuprofen and ISDN in Duchenne muscular dystrophy affected patients. To assess the efficacy of the combination of ibuprofen and ISDN assessed by other muscular, cardiac and pulmonary measures of functionality and also its impact on quality of life ;Primary end point(s): The primary outcome will be the mean change (%) from baseline to the end of the study in the MFM score;Timepoint(s) of evaluation of this end point: Treatment period: 18 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary outcomes include other muscular measures, cardiac and pulmonary function and Quality of Life. ;Timepoint(s) of evaluation of this end point: 18 months | — |
Countries
Italy
Contacts
Public ContactPaola Vietti
TFS Trial Form Support S.r.l
Outcome results
None listed