Study within children with Duchenne Muscular Dystrophy

• Register: EUCTR
• Last refreshed on: 25 January 2016
• Main ID: EUCTR2009-009871-36-DE
• Date of registration: 06/08/2009
• Prospective Registration: Yes
• Primary sponsor: Friedrich- Alexander- Universität Erlangen Nürnberg
• Public title: Study within children with Duchenne Muscular Dystrophy
• Scientific title: Effect and Safety of preventive Treatment with ACE-Inhibitor and Beta-Blocker on the onset of Left Ventricular Dysfunction in Duchenne Muscular Dystrophy - DMD-Kardio
• Recruitment status: Not Recruiting
• URL: https://www.clinicaltrialsregister.eu/ctr-search/search?query=eudract_number:2009-009871-36
• Study type: Interventional clinical trial of medicinal product
• Study design: Controlled: yes Randomised: yes Open: no Single blind: no Double blind: yes Parallel group: yes Cross over: no Other: no If controlled, specify comparator, Other Medicinial Product: no Placebo: yes Other: no Number of treatment arms in the trial: 2

Countries of recruitment

Germany

Contacts

• Name: LKP
• Affiliation: Universitätsklinikum Erlangen, Kinderkardiologie

• Name: LKP
• Affiliation: Universitätsklinikum Erlangen, Kinderkardiologie

Key inclusion & exclusion criteria

Inclusion criteria:
Male patients with DMD (confirmed by genetic analysis or muscle biopsy) • aged 10 – 14 years • Normal left ventricular function (fraction shortening = 30%) • Normal renal function (GFR > 30 ml/min/1,73 m2) • Informed Consent of both parents, assent of patient

Are the trial subjects under 18? yes
Number of subjects for this age range: 55
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:
Dilated left ventricle (size > 97. Percentile) or Left Ventricular Dysfunction (fraction shortening <30%) • contraindications for ACE-Inhibitors/Beta-Blocker • previous therapy with ACE-Inhibitors, AT II Antagonists, Beta-Blockers • Other medical reasons which render participation not reasonable for the patient


Age minimum:
Age maximum:
Gender:
Female: no
Male: yes

Health Condition(s) or Problem(s) studied

Therapeutic area: Diseases [C] - Musculoskeletal Diseases [C05]

Intervention(s)

Trade Name: EnalHexal®, 5 mg
Pharmaceutical Form: Tablet
INN or Proposed INN: ENALAPRIL
CAS Number: 75847733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Trade Name: EnaHexal®, 10mg
Pharmaceutical Form: Tablet
INN or Proposed INN: ENALAPRIL
CAS Number: 75847733
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 10-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Trade Name: MetoHEXAL® Succ ® 23,75 mg Retardtabletten
Pharmaceutical Form: Tablet
INN or Proposed INN: METOPROLOL SUCCINATE
CAS Number: 98418474
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 23.75-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Trade Name: MetoHEXAL® Succ ® 47,5 mg Retardtabletten
Pharmaceutical Form: Tablet
INN or Proposed INN: METOPROLOL SUCCINATE
CAS Number: 98418474
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 47.5-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Trade Name: MetoHEXAL® Succ ® 95 mg Retardtabletten
Pharmaceutical Form: Tablet
INN or Proposed INN: METOPROLOL SUCCINATE
CAS Number: 98418474
Concentration unit: mg milligram(s)
Concentration type: equal
Concentration number: 95-
Pharmaceutical form of the placebo: Tablet
Route of administration of the placebo: Oral use

Primary Outcome(s)

Main Objective: Efficacy of prophylactic anticongestive therapy: time from randomisation to left ventricular dysfunction, defined as left-ventricular shortening fraction <28%(6 monthly control visits)

Primary end point(s): efficacy of prophylactic anticongestive therapy, time from randomisation to left ventricular dysfunction,defined as left-ventricular shortening fraction < 28% (6 monthly control visits)

Secondary Objective: - Comparison of safety and tolerability
- Comparison of quality of life (Kindl questionnaire12-16)
- Comparison of diagnostics: Tissue Doppler vs 2D Echocardiography
- Comparison of total survival in both arms
- Comparison of pathophysiological changes in neuromuscular regulation (neurohumeral parameters like pro- BNP, Angiotensin II, Noradrenalin)
- Comparison of drop of heart frequency rate and heart frequency variability (24h ECG, Tissue Doppler)

Timepoint(s) of evaluation of this end point: from baseline R4 until study end

Secondary Outcome(s)

Secondary end point(s): comparison of safety and tolerability
comparison of quality of life (Kindl)
comparison of diagnostics tissue doppler vs 2 D Echo
comparison of pro-BNP, Angiotensin II, Noradrenalin

Timepoint(s) of evaluation of this end point: change of LVSF from baseline

Secondary ID(s)

140/540

Source(s) of Monetary Support

Universitätsklinikum Erlangen