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Effect of Modulating the nNOS System on Cardiac, Muscular and Cognitive Function in Becker Muscular Dystrophy Patients

NCT01350154 · Status: COMPLETED · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 17

Last updated 2013-04-10

No results posted yet for this study

Summary

This study is done to evaluate whether treatment with the drug sildenafil (Revatio®) can improve muscular, cardiac, cerebrovascular or cognitive function in patients with Beckers muscular dystrophy when compared to placebo (inactive medication). The study is based on the recent findings of an improved cardiac function in a mouse model of muscular dystrophy (Adamo et al 2010) and the previous findings of changed cognitive function in people with Becker dystrophy.

In muscular dystrophy, the cellular protein, dystrophin is affected. During normal conditions, the enzyme neuronal nitric oxide synthase (nNOS), which produce nitric oxide (NO), is attached to dystrophin. NO is important in normal vascular function in each of muscle, heart and brain by stimulating production of cyclic GMP. However, in muscular dystrophy with dystrophin deficiency, nNOS do not have the normal cellular anchor, resulting in decreased NO levels and subsequent reduced cyclic GMP production. Sildenafil inhibits degradation of cGMP thus prolonging and increasing a cGMP response. Such effects are the basis for use of sildenafil in pulmonary hypertension and erectile dysfunction. Current hypothesis: Sildenafil restores the cyclic GMP function affected in muscular dystrophy wit nNOS deficiency resulting in improved muscle, cardiac, cerebrovascular and cognitive function.

Conditions

  • Becker Muscular Dystrophy

Interventions

DRUG

Sildenafil

20 mg in gelatine capsules, oral, three times daily

DRUG

Placebo

Lactose monohydrate oral in gelatine capsules, 3 times daily

Sponsors & Collaborators

  • Glostrup University Hospital, Copenhagen

    collaborator OTHER

  • Rigshospitalet, Denmark

    lead OTHER

Principal Investigators

  • John Vissing, MD, DMSci · Neuromuscular Clinic and Research Unit, Dept. Neurology, Rigshospitalet

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
CROSSOVER

Eligibility

Min Age
18 Years
Max Age
80 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2011-11-30
Primary Completion
2013-04-30
Completion
2013-04-30

Countries

  • Denmark

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT01350154 on ClinicalTrials.gov