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Clinical investigation in Duchenne dystrophy. VI. Double-blind controlled trial of nifedipine
Abstract:
Excessive accumulation of intracellular calcium in Duchenne muscular dystrophy (DMD) may be a necessary step in the process that causes muscle damage in this disease. Because of this possibility, a controlled trial of the calcium channel blocking agent nifedipine was undertaken. One hundred and five patients were randomized and treated in a double-blind manner for 18 months. Muscle strength, contractures, functional ability, cardiopulmonary changes, and laboratory data were monitored. The dose of nifedipine was 0.75-1 mg/kg/day in the first 6 months and 1.5-2 mg/kg/day for the next 12 months. Satisfactory blood levels of nifedipine were attained. The study had a power greater than 0.99 to detect a slowing of the illness to 25% of its original rate of progression. No significant improvement was demonstrated in the treated group. One or more of the frequent mild side effects of flushing, dizziness, and leg edema, often associated with the use of nifedipine in adults, occurred transiently in approximately one-half of the patients in the nifedipine group and in 21% of the placebo group. Four patients died, two on nifedipine and two on placebo. This study demonstrates that nifedipine is safe to administer in children, but that it is without beneficial effect on the course of DMD.
Insights
Nifedipine, a calcium channel blocker, did not improve muscle strength or slow disease progression in Duchenne muscular dystrophy (DMD) patients over 18 months. The drug was safe but ineffective for treating DMD.
Area of Science:
- Neurology
- Cardiovascular Pharmacology
Background:
- Intracellular calcium accumulation is implicated in Duchenne muscular dystrophy (DMD) pathogenesis.
- Calcium channel blockers may mitigate this cellular damage.
Purpose of the Study:
- To evaluate the efficacy and safety of nifedipine in treating Duchenne muscular dystrophy.
- To determine if nifedipine can slow disease progression in pediatric DMD patients.
Main Methods:
- A double-blind, randomized controlled trial involving 105 pediatric patients with DMD.
- Patients received nifedipine or placebo for 18 months, with escalating doses.
- Monitoring included muscle strength, contractures, functional ability, and cardiopulmonary parameters.
Main Results:
- Nifedipine treatment did not demonstrate significant improvement in muscle strength or functional ability compared to placebo.
- The study achieved high statistical power to detect even a 25% slowing of disease progression.
- Mild side effects like flushing and dizziness were observed, with transient occurrences in approximately 50% of the nifedipine group.
Conclusions:
- Nifedipine is safe for administration in children with Duchenne muscular dystrophy.
- Nifedipine does not offer beneficial effects on the clinical course or progression of DMD.