Actively Recruiting
A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
Led by Gianni Soraru · Updated on 2024-04-15
90
Participants Needed
1
Research Sites
26 weeks
Total Duration
On this page
Sponsors
G
Gianni Soraru
Lead Sponsor
M
Mario Negri Institute for Pharmacological Research
Collaborating Sponsor
AI-Summary
What this Trial Is About
Spinal and Bulbar Muscular Atrophy (SBMA) is a rare condition with no current cure to stop or slow its progression. Researchers are studying the potential of beta2-agonist drugs, specifically clenbuterol, to improve muscle function in people with SBMA. This phase II, multicenter, randomized, double-blind, placebo-controlled trial aims to evaluate the safety and effectiveness of clenbuterol for this condition, addressing concerns from earlier small-scale studies. In this trial, 90 participants with genetically confirmed SBMA will be randomly assigned to one of two groups. One group will receive clenbuterol tablets at a dose of 0.04 mg per day, while the other group will receive placebo tablets. Both groups will be treated for 48 weeks under a quadruple-blind design to compare the effects of clenbuterol against placebo. Participants will be involved for 48 weeks during which their motor function and quality of life will be closely monitored. Assessments include a 6-minute walk test, SBMA-FRS scale, Adult Myopathy Assessment Tool, lung function tests, serum creatinine levels, and neuromuscular quality of life questionnaires. Safety and efficacy data collected will help determine the potential benefits and risks of clenbuterol in SBMA patients.
CONDITIONS
Brief Title
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Males with genetically confirmed diagnosis of SBMA (AR CAG repeat number >= 38)
- Aged between 18 and 75 years
- Showing one or more clinical symptoms: muscle atrophy, limb weakness, bulbar palsy
- Able to walk independently with or without a cane or other supporting device (wheelchair use not allowed)
- Providing written informed consent
You will not qualify if you...
- Cardiovascular disease preventing use of beta2 agonists as decided by investigators
- Glaucoma, severe prostatic hypertrophy, hyperthyroidism, pheochromocytoma, or other conditions posing risk or preventing study completion
- Concurrent treatment with beta-blockers or sympathomimetic drugs (must discontinue beta-blockers 3 weeks before randomization)
- Unable to walk or only able to walk with caregiver support
- Use of beta2 agonists in the last 6 months
- Participation in another interventional trial in the last 3 months
- Having neuromuscular diseases other than SBMA
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - 48 weeks
Participants receive either Clenbuterol or a placebo daily and are monitored for efficacy and safety.
Regular visits during treatment period
Trial Site Locations
Total: 1 location
1
Azienda Ospedale Università di Padova
Padova, PD, Italy, 35134
Actively Recruiting
Research Team
G
Gianni Sorarù, MD
E
Elisabetta Pupillo, PharmD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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