Actively Recruiting
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
Led by Gianni Soraru · Updated on 2024-04-15
90
Participants Needed
1
Research Sites
193 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
There is no cure to arrest or delay SBMA progression. It is estimated that \~1000 individuals are affected by SBMA in Italy at any given time (prevalence: 1.5/100000) with an annual incidence of 0.19/100000 males. Here, we are going to test the potential of beta2-agonist stimulation on muscle as a therapeutic avenue for SBMA. We have provided pre-clinical evidence that β-agonist stimulation may be a therapeutic strategy for SBMA. Moreover, we have shown that beta2-agonists are effective in improving motor function without relevant adverse events in a small cohort of SBMA patients. To establish safety and efficacy of clenbuterol as a cure for SBMA, we are conducting a multicenter, phase II, randomized, double-blind, parallel-group, single dose, placebo-controlled trial. Indeed, based on our preliminary data, some concerns remain to be addressed.
CONDITIONS
Official 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 a genetically confirmed diagnosis of SBMA (AR CAG repeat number ≥ 38)
- Aged between 18 and 75 years
- Showing one or more symptoms: muscle atrophy, limb weakness, or bulbar palsy
- Able to walk independently with or without a cane or other supporting device (excluding wheelchairs)
- Able to provide written informed consent
You will not qualify if you...
- Having cardiovascular disease that prevents use of beta2 agonists as judged by investigators
- Diagnosed with glaucoma, severe prostatic hypertrophy, hyperthyroidism, pheochromocytoma, or other conditions increasing risk or preventing study completion
- Currently treated with beta-blockers or sympathomimetic drugs (must stop beta-blockers 3 weeks before randomization if used)
- Unable to walk or requiring caregiver support to walk
- Used beta2 agonists in the past 6 months
- Participated in another interventional trial within 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
Trial Site Locations
Total: 1 location
1
Azienda Ospedale Università di Padova
Padova, PD, Italy, 35134
Actively Recruiting
Research Team
G
Gianni Sorarù, MD
CONTACT
E
Elisabetta Pupillo, PharmD
CONTACT
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
Not the Right Trial for You?
Explore thousands of other clinical trials that might be a better match.
Sign up to get personalized trial recommendations delivered to your inbox.
Already have an account? Log in here