Testing clenbuterol for treating spinal and bulbar muscular atrophy
A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
This study is testing if the drug clenbuterol can help men with spinal and bulbar muscular atrophy feel stronger and improve their muscle function.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 90 (estimated) |
| Ages | 18 Years to 75 Years |
| Sex | All |
| Sponsor | University Hospital Padova Academic / other |
| Locations | 1 site (Padova, PD) |
| Trial ID | NCT06169046 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the safety and efficacy of clenbuterol, a beta2-agonist, in patients diagnosed with spinal and bulbar muscular atrophy (SBMA). The study is a multicenter, phase II, randomized, double-blind, placebo-controlled trial involving male participants aged 18 to 75 who exhibit symptoms of muscle atrophy or weakness. The trial will assess the potential of clenbuterol to improve motor function and address concerns raised in preliminary studies. Participants will receive either clenbuterol or a placebo in a controlled setting to determine the treatment's effectiveness.
Who should consider this trial
Good fit: Ideal candidates are males aged 18 to 75 with a genetically confirmed diagnosis of SBMA and specific clinical symptoms.
Not a fit: Patients with cardiovascular diseases or other medical conditions that contraindicate the use of beta2-agonists may not benefit from this study.
Why it matters
Potential benefit: If successful, this treatment could provide a new therapeutic option to improve motor function in patients with SBMA.
How similar studies have performed: Preliminary studies have shown some success with beta2-agonists in improving motor function in SBMA patients, indicating potential for this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: 1. males who have received a genetically confirmed diagnosis of SBMA (AR CAG repeat number \>= 38); 2. aged between 18 and 75 (+364 days) years; 3. displaying one or more of the following clinical symptoms: muscle atrophy, limb weakness, bulbar palsy; 4. able to walk independently with or without a cane or other supporting device (all supporting devices are acceptable except on wheelchair); 5. providing a written informed consent. Exclusion Criteria: 1. a documented cardiovascular disease precluding the use of beta2 agonists (in the judgment of the investigators); 2. glaucoma, severe prostatic hypertrophy, hyperthyroidism, pheochromocytoma, and other medical conditions that, in the judgment of the investigators, would expose the patient to undue risk of harm or prevent the patient from completing the study; 3. concomitant treatment with either beta-blockers or sympathomimetic drugs (If a beta-blockers concomitant medication is ongoing before the study inclusion, the patient can be enrolled if the beta-blocker is discontinued for 3 weeks prior to randomization visit); 4. inability to walk or walking only with the support of a caregiver; 5. use of beta2 agonists in the preceding 6 months; 6. participation to an interventional trial in the preceding 3 months; 7. neuromuscular disease other than SBMA.
Where this trial is running
Padova, PD
- Azienda Ospedale Università di Padova — Padova, Pd, Italy (Recruiting)
Study contacts
- Principal investigator: Gianni Sorarù, MD — Azienda Ospedale Università di Padova
- Study coordinator: Gianni Sorarù, MD
- Email: gianni.soraru@unipd.it
- Phone: 0498213600
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.