NCT06169046RecruitingPhase 2
A Placebo-controlled Study of Clenbuterol in 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)
Source: ClinicalTrials.gov · Last updated 15 Apr 2024 · Not updated for over 12 months - ask the site whether it is still open
Brief summary (registry, in English)
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.
- Status (registry)
- Recruiting
- Phase (registry)
- Phase 2
- Study type
- Interventional
- Enrollment
- 90 (estimated)
- First submitted
- 5 Dec 2023
- Start date
- 13 Apr 2024
- Primary completion date
- 30 Jun 2027
- Lead sponsor
- Gianni Soraru
- NCT number
- NCT06169046
- EU CT number
- 2024-516563-86-00
- EudraCT number
- 2017-005103-27
What you commit to
- Study type: Interventional · Phase 2
- Planned participants: 90
- From 13 Apr 2024 to the primary completion date, 30 Jun 2027
- 7 sites in 1 country
- Visits and tests: ask the trial team, the registry does not list them
Built from the registry fields of ClinicalTrials.gov. It describes the study, not your situation.
Who cannot take part
Copied verbatim from ClinicalTrials.gov and grouped by topic. Only the trial team decides who can take part.
Prior treatment
- 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);
Other illness
- a documented cardiovascular disease precluding the use of beta2 agonists (in the judgment of the investigators);
- 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;
- neuromuscular disease other than SBMA.
Other
- inability to walk or walking only with the support of a caregiver;
- use of beta2 agonists in the preceding 6 months;
- participation to an interventional trial in the preceding 3 months;
Who the study is looking for
- males who have received a genetically confirmed diagnosis of SBMA (AR CAG repeat number \>= 38);
- aged between 18 and 75 (+364 days) years;
- displaying one or more of the following clinical symptoms: muscle atrophy, limb weakness, bulbar palsy;
- able to walk independently with or without a cane or other supporting device (all supporting devices are acceptable except on wheelchair);
- providing a written informed consent.
Age in the registry: 18 years to 75 years · Sex: All · Does not accept healthy volunteers
Sites (7)
Sites in registry order. Pick your city on the disease page to sort them by distance.
| Site | City | Country | Site status |
|---|---|---|---|
| IRCCS Foundation Istituto Neurologico Carlo Besta Coordinator of this site? Confirm its status | Milan | Italy | Active, not recruiting |
| AOU di Modena Nuovo Ospedale Civile S. Agostino Estense di Modena - Ospedale di Baggiovara Coordinator of this site? Confirm its status | Modena | Italy | Active, not recruiting |
| Azienda Ospedaliero- Universitaria Maggiore della Carità di Novara Clinica Neurologica Centro SLA Coordinator of this site? Confirm its status | Novara | Italy | Active, not recruiting |
| Azienda Ospedaliera di Padova Coordinator of this site? Confirm its status | Padova | Italy | Active, not recruiting |
| Azienda Ospedaliero Universitaria Pisana Coordinator of this site? Confirm its status | Pisa | Italy | Active, not recruiting |
| Fondazione Policlinico Universitario Agostino Gemelli IRCCS Coordinator of this site? Confirm its status | Rome | Italy | Active, not recruiting |
| Azienda Ospedaliero Universitaria Città della Salute e della Scienza di Torino Dipartimento di Neur Coordinator of this site? Confirm its status | Torino | Italy | Active, not recruiting |
This page is information, not medical advice. Discuss any trial with your doctor.