A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy

NCT ID: NCT06169046

Last Updated: 2024-04-15

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 90 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2024-04-13
• Study Completion Date: 2027-12-31

Brief Summary

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

Spinal and Bulbar Muscular Atrophy

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

clenbuterole

45 patients will receive Clenbuterol at a final dosage of 0.04 mg/day, treated for a total of 48 weeks

Group Type: EXPERIMENTAL

Clenbuterol

Intervention Type: DRUG

tablets

placebo

45 patients will receive placebo, treated for a total of 48 weeks

Group Type: PLACEBO_COMPARATOR

Placebo

Intervention Type: DRUG

tablets

Interventions

Clenbuterol

tablets

Intervention Type: DRUG

Placebo

tablets

Intervention Type: DRUG

Other Intervention Names

active

Eligibility 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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Mario Negri Institute for Pharmacological Research

OTHER

Sponsor Role: collaborator

Gianni Soraru

OTHER

Sponsor Role: lead

Responsible Party

Gianni Soraru

Professor

Responsibility Role: SPONSOR_INVESTIGATOR

Principal Investigators

Gianni Sorarù, MD

Role: PRINCIPAL_INVESTIGATOR

Azienda Ospedale Università di Padova

Locations

Azienda Ospedale Università di Padova

Padua, PD, Italy

Site Status: RECRUITING

Countries

Italy

Central Contacts

Gianni Sorarù, MD

Role: CONTACT

gianni.soraru@unipd.it

0498213600

Elisabetta Pupillo, PharmD

Role: CONTACT

elisabetta.pupillo@marionegri.it

0239014605

Facility Contacts

Gianni Sorarù

Role: primary

gianni.soraru@unipd.it

3478889974

Other Identifiers

EudraCT: 2017-005103-27

Identifier Type: -

Identifier Source: org_study_id