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

Spinal and Bulbar Muscular Atrophy Clinical Trial

— BetaSBMA  

Official title:

A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT06169046
• Other study ID #: EudraCT: 2017-005103-27
• Status: Recruiting
• Phase: Phase 2
• Start date: April 13, 2024
• Est. completion date: December 31, 2027

Study information

• Verified date: April 2024
• Source: University Hospital Padova
• Contact: Gianni Sorarù, MD
• Phone: 0498213600
• Email: gianni.soraru[@]unipd.it
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 90
• Est. completion date: December 31, 2027
• Est. primary completion date: June 30, 2027
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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.

Related Conditions & MeSH terms

• Atrophy
• Bulbo-Spinal Atrophy, X-Linked
• Muscular Atrophy
• Spinal and Bulbar Muscular Atrophy

Intervention

Drug:
• Clenbuterol
tablets
• Placebo
tablets

Locations

Country	Name	City	State
Italy	Azienda Ospedale Università di Padova	Padova	PD

Sponsors (2)

Lead Sponsor	Collaborator
Gianni Soraru	Mario Negri Institute for Pharmacological Research

Country where clinical trial is conducted

Italy, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	6-minute-walk test	to assess the efficacy of chronic treatment with clenbuterol in patients with SBMA by the 6MW test	48 weeks
Secondary	SBMA-FRS scale	SBMA-FRS total score during the 12-months treatment period (from V2 to V7) in the two treatment arms	48 weeks
Secondary	Adult Myopathy Assessment Tool (AMAT)	AMAT total score during the 12-months treatment period (from V2 to V7) in the two treatment arms	48 weeks
Secondary	FVC	FVC during the 12-months treatment period (from V2 to V7) in the two treatment arms	48 weeks
Secondary	6K scale	6K total score during the 12-months treatment period (from V2 to V7) in the two treatment arms	48 weeks
Secondary	Serum creatinine levels	serum creatinine levels during the 12-months treatment period (from V2 to V7) in the two treatment arms	48 weeks
Secondary	ALSAQ-40	ALSAQ-40 total score during the 12-months treatment period (from V2 to V7) in the two treatment arms	48 weeks
Secondary	individualized neuromuscular quality of life (INQoL) questionnaire	INQOL total score during the 12-months treatment period (from V2 to V7) in the two treatment arms	48 weeks