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

NCT06169046 | Recruiting Phase 2 DMC

• 1 other identifier: EudraCT: 2017-005103-27
• Study Type: interventional
• Target: 90
• Locations: 1 country
• Sites: 1

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

Keywords

SBMA; clenbuterol; trial

Outcome Measures

Primary Outcomes (1)

• 6-minute-walk test

  to assess the efficacy of chronic treatment with clenbuterol in patients with SBMA by the 6MW test

  48 weeks

Secondary Outcomes (7)

• SBMA-FRS scale

  48 weeks

• Adult Myopathy Assessment Tool (AMAT)

  48 weeks

• FVC

  48 weeks

• 6K scale

  48 weeks

• Serum creatinine levels

  48 weeks

Study Arms (2)

clenbuterole

EXPERIMENTAL

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

Drug: Clenbuterol

placebo

PLACEBO COMPARATOR

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

Drug: Placebo

Interventions

Clenbuterol DRUG

tablets

Also known as: active

clenbuterole

Placebo DRUG

tablets

placebo

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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;
• 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;
• neuromuscular disease other than SBMA.

Sponsors & Collaborators

• Gianni Soraru: lead
• Mario Negri Institute for Pharmacological Research: collaborator

Study Sites (1)

Azienda Ospedale Università di Padova

Padua, PD, 35134, Italy

RECRUITING

MeSH Terms

Conditions

Bulbo-Spinal Atrophy, X-Linked

Interventions

Clenbuterol; Exercise

Condition Hierarchy (Ancestors)

Muscular Atrophy, Spinal; Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Motor Neuron Disease; Neuromuscular Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Ethanolamines; Amino Alcohols; Alcohols; Organic Chemicals; Amines; Motor Activity; Movement; Musculoskeletal Physiological Phenomena; Musculoskeletal and Neural Physiological Phenomena

Study Officials

• Gianni Sorarù, MD

  Azienda Ospedale Università di Padova

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Gianni Sorarù, MD

CONTACT

0498213600; gianni.soraru@unipd.it

Elisabetta Pupillo, PharmD

CONTACT

0239014605; elisabetta.pupillo@marionegri.it

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR INVESTIGATOR
• PI Title: Professor

Model Details: 2 parallel groups

Study Record Dates

• First Submitted: December 5, 2023
• First Posted: December 13, 2023
• Study Start: April 13, 2024
• Primary Completion (Estimated): June 30, 2027
• Study Completion (Estimated): December 31, 2027
• Last Updated: April 15, 2024

Record last verified: 2024-04

Locations

IT (1)