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

NCT06169046 · Status: RECRUITING · Phase: PHASE2 · Type: INTERVENTIONAL · Enrollment: 90

Last updated 2024-04-15

No results posted yet for this study

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

Interventions

DRUG

Clenbuterol

tablets

DRUG

Placebo

tablets

Sponsors & Collaborators

  • Mario Negri Institute for Pharmacological Research

    collaborator OTHER

  • Gianni Soraru

    lead OTHER

Principal Investigators

  • Gianni Sorarù, MD · Azienda Ospedale Università di Padova

Study Design

Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Model
PARALLEL

Eligibility

Min Age
18 Years
Max Age
75 Years
Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2024-04-13
Primary Completion
2027-06-30
Completion
2027-12-31

Countries

  • Italy

Study Locations

More Related Trials

Entities

Drugs

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT06169046 on ClinicalTrials.gov