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

ConditionSpinal and Bulbar Muscular Atrophy
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-75
SponsorGianni Soraru

About this trial

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.

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

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);

Disqualifiers

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;

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);

inability to walk or walking only with the support of a caregiver;

Trial design

Design model

Parallel

Treatments tested in this trial

  • Clenbuterol

    Drug

    tablets

  • Placebo

    Drug

    tablets

Treatment groups

90 Participants
are divided into 2 treatment groups
Group A: clenbuteroleExperimental treatment 1 intervention
Group B: placeboPlacebo comparator 1 intervention

Trial outcomes

Primary outcomes

1

6-minute-walk test

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

Time frame
48 weeks

Secondary outcomes

1

SBMA-FRS scale

SBMA-FRS total score during the 12-months treatment period (from V2 to V7) in the two treatment arms

Time frame
48 weeks
2

Adult Myopathy Assessment Tool (AMAT)

AMAT total score during the 12-months treatment period (from V2 to V7) in the two treatment arms

Time frame
48 weeks
3

FVC

FVC during the 12-months treatment period (from V2 to V7) in the two treatment arms

Time frame
48 weeks
4

6K scale

6K total score during the 12-months treatment period (from V2 to V7) in the two treatment arms

Time frame
48 weeks

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

Gianni Soraru

Lead sponsor

University Hospital Padova

Sponsor institution

Mario Negri Institute for Pharmacological Research

Collaborator

Source ClinicalTrials.gov
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