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

Conditions

Spinal and Bulbar Muscular Atrophy

Brief summary

A responder to the assigned treatment (clenbuterol or placebo) will be defined as a subject with a 15% increase at V7 (48 weeks), compared to baseline, in the distance covered in six minutes at the 6MWT. The primary endpoint will be the percentage of responders in the two treatment arms.

Detailed description

6MWT during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, SBMA-FRS total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, AMAT total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, FVC during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, 6K total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, serum creatinine levels during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, ALSAQ-40 total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, INQOL total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms

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Interventions

DRUGMonores® 20 microgrammi Compresse

DRUGplacebo

Eligibility

Sex/Gender

Male

Age

18 Years to No maximum

Design outcomes

Primary

Measure	Time frame
A responder to the assigned treatment (clenbuterol or placebo) will be defined as a subject with a 15% increase at V7 (48 weeks), compared to baseline, in the distance covered in six minutes at the 6MWT. The primary endpoint will be the percentage of responders in the two treatment arms.	—

Secondary

Measure	Time frame
6MWT during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, SBMA-FRS total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, AMAT total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, FVC during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, 6K total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, serum creatinine levels during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, ALSAQ-40 total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, INQOL total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms	—

Measure

Time frame

6MWT during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, SBMA-FRS total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, AMAT total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, FVC during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, 6K total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, serum creatinine levels during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, ALSAQ-40 total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms, INQOL total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms

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Countries

Italy

Outcome results

None listed