Not recruitingRare disease

Study of Clenbuterol vs Placebo in Adult Patients with Spinal and Bulbar Muscular Atrophy (SBMA)

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What is this trial about?

A plain-language summary of the goals, design and what participants do

This study focuses on Spinal and Bulbar Muscular Atrophy (SBMA), a rare genetic condition that causes muscle weakness and wasting, particularly affecting the muscles used for speaking, swallowing, and limb movement. The study will test a medication called clenbuterol, comparing it with a placebo to determine if it can help people with this condition.

The main purpose is to evaluate how well clenbuterol works and how safe it is when used as a long-term treatment for patients with SBMA. During the study, participants will receive either clenbuterol tablets or placebo tablets for a period of 48 weeks. The medication will be taken by oral route, with a maximum daily dose of 40 micrograms.

Throughout the study, various measurements will be taken to assess how participants respond to the treatment, including their ability to walk and perform daily activities. The study will also monitor breathing function and overall quality of life. Regular check-ups will be conducted to ensure participant safety and to track any changes in their condition.

The research process

The trial runs in 4 steps – from screening to follow-up. Each step says what happens and what the team monitors.

Step 1
Initial trial participation
You will begin a 48-week treatment period after being confirmed eligible for the study
You will be randomly assigned to receive either Monores® tablets (containing clenbuterol) or a placebo tablet
The medication will be taken by mouth
Step 2
Treatment period
The treatment will continue for 48 weeks
Regular assessments will be conducted to monitor your progress
Your ability to walk will be measured through a six-minute walking test
Your breathing capacity will be evaluated
Your muscle strength and function will be assessed
Blood tests will be performed to check creatinine levels
Step 3
Assessment visits
You will need to attend multiple assessment visits during the 48-week period
During these visits, your overall physical function will be evaluated
Your quality of life will be assessed through questionnaires
Your muscle strength and walking ability will be tested
Step 4
Final evaluation
At week 48, a final assessment will be conducted
The main focus will be on measuring the distance you can walk in six minutes
This distance will be compared to your initial walking ability
Additional measurements of muscle strength, breathing capacity, and overall function will be recorded

Who can join the trial?

8 criteria

Must be male
Must have a confirmed genetic diagnosis of Spinal and Bulbar Muscular Atrophy with at least 38 CAG repeats in the AR gene
Must be between 18 and 75 years old
Must have at least one of these symptoms:
- Muscle atrophy (decrease in muscle size)
- Limb weakness (reduced strength in arms or legs)
- Bulbar palsy (difficulty with speech, chewing, or swallowing)
- Must be able to walk independently, with or without support devices like canes or walkers (wheelchairs not allowed)
- Must be able and willing to provide written informed consent (formal agreement to participate in the study)

Who cannot join the trial?

12 criteria

Female patients (only male patients can participate)
Individuals under 18 years of age
Patients with other forms of muscular atrophy (conditions causing muscle weakness and loss) not related to SBMA
Patients with severe heart conditions, as clenbuterol may affect heart function
Patients currently taking medications that could interact with clenbuterol
Individuals with uncontrolled high blood pressure
Patients with severe liver or kidney dysfunction
Individuals participating in other clinical trials
Patients with known allergies to clenbuterol or similar medications
Individuals unable to provide informed consent
Patients with severe psychiatric conditions that could affect their ability to follow study procedures
Individuals with conditions that could interfere with the evaluation of the study results

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Verified sites

All sites with verified contact details – recruitment status may not be available; ask directly

Azienda Ospedaliera Universitaria Citta' Della Salute E Della Scienza Di Torino
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Turin, Italy
Azienda Ospedaliera di Padova
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Padua, Italy
Azienda Ospedaliero Universitaria Pisana
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Pisa, Italy

Trial locations

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Investigated drugs

Clenbuterol is a medication that belongs to a class of drugs called beta-2 agonists. It works by relaxing the smooth muscles and improving breathing. In this trial, it is being studied for its potential benefits in patients with Spinal and Bulbar Muscular Atrophy (SBMA), a rare genetic condition that affects muscle strength and function. The medication may help improve muscle strength and function in patients with this condition.

Clenbuterol Hydrochloride

What is already known about the treatment

Clenbuterol - A beta-2 adrenergic agonist typically administered orally in tablet form, which was originally developed for treating breathing disorders such as asthma. In clinical trials, it is being investigated for potential therapeutic benefits in patients with Spinal and Bulbar Muscular Atrophy (SBMA), where it may help improve muscle strength and function. The medication works by stimulating beta-2 receptors, leading to increased protein synthesis in muscle cells and potential reduction of protein breakdown, while also having bronchodilating properties. Currently classified as a sympathomimetic amine, it is being studied in controlled trials to evaluate its efficacy and safety profile specifically for neuromuscular conditions, though it's important to note that its use for muscle-related conditions is still experimental in many countries.

Investigated diseases

Spinal and Bulbar Muscular Atrophy - A genetic neuromuscular disorder that primarily affects males and is characterized by progressive muscle weakness and wasting. The condition begins in the muscles of the shoulders and hips, gradually spreading to other muscle groups including those involved in speaking and swallowing. The disease is caused by a mutation in the androgen receptor gene on the X chromosome. Symptoms typically appear in adulthood, usually between the ages of 30 and 50. Also known as Kennedy's disease, it causes muscle cramps, tremors, and twitching, along with gradual muscle loss in arms and legs.