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

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What is this study about?

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.

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

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

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

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 Study?

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 Study?

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

Where you can join this trial?

Verified and Recommended Sites

No sites found in this category

Verified Sites

Site Name	City	Country	Status
Fondazione Policlinico Universitario Agostino Gemelli IRCCS	Rome	Italy

Other Sites

Site Name	City	Country
Azienda Ospedaliera di Padova	Padua	Italy
Azienda Ospedaliero-Universitaria Maggiore Della Carita	Novara	Italy
Fondazione I.R.C.C.S. Istituto Neurologico Besta	Milan	Italy
Azienda Ospedaliero Universitaria Pisana	Pisa	Italy
Amyhqpk Ogevxwqpncn Uqvlfmwdgvyqm Cquqzzjwusmc Dryki Shhuqa E Dfojv Sdmtglf Dd Ttpuoe	Turin	Italy

Trial status

Country	Status	Recruitment Start
Italy	Recruiting	08.04.2024

Trial locations

Investigated drugs:

Clenbuterol Hydrochloride

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.

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.

Trial ID:

2024-516563-86-00

Trial Phase:

Therapeutic exploratory (Phase II)

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

What is this study about?

Who Can Join the Study?

Who Cannot Join the Study?