Spinal and Bulbar Muscular AtrophyPhase 2BetaSBMANCT06169046
A Placebo-controlled Study of Clenbuterol in Spinal and Bulbar Muscular Atrophy
A Phase 2 study testing Clenbuterol in people with Spinal and Bulbar Muscular Atrophy, run by Gianni Soraru at 1 site in Italy.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Spinal and Bulbar Muscular Atrophy
- What is being tested?
- Clenbuterol
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 90 participants
- Where
- 1 siteItaly
- Study dates
- 13 Apr 2024to 31 Dec 2027
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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);
- providing a written informed consent.
Who cannot join
- 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;
- use of beta2 agonists in the preceding 6 months;
- participation to an interventional trial in the preceding 3 months;
- neuromuscular disease other than SBMA.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Italy1 site, 1 recruiting
- Recruiting
Azienda Ospedale Università di Padova
Padova, PD, 35134
Gianni Sorarù, 3478889974, gianni.soraru@unipd.it
About this study
Official title: A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
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
- Being tested
- Clenbuterol, Placebo
- Keywords
- SBMA, clenbuterol, trial
- Medical subject headings
- Bulbo-Spinal Atrophy, X-Linked
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ClenbuterolDrug
Also called active
tablets
PlaceboDrug
tablets
| Study group | Role | What they receive |
|---|---|---|
| clenbuterole | Experimental | 45 patients will receive Clenbuterol at a final dosage of 0.04 mg/day, treated for a total of 48 weeks Receives: Drug: Clenbuterol |
| placebo | Placebo comparator | 45 patients will receive placebo, treated for a total of 48 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
6-minute-walk test
to assess the efficacy of chronic treatment with clenbuterol in patients with SBMA by the 6MW test
Measured: 48 weeks
Secondary outcomes (7)
SBMA-FRS scale
SBMA-FRS total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Measured: 48 weeks
Adult Myopathy Assessment Tool (AMAT)
AMAT total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Measured: 48 weeks
FVC
FVC during the 12-months treatment period (from V2 to V7) in the two treatment arms
Measured: 48 weeks
6K scale
6K total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Measured: 48 weeks
Serum creatinine levels
serum creatinine levels during the 12-months treatment period (from V2 to V7) in the two treatment arms
Measured: 48 weeks
ALSAQ-40
ALSAQ-40 total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Measured: 48 weeks
individualized neuromuscular quality of life (INQoL) questionnaire
INQOL total score during the 12-months treatment period (from V2 to V7) in the two treatment arms
Measured: 48 weeks
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Dec 2023
- Study start13 Apr 2024
- Primary completion30 Jun 2027 (estimated)
- Study completion31 Dec 2027 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal and Bulbar Muscular AtrophyStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- 2 parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 90 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Gianni Soraru
- Sponsor type
- University, hospital or other
- Collaborators
- Mario Negri Institute for Pharmacological Research
- Responsible party
- Sponsor-investigator, Gianni Soraru, University Hospital Padova
- Investigators
- Gianni Sorarù, MD, Principal investigator, Azienda Ospedale Università di Padova
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06169046
- Sponsor's protocol ID
- EudraCT: 2017-005103-27
- Acronym
- BetaSBMA
- Status last verified
- Apr 2024
- First submitted
- 5 Dec 2023
- Last updated
- 15 Apr 2024
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 15 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Placebo-controlled Study to Evaluate the Efficacy and Safety of Clenbuterol in Patients With Spinal and Bulbar Muscular Atrophy (SBMA)
- ClinicalTrials.gov ID
- NCT06169046
- Acronym / sponsor ID
- BetaSBMA / EudraCT: 2017-005103-27
- Conditions
- Spinal and Bulbar Muscular Atrophy
- Keywords
- SBMA, clenbuterol, trial
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 90 participants (estimated)
- Lead sponsor
- Gianni SoraruUniversity, hospital or other
- Collaborators
- Mario Negri Institute for Pharmacological Research
- Study start
- 13 Apr 2024 (actual)
- Primary completion
- 30 Jun 2027 (estimated)
- Study completion
- 31 Dec 2027 (estimated)
- First posted
- 13 Dec 2023 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
- Model description
- 2 parallel groups
| Arm | Type | Receives |
|---|---|---|
| clenbuterole | Experimental | Drug: Clenbuterol45 patients will receive Clenbuterol at a final dosage of 0.04 mg/day, treated for a total of 48 weeks |
| placebo | Placebo comparator | Drug: Placebo45 patients will receive placebo, treated for a total of 48 weeks |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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);
- providing a written informed consent.
Key exclusion criteria (7 listed)
- 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;
- use of beta2 agonists in the preceding 6 months;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ClenbuterolAlso: activetablets | Drug | clenbuterole |
| Placebotablets | Drug | placebo |
Primary outcome
6-minute-walk test
Time frame: 48 weeks
to assess the efficacy of chronic treatment with clenbuterol in patients with SBMA by the 6MW test
7 secondary outcomes
SBMA-FRS scale
Time frame: 48 weeks
Adult Myopathy Assessment Tool (AMAT)
Time frame: 48 weeks
FVC
Time frame: 48 weeks
6K scale
Time frame: 48 weeks
Serum creatinine levels
Time frame: 48 weeks
ALSAQ-40
Time frame: 48 weeks
individualized neuromuscular quality of life (INQoL) questionnaire
Time frame: 48 weeks
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 15 Apr 2024.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Gianni Sorarù, MD principal investigator
Azienda Ospedale Università di Padova
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Clenbuterol in people with Spinal and Bulbar Muscular Atrophy, run by Gianni Soraru at 1 site in Italy.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 7 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 15 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Italy. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Gianni Sorarù, MD (0498213600, gianni.soraru@unipd.it), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
6-minute-walk test (time frame: 48 weeks). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 15 Apr 2024.