Clinical trial • Phase II • Neurology
Clenbuterol hydrochloride for Spinal and bulbar muscular atrophy
Phase II trial of Clenbuterol hydrochloride for Spinal and bulbar muscular atrophy.
Overview
- Trial Therapeutic Area
- Neurology
- Trial Disease
- Spinal and bulbar muscular atrophy
- Trial Stage
- Phase II
- Drug Modality
- Small molecule|Other
Key dates
- Initial CTIS Submission Date
- 25-07-2024
- First CTIS Authorization Date
- 16-09-2024
Trial design
Clenbuterol (Monores® 20 microgrammi Compresse; active substance: clenbuterol hydrochloride) vs placebo. Product information indicates 20 µg tablet strength and a maximum daily dose amount recorded as 40 µg; no detailed dosing schedule or administration schedule explicitly stated in the CTIS record.-controlled Phase II trial in Italy.
- Comparator
- Clenbuterol (Monores® 20 microgrammi Compresse; active substance: clenbuterol hydrochloride) vs placebo. Product information indicates 20 µg tablet strength and a maximum daily dose amount recorded as 40 µg; no detailed dosing schedule or administration schedule explicitly stated in the CTIS record.
- Target Sample Size
- 90
- Trial Duration For Participant
- 336
Eligibility
Recruits 90 No vulnerable populations selected (isVulnerablePopulationSelected:false). Participants must provide written informed consent; only adults (aged between 18 and 75 years) are eligible, so no assent/minor consent arrangements are specified..
- Vulnerable Population
- No vulnerable populations selected (isVulnerablePopulationSelected:false). Participants must provide written informed consent; only adults (aged between 18 and 75 years) are eligible, so no assent/minor consent arrangements are specified.
Inclusion criteria
- {"criterion_text":"- males who have received a genetically confirmed diagnosis of SBMA (AR CAG repeat number >= 38);\n- aged between 18 and 75 (+364 days) years;\n- displaying one or more of the following clinical symptoms: muscle atrophy, limb weakness, bulbar palsy;\n- able to walk independently with or without a cane or other supporting device (all supporting devices are acceptable except on wheelchair);\n- providing a written informed consent."}
Exclusion criteria
- {"criterion_text":"- a documented cardiovascular disease precluding the use of beta2 agonists (in the judgment of the investigators);\n- 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;\n- 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);\n- inability to walk or walking only with the support of a caregiver;\n- use of beta2 agonists in the preceding 6 months;\n- participation to an interventional trial in the preceding 3 months;\n- neuromuscular disease other than SBMA."}
Endpoints
Primary endpoints
- {"endpoint_text":"- 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.","definition_or_measurement_approach":"Responder defined as ≥15% increase at V7 (48 weeks) vs baseline in distance covered on the 6-minute walk test (6MWT); primary outcome is percentage of responders in each treatment arm."}
Secondary endpoints
- {"endpoint_text":"- 6MWT during the 48-weeks treatment period (from V2 to V7) in the two treatment arms","definition_or_measurement_approach":"6-minute walk test measured at visits from V2 to V7 over the 48-week treatment period, compared between treatment arms."}
- {"endpoint_text":"- SBMA-FRS total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms","definition_or_measurement_approach":"SBMA Functional Rating Scale (total score) assessed at visits V2–V7 across 48 weeks, compared between arms."}
- {"endpoint_text":"- AMAT total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms","definition_or_measurement_approach":"Adult Myopathy Assessment Tool (AMAT) total score assessed at V2–V7 (48 weeks), compared between arms."}
- {"endpoint_text":"- FVC during the 48-weeks treatment period (from V2 to V7) in the two treatment arms","definition_or_measurement_approach":"Forced vital capacity (FVC) measured at visits V2–V7 over 48 weeks, compared between arms."}
- {"endpoint_text":"- 6K total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms","definition_or_measurement_approach":"6K total score assessed at V2–V7 across 48 weeks, compared between arms."}
- {"endpoint_text":"- serum creatinine levels during the 48-weeks treatment period (from V2 to V7) in the two treatment arms","definition_or_measurement_approach":"Serum creatinine measured at visits V2–V7 during the 48-week treatment period, compared between arms."}
- {"endpoint_text":"- ALSAQ-40 total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms","definition_or_measurement_approach":"ALSAQ-40 total score assessed at V2–V7 across 48 weeks, compared between arms."}
- {"endpoint_text":"- INQOL total score during the 48-weeks treatment period (from V2 to V7) in the two treatment arms","definition_or_measurement_approach":"INQoL total score assessed at visits V2–V7 during the 48-week treatment period, compared between arms."}
Recruitment
- Planned Sample Size
- 90
- Recruitment Window Months
- 32
- Consent Approach
- Participants must provide written informed consent. Subject information and informed consent form available (document 'L1_ SIS and ICF_adults'). Only adults (18-75 years) are eligible; no assent or minor-consent processes are specified. Languages of consent documents not specified in the CTIS record.
Geography
- Total Number Of Sites
- 7
- Total Number Of Participants
- 90
Italy
- Earliest CTIS Part Ii Submission Date
- 02-08-2024
- Latest Decision Or Authorization Date
- 04-11-2024
- Processing Time Days
- 94
- Number Of Sites
- 7
- Number Of Participants
- 90
Sites
- Site Name
- Azienda Ospedaliero Universitaria Città della Salute e della Scienza di Torino Dipartimento di Neur
- Department Name
- Dipartimento di Neuroscienze CRESLA
- Principal Investigator Name
- Adriano Chiò
- Principal Investigator Email
- adriano.chio@unito.it
- Contact Person Name
- Adriano Chiò
- Contact Person Email
- adriano.chio@unito.it
- Site Name
- Fondazione Policlinico Universitario Agostino Gemelli IRCCS
- Department Name
- Centro Clinico Nemo Adulti
- Principal Investigator Name
- Mario Sabatelli
- Principal Investigator Email
- mario.sabatelli@unicatt.it
- Contact Person Name
- Mario Sabatelli
- Contact Person Email
- mario.sabatelli@unicatt.it
- Site Name
- Azienda Ospedaliero Universitaria Pisana
- Department Name
- U.O. di Neurologia
- Principal Investigator Name
- Gabriele Siciliano
- Principal Investigator Email
- g.siciliano@med.unipi.it
- Contact Person Name
- Gabriele Siciliano
- Contact Person Email
- g.siciliano@med.unipi.it
- Site Name
- Azienda Ospedaliera di Padova
- Department Name
- Clinica Neurologica
- Principal Investigator Name
- Gianni Sorarù
- Principal Investigator Email
- gianni.soraru@unipd.it
- Contact Person Name
- Gianni Sorarù
- Contact Person Email
- gianni.soraru@unipd.it
- Site Name
- AOU di Modena Nuovo Ospedale Civile S. Agostino Estense di Modena- Ospedale di Baggiovara
- Department Name
- U.O. di Neurologia
- Principal Investigator Name
- Jessica Mandrioli
- Principal Investigator Email
- j.mandrioli@ausl.mo.it
- Contact Person Name
- Jessica Mandrioli
- Contact Person Email
- j.mandrioli@ausl.mo.it
- Site Name
- IRCCS Foundation Istituto Neurologico Carlo Besta
- Department Name
- Neuroscience
- Principal Investigator Name
- Davide Pareyson
- Principal Investigator Email
- davide.pareyson@istituto-besta.it
- Contact Person Name
- Davide Pareyson
- Contact Person Email
- davide.pareyson@istituto-besta.it
- Site Name
- Azienda Ospedaliero- Universitaria Maggiore della Carità di Novara Clinica Neurologica Centro SLA
- Department Name
- Clinica Neurologica Centro SLA
- Principal Investigator Name
- Fabiola De Marchi
- Principal Investigator Email
- fabiola.demarchi@uniupo.it
- Contact Person Name
- Fabiola De Marchi
- Contact Person Email
- fabiola.demarchi@uniupo.it
Sponsor
Primary sponsor
- Full Name
- Azienda Ospedaliera di Padova
- Organisation Type
- Hospital/Clinic/Other health care facility
- Country Of Registered Address
- Italy
Investigational products
- Investigational Product Name
- Monores® 20 microgrammi Compresse
- Active Substance
- Clenbuterol hydrochloride
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Marketing authorisation in Italy (marketingAuthNumber: 024217034; MIA: IT176H2023)
- Maximum Dose
- 40 µg per day
- Investigational Product Name
- placebo
- Modality
- Other
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