Clinical trial • Phase III • Endocrinology | Rare Disease
Biotin for Methylcrotonylglycinuria | Biotinidase deficiency
Phase III trial of Biotin for Methylcrotonylglycinuria | Biotinidase deficiency.
Overview
- Trial Therapeutic Area
- Endocrinology | Rare Disease
- Trial Disease
- Methylcrotonylglycinuria | Biotinidase deficiency
- Trial Stage
- Phase III
- Drug Modality
- Small molecule
- Paediatric Trial
- Yes
Key dates
- Initial CTIS Submission Date
- 27-11-2024
- First CTIS Authorization Date
- 12-03-2025
Trial design
Randomised, open-label, medebiotin fuerte solución inyectable (commercial comparator, active substance: biotin sodium; marketing authorisation es 34.236) and medebiotín fuerte comprimidos (commercial comparator, active substance: biotin; marketing authorisation es 24616). usual prescribed treatment vs biotin master formula (chewable formulation). maximum daily dose listed as 15 mg; treatment duration per arm 4 months then cross to other arm for 4 months (crossover).-controlled, crossover Phase III trial in Spain.
- Randomised
- Yes
- Open Label
- Yes
- Comparator
- Medebiotin Fuerte solución inyectable (commercial comparator, active substance: biotin sodium; marketing authorisation ES 34.236) and Medebiotín Fuerte Comprimidos (commercial comparator, active substance: biotin; marketing authorisation ES 24616). Usual prescribed treatment vs biotin master formula (chewable formulation). Maximum daily dose listed as 15 mg; treatment duration per arm 4 months then cross to other arm for 4 months (crossover).
- Crossover
- Yes
- Target Sample Size
- 14
- Trial Duration For Participant
- 240
Eligibility
Recruits 14 paediatric patients.
- Vulnerable Population
- Paediatric participants aged 3-18 years. Consent is provided by parent(s) or guardian(s). Assent documents are available for adolescents (assent forms for 12-17 years are listed). Separate subject information and informed consent forms for parents/guardians and adults are included (documents in Spanish).
Inclusion criteria
- {"criterion_text":"- Outpatients of both sexes, between 3-18 years of age (both ages included within the range). Diagnosis of one of the two rare metabolic diseases under study: methylcrotonylglycinuria treated with Medebiotin tablets or injectable solution, biotinidase deficiency treated with Medebiotin tablets or injectable solution. The patient has no swallowing problems at the time of signing the informed consent document by the parent(s) or guardian(s). Good compliance with the treatment prescribed by the specialist for each patient during the last year (Medebiotin tablets or injectable solution). This implies that the patient has not presented any metabolic decompensation in the last year."}
Exclusion criteria
- {"criterion_text":"- Known hypersensitivity to any of the excipients of the biotin master formulation. Any disorder or situation (severe metabolic decompensation) which, in the opinion of the investigating physician, would pose a risk for the subject not to comply with the prescribed treatment."}
Endpoints
Primary endpoints
- {"endpoint_text":"- visual scale and in the collection of subjective symptoms reported by the patient.","definition_or_measurement_approach":"Measured by visual scale and collection of subjective symptoms reported by the patient (as stated in endpoint text)."}
Secondary endpoints
- {"endpoint_text":"- Determination of organic acids","definition_or_measurement_approach":"Laboratory determination of organic acids (as stated)."}
- {"endpoint_text":"- plasma biotin levels","definition_or_measurement_approach":"Measurement of biotin concentration in plasma (as stated)."}
Recruitment
- Planned Sample Size
- 14
- Recruitment Window Months
- 9
- Consent Approach
- Informed consent obtained from parent(s) or guardian(s). Assent forms available for adolescents (assent documents for 12-17 years are listed). Subject information and informed consent forms for parents/guardians and adults are provided (documents in Spanish). Contact and consent documents versions are included in the submission (multiple ICF/assent documents).
Geography
- Total Number Of Sites
- 1
- Total Number Of Participants
- 14
Spain
- Earliest CTIS Part Ii Submission Date
- 18-02-2025
- Latest Decision Or Authorization Date
- 12-03-2025
- Processing Time Days
- 22
- Number Of Sites
- 1
- Number Of Participants
- 14
Sites
- Site Name
- Complexo Hospitalario Universitario De Santiago
- Department Name
- Unidad de Enfermedades Metabólicas Congénitas
- Principal Investigator Name
- Maria Luz Couce Pico
- Principal Investigator Email
- Maria.Luz.Couce.Pico@sergas.es
- Contact Person Name
- Maria Luz Couce Pico
- Contact Person Email
- Maria.Luz.Couce.Pico@sergas.es
- Number Of Participants
- 14
Sponsor
Primary sponsor
- Full Name
- Fundacion Publica Galega Instituto De Investigacion Sanitaria De Santiago De Compostela
- Organisation Type
- Hospital/Clinic/Other health care facility
- Country Of Registered Address
- Spain
Investigational products
- Investigational Product Name
- BIOTIN (chewable tablet)
- Active Substance
- Biotin
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- No marketing authorisation (marketingAuthNumber = '-')
- Maximum Dose
- 15 mg
- Investigational Product Name
- Medebiotin Fuerte solución inyectable (SOLUTION FOR INJECTION)
- Active Substance
- Biotin sodium
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Marketing authorisation ES 34.236
- Maximum Dose
- 15 mg
- Investigational Product Name
- Medebiotín Fuerte Comprimidos (TABLET)
- Active Substance
- Biotin
- Modality
- Small molecule
- Routes Of Administration
- Oral
- Route
- Oral
- Authorisation Status
- Marketing authorisation ES 24616
- Maximum Dose
- 15 mg
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