Overview
Sponsor-declared trial summary
Phase
Therapeutic confirmatory (Phase III)
Status
Authorised, recruitment pending
Participants planned
14
Countries
1
Sites
1
Metilcrotonilglicinuria
The aim is to compare the tolerability of the commercial biotin drug prescribed as usual treatment in pediatric patients at the Hospital Clínico Universitario de Santiago de Compostela with respect to the biotin master formula (chewable formulation) prepared in the Pharmacy Service of the same center, in order to impro…
Key facts
- Sponsor
- Fundacion Publica Galega Instituto De Investigacion Sanitaria De Santiago De Compostela
- Participant type
- Pediatric, Patients
- Age range
- 0-17 years
- Gender
- Male and Female
- Therapeutic area
- Diseases [C] - Nutritional and Metabolic Diseases [C18]
- Decision date (initial)
- 2025-03-12
- Transition trial
- No
- Low-intervention
- No
- Rare-disease indication
- Yes
- Vulnerable population
- No
Trial design
CTIS Part I — objectives, methods, condition coding
Main objective
Scope: Others
The aim is to compare the tolerability of the commercial biotin drug prescribed as usual treatment in pediatric patients at the Hospital Clínico Universitario de Santiago de Compostela with respect to the biotin master formula (chewable formulation) prepared in the Pharmacy Service of the same center, in order to improve the care and adherence of pediatric patients diagnosed with the rare hereditary metabolic disease
Secondary objectives 2
- Assess acceptability and adherence to treatment
- To evaluate the safety of chewable master formulations prepared by 3D printing
Conditions and MedDRA coding
Metilcrotonilglicinuria
| Version | Level | Code | Term | System organ class |
|---|---|---|---|---|
| 20.0 | PT | 10071434 | Biotinidase deficiency | 100000004850 |
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Treatment of study Patient was randomization in two arm and started treatment during 4 month and them cross ther treatment with the other arm during 4 month more.
|
Randomised Controlled | None | Medebiotin comprimidos o solucion inyectable: Medebiotin comprimidos o solucion inyectable Formula magistral masticable de biotina: Formula magistral masticable de biotina |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 1
- 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 1
- 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 and secondary outcome measures (English text)
Primary endpoints 1
- visual scale and in the collection of subjective symptoms reported by the patient.
Secondary endpoints 2
- Determination of organic acids
- plasma biotin levels
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
SUB05841MIG · Substance
- Active substance
- Biotin
- Pharmaceutical form
- CHEWABLE TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 15 mg milligram(s)
- Max total dose
- 15 mg milligram(s)
- Max treatment duration
- 8 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Comparator 2
Medebiotin Fuerte solución inyectable
PRD345970 · Product
- Active substance
- Biotin Sodium
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- ORAL USE
- Max daily dose
- 15 mg milligram(s)
- Max total dose
- 15 mg milligram(s)
- Max treatment duration
- 8 Month(s)
- Authorisation status
- Authorised
- ATC code
- A11HA05 — BIOTIN
- Marketing authorisation
- 34.236
- MA holder
- LABORATORIO REIG JOFRE, S.A.
- MA country
- Spain
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
PRD345969 · Product
- Active substance
- Biotin
- Pharmaceutical form
- TABLET
- Route of administration
- ORAL USE
- Max daily dose
- 15 mg milligram(s)
- Max total dose
- 15 mg milligram(s)
- Max treatment duration
- 8 Month(s)
- Authorisation status
- Authorised
- ATC code
- A11HA05 — BIOTIN
- Marketing authorisation
- 24616
- MA holder
- LABORATORIO REIG JOFRE, S.A.
- MA country
- Spain
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Fundacion Publica Galega Instituto De Investigacion Sanitaria De Santiago De Compostela
- Sponsor organisation
- Fundacion Publica Galega Instituto De Investigacion Sanitaria De Santiago De Compostela
- Address
- Travesia Da Choupana S/N
- City
- Santiago De Compostela
- Postcode
- 15706
- Country
- Spain
Scientific contact point
- Organisation
- Fundacion Publica Galega Instituto De Investigacion Sanitaria De Santiago De Compostela
- Contact name
- Dra Maria Luz Couce Pico
Public contact point
- Organisation
- Fundacion Publica Galega Instituto De Investigacion Sanitaria De Santiago De Compostela
- Contact name
- Dra Maria Luz Couce Pico
Locations
1 EU/EEA country · 1 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Authorised, recruitment pending | 14 | 1 |
| Rest of world | — | 0 | — |
Investigational sites
Complexo Hospitalario Universitario De Santiago
Unidad de Enfermedades Metabólicas Congénitas, Calle Choupana Da S/n, 15706, Santiago De Compostela
Results and documents
Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial
Documents 17 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | Biotine Protocolo_v06_18022025 clean | 6 |
| Protocol (for publication) | Biotine Protocolo_v06_18022025 con cambios | 6 |
| Protocol (for publication) | Protocolo_EUCT 2024-519378-37-00_ v05 04112024 | 5 |
| Recruitment arrangements (for publication) | Recruitment material description v1 | 1 |
| Subject information and informed consent form (for publication) | IMPRIMIDO Asentimiento 12 a 17 anos v6 de 18022025 clean | 6 |
| Subject information and informed consent form (for publication) | IMPRIMIDO Asentimiento 12 a 17 anos v6 de 18022025 Con cambios | 6 |
| Subject information and informed consent form (for publication) | IMPRIMIDO HIPCI Adultos v6 de 18022025 clean | 6 |
| Subject information and informed consent form (for publication) | IMPRIMIDO HIPCI Adultos v6 de 18022025 con cambios | 6 |
| Subject information and informed consent form (for publication) | IMPRIMIDO HIPCI Padrestutores v6 de 18022025 con cambios | 6 |
| Subject information and informed consent form (for publication) | IMPRIMIDO HIPCI Padrestutores v6 de18022025 clean | 6 |
| Subject information and informed consent form (for publication) | SIC and ICF adult tutors v5 04112024 | 5 |
| Subject information and informed consent form (for publication) | SIC and ICF adults v5 04112024 | 5 |
| Subject information and informed consent form (for publication) | SIC and ICF Assent 12 to 17 years v5 04112024 | 5 |
| Summary of Product Characteristics (SmPC) (for publication) | SmPC_Biotina Acofarma | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | SmPC_Medebiotin comprimidos 2019 02 | 1 |
| Summary of Product Characteristics (SmPC) (for publication) | SmPC_Medebiotin solucion inyectable 2005 07 01 | 1 |
| Synopsis of the protocol (for publication) | Protocolo Sinopsis_EUCT 2024-519378-37-00_ v05 04112024 | 5 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2024-11-27 | Spain | Acceptable with conditions 2025-03-12
|
2025-03-12 |