Overview
Sponsor-declared trial summary
Phase
Phase II and Phase III (Integrated)
Status
Authorised, recruitment pending
Participants planned
6
Countries
1
Sites
4
Hydroxycobalamin deficiency
to determine whether the administration of high doses of hydroxycobalamin decreases the blood concentration of tHcy and to compare the decrease with treatment at standard doses.
Key facts
- Sponsor
- Fundacion 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)
- 2025-06-09
- 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: Therapy
to determine whether the administration of high doses of hydroxycobalamin decreases the blood concentration of tHcy and to compare the decrease with treatment at standard doses.
Secondary objectives 3
- To evaluate the efficacy of high-dose hydroxycobalamin on visual function, neurocognitive abilities and auxological parameters compared to standard doses
- To assess parental stress and quality of life of patients and caregivers in both treatment groups and to determine if there are differences between doses
- To assess the safety and tolerability of high doses of hydroxycobalamin in the long term, in terms of pain at the procedure and injection site compared to the standard dose
Conditions and MedDRA coding
Hydroxycobalamin deficiency
Study design 1 period
| # | Title | Allocation | Blinding | Roles blinded | Arms |
|---|---|---|---|---|---|
| 1 | Treatment of study LA HIDROXOCOBALAMINA EN UNA NUEVA CONCENTRACIÓN
|
Randomised Controlled | None | Hidroxocobalamina 1 mg+ Hidroxocobalamina 25 mg: Hidroxocobalamina 1 mg durante un año + Hidroxocobalamina 25 mg durante un año adicional Hidroxocobalamina 10 mg+ Hidroxocobalamina 25 mg: Hidroxocobalamina 10 mg durante un año+ Hidroxocobalamina 25 mg durante un año mas |
Eligibility criteria
Principal inclusion / exclusion criteria as submitted by sponsor
Inclusion criteria 4
- Pediatric patients under 2 years of age.
- Patients with genetic diagnosis of any of the Cbl-RD diseases with early diagnosis who present ocular manifestations
- Patients whose legal representative or, failing that, caregivers of the patient give written informed consent
- Patients who, at the investigator's discretion, can complete the total follow-up of the study
Exclusion criteria 4
- Premature patients born before 32 weeks of gestation
- Patients with any other relevant pathology not associated with Cbl-RD diseases who, according to the physician's criteria, are not candidates to participate in the study.
- Patients who present any contraindication to receive treatment with hydroxycobalamin, including hypersensitivity to the active ingredient (hydroxycobalamin) or to any of its excipients, according to the specifications of its data sheet
- Participation in another clinical trial that prevents the patient's inclusion in the study
Endpoints
Primary and secondary outcome measures (English text)
Primary endpoints 1
- measurement of tHcy blood concentration at baseline and at 3-, 6-, 9-, 12-, 12-, 15-, 18- and 24-month follow-up and comparison of concentrations in both treatment arms
Investigational products
Investigational medicinal products (IMPs), comparators, placebo, auxiliary
Test 1
SUB32920 · Substance
- Active substance
- Cobalamin
- Pharmaceutical form
- SOLUTION FOR INJECTION
- Route of administration
- INTRAMUSCULAR INJECTION
- Max daily dose
- 25 mg milligram(s)
- Max total dose
- 25 mg milligram(s)
- Max treatment duration
- 24 Month(s)
- Authorisation status
- Authorised
- ATC code
- - — -
- Marketing authorisation
- -
- Paediatric formulation
- No
- Orphan designation
- No
- Modified vs. Marketing Authorisation
- No
Sponsors and contacts
Sponsor organisations, regulatory contacts, third parties
Fundacion Instituto De Investigacion Sanitaria De Santiago De Compostela
- Sponsor organisation
- Fundacion 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 Instituto De Investigacion Sanitaria De Santiago De Compostela
- Contact name
- Dra Maria Luz Couce Pico
Public contact point
- Organisation
- Fundacion Instituto De Investigacion Sanitaria De Santiago De Compostela
- Contact name
- Dra Maria Luz Couce Pico
Locations
1 EU/EEA country · 4 investigational sites
By country
| Country | MS status | Planned subjects | Sites |
|---|---|---|---|
| Spain | Authorised, recruitment pending | 6 | 4 |
| Rest of world | — | 0 | — |
Investigational sites
Hospital Sant Joan De Deu Barcelona
neurologia pediatrica, Passeig De Sant Joan De Deu 2, 08950, Esplugues De Llobregat
Hospital Universitario Marques De Valdecilla
neurologia pediatrica, Avenida Valdecilla Sn, 39008, Santander
Hospital Universitario De Cruces
neurologia pediatrica, Cruces Plaza S/n, 48903, Barakaldo
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 22 files
Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.
| Type | Title | Version |
|---|---|---|
| Protocol (for publication) | Apendice 11_ethical_consid_ct_with_minors_0 | 1 |
| Protocol (for publication) | Apendice 3 MLC-HYC2024Variables CRD | 1 |
| Protocol (for publication) | Apendice 4 SAE Report Form MLC-HyC | 1 |
| Protocol (for publication) | Apendice 5 Escala VFCS Spanish | 1 |
| Protocol (for publication) | Apendice 6 Escala de BAYLEY de desarrollo infantil -III | 1 |
| Protocol (for publication) | Apendice 7 Escala de Impacto en la familia IFS | 1 |
| Protocol (for publication) | Apendice 8 Escala de evaluacion de calidad de vida SF-36 | 1 |
| Protocol (for publication) | Apendice 9 EscalaFLACC | 1 |
| Protocol (for publication) | Protocol MLC HyC2024 v1 of 13022025 | 1 |
| Protocol (for publication) | Protocolo ensayo clinico MLCHyC 2024 V3 del 04062025 | 3 |
| Protocol (for publication) | Protocolo ensayo clinico MLCHyC 2024 V3 del 04062025 CC | 3 |
| Protocol (for publication) | Protocolo ensayo clinico MLCHyC2024 V2 08052025 | 2 |
| Protocol (for publication) | Protocolo ensayo clinico MLCHyC2024 V2 08052025 CC | 2 |
| Recruitment arrangements (for publication) | MLC-Hcy-2024 Recruitment arrangement _v1 11102024 | 1 |
| Subject information and informed consent form (for publication) | MLC HYC2024 HIP CI Padres V2 08052025 | 1 |
| Subject information and informed consent form (for publication) | MLC HYC2024 HIP CI Padres V2 08052025 CC | 1 |
| Subject information and informed consent form (for publication) | MLC HYC2024 HIP CI Padres_def | 1 |
| Subject information and informed consent form (for publication) | MLC HYC2024 HIP CI Padres_V3 04062025 | 3 |
| Subject information and informed consent form (for publication) | MLC HYC2024 HIP CI Padres_V3 04062025 CC | 3 |
| Summary of Product Characteristics (SmPC) (for publication) | RESUMEN DE LAS CARACTERISTICAS DEL PRODUCTO EXPERIMENTAL | 2 |
| Summary of Product Characteristics (SmPC) (for publication) | SmPC Hidroxicobalamina Mar 2023 | 1 |
| Synopsis of the protocol (for publication) | Synopsis protocol MLC HyC 2024 V1 of 13022025 | 1 |
Application history
1 submissions — initial application plus substantial / non-substantial modifications
| # | Type | Code | Submitted | Reference MS | Conclusion | Decision date |
|---|---|---|---|---|---|---|
| 1 | INITIAL | IN | 2025-03-03 | Spain | Acceptable with conditions 2025-06-09
|
2025-06-09 |