NCT04478734 Trial of the Combined Use of Thiamine and Biotin in Patients With Huntington's Disease
| NCT ID | NCT04478734 |
| Status | Recruiting |
| Phase | Phase 2 |
| Sponsor | Fundación Pública Andaluza para la gestión de la Investigación en Sevilla |
| Condition | Huntington Disease |
| Study Type | INTERVENTIONAL |
| Enrollment | 24 participants |
| Start Date | 2023-04-12 |
| Primary Completion | 2025-06-30 |
Eligibility & Interventions
Eligibility Fast-Check
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What to Expect as a Participant
You will actively receive the study intervention — which may be a drug, biologic, device, or procedure.
In Phase 2, researchers evaluate early signs of effectiveness. You may be randomized to receive the active treatment or a comparator. Monitoring continues closely.
This trial targets 24 participants in total. It began in 2023-04-12 with a primary completion date of 2025-06-30.
⚠ This information is for research awareness only. Always consult your physician before joining any clinical trial. Participation is voluntary and you may withdraw at any time.
Brief Summary
Evaluate the safety and tolerability of combined oral thiamine with biotin therapy in patients with Huntington´s disease in mild to moderate stages and it is intended to evaluate the biological effect of the treatment in the central nervous system of these patients using as the main biomarker the increase in the level of thiamine monophosphate (TMP) in cerebrospinal fluid (CSF) of these patients with Huntington Disease (HD) during a follow-up period of one year. Our main hypothesis is that combined thiamine-biotin oral therapy is a secure and well-tolerated treatment, potentially capable of modifying the disease course or avoiding the progression of symptoms in early-stages HD patients
Eligibility Criteria
Inclusion Criteria: * Patients of legal age with manifest Huntington's disease with motor symptoms (chorea, dystonia or bradykinesia) and/or neuropsychiatric; and genetic confirmation of a number of repetitions of the cytosine-adenine-guanine trinucleotide (CAG triplet) in the HTT gene (coding for HTT) greater than or equal to 39 * Patients should be capable of giving informed consent and attending the planned visit of the study. * Women of childbearing age should obtain a negative result in the serum or urine pregnancy test at the screening visit. They must also accept the use of appropriate contraceptive methods during the course of the clinical trial and men who have a partner of childbearing age, accept the use of contraceptive methods Exclusion Criteria: * Medical comorbidities considered clinically significant by the clinical judgment of the investigators. * Pregnancy or lactation * Patients with HD dependents on the basic routine daily life activities (UHDRS TFC \< 7) or a severe cognitive decline. * Active psychosis at the moment of the screening evaluation. * Severe renal failure. * Patients previously treated with thiamine and/or biotin or enrolled in other HD clinical trial with oligonucleotide antisense (IONIS-HTTRX (RG6042).
Contact & Investigator
Pablo Mir Rivera, MD/PhD
STUDY DIRECTOR
Institute of Biomedicine of Seville (IBiS)
Frequently Asked Questions
Who can join the NCT04478734 clinical trial?
This trial is open to participants of all sexes, aged 18 Years or older, studying Huntington Disease. Full inclusion and exclusion criteria are listed in the Eligibility Criteria section. Always confirm your eligibility with the research team before applying.
What phase is the NCT04478734 trial and what does that mean for participants?
Phase 2 trials evaluate whether the treatment shows signs of effectiveness while continuing to monitor safety. More participants are enrolled than in Phase 1 to help refine the treatment protocol.
Is NCT04478734 currently recruiting?
Yes, NCT04478734 is actively recruiting participants. Contact the research team at pmir@us.es for enrollment information.
Where is the NCT04478734 trial being conducted?
This trial is being conducted at San Sebastián, Spain, Seville, Spain, Madrid, Spain.
Who is sponsoring the NCT04478734 clinical trial?
NCT04478734 is sponsored by Fundación Pública Andaluza para la gestión de la Investigación en Sevilla. The principal investigator is Pablo Mir Rivera, MD/PhD at Institute of Biomedicine of Seville (IBiS). The trial plans to enroll 24 participants.