Combined Thiamine and Biotin Treatment for Huntington's Disease

Multicentric Trial on the Use of Combined Therapy of Thiamine and Biotine in Patients With Huntington´s Disease

This study is testing if a combination of thiamine and biotin can help slow down symptoms in people with mild to moderate Huntington's disease.

Quick facts

• Phase: Phase 2
• Study type: Interventional
• Enrollment: 24 (estimated)
• Ages: 18 Years and up
• Sex: All
• Sponsor: Fundación Pública Andaluza para la gestión de la Investigación en Sevilla (Academic / other)
• Locations: 3 sites (San Sebastián, San Sebastian and 2 other locations)
• Trial ID: NCT04478734 on ClinicalTrials.gov

What this trial studies

This trial evaluates the safety and tolerability of combined oral thiamine and biotin therapy in patients with mild to moderate Huntington's disease. The study aims to assess the biological effects of the treatment by measuring thiamine monophosphate levels in cerebrospinal fluid over a one-year follow-up period. Participants will undergo periodic clinical examinations and monitoring for adverse effects, alongside analytical assessments of thiamine levels in both cerebrospinal fluid and blood. The ultimate goal is to determine if this combination therapy can modify the disease course or slow symptom progression in early-stage Huntington's disease patients.

Who should consider this trial

Good fit: Ideal candidates are adults with manifest Huntington's disease exhibiting motor symptoms and genetic confirmation of the condition.

Not a fit: Patients with significant medical comorbidities or those who are pregnant or lactating may not benefit from this study.

Why it matters

Potential benefit: If successful, this treatment could potentially slow the progression of Huntington's disease symptoms in early-stage patients.

How similar studies have performed: While this approach is novel, other studies have explored thiamine and biotin individually, but combined therapy in this context is less common.

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).

Where this trial is running

San Sebastián, San Sebastian and 2 other locations

• Hospital Universitario de San Sebastián — San Sebastián, San Sebastian, Spain (Recruiting)
• Virgen del Rocío Hospital — Seville, Seville, Spain (Recruiting)
• Hospital Ramón y Cajal — Madrid, Spain (Recruiting)

Study contacts

• Principal investigator: Clara M. Rosso Fernández — Virgen del Rocío University Hospital Research and Clinical Trials Unit
• Study coordinator: Pablo Mir Rivera, MD/PhD

Conditions Huntington Disease; thiamine and biotine; combination therapy