Trial of the Combined Use of Thiamine and Biotin in Patients With Huntington's Disease

• Status: Not yet recruiting
• Trial end date: 2023-06-01
• Target enrollment: 0
• Participant gender: All

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

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Fundación Pública Andaluza para la gestión de la Investigación en Sevilla
• Collaborator: Ciberned (Centro de Investigación Biomédica en Red)
• Treatments: Biotin; Thiamine

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).