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

Who is this study for? Patients with Huntington´s disease
What treatments are being studied? Thiamine+Biotine
Status: Recruiting
Location: See all (3) locations...
Intervention Type: Drug
Study Type: Interventional
Study Phase: Phase 2
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
Participation Requirements
Sex: All
Minimum Age: 18
Healthy Volunteers: f
View:

• 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

Locations
Other Locations
Spain
Hospital Ramón y Cajal
RECRUITING
Madrid
Hospital Universitario de San Sebastián
RECRUITING
San Sebastián
Virgen del Rocío Hospital
RECRUITING
Seville
Contact Information
Primary
Pablo Mir Rivera, MD/PhD
pmir@us.es
+34 955923039
Backup
Clara M. Rosso Fernández, MD/PhD
claram.rosso.sspa@juntadeandalucia.es
+34 955012144
Time Frame
Start Date: 2023-04-12
Estimated Completion Date: 2025-12-30
Participants
Target number of participants: 24
Treatments
Experimental: Moderate doses
moderate doses of combination therapy applying the minimum average dosage of thiamine and biotin used in patients with BTBGD
Experimental: High doses
high doses of the combination therapy applying the average standard dosage of thiamine and biotin used in patients with BTBGD.
Sponsors
Collaborators: Ciberned (Centro de Investigación Biomédica en Red)
Leads: Fundación Pública Andaluza para la gestión de la Investigación en Sevilla

This content was sourced from clinicaltrials.gov