Combined Use of Thiamine and Biotin in Patients With Huntington's Disease (HUNTIAM)
Recruiting
SPONSOR
Fundación Pública Andaluza para la gestión de la Investigación en Sevilla
PARTICIPANTS
24
This is a phase 2 study to look at the safety and how well tolerated a combined thiamine with biotin therapy will be in HD patients in mild to moderate stages. They will evaluate the biological effect of the treatment in the central nervous system 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. The treatment will be oral(by mouth) over a year.
Our main hypothesis is that the 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
Ages Eligible
for Study:
18 Years and older (Adult, Older Adult )
Sexes Eligible
for Study:
All
Accepts Healthy Volunteers:
No
- 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
- 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
LOCATIONS
SPAIN
TRIAL SITE: Hospital y Cajal
Address: Madrid, Spain
Contact: Jose Luis Lopez-Sendon, MD
TRIAL SITE: Hospital Universitário de San Sebastian
Address: San Sebastain, Spain
Contact: Javier Ruiz Martinez, MD
TRIAL SITE: Virgen Del Ricio Hospital
Address: Sevilla, Spain
Contact: Pablo Mir Rivera
email: pmir@us.es