Clinical trial
Multicentric Trial on the Use of Combined Therapy of Thiamine and Biotine in Patients With Huntington´s Disease
Name
HUNTIAM
Description
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
Trial arms
Trial start
2023-04-12
Estimated PCD
2025-06-30
Trial end
2025-12-30
Status
Recruiting
Phase
Early phase I
Treatment
Moderate doses of Thiamine y Biotin
Thiamine 600 mg every day + Biotin 150mg every day
Arms:
Moderate doses
Other names:
moderate doses of combination therapy
High doses of Thiamine y Biotin
Thiamine 1200 mg every day + Biotin 300mg every day
Arms:
High doses
Other names:
high doses of the combination therapy
Size
24
Primary endpoint
Incidence of Treatment-Emergent Adverse Events as assessed by clinical examination anamnesis and Analytical monitoring with hematological and biochemical control (hepatic and renal function)
From signature of informed consent form, at all scheduled visits, to end of follow up week 52
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).
Protocol
{'studyType': 'INTERVENTIONAL', 'phases': ['PHASE2'], 'designInfo': {'allocation': 'RANDOMIZED', 'interventionModel': 'PARALLEL', 'primaryPurpose': 'TREATMENT', 'maskingInfo': {'masking': 'NONE'}}, 'enrollmentInfo': {'count': 24, 'type': 'ESTIMATED'}}
Updated at
2024-06-05
1 organization
1 product
1 indication
Product
Thiamine + BiotinIndication
Huntington's disease