Trial of the Combined Use of Thiamine and Biotin in Patients With Huntington's Disease (HUNTIAM)
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|ClinicalTrials.gov Identifier: NCT04478734|
Recruitment Status : Recruiting
First Posted : July 21, 2020
Last Update Posted : May 6, 2023
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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
|Condition or disease||Intervention/treatment||Phase|
|Huntington Disease||Drug: Moderate doses of Thiamine y Biotin Drug: High doses of Thiamine y Biotin||Phase 2|
|Study Type :||Interventional (Clinical Trial)|
|Estimated Enrollment :||24 participants|
|Intervention Model:||Parallel Assignment|
|Masking:||None (Open Label)|
|Official Title:||Multicentric Trial on the Use of Combined Therapy of Thiamine and Biotine in Patients With Huntington´s Disease|
|Actual Study Start Date :||April 12, 2023|
|Estimated Primary Completion Date :||June 30, 2025|
|Estimated Study Completion Date :||December 30, 2025|
Experimental: Moderate doses
moderate doses of combination therapy applying the minimum average dosage of thiamine and biotin used in patients with BTBGD
Drug: Moderate doses of Thiamine y Biotin
Thiamine 600 mg every day + Biotin 150mg every day
Other Name: moderate doses of combination therapy
Experimental: High doses
high doses of the combination therapy applying the average standard dosage of thiamine and biotin used in patients with BTBGD.
Drug: High doses of Thiamine y Biotin
Thiamine 1200 mg every day + Biotin 300mg every day
Other Name: high doses of the combination therapy
- Incidence of Treatment-Emergent Adverse Events as assessed by clinical examination anamnesis and Analytical monitoring with hematological and biochemical control (hepatic and renal function) [ Time Frame: From signature of informed consent form, at all scheduled visits, to end of follow up week 52 ]Patient´s condition and emergence of comorbidity by clinical examination and anamnesis directed by a neurologist, by measuring of vital signs (blood pressure, heart rate,breath rate weight and height)
- The evaluation of the efficacy of treatment with combined oral thiamine and biotin therapy in increasing thiamine monophosphate (TMP) levels in CSF of patients with HD [ Time Frame: At baseline (week 0) and visit 8 (week 48) ]Determination and comparison of thiamine levels (free, TMP and TTP) in CSF and blood at the beginning and the end of the treatment.
- Evaluate the biological effect of the combined thiamine-biotin oral therapy in the neurodegeration of HD patients [ Time Frame: At baseline (week 0) and visit 8 (week 48) ]Measurement of the change in CSF NfL levels of patients with HD treated with the combined thiamine-biotin therapy
- Evaluate the biological effect of the combined thiamine-biotin oral therapy in the neuroimaging progression markers in patients with HD [ Time Frame: At baseline (week 0) and visit 8 (week 48) ]Measurement of the change in the volume of the caudate nucleus, white matter and cortical thickness, as well as in the combined cerebral atrophy score; comparing these values with those described in prospective registries of patients with HD.
- Evaluate the effect of the combined thiamine-biotin oral therapy in the quality of life of patients with HD [ Time Frame: At baseline (week 0), week 24 and week 48 ]Changes in the 36-Item Short Form Health Survey (SF-36) score of the quality of life. Short Form 36 Health Survey (SF-36 ) consists of 35 punctually items, divided into 8 dimensions: Physical Function, Physical Role, Emotional Role, Social Function, Mental Health, General Health, Body Pain and Vitality. It also contains an additional item that is not part of any dimension and that measures the change in health over time. The scores of the 8 dimensions of SF-36 are arranged in such a way that the higher the value recorded, the better the corresponding health status. scale (SF-36).
- Evaluate the clinical effect of the combined thiamine-biotin oral therapy in the severity of motor symptoms of patients with HD [ Time Frame: At baseline (week 0), week 24 and week 48 ]Evaluate changes in the score of the UHDRS motor scale and UHDRS- Total functional capacity.
- Evaluate the clinical effect of the combined thiamine-biotin oral therapy in the severity of bradykinesia of patients with HD. [ Time Frame: At baseline (week 0), week 12, week 24, week 36 and week 48 ]Evaluate changes in the score of Quantitative-motor assessments (Q-motor). Measurement of bradykinesia will be done through quantitative movement measurement techniques.
- Evaluate the effect of the combined thiamine-biotin oral therapy in the global severity of disease of patients with HD [ Time Frame: At baseline (week 0), week 24 and week 48 ]
Changes in the score of The Clinical Global Impressions - Severity scale-S (CGI-C).
The CGI provides a brief of the patient's global functioning prior to and after initiating a study medication.
Comprises two measures evaluating the following: (a) severity of psychopathology from 1 to 7 and (b) change from the initiation of treatment on a similar seven-point scale. CGI-C scores range from 1 (very much improved) through to 7 (very much worse).
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
|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 (IONIS-HTTRX (RG6042).
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04478734
|Contact: Pablo Mir Rivera, MD/PhD||+34 email@example.com|
|Contact: Clara M. Rosso Fernández, MD/PhD||+34 firstname.lastname@example.org|
|Virgen del Rocío Hospital||Not yet recruiting|
|Sevilla, Seville, Spain, 41013|
|Contact: Clara M Rosso Fernández, MD/PhD +34 955012144 email@example.com|
|Contact: Pablo Mir Rivera, MD/PhD +34955923039 firstname.lastname@example.org|
|Principal Investigator: Pablo Mir Rivera, MD/PhD|
|Hospital Virgen del Rocio||Recruiting|
|Sevilla, Spain, 41013|
|Contact: Pablo Mir Rivera, MD 679150952 email@example.com|
|Study Director:||Pablo Mir Rivera, MD/PhD||Institute of Biomedicine of Seville (IBiS)|
|Principal Investigator:||Clara M. Rosso Fernández||Virgen del Rocío University Hospital Research and Clinical Trials Unit|
|Responsible Party:||Fundación Pública Andaluza para la gestión de la Investigación en Sevilla|
|Other Study ID Numbers:||
|First Posted:||July 21, 2020 Key Record Dates|
|Last Update Posted:||May 6, 2023|
|Last Verified:||May 2023|
|Individual Participant Data (IPD) Sharing Statement:|
|Plan to Share IPD:||Yes|
|Plan Description:||Once the study is completed and the data processed, the results will be shared|
Clinical Study Report (CSR)
|Time Frame:||After the primary completion date and submit results information|
|Access Criteria:||collaborating researchers|
|Studies a U.S. FDA-regulated Drug Product:||No|
|Studies a U.S. FDA-regulated Device Product:||No|
|Product Manufactured in and Exported from the U.S.:||No|
thiamine and biotine
Basal Ganglia Diseases
Central Nervous System Diseases
Nervous System Diseases
Heredodegenerative Disorders, Nervous System
Genetic Diseases, Inborn
Vitamin B Complex
Physiological Effects of Drugs