Investigating Nicotinamide Riboside for Huntington's Disease
The NAD-HD Study: A Parallel-group, Phase 2, Double-blind Study to Investigate the Efficacy and Safety of Oral Nicotinamide Riboside Compared With Placebo in Participants Aged 18 to 80 Years With Huntington's Disease
This study is testing if a vitamin B3 supplement called nicotinamide riboside can help slow down Huntington's disease in adults compared to a placebo.
Quick facts
| Phase | Phase 2 |
|---|---|
| Study type | Interventional |
| Enrollment | 120 (estimated) |
| Ages | 18 Years to 80 Years |
| Sex | All |
| Sponsor | Oslo University Hospital Academic / other |
| Locations | 1 site (Oslo) |
| Trial ID | NCT06853743 on ClinicalTrials.gov |
What this trial studies
This clinical trial aims to evaluate the efficacy and safety of nicotinamide riboside (NR), a form of vitamin B3, in slowing the progression of Huntington's disease in adults. Participants will receive either 2000mg of NR or a placebo daily for two years, with regular clinic visits for assessments and brain imaging. The study will measure overall symptom burden, specific symptom domains, and potential side effects, as well as analyze biomarkers related to the disease. By comparing NR to a placebo, researchers hope to determine its effectiveness in treating Huntington's disease.
Who should consider this trial
Good fit: Ideal candidates are adults with confirmed Huntington's disease gene expansion and early to mid-stage symptoms.
Not a fit: Patients with late-stage Huntington's disease or those without confirmed gene expansion may not benefit from this study.
Why it matters
Potential benefit: If successful, this study could provide a new therapeutic option to slow the progression of Huntington's disease.
How similar studies have performed: Other studies have explored similar neuroprotective approaches, but the specific use of nicotinamide riboside in Huntington's disease is relatively novel.
Eligibility criteria
Show full inclusion / exclusion criteria
Inclusion Criteria: * Confirmed HD gene expansion carrier status with a diagnostic genetic test confirming ≥36 CAG-repeats in HTT. * Clinically manifest HD with objective neurological signs corresponding to Diagnostic Confidence Level 4 based on the Total Motor Score of the UHDRS. * Early or mid-stage disease corresponding to Shoulson-Fahn stage 1-340 and Total Functional Capacity (TFC) \> 2. * Ability to walk indoors unassisted or by the help of walking aids only as determined at screening and baseline visits. * Ability to write and perform pen-and-paper tests (SDMT, SWRT, MoCA) and complete questionnaires (HADS-SIS, SF-12) included in the study protocol. * Ability to follow up telephone appointments and reliably attend study visits independently or with the assistance of a reliable partner (family member, friend, or assistant). * Ability to tolerate blood draws. * Participants who are women of childbearing potential should use an approved method for highly effective birth control throughout the study intervention period * Capable of giving signed informed consent Exclusion Criteria: * Presence of other co-morbid neurological or psychiatric disorders considered clinically significant by the investigator, including, but not limited to psychotic disorders, brain tumor or inflammatory neurological disease. * Attempted suicide or active suicidal ideation within 12 months prior to screening. * A history of alcohol or substance use within a 12 month period prior to the baseline visit, fulfilling criteria of dependence or abuse under the International Classification of Diseases (ICD-10) F10-F19 (not including tobacco dependence). * Any malignancy within 5 years prior to screening (except basal or squamous cell carcinoma of the skin or carcinoma in situ of the cervix that has been successfully treated) or history of a previously treated malignant disorder \> 5 years prior to screening with a remaining clinically significant recurrence risk. * Established coronary artery disease or clinically significant cerebrovascular disease as deemed by the investigator. * High age-specific cardiovascular risk as defined by the NORRISK2 algorithm based on smoking status, systolic blood pressure, serum total cholesterol and family history, modeled on the Norwegian population * Any medical condition which, in the investigator's judgement, may preclude the subject's safe participation in the study, interfere with the ability to comply with study procedures or confound the interpretation of study results. * Use of vitamin B3 supplements in any form or dose during the study or within 3 months prior to screening. * Planned major surgery of any kind during the study period that is likely to affect clinical ratings, including, but not limited to, joint replacement and spinal surgery. * Electroconvulsive therapy. * Any medical therapy with known severe side effects that, in the investigator's judgement, may preclude the subject's safe participation in the study, interfere with the ability to comply with study procedures or confound the interpretation of study results. * Any history of gene therapy exposure for the treatment of HD.
Where this trial is running
Oslo
- Oslo University Hospital — Oslo, Norway (Recruiting)
Study contacts
- Principal investigator: Lasse Pihlstrøm, PhD — Oslo University Hospital
- Study coordinator: Ellen H Maurtveten, BSc
- Email: emaurtve@ous-hf.no
- Phone: +47 95143795
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.