NCT05509153 · Phase 2 · RECRUITING
A Randomised Controlled Trial, Of N-Acetyl Cysteine (NAC), for Premanifest Huntingtin Gene Expansion Carriers
This trial is testing whether N-Acetyl Cysteine, a supplement-derived antioxidant drug, can slow early brain changes in people who carry the Huntington disease gene but have not yet developed symptoms. Researchers will track clinical and MRI outcomes over three years. This is a Phase 2 trial, meaning they are gathering evidence on whether it works and is safe, not a proven or approved treatment.
You may qualify if
- Able to provide informed consent
- Huntingtin gene expansion carrier with >= 39 CAG repeats
- Absence of unequivocal motor signs of HD - that is, UHDRS
- Diagnostic Confidence Level needs to be \<4 upon enrolment
- Expected to develop clinical HD within 10 years of trial enrolment using the Langbehn formula
- Availability of an informant for corroborative history
- Negative serum pregnancy test for women of childbearing potential
- If of childbearing potential, is able and agrees to remain abstinent or use adequate contraceptive methods
- Ability to tolerate MRI scans
- Ability to tolerate blood draws
- Able to comply with all study protocol requirements, according to the investigators judgement
- In the opinion of the investigator, medically, psychiatrically and neurologically stable at the time of enrolment
You're excluded if
- Diagnosis of clinical HD
- Known hypersensitivity to NAC
- Pregnancy, breastfeeding or intention to do so prior to the end of the study
- Exposure to any investigational drugs within 30 days of Baseline Visit
- Use of supplemental NAC
- Abnormalities in laboratory measurements, ECG or vital signs at screening, which precludes safe participation in the study
- Current or history of substance abuse within one year of Baseline visit
- Unstable psychiatric or acute medical illness including cancer, as determined by investigator
- Current use of antipsychotic medications or Tetrabenazine
- History of gene therapy, cell transplantation, or any experimental brain surgery
- History of attempted suicide or suicidal ideation within 12 months prior to screening
- Pre-existing structural brain lesion as assessed by a centrally read MRI scan during the screening period
The sponsor's own eligibility wording, lightly reformatted. The study team makes the final eligibility decision — worth discussing with your doctor.
Eligibility criteria as of 2025-02-28