CompletedPhase 2

Homocystinuria: Treatment With N-Acetylcysteine

Canada5 participantsStarted 2007-11

Plain-language summary

The purpose of this study is determine if oral N-acetylcysteine is effective in lowering homocysteine in individuals with homocystinuria.

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion Criteria: * Homocystinuria (lens dislocation and hyperhomocysteinemia) * Age ≥ 18 (the age of majority in Canada) Exclusion Criteria: * Nursing mothers or pregnant women * Chronic liver disease * Taking nitrates * Cystine stone formers * History of active peptic ulcer disease * Subjects receiving carbamazepine and metoclopramide * Use of other products containing cysteine or N-acetylcysteine (e.g. nebulized NAC, cysteine supplements, methionine restriction) * Hypersensitivity to any ingredient in the study product * Clinically significant, abnormal laboratory test on screening (Visit 2) Other Criteria: * Women of child-bearing capacity must be using an acceptable method of birth control and have a negative pregnancy test before being enrolled

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Lowering plasma total homocysteine

Timeframe: 3 months

Trial details

NCT IDNCT00483314

SponsorMcGill University Health Centre/Research Institute of the McGill University Health Centre

Sponsor typeOTHER

Study typeINTERVENTIONAL

Primary completion2008-12

View on ClinicalTrials.gov More Homocystinuria trials

Who can participate

Age range

18 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.