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| Sponsor: | McGill University Health Center |
|---|---|
| Collaborator: |
March of Dimes |
| Information provided by: | McGill University Health Center |
| ClinicalTrials.gov Identifier: | NCT00483314 |
Purpose
The purpose of this study is determine if oral N-acetylcysteine is effective in lowering homocysteine in individuals with homocystinuria.
| Condition | Intervention | Phase |
|---|---|---|
|
Homocystinuria |
Drug: N-acetylcysteine |
Phase II |
| Study Type: | Interventional |
| Study Design: | Treatment, Non-Randomized, Open Label, Uncontrolled, Single Group Assignment, Efficacy Study |
| Official Title: | Homocystinuria: Treatment With N-Acetylcysteine |
| Enrollment: | 5 |
| Study Start Date: | November 2007 |
| Study Completion Date: | February 2009 |
| Primary Completion Date: | December 2008 (Final data collection date for primary outcome measure) |
| Arms | Assigned Interventions |
|---|---|
| 1: Experimental |
Drug: N-acetylcysteine
2 g p.o. BID x 60 days
|
Homocystinuria (MIM 236200) due to CBS deficiency is the most common inborn error of sulfur amino acid metabolism with severe clinical manifestations. We propose:
An open-label pilot study of N-acetylcysteine (NAC) to lower plasma homocysteine levels in those that have not responded to conventional treatment which includes betaine (Cystadane®, Orphan Medical Inc.), which while lowering Hcy levels does not normalize it, and is very expensive. There are no known contraindications to NAC used for nutritional supplementation and it is relatively inexpensive.
Oral NAC has reduced total plasma homocysteine in healthy subjects in a dose-dependent fashion.
Eligibility| Ages Eligible for Study: | 18 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
Exclusion Criteria:
Other Criteria:
Contacts and Locations| Canada, Quebec | |
| Royal Victoria Hospital | |
| Montreal, Quebec, Canada, H3A 1A1 | |
| MUHC-Royal Victoria Hospital | |
| Montreal, Quebec, Canada, H3A 1A1 | |
| Principal Investigator: | Brian M GILFIX, MD, PhD | McGill University Health Center |
More Information
| Responsible Party: | McGill University Health Centre ( Brian M. Gilfix, Principal Investigator ) |
| Study ID Numbers: | #6-FY06-317 |
| Study First Received: | June 5, 2007 |
| Last Updated: | February 16, 2009 |
| ClinicalTrials.gov Identifier: | NCT00483314 History of Changes |
| Health Authority: | Canada: Health Canada |
|
Respiratory System Agents Anti-Infective Agents Antioxidants Molecular Mechanisms of Pharmacological Action Physiological Effects of Drugs Brain Diseases Metabolism, Inborn Errors Therapeutic Uses Free Radical Scavengers Connective Tissue Diseases Acetylcysteine Brain Diseases, Metabolic, Inborn Antidotes |
Metabolic Diseases Amino Acid Metabolism, Inborn Errors Nervous System Diseases Central Nervous System Diseases Homocystinuria Antiviral Agents Protective Agents Pharmacologic Actions Genetic Diseases, Inborn Expectorants N-monoacetylcystine Brain Diseases, Metabolic |