Premanifest Huntington's Disease Extension Study II: Creatine Safety & Tolerability (Pre-CREST-2X)
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Purpose
The purpose of this clinical trial is to extend the Pre-Crest-X study to further assess the long-term safety and tolerability of up to 30 grams daily creatine in individuals at-risk for Huntington's Disease (HD) and to assess whether biomarkers responsive to creatine in symptomatic individuals are informative in premanifest individuals over a longer duration.
| Condition | Intervention | Phase |
|---|---|---|
|
Huntington's Disease |
Drug: Creatine monohydrate |
Phase 2 |
| Study Type: | Interventional |
| Study Design: | Endpoint Classification: Safety Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment |
| Official Title: | Premanifest Huntington's Disease Extension Study II: Creatine Safety & Tolerability |
- Safety [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]Frequency of adverse events
- Tolerability [ Time Frame: 24 months ] [ Designated as safety issue: Yes ]Proportion of subjects completing the extension study at given dose level
- Clinical measures [ Time Frame: 24 months ] [ Designated as safety issue: No ]Components of the UHDRS (Unified Huntington Disease Rating Scale)
- Biological Markers of Disease Progression [ Time Frame: 24 months ] [ Designated as safety issue: No ]Biological indicators that creatine treatment might affect the progression of HD: plasma levels of creatine, serum levels of 8OH2'dG and 8OHG, magnetic resonance imaging (MRI), morphometric neuroimaging (biomarker of neurodegeneration), metabolomic profiling, and gene expression analysis to assess transcriptional effects of HD and creatine therapy.
| Estimated Enrollment: | 60 |
| Study Start Date: | April 2010 |
| Estimated Study Completion Date: | March 2013 |
| Estimated Primary Completion Date: | March 2013 (Final data collection date for primary outcome measure) |
-
Drug: Creatine monohydrate
Extensive evidence exists that neurodegeneration begins many years before HD can be diagnosed clinically. Therefore, it is most desirable to begin a neuroprotective therapy before or during this premanifest period with the aim of delaying onset, as well as slowing functional decline. Cellular energy depletion is present early in HD and can be ameliorated by creatine, which helps regenerate cellular ATP. Preclinical evidence for creatine's potential neuroprotective effects in animal models of HD has been well-documented. Before the clinical efficacy of creatine can be tested in premanifest HD, its long-term safety and tolerability must be assessed in these individuals and its ability to favorably modify biomarkers of HD should also be confirmed. This extension trial will continue to follow eligible individuals who completed the Pre-CREST-X extension study on open-label creatine (up to 30 grams daily) for long term safety and tolerability for an additional 24 months. Other biological and imaging biomarkers of disease progression and potential response to treatment will also be assessed.
Eligibility| Ages Eligible for Study: | 26 Years and older |
| Genders Eligible for Study: | Both |
| Accepts Healthy Volunteers: | No |
Inclusion Criteria:
- Individuals who have completed the Pre-CREST Study.
- Individuals capable of providing independent informed consent and complying with trial procedures.
Exclusion Criteria:
-Clinical evidence of unstable medical or psychiatric illness in the investigator's judgment.
Additional eligibility criteria apply.
Contacts and Locations| United States, Massachusetts | |
| Massachusetts General Hospital | |
| Charlestown, Massachusetts, United States, 02129 | |
| Principal Investigator: | Diana Rosas, MD, MS | Massachusetts General Hospital |
More Information
Publications:
| Responsible Party: | Steven M. Hersch, Professor of Neurology, Massachusetts General Hospital |
| ClinicalTrials.gov Identifier: | NCT01411163 History of Changes |
| Other Study ID Numbers: | 2010P000511 |
| Study First Received: | August 4, 2011 |
| Last Updated: | December 11, 2012 |
| Health Authority: | United States: Food and Drug Administration United States: Institutional Review Board United States: Federal Government |
Keywords provided by Massachusetts General Hospital:
|
Premanifest At-Risk Huntington's Disease HD |
Additional relevant MeSH terms:
|
Huntington Disease Basal Ganglia Diseases Brain Diseases Central Nervous System Diseases Nervous System Diseases Dementia Chorea Dyskinesias |
Movement Disorders Heredodegenerative Disorders, Nervous System Neurodegenerative Diseases Genetic Diseases, Inborn Cognition Disorders Delirium, Dementia, Amnestic, Cognitive Disorders Mental Disorders |
ClinicalTrials.gov processed this record on May 21, 2013