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NCT00975689 · ClinicalTrials.gov registry record · Phase 1
Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine
A Phase 1 study of Niemann-Pick Disease, Type C, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- Completed
- Registry status
- Phase 1
- Development phase
- 35
- Enrollment target
- 1
- Study location
NCT00975689 is a Phase 1 study of Niemann-Pick Disease, Type C that has completed, run by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD). The registered enrollment target is 35 participants, below the 351-participant average among 3 other Niemann-Pick Disease, Type C trials with a reported enrollment target (90% lower). The trial reports 1 study location across 1 state.
The verdict
NCT00975689, a Phase 1 study of Niemann-Pick Disease, Type C, has completed, sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD).
- COMPLETED
- Registry status
- Phase 1
- Development phase
- 35 participants
- Enrollment target
- 1
- Study location
Study Summary
Background: * Niemann-Pick disease type C (NPC) is a genetic disorder that results in progressive loss of nervous system function by affecting the membranes of nerve cells. There is no known cure for NPC. * N-acetyl cysteine (NAC) is a drug that has been approved by the Food and Drug Administration to use either orally or IV for the treatment of acetaminophen (Tylenol) poisoning or as an aerosol to reduce the stickiness of mucous in patients with cystic fibrosis. In the body, NAC is converted to an amino acid called cysteine, which cells can convert to a chemical called glutathione. Glutathione is important in helping cells deal with oxidative stress. Based on a number of experiments in cells, mice and patients with NPC, we believe that oxidative stress is increased in NPC. If we can increase glutathione levels, we may be able to decrease the oxidative stress. Objectives: \- To test the safety and effectiveness of N-acetyl cysteine to treat Niemann-Pick disease (type C). Eligibility: \- Individuals at least 1 year of age who have been diagnosed with NPC. Design: * Patients entering this study will be seen at the National Institutes of Health Clinical Center four times during the 20 weeks of the study. These admissions will occur at the start of the study and at weeks 8, 12, and 20. The first NIH visit will last 2 days, and the other visits will last 1 day. * Patients will participate in a two-stage study: a period of 8 weeks receiving NAC and a second period of 8 weeks when receiving a placebo (a pill without NAC). Every patient participating in this study will receive NAC during one of the two time periods. * The two treatment periods will be separated by a wash-out period, 4 weeks when patients will receive neither NAC nor placebo. Patients will also have a 4-week wash-out period at the beginning of the study. Most physician-prescribed medications, such as seizure medications, will be allowed. * During each visit, examinations, procedures, and tests will be
Conditions Studied
Interventions
- DRUG N-Acetyl Cysteine
Study Locations (1)
Maryland
- National Institutes of Health Clinical Center, 9000 Rockville Pike - Bethesda
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 35 participants |
| Start Date | 2009-08 |
| Est. Completion | 2010-11 |
| Phase | Phase 1 |
Sponsor
Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD)305 total trials
Interested in This Trial?
Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00975689
The ClinicalTrials.gov registry entry for NCT00975689 describes a study currently listed as completed, categorized as Phase 1. The registered enrollment target is 35 participants, a figure that helps gauge the scale of data the investigators plan to collect, below the 351-participant average among 3 other Niemann-Pick Disease, Type C trials with a reported enrollment target (90% lower). The listed sponsor is Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total studies on file at ClinicalTrials.gov.
The record links to 1 condition, with Niemann-Pick Disease, Type C appearing as the primary indexed condition, and to 1 intervention - of which N-Acetyl Cysteine is the first listed.
NCT00975689 reports 1 study location spanning 1 distinct geographic area - top geographies include Maryland.
Frequently Asked Questions
What is clinical trial NCT00975689 about?
NCT00975689 is a clinical study titled "Biomarker Validation for Niemann-Pick Disease, Type C: Safety and Efficacy of N-Acetyl Cysteine". Background: * Niemann-Pick disease type C (NPC) is a genetic disorder that results in progressive loss of nervous system function by affecting the membranes of nerve cells. There is no known cure for NPC. * N-acetyl cysteine (NAC) is a drug that has been approved by the Food and Drug Administration...
What is the current status of trial NCT00975689?
This trial is currently completed. It is a Phase 1 study. The enrollment target is 35 participants. The study started on 2009-08. Estimated completion is 2010-11.
What conditions does trial NCT00975689 study?
This clinical trial studies the following conditions: Niemann-Pick Disease, Type C.
What interventions are being tested in trial NCT00975689?
The interventions under investigation include: N-Acetyl Cysteine (DRUG).
Who is sponsoring clinical trial NCT00975689?
This trial is sponsored by Eunice Kennedy Shriver National Institute of Child Health and Human Development (NICHD), which has 305 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT00975689 being conducted?
This trial has 1 study location across Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
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