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NCT00107783 · ClinicalTrials.gov registry record · Phase 2
Long-Term Study of Nitisinone to Treat Alkaptonuria
A Phase 2 study, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 2
- Development phase
- 40
- Enrollment target
NCT00107783 is a Phase 2 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 40 participants.
The verdict
NCT00107783, a Phase 2 study, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 40 participants
- Enrollment target
Study Summary
This 3-year study will examine the safety and effectiveness of long-term use of nitisinone (Orfadin) for treating joint problems in patients with alkaptonuria, an inherited disease in which a compound called homogentisic acid accumulates. The excess homogentisic acid causes arthritis and limited joint movement. It can also cause heart valve damage and kidney stones. Patients between 30 and 80 years of age with alkaptonuria may be eligible for this study. Patients must have hip involvement, but at least one remaining hip joint. Candidates are recruited from among patients enrolled in protocol 00-HG-0141, "Clinical, Biochemical, and Molecular Investigations into Alkaptonuria." Participants may enter both protocols simultaneously. Participants are randomly assigned to one of two treatment groups: one group takes their regular medicines plus a 2-mg nitisinone capsule daily; the other group takes only their regular medicines. Patients taking nitisinone have blood tests to measure liver function 2 weeks and 6 weeks after starting treatment. Before starting therapy, all patients are admitted to the NIH Clinical Center for 4-5 days to undergo the following procedures: * Medical history and physical examination * 24-hour urine collection to test for sugar, protein, and other molecules * Blood tests for liver and thyroid function, blood counts, and blood chemistries * Blood and urine tests to measure tyrosine and other amino acids and homogentisic acid * Bone x-rays * Spiral CT (computed tomography) of the abdomen to detect kidney stones * Eye examination and evaluations by specialists in rehabilitation medicine and pain, plus other consults in skin, brain, lung, heart, and kidney, as needed All patients, whether or not they receive nitisinone, return to the Clinical Center for a 2-3 day follow-up admission every 4 months for a history and physical examination, blood tests, and two 24-hour urine collections. Every 12 months (12, 24 and 36 months after starting the study),
Interventions
- DRUG Nitisinone (NTBC)
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 40 participants |
| Start Date | 2005-01 |
| Est. Completion | 2009-04 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00107783
The ClinicalTrials.gov registry entry for NCT00107783 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 40 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Human Genome Research Institute (NHGRI), which has 185 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Nitisinone (NTBC) is the first listed.
NCT00107783 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00107783 about?
NCT00107783 is a clinical study titled "Long-Term Study of Nitisinone to Treat Alkaptonuria". This 3-year study will examine the safety and effectiveness of long-term use of nitisinone (Orfadin) for treating joint problems in patients with alkaptonuria, an inherited disease in which a compound called homogentisic acid accumulates. The excess homogentisic acid causes arthritis and limited joi...
What is the current status of trial NCT00107783?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 40 participants. The study started on 2005-01. Estimated completion is 2009-04.
What interventions are being tested in trial NCT00107783?
The interventions under investigation include: Nitisinone (NTBC) (DRUG).
Who is sponsoring clinical trial NCT00107783?
This trial is sponsored by National Human Genome Research Institute (NHGRI), which has 185 total clinical trials registered on ClinicalTrials.gov.
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