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NCT00076102 · ClinicalTrials.gov registry record · Phase 2
Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas
A Phase 2 study, sponsored by National Cancer Institute (NCI).
- Completed
- Registry status
- Phase 2
- Development phase
- 36
- Enrollment target
NCT00076102: Completed Phase 2 study, sponsored by National Cancer Institute (NCI).
NCT00076102 is a Phase 2 study that has completed, run by National Cancer Institute (NCI). The registered enrollment target is 36 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT00076102, a Phase 2 study, has completed, sponsored by National Cancer Institute (NCI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 36 participants
- Enrollment target
Study Summary
Background: Neurofibromatosis Type 1 (NF1) is an autosomal dominant, progressive genetic disorder characterized by diverse clinical manifestations. Patients with NF1 have an increased risk of developing tumors of the central and peripheral nervous system including plexiform neurofibromas, which are benign nerve sheath tumors that may cause severe morbidity and possible mortality. The histopathology of these tumors suggests that events connected with formation of fibroblasts might constitute a point of molecular vulnerability. Gene profile analysis demonstrates overexpression of fibroblast growth factor, epidermal growth factor, and platelet-derived growth factor in plexiform neurofibromas in patients with NF1. Pirfenidone is a novel antifibrotic agent that inhibits these and other growth factors. Clinical experience in adults has demonstrated that pirfenidone is effective in a variety of fibrosing conditions and pirfenidone is presently under study in a phase II trial for adults with progressive plexiform neurofibromas. A phase I trial of pirfenidone in children and young adults with NF1 and plexiform neurofibromas was completed, and has established the phase II dose (the dose resulting in a mean drug exposure \[AUC\] not more than 1 standard deviation below the mean drug exposure \[AUC\] in adults who received pirfenidone at the dose level demonstrating activity in fibrosing conditions). Pirfenidone has been well tolerated. Objectives: To determine whether pirfenidone increases the time to disease progression based on volumetric measurements in children and young adults with NF1 and growing plexiform neurofibromas. To define the objective response rate to pirfenidone in NF1-related plexiform neurofibromas. To describe and define the toxicities of pirfenidone. Eligibility: Individuals (greater than or equal to 3 years to less than or equal to 21 years of age) with a clinical diagnosis of NF1 and inoperable, measurable, and progressive plexiform neurofibromas
Interventions
- DRUG Pirfenidone
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 36 participants |
| Start Date | 2004-07-21 |
| Est. Completion | 2010-04-01 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT00076102
The ClinicalTrials.gov registry entry for NCT00076102 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 36 participants, a figure that helps gauge the scale of data the investigators plan to collect. The listed sponsor is National Cancer Institute (NCI), which has 3,257 total studies on file at ClinicalTrials.gov.
The record links to 0 conditions, and to 1 intervention - of which Pirfenidone is the first listed.
NCT00076102 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT00076102 about?
NCT00076102 is a clinical study titled "Pirfenidone in Children and Young Adults With Neurofibromatosis Type I and Progressive Plexiform Neurofibromas". Background: Neurofibromatosis Type 1 (NF1) is an autosomal dominant, progressive genetic disorder characterized by diverse clinical manifestations. Patients with NF1 have an increased risk of developing tumors of the central and peripheral nervous system including plexiform neurofibromas, which are...
What is the current status of trial NCT00076102?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 36 participants. The study started on 2004-07-21. Estimated completion is 2010-04-01.
What interventions are being tested in trial NCT00076102?
The interventions under investigation include: Pirfenidone (DRUG).
Who is sponsoring clinical trial NCT00076102?
This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.
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