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NCT02281760 · ClinicalTrials.gov registry record · Phase 2
Dabrafenib and Trametinib in People With BRAF V600E Mutation Positive Lesions in Erdheim Chester Disease
A Phase 2 study, sponsored by National Human Genome Research Institute (NHGRI).
- Completed
- Registry status
- Phase 2
- Development phase
- 9
- Enrollment target
NCT02281760 is a Phase 2 study that has completed, run by National Human Genome Research Institute (NHGRI). The registered enrollment target is 9 participants.
The verdict
NCT02281760, a Phase 2 study, has completed, sponsored by National Human Genome Research Institute (NHGRI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 9 participants
- Enrollment target
Study Summary
Erdheim-Chester Diseases (ECD) is a very rare non-Langerhans cell histiocytosis of unknown origin and pathogenesis. It has been reported mainly in adult males over the age of 40 years, although cases have been reported in females as well. Children are rarely affected. Mutation of the BRAF gene, specifically BRAFV600E, has been recently identified in 50% of Erdheim Chester lesions in a French cohort. This somatic mutation is believed to be the driver mutation in positive cases. The clinical characteristics of ECD range from asymptomatic to multisystemic involvement; longitudinal progression and natural history are becoming better understood. ECD commonly affects the bones, kidneys, retroperitoneal space, skin and brain. If untreated, the disease progresses rapidly, causing fatal outcomes due to severe lung disease, chronic renal failure, cardiomyopathy and other complications. The diagnosis of ECD relies upon imaging studies and specific pathologic findings in biopsies of affected organs, i.e., fibrosis and infiltration of tissues with foamy histiocytes, lymphocytes, and plasma cells. Immunohistochemistry reveals cells positive for CD68 and CD163 and negative for CD1a, with 20% positivity to S-100. There is no standard treatment for ECD, although chemotherapy, radiation, stem cell transplantation, alpha-interferon, anakinra, imatinib and sirolimus have been proposed. The recent discovery of the BRAFV600E mutation in several ECD patients has opened a new area for treatment options. Vemurafenib, an FDA approved BRAF inhibitor for the treatment of patients with metastatic or unresectable melanoma with the V600E mutation, binds to this form of mutated BRAF causing protein inactivation. The use of vemurafenib in patients with ECD has been reported in 3 patients who experienced remission of the disease, and is currently being studied in the U.S. and Europe as monotherapy. Tumor/disease resistance to vemurafenib has occurred in melanoma and other cancers, although it has no
Interventions
- DRUG Dabrafenib Mesylate
- DRUG Trametinib Dimethyl Sulfoxide
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 9 participants |
| Start Date | 2014-11-01 |
| Est. Completion | 2018-08-29 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT02281760
The ClinicalTrials.gov registry entry for NCT02281760 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 9 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 2 interventions - of which Dabrafenib Mesylate is the first listed.
NCT02281760 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT02281760 about?
NCT02281760 is a clinical study titled "Dabrafenib and Trametinib in People With BRAF V600E Mutation Positive Lesions in Erdheim Chester Disease". Erdheim-Chester Diseases (ECD) is a very rare non-Langerhans cell histiocytosis of unknown origin and pathogenesis. It has been reported mainly in adult males over the age of 40 years, although cases have been reported in females as well. Children are rarely affected. Mutation of the BRAF gene, spec...
What is the current status of trial NCT02281760?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 9 participants. The study started on 2014-11-01. Estimated completion is 2018-08-29.
What interventions are being tested in trial NCT02281760?
The interventions under investigation include: Dabrafenib Mesylate (DRUG), Trametinib Dimethyl Sulfoxide (DRUG).
Who is sponsoring clinical trial NCT02281760?
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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