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NCT01981551 · ClinicalTrials.gov registry record · Phase 2
Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis
A Phase 2 study, sponsored by National Cancer Institute (NCI).
- Completed
- Registry status
- Phase 2
- Development phase
- 17
- Enrollment target
NCT01981551: Completed Phase 2 study, sponsored by National Cancer Institute (NCI).
NCT01981551 is a Phase 2 study that has completed, run by National Cancer Institute (NCI). The registered enrollment target is 17 participants. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT01981551, a Phase 2 study, has completed, sponsored by National Cancer Institute (NCI).
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 17 participants
- Enrollment target
Study Summary
Background: * Desmoid tumors (also known as aggressive fibromatosis), are rare, locally invasive, slow-growing soft-tissue tumors. The disease can be either asymptomatic or be associated with severe loss of organ function and significant morbidity. * Treatment with the selective small-molecule Gamma-secretase inhibitor PF-03084014 caused significant tumor shrinkage in patients with unresectable desmoid tumors in an early phase clinical trial. * The Notch pathway is a key regulator of cell differentiation, proliferation and apoptosis; aberrant signaling via the Notch pathway is associated with carcinogenesis. Objectives: * Primary: Determine the response rate (Complete Response (CR)+Partial Response (PR)) of PF-03084014 in patients with desmoid tumors/aggressive fibromatosis * Secondary: Assess symptom measures at baseline and on study; perform genotyping for germline and somatic mutations in adenomatous polyposis coli gene (APC) and catenin-beta 1 (CTNNB1) genes; correlate clinical response to therapy with genotyping data; and assess modulation of the Notch pathway by evaluating notch response genes in tumor biopsies at baseline and after drug administration Eligibility: * Age greater than or equal to18; histologically confirmed desmoid tumor not amenable to curative resection or definitive radiation therapy that has progressed after receiving at least one line of standard treatment; adequate organ function * Willingness to provide blood samples and 10 unstained slides or a tumor block for genetic research studies Study Design: * This is an open-label Phase II trial of PF-03084014; study drug will be administered orally at 150 mg twice a day in 21-day cycles * Optional tumor biopsies for research purposes will be performed at baseline prior to study treatment and at the beginning of cycle 7 (+/- one cycle) * Restaging scans (magnetic resonance imaging (MRI) with diffusion weighting) will be performed at baseline, at the end of cycles 1 and 6, and then every 6
Interventions
- DRUG PF-03084014
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 17 participants |
| Start Date | 2013-10-31 |
| Est. Completion | 2023-12-01 |
| Phase | Phase 2 |
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Full Details on ClinicalTrials.gov ↗What the Registry Record Tells You About NCT01981551
The ClinicalTrials.gov registry entry for NCT01981551 describes a study currently listed as completed, categorized as Phase 2. The registered enrollment target is 17 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 PF-03084014 is the first listed.
NCT01981551 reports 0 study locations.
Frequently Asked Questions
What is clinical trial NCT01981551 about?
NCT01981551 is a clinical study titled "Phase II Trial of the Gamma-Secretase Inhibitor PF-03084014 in Adults With Desmoid Tumors/Aggressive Fibromatosis". Background: * Desmoid tumors (also known as aggressive fibromatosis), are rare, locally invasive, slow-growing soft-tissue tumors. The disease can be either asymptomatic or be associated with severe loss of organ function and significant morbidity. * Treatment with the selective small-molecule Gamm...
What is the current status of trial NCT01981551?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 17 participants. The study started on 2013-10-31. Estimated completion is 2023-12-01.
What interventions are being tested in trial NCT01981551?
The interventions under investigation include: PF-03084014 (DRUG).
Who is sponsoring clinical trial NCT01981551?
This trial is sponsored by National Cancer Institute (NCI), which has 3,257 total clinical trials registered on ClinicalTrials.gov.
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