MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1
Public ClinicalTrials.gov record NCT02096471. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official title
A Phase 2 Trial of the MEK Inhibitor PD-0325901 in Adolescents and Adults With NF1-Associated Morbid Plexiform Neurofibromas
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
This phase II open label study will evaluate adolescents (≥ 16 years of age) and adults with neurofibromatosis type-1 (NF1) and plexiform neurofibromas treated with the MEK inhibitor PD-0325901. The primary aim of the study will be to assess quantitative radiographic response in a target lesion. Subjects will receive PD-0325901 by mouth on a bid dosing schedule of 2 mg/m2/dose with a maximum dose of 4 mg bid. Each course is 4 weeks duration, and subjects will receive drug on a 3 week on/1 week off schedule. Subjects may receive additional courses beyond course 8 only if there is at least 15% reduction in volume of the target tumor. Subjects who have a 20% or greater reduction in target tumor volume at the end of 12 courses can continue on therapy for up to an additional year (maximum of 24 total courses). However, subjects who do not achieve at least 15% reduction in volume of the target tumor after 8 courses (\~8 months) will be considered treatment failures and taken off study. The Primary purpose of this protocol is to determine whether PD-0325901 results in objective radiographic responses based on volumetric MRI measurements in adolescents and adults with NF1 and growing or symptomatic inoperable PN. There are several secondary aims of this protocol: To evaluate the feasibility and toxicity of chronic PD-0325901 administration in this patient population To estimate the objective response rate of up to 2 non-target plexiform neurofibromas to PD-0325901 by MRI To characterize the pharmacokinetic profile of PD-0325901 when administered to this patient population To evaluate quality of life and pain during treatment with PD-0325901
Study identification
- NCT ID
- NCT02096471
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 19 participants
Conditions and interventions
Eligibility (public fields only)
- Age range
- 16 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- May 31, 2014
- Primary completion
- Aug 8, 2017
- Completion
- Jul 31, 2018
- Last update posted
- Jan 9, 2019
2014 – 2018
United States locations
- U.S. sites
- 11
- U.S. states
- 11
- U.S. cities
- 11
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Children's Hospital Los Angeles | Los Angeles | California | 90027 | — |
| Children's National Medical Center | Washington D.C. | District of Columbia | 20010 | — |
| University of Chicago | Chicago | Illinois | 60637 | — |
| Indiana Unversity | Indianapolis | Indiana | 46202 | — |
| National Cancer Institute (NCI) | Bethesda | Maryland | 20892 | — |
| Children's Hospital Boston | Boston | Massachusetts | 02115 | — |
| Washington University - St. Louis | St Louis | Missouri | 63110 | — |
| New York University Medical Center | New York | New York | 10016 | — |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | — |
| Children's Hospital of Philadelphia | Philadelphia | Pennsylvania | 19096 | — |
| University of Utah | Salt Lake City | Utah | 84132 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT02096471, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Jan 9, 2019 · Synced Sep 10, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT02096471 live on ClinicalTrials.gov.