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NCT02096471 · ClinicalTrials.gov registry record · Phase 2
MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1
A Phase 2 study of Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN, sponsored by University of Alabama at Birmingham.
- Completed
- Registry status
- Phase 2
- Development phase
- 19
- Enrollment target
- 11
- Study locations
NCT02096471: Completed Phase 2 study of Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN, sponsored by University of Alabama at Birmingham.
NCT02096471 is a Phase 2 study of Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN that has completed, run by University of Alabama at Birmingham. The registered enrollment target is 19 participants, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (86% lower). The trial reports 11 study locations across 11 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT02096471, a Phase 2 study of Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN, has completed, sponsored by University of Alabama at Birmingham.
- COMPLETED
- Registry status
- Phase 2
- Development phase
- 19 participants
- Enrollment target
- 11
- Study locations
Study Summary
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
Primary Outcome
Response is assessed by the NCI-POB at the time that follow-up 3D-MRI scans are performed (after course 4, 8, 12, and then after completion of every 6 courses thereafter). For the purpose of determining the level of response (complete, partial, etc.) measurements from the follow-up scans are compared to the target lesion size in the pretreatment MRI scan using 3D data analysis.Complete Response (CR): A complete resolution of the target plexiform neurofibroma for ≥ 4 weeks Partial Response (PR):
Conditions Studied
Interventions
- DRUG PD-0325901
Study Locations (11)
California
- Children's Hospital Los Angeles - Los Angeles
District of Columbia
- Children's National Medical Center - Washington D.C.
Illinois
- University of Chicago - Chicago
Indiana
- Indiana Unversity - Indianapolis
Maryland
- National Cancer Institute (NCI) - Bethesda
Massachusetts
- Children's Hospital Boston - Boston
Missouri
- Washington University - St. Louis - St Louis
New York
- New York University Medical Center - New York
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 19 participants |
| Start Date | 2014-06 |
| Est. Completion | 2018-08-01 |
| Phase | Phase 2 |
What the finished NCT02096471 record still lists
NCT02096471 is an interventional study that assigns participants to a tested intervention. The registry caps enrollment at 19 participants, a relatively small participant target, below the 133-participant average among 30,622 other Phase 2 trials with a reported enrollment target (86% lower).
The record links to 1 condition, with Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN appearing as the primary indexed condition, and to 1 intervention - of which PD-0325901 is the first listed.
NCT02096471 lists 11 locations in 11 states (California, District of Columbia, Illinois).
Frequently Asked Questions
What is clinical trial NCT02096471 about?
NCT02096471 is a clinical study titled "MEK Inhibitor PD-0325901 Trial in Adolescents and Adults With NF1". 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. Sub...
What is the current status of trial NCT02096471?
This trial is currently completed. It is a Phase 2 study. The enrollment target is 19 participants. The study started on 2014-06. Estimated completion is 2018-08-01.
What conditions does trial NCT02096471 study?
This clinical trial studies the following conditions: Neurofibromatosis Type 1 and Growing or Symptomatic, Inoperable PN.
What interventions are being tested in trial NCT02096471?
The interventions under investigation include: PD-0325901 (DRUG).
Who is sponsoring clinical trial NCT02096471?
This trial is sponsored by University of Alabama at Birmingham, which has 1,160 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT02096471 being conducted?
This trial has 11 study locations across California, District of Columbia, Illinois, Indiana, Maryland. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
How this trial's enrollment target compares
Where NCT02096471's enrollment target sits among peer trials
19 1894th of 2000 higher than 103 of 2,000 other Phase 2 trials
participants (enrollment target), bucketed by value
Each bar is a band; taller bars hold more other Phase 2 trials. The dashed line + filled bar mark this entry. Hover or tap any bar for its full count and share, and where it sits relative to this entry.
Source ClinicalTrials.gov registry export · 2026-08-08
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