Medical Information Only. Always consult your healthcare provider before enrolling in any clinical trial.
NCT03962543 · ClinicalTrials.gov registry record · Phase 2
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
A Phase 2 study of Plexiform Neurofibroma and Neurofibromatosis Type 1 (NF1), sponsored by SpringWorks Therapeutics.
- Active
- Registry status
- Phase 2
- Development phase
- 114
- Enrollment target
- 20
- Study locations
NCT03962543: Active Phase 2 study of Plexiform Neurofibroma and Neurofibromatosis Type 1 (NF1), sponsored by SpringWorks Therapeutics.
NCT03962543 is a Phase 2 study of Plexiform Neurofibroma and Neurofibromatosis Type 1 (NF1) that is active but no longer recruiting, run by SpringWorks Therapeutics. The registered enrollment target is 114 participants, below the 240-participant average among 7 other Plexiform Neurofibroma trials with a reported enrollment target (52% lower). The trial reports 20 study locations across 10 states. According to ClinicalTrials.gov, the official US trial registry.
The verdict
NCT03962543, a Phase 2 study of Plexiform Neurofibroma and Neurofibromatosis Type 1 (NF1), is active but no longer recruiting, sponsored by SpringWorks Therapeutics.
- ACTIVE NOT RECRUITING
- Registry status
- Phase 2
- Development phase
- 114 participants
- Enrollment target
- 20
- Study locations
Study Summary
This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901). Eligible participants may continue in a long-term follow-up phase.
Primary Outcome
Response will be determined by a blinded centralized review of volumetric MRI. The confirmed objective response rate (complete or partial response) by the end of Treatment Phase (i.e., Cycle 24) is defined as the proportion of participants who have a confirmed ≥ 20% reduction in target tumor volume as compared to baseline as assessed by a BICR, and the response needs to be confirmed by BICR in a consecutive tumor assessment within 2 - 6 months. Partial response is defined as a ≥ 20% reduction in
Conditions Studied
Interventions
- DRUG Mirdametinib (PD-0325901) oral capsule or dispersible tablet
Study Locations (20)
California
- Children's Hospital Los Angeles - Los Angeles
- UCLA Oncology Center - Los Angeles
- University of California - Irvine Health - Orange
- Children's Hospital of Orange County - Orange
- Lucile Packard Children's Hospital Stanford - Palo Alto
- University of California - Davis Comprehensive Cancer Center - Sacramento
Florida
- University of Florida Clinical Research Center - Gainesville
- Nicklaus Children's Hospital - Miami
- AdventHealth Pediatric Oncology Hematology at Orlando - Orlando
- Orlando Health, Inc. - Orlando
- Johns Hopkins All Children's Hospital - St. Petersburg
- Moffitt Cancer Center - Tampa
Alabama
- University of Alabama at Birmingham/Children's of Alabama - Birmingham
Arizona
- Mayo Clinic Hospital - Phoenix
Arkansas
- Arkansas Children's Hospital - Little Rock
Colorado
- Children's Hospital Colorado - Aurora
Delaware
- Nemours A. I. duPont Hospital for Children - Wilmington
District of Columbia
- Children's National Medical Center - Washington D.C.
Trial Details
| Field | Value |
|---|---|
| Enrollment Target | 114 participants |
| Start Date | 2019-09-29 |
| Est. Completion | 2028-12-22 |
| Phase | Phase 2 |
What the registry record for NCT03962543 still lists
NCT03962543 is an interventional study that assigns participants to a tested intervention. The registered 114 participants enrollment target is mid-sized for trials with a published cap, below the 240-participant average among 7 other Plexiform Neurofibroma trials with a reported enrollment target (52% lower).
The record links to 2 conditions, with Plexiform Neurofibroma appearing as the primary indexed condition, and to 1 intervention - of which Mirdametinib (PD-0325901) oral capsule or dispersible tablet is the first listed.
NCT03962543 names 20 study sites across 10 states, led by California, Florida, Alabama.
Frequently Asked Questions
What is clinical trial NCT03962543 about?
NCT03962543 is a clinical study titled "MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas". This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901). Eligible participants may continue in a long-term follow-up phase.
What is the current status of trial NCT03962543?
This trial is currently active not recruiting. It is a Phase 2 study. The enrollment target is 114 participants. The study started on 2019-09-29. Estimated completion is 2028-12-22.
What conditions does trial NCT03962543 study?
This clinical trial studies the following conditions: Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1).
What interventions are being tested in trial NCT03962543?
The interventions under investigation include: Mirdametinib (PD-0325901) oral capsule or dispersible tablet (DRUG).
Who is sponsoring clinical trial NCT03962543?
This trial is sponsored by SpringWorks Therapeutics, which has 7 total clinical trials registered on ClinicalTrials.gov.
Where is trial NCT03962543 being conducted?
This trial has 20 study locations across Alabama, Arizona, Arkansas, California, Colorado. Contact the study sites directly through ClinicalTrials.gov for enrollment availability.
Learn More About Clinical Trials
Similar trials for Plexiform Neurofibroma
Matched on the same primary condition, ranked to surface studies in the same phase first, then by recruiting status, no relevance scoring or editorial curation.
-
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
RECRUITING · Phase 2
-
Neurofibromatosis Type 1 Tumor Early Detection Study
RECRUITING
-
A Study of Avutometinib for People With Solid Tumor Cancers
RECRUITING · Phase 1
-
Natural History Study of Patients With Neurofibromatosis Type I
ACTIVE NOT RECRUITING
-
Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1
ACTIVE NOT RECRUITING · Phase 1
-
Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1
ACTIVE NOT RECRUITING · NA
Nationwide trials with similar profiles
Cross-condition peers matched on enrollment target and registry start date, not the same-condition list above.
Similar enrollment target
- NCT04522791 · 114 participants · Phase 2
Breathing, Relaxation, Attention Training, & Health in Older Adults (BREATHE)
- NCT04875052 · 114 participants · NA
Vibration and Post-traumatic Osteoarthritis Risk Following ACL Injury
- NCT05100342 · 114 participants
Prospective Validation of a Points Score System Predicting 30-day Survival
- NCT05224089 · 114 participants · Phase 4
Bilateral TAP and RS Blocks Using Liposomal Bupivacaine/Bupivacaine vs. Regular Bupivacaine in Laparoscopic Colectomy
Similar registry start date
- NCT05962346 · started 2026-12 · NA
Fetal Endoscopic Tracheal Occlusion for Congenital Diaphragmatic Hernia
- NCT07125183 · started 2026-12 · Phase 2
Study on Efficacy and Tolerability of Weekly Doxorubicin in Elderly Patients With Advanced or Metastatic Leiomyosarcoma
- NCT07292298 · started 2026-11 · Phase 2
Phase 2 Single-Arm Rectal Cancer Brachytherapy for Patients With Low-Lying Residual Adenocarcinoma After Total Neoadjuvant Therapy to Improve Organ Preservation Rates
- NCT04263285 · started 2026-10 · NA
Treatment of Depression Post-SCI