Neurofibroma, Plexiform clinical trials
The registry lists 63 studies for Neurofibroma, Plexiform across more than 10 countries. 19 are recruiting or about to start. 19 of them are mainly about Neurofibroma, Plexiform (how this is decided). Per ClinicalTrials.gov, last updated 2026-11-09.
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Who runs them
National Cancer Institute (NCI) (18) · AstraZeneca (6) · SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany (3) · Indiana University (3) · Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd. (3) · Novartis Pharmaceuticals (3) · University of Alabama at Birmingham (3) · Children's Hospital of Philadelphia (2)
What is being tested here
Drugs under test in Neurofibroma, Plexiform studies, those with the most open studies first. Medicines only given beside them are not counted. How drugs are counted
| Drug | Open | Phase 3 | All |
|---|---|---|---|
| AZD 6244 | 3 | 1 | 10 |
| Luvometinib | 1 | 1 | 3 |
| Cabozantinib | 1 | 0 | 2 |
| Pexidartinib | 1 | 0 | 1 |
| Tunlametinib | 1 | 0 | 1 |
| Trametinib | 0 | 0 | 4 |
| Pirfenidone | 0 | 0 | 3 |
| Everolimus | 0 | 0 | 2 |
| Imatinib | 0 | 0 | 2 |
| Mirdametinib | 0 | 0 | 1 |
Drug classes tested here Tyrosine kinase inhibitors (TKIs) 2 open
Coming up
Studies recruiting or running, from their records. “Main data collection” ends at the primary completion date, the sponsor’s own estimate, which often moves.
All 9
- Nov 2026Pediatric Long-Term Follow-up and Rollover Study · Novartis Pharmaceuticals
- Dec 2026Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ... · National Cancer Institute (NCI)
- Jan 2027AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors · National Cancer Institute (NCI)
- Mar 2027Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway. · St. Justine's Hospital
- May 2027A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1) · National Cancer Institute (NCI)
- Jun 2027Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1 · Ann & Robert H Lurie Children's Hospital of Chicago
- Jun 2027A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1 · Healx Limited
- Jun 2027Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs · AstraZeneca
- Jun 2027Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs) · National Cancer Institute (NCI)
Who these studies take
Of the 13 Neurofibroma, Plexiform studies recruiting now, 7 (54%) set no upper age limit and 2 (15%) stop at 75 or younger. 9 (69%) take children under 18. 0 (0%) are for women only and 0 (0%) for men only; 1 (8%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 6 (46%) | 6 (46%) |
| 18 | 12 (92%) | 12 (92%) |
| 30 | 12 (92%) | 12 (92%) |
| 40 | 11 (85%) | 11 (85%) |
| 50 | 11 (85%) | 11 (85%) |
| 65 | 11 (85%) | 11 (85%) |
| 75 | 11 (85%) | 11 (85%) |
| 85 | 10 (77%) | 10 (77%) |
Across all 63 studies, recruiting or not: 51% stop at 75 or younger and 46% at 65; 79% take children; 0% are for women only and 0% for men only; 3% take healthy volunteers. From each study’s registry entry; the criteria on its page decide.
Studies
Recruiting first, then most recently updated. Showing 50 of 63. Browse all 63 → or search them.
- Phase I Trial of TURALIO(R) (Pexidartinib, PLX3397) in Children and Young Adults With Refractory Leukemias and Refractory Solid Tumors Including Neurofibromatosis Type 1 (NF1) Associated Plexiform Neurofibromas (PN) and Tenosynovial Giant Cell Tumor ...RecruitingNCT02390752Phase 11 country
- Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)RecruitingNCT062222031 country
- Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)RecruitingNCT025440221 country
- PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform NeurofibromasRecruitingNCT06961565Phase 13 countries
- A Study of Mirdametinib in Infants and Toddlers With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasNot yet recruitingNCT07850635Phase 11 country
- A Study to Evaluate the Feasibility of a Physiologic Biomarker to Assess Pain and Other Sensory Problems Using Pupillometry in Participants With Neurofibromatosis Type 1 (NF1)RecruitingNCT06507748Not applicable1 country
- Individual Patient Compassionate Use of MirdametinibAvailableNCT050281662 countries
- Study of Cabozantinib With Selumetinib for Plexiform NeurofibromasRecruitingNCT06502171Phase 11 country
- Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1Enrolling by invitationNCT052389091 country
- Neurofibromatosis Type 1 Tumor Early Detection StudyEnrolling by invitationNCT065158601 country
- Real-World Treatment Study of Koselugo (Selumetinib)RecruitingNCT063604061 country
- A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1RecruitingNCT06541847Phase 21 country
- Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1RecruitingNCT06188741Phase 21 country
- Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform NeurofibromasRecruitingNCT07407803Phase 31 country
- Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1Not yet recruitingNCT07024394Phase 1/21 country
- Low-dose Selumetinib for the Treatment of Plexiform Neurofibromas in Chinese ChildrenNot yet recruitingNCT06763315Phase 21 country
- A Retrospective Study on Epidemiological Characteristics of Chinese NF1 Patients in Real World (PROMISE)RecruitingNCT063792301 country
- Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1RecruitingNCT05199376Not applicable1 country
- HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasRecruitingNCT05331105Phase 21 country
- Natural History Study of Patients With Neurofibromatosis Type IActive, not recruitingNCT009241961 country
- A Study of Avutometinib for People With Solid Tumor CancersCompletedNCT06104488Phase 11 country
- AZD6244 Hydrogen Sulfate for Children With Nervous System TumorsActive, not recruitingNCT01362803Phase 1/21 country
- MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasActive, not recruitingNCT03962543Phase 21 country
- Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNsActive, not recruitingNCT061756371 country
- MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform NeurofibromasActive, not recruitingNCT02407405Phase 21 country
- Pediatric Long-Term Follow-up and Rollover StudyActive, not recruitingNCT03975829Phase 419 countries
- A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)Active, not recruitingNCT04590235Phase 11 country
- Trametinib for Pediatric Neuro-oncology Patients With Refractory Tumor and Activation of the MAPK/ERK Pathway.Active, not recruitingNCT03363217Phase 21 country
- Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform NeurofibromasActive, not recruitingNCT04924608Phase 313 countries
- Targeting the Mechanisms Underlying Cutaneous Neurofibroma Formation in NF1: A Clinical Translational Approach.CompletedNCT027777751 country
- Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform NeurofibromasCompletedNCT03231306Phase 21 country
- US Selumetinib RegistryTerminatedNCT056836781 country
- FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform NeurofibromasActive, not recruitingNCT05913037Phase 31 country
- Study to Evaluate the Safety, Tolerability, PK Characteristics and Anti-tumor Activity of FCN-159 in Adult and Pediatric Participants With Neurofibromatosis Type 1Active, not recruitingNCT04954001Phase 1/23 countries
- Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment TrialsCompletedNCT03531814Not applicable1 country
- Treatment of NF1-related Plexiform Neurofibroma With TrametinibActive, not recruitingNCT03741101Phase 21 country
- Intermittent Dosing Of Selumetinib In Childhood NF1 Associated TumoursCompletedNCT03326388Phase 1/21 country
- Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled StudyWithdrawnNCT05825365Phase 2
- Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and AdultsCompletedNCT02101736Phase 21 country
- Acceptance and Commitment Training for Adolescents and Young Adults With Neurofibromatosis Type 1, Plexiform Neurofibromas, and Chronic PainCompletedNCT02471339Phase 31 country
- Study to Investigate Safety, Pharmacokinetic (PK), Pharmacodynamic (PD) and Clinical Activity of Trametinib in Subjects With Cancer or Plexiform Neurofibromas and Trametinib in Combination With Dabrafenib in Subjects With Cancers Harboring V600 MutationsCompletedNCT02124772Phase 1/25 countries
- PET/MRI in CNS and Extra-CNS Tumors of Patients With Neurofibromatosis-1 (NF1)CompletedNCT018000321 country
- Whole Body MRI to Identify Atypical Neurofibromas in Patients With NF1Active, not recruitingNCT03820778Not applicable1 country
- Study of Imatinib in Children With Neurofibromatosis and Airway TumorsWithdrawnNCT03688568Phase 21 country
- Clinical Sequencing of Cancer and Tissue Repository: OncoGenomicsWithdrawnNCT030274011 country
- Study of Plexiform Neurofibromas in Neurofibromatosis Type 1CompletedNCT000064351 country
- An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1Approved for marketingNCT032596331 country
- Childhood Cancer and Plexiform Neurofibroma Tissue Microarray for Molecular Target Screening and Clinical Drug DevelopmentCompletedNCT003405221 country
- Sorafenib to Treat Children and Young Adults With Neurofibromatosis Type 1 and Inoperable Plexiform NeurofibromasCompletedNCT00727233Phase 11 country
- Study of Imatinib Mesylate in Neurofibromatosis Type I Patients Aged 2 to 21 With Plexiform NeurofibromasTerminatedNCT02177825Phase 21 country