Neurofibromatosis type 1 trials in United States

In United States 27 Also abroad 40

27 trials · From ClinicalTrials.gov, checked 15 November 2026. Ask the study team whether a trial fits you.

Phase 3Taking people
A Study of the Drugs Selumetinib vs. Carboplatin and Vincristine in Patients With Low-Grade Glioma
Where
Akron, Albany, Ann Arbor, Atlanta, Aurora, Austin, Baltimore, Bangor, Bethesda, Birmingham, Boise, Boston, Buffalo, Burlington, Chapel Hill, Charleston, Charlotte, Chicago, Cincinnati, Cleveland, Columbia, Columbus, Dallas, Danville, Dayton, Des Moines, Detroit, Durham, El Paso, Fargo, Fort Myers, Fort Worth, Gainesville, Grand Rapids, Greenville, Hartford, Hershey, Hollywood, Honolulu, Houston, Indianapolis, Iowa City, Jackson, Jacksonville, Kansas City, Knoxville, Lexington, Little Rock, Loma Linda, Long Beach, Los Angeles, Louisville, Lubbock, Madison, Memphis, Mesa, Miami, Milwaukee, Mineola, Minneapolis, Morgantown, Morristown, Nashville, New Haven, New Hyde Park, New Orleans, New York, Norfolk, Oakland, Oklahoma City, Omaha, Orange, Orlando, Palo Alto, Pensacola, Peoria, Philadelphia, Phoenix, Pittsburgh, Portland, Providence, Richmond, Roanoke, Rochester, Royal Oak, Salt Lake City, San Antonio, San Diego, San Francisco, Scarborough, Seattle, Spokane, Springfield, St Louis, St. Petersburg, Syracuse, Tacoma, Tampa, Valhalla, Washington D.C., Wilmington, Winston-Salem
Age
2 years to 21 years
People
170 worldwide
Run by
National Cancer Institute (NCI)
See details and how to joinNCT04166409
Phase 1/2Taking people
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
Where
Bethesda
Age
12 years and over
People
55
Run by
National Cancer Institute (NCI)
See details and how to joinNCT04750928
Phase 1Taking people
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 ...
Where
Bethesda
Age
3 years to 35 years
People
54
Run by
National Cancer Institute (NCI)
See details and how to joinNCT02390752
No phaseTaking people
Surveillance for Malignant Transformation of Neurofibromatosis Type 1 (NF1) Related Peripheral Nerve Sheath Tumors (PNST)
Where
Bethesda
Age
3 years to 120 years
People
225
Run by
National Cancer Institute (NCI)
See details and how to joinNCT06222203
No phaseTaking people
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
Where
Baltimore, Bethesda, Chicago, Cincinnati, Philadelphia, Washington D.C.
Age
5 years and over
People
476
Run by
National Cancer Institute (NCI)
See details and how to joinNCT02544022
Phase 1Taking people
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
Where
Birmingham
Age
18 years and over
People
56 worldwide
Run by
Pasithea Therapeutics Corp.
See details and how to joinNCT06961565
No phaseTaking people
The NF Registry Sponsored by the Children's Tumor Foundation
Where
New York
People
20,000
Run by
The Children's Tumor Foundation
See details and how to joinNCT01885767
No phaseTaking people
Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial
Where
Bethesda
Age
18 years and over
People
70
Run by
National Cancer Institute (NCI)
See details and how to joinNCT05361811
No phaseTaking people
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)
Where
Bethesda, Washington D.C.
Age
1 year to 120 years
People
70
Run by
National Cancer Institute (NCI)
See details and how to joinNCT06507748
No phaseTaking people
Development of Patient-Reported Outcome Measures Assessing Tumor Visibility and Appearance Concerns in Neurofibromatosis Type 1: A Qualitative Study
Where
Bethesda
Age
5 years to 120 years
People
110
Run by
National Cancer Institute (NCI)
See details and how to joinNCT06880991
Phase 2Not yet taking people in United States
Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma
Where
Birmingham
Age
12 years to 89 years
People
55
Run by
University of Alabama at Birmingham
See details and how to joinNCT07521657
Phase 1Taking people
Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas
Where
Birmingham, Boston, Cincinnati, Dallas, Indianapolis, Los Angeles, Philadelphia, Washington D.C.
Age
16 years and over
People
30
Run by
Girish Dhall, MD
See details and how to joinNCT06502171
No phaseEnrolling by invitation
Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1
Where
Chicago
People
200
Run by
Ann & Robert H Lurie Children's Hospital of Chicago
See details and how to joinNCT05238909
Phase 1/2Taking people
A Phase I/II Study of Trametinib and Azacitidine for Patients With Newly Diagnosed Juvenile Myelomonocytic Leukemia
Where
Ann Arbor, Atlanta, Baltimore, Charlotte, Chicago, Cincinnati, Dallas, Denver, Indianapolis, Kansas City, Los Angeles, Memphis, Miami, New York, Philadelphia, Phoenix, Portland, Salt Lake City, San Francisco, Seattle, Washington D.C.
Age
1 month to 21 years
People
58
Run by
Therapeutic Advances in Childhood Leukemia Consortium
See details and how to joinNCT05849662
No phaseEnrolling by invitation
Neurofibromatosis Type 1 Tumor Early Detection Study
Where
Boston
Age
18 years and over
People
1,000
Run by
David Miller
See details and how to joinNCT06515860
Phase 2Taking people
Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas
Where
Birmingham
Age
18 years to 99 years
People
41
Run by
University of Alabama at Birmingham
See details and how to joinNCT05253131
Phase 2Taking people
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
Where
Aurora, Baltimore, Birmingham, Chicago, Cincinnati, Dallas, Indianapolis, Los Angeles, New York, Palo Alto, Philadelphia, St Louis, Washington D.C.
Age
12 years and over
People
25
Run by
Healx Limited
See details and how to joinNCT06541847
Phase 1/2Taking people
Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST
Where
Dallas, Washington D.C.
Age
18 years and over
People
45
Run by
AeRang Kim
See details and how to joinNCT06735820
No phaseTaking people
Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy
Where
Boston
Age
18 years and over
People
50
Run by
Massachusetts General Hospital
See details and how to joinNCT07233408
No phaseTaking people
Familial Investigations of Childhood Cancer Predisposition
Where
Memphis
People
1,500
Run by
St. Jude Children's Research Hospital
See details and how to joinNCT03050268
Phase 2Taking people
Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1
Where
Baltimore, Bethesda, Birmingham, Boston, Chicago, Cincinnati, Dallas, Indianapolis, Los Angeles, Palo Alto, Philadelphia, Rochester, St Louis, Washington D.C.
Age
1 year to 8 years
People
200
Run by
University of Alabama at Birmingham
See details and how to joinNCT06188741
Phase 1Taking people
Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
Where
Baltimore
Age
18 years and over
People
10
Run by
Johns Hopkins University
See details and how to joinNCT07102394
No phaseTaking people
Identification of Pre-Malignant Lesions In Pediatric Patients With Neurofibromatosis Type 1 Using Novel Magnetic Resonance Imaging Techniques Paired With Artificial Intelligence
Where
Los Angeles
Age
5 years to 17 years
People
15
Run by
Nicole Baca
See details and how to joinNCT04763109
Phase 1Enrolling by invitation
Assessing the Efficacy of Repeat, Monthly Treatments of Deoxycholate for NF1 Associated Cutaneous Neurofibromas (cNFs)
Where
Boston
Age
18 years to 85 years
People
15
Run by
Massachusetts General Hospital
See details and how to joinNCT06300502
Phase 1/2Taking people
Mirdametinib Monotherapy in Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF).
Where
Baltimore
Age
18 years and over
People
24
Run by
Johns Hopkins University
See details and how to joinNCT06159166