Neurofibromatoses clinical trials
The registry lists 57 studies for Neurofibromatoses across more than 10 countries. 14 are recruiting or about to start. 23 of them are mainly about Neurofibromatoses (how this is decided). Per ClinicalTrials.gov, last updated 2026-10-29. Counting its 2 narrower conditions too, such as Neurofibromatosis 1: 247 studies, 58 open.
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Who runs them
Massachusetts General Hospital (8) · National Cancer Institute (NCI) (5) · Indiana University (3) · Johns Hopkins University (2) · Memorial Sloan Kettering Cancer Center (2) · University of Alabama at Birmingham (2) · Assistance Publique - Hôpitaux de Paris (2) · SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany (2)
What is being tested here
Drugs under test in Neurofibromatoses 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 |
|---|---|---|---|
| Mirdametinib | 1 | 0 | 2 |
| Talimogene laherparepvec | 1 | 0 | 1 |
| Imatinib | 0 | 1 | 3 |
| Sunitinib | 0 | 0 | 2 |
| Binimetinib | 0 | 0 | 1 |
| Diphenylcyclopropenone | 0 | 0 | 1 |
| Cabozantinib | 0 | 0 | 1 |
| Aminolevulinic Acid | 0 | 0 | 1 |
| Dexmedetomidine | 0 | 0 | 1 |
| Tipifarnib | 0 | 0 | 1 |
Drug classes tested here Tyrosine kinase inhibitors (TKIs) 9
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 6
- Dec 2026Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1 · Johns Hopkins University
- May 2027Development of Non-Invasive Prenatal Diagnosis for Single Gene Disorders · Assistance Publique - Hôpitaux de Paris
- May 2027Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy · Massachusetts General Hospital
- Jul 2027Glioma Developmental and HyperActive Ras Tumor (DHART) Board · Memorial Sloan Kettering Cancer Center
- Sep 2027Neurocutaneous Syndromes in Children: Prevalence and Clinical Characteristics · Assiut University
- Oct 2027Mirdametinib in Patients With Advanced NF1-mutant Melanoma · Kevin Kim, MD
Who these studies take
Of the 10 Neurofibromatoses studies recruiting now, 9 (90%) set no upper age limit and 1 (10%) stop at 75 or younger. 3 (30%) take children under 18. 1 (10%) are for women only and 0 (0%) for men only; 0 (0%) take healthy volunteers.
| Age | Women | Men |
|---|---|---|
| 10 | 3 (30%) | 3 (30%) |
| 18 | 10 (100%) | 9 (90%) |
| 30 | 10 (100%) | 9 (90%) |
| 40 | 10 (100%) | 9 (90%) |
| 50 | 10 (100%) | 9 (90%) |
| 65 | 10 (100%) | 9 (90%) |
| 75 | 10 (100%) | 9 (90%) |
| 85 | 9 (90%) | 8 (80%) |
Across all 57 studies, recruiting or not: 35% stop at 75 or younger and 28% at 65; 58% take children; 2% are for women only and 0% for men only; 16% 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 57. Browse all 57 → or search them.
- PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform NeurofibromasRecruitingNCT06961565Phase 13 countries
- The NF Registry Sponsored by the Children's Tumor FoundationRecruitingNCT018857671 country
- Development of Non-Invasive Prenatal Diagnosis for Single Gene DisordersRecruitingNCT061474141 country
- Natural History Study of Patients With Neurofibromatosis Type 2RecruitingNCT005983511 country
- Individual Patient Compassionate Use of MirdametinibAvailableNCT050281662 countries
- Neurocutaneous Syndromes in Children: Prevalence and Clinical CharacteristicsNot yet recruitingNCT07843823
- Clinical and Demographic Characteristics of Adult p Atients With NEurofibromatosis in RUSsiaRecruitingNCT070889911 country
- How Can the Experience of Hospital Outpatient Departments be Improved?Not yet recruitingNCT078182131 country
- Glioma Developmental and HyperActive Ras Tumor (DHART) BoardRecruitingNCT054897831 country
- Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health LiteracyRecruitingNCT07233408Not applicable1 country
- Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1RecruitingNCT07102394Phase 11 country
- Mirdametinib in Patients With Advanced NF1-mutant MelanomaRecruitingNCT07237100Phase 21 country
- GROWing Up With Rare GENEtic SyndromesRecruitingNCT044633161 country
- The Neurofibromatosis-associated Tumor BiobankNot yet recruitingNCT03359304
- Transformation of Plexiform Neurofibromas to Malignant Peripheral Nerve Sheath Tumors in Neurofibromatosis Type 1CompletedNCT02211768Phase 11 country
- Natural History and Biology of Skin Neurofibromas in Neurofibromatosis Type 1CompletedNCT003141191 country
- Natural History Study of Cutaneous Neurofibromas in People With NF1Active, not recruitingNCT055815111 country
- MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform NeurofibromasActive, not recruitingNCT03962543Phase 21 country
- A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1CompletedNCT06262113Not applicable1 country
- Resiliency Training in Adolescents With NF1 and NF2CompletedNCT03873610Not applicable1 country
- Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs)SuspendedNCT06934928Not applicable1 country
- Resiliency Training for Adults With Neurofibromatosis Via Live VideoconferencingCompletedNCT03406208Not applicable1 country
- Phase II Study of Binimetinib in Children and Adults With NF1 Plexiform NeurofibromasCompletedNCT03231306Phase 21 country
- Clinical Hypnosis and Home Blood Pressure Monitoring in Children With Neurofibromatosis Type 1CompletedNCT05196854Not applicable1 country
- Validating Pain Scales in Children and Young AdultsCompletedNCT016399501 country
- DPCP to Treat Cutaneous Neurofibromas Associated With NF1CompletedNCT05438290Phase 11 country
- A Clinical Trial to Evaluate the Safety and Efficacy of AL2846 Capsules in Chinese Patients With Type I NeurofibromatosisTerminatedNCT05011019Phase 1/21 country
- Quality Improvement Project - "My Logbook! - I Know my Way Around!"; ("Mein Logbuch - Ich Kenne Mich Aus!")CompletedNCT04474678Not applicable4 countries
- Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1CompletedNCT05377008Not applicable1 country
- Cabozantinib for Plexiform Neurofibromas (PN) in Subjects With NF1 in Children and AdultsCompletedNCT02101736Phase 21 country
- Mindful Parenting - A Single Case Experimental Design StudyStatus unknownNCT05779527Not applicable1 country
- A Long-term Study of NPC-12G Gel in Neurofibromatosis Type ITerminatedNCT04461886Phase 31 country
- A Clinical Trial to Evaluate the Tolerability and Pharmacokinetics of TQ-B3234 in Patients With Type I NeurofibromatosisStatus unknownNCT05107037Phase 11 country
- Phase I Photodynamic Therapy (PDT) for Benign Dermal Neurofibromas (NF1)CompletedNCT01682811Phase 11 country
- Dexmedetomidine and Propofol for Pediatric MRI SedationCompletedNCT03513757Phase 41 country
- The Use of a Distress Thermometer in a Pediatric Research Setting: An Exploratory Pilot StudyCompletedNCT008242781 country
- Using Positron Emission Tomography to Predict Intracranial Tumor Growth in Neurofibromatosis Type II PatientsCompletedNCT012227281 country
- Acceptance and Commitment Therapy for Adolescents and Young Adults With Neurofibromatosis and Chronic PainCompletedNCT01633008Early Phase 11 country
- PTC299 for Treatment of Neurofibromatosis Type 2TerminatedNCT00911248Phase 21 country
- Neurofibromatosis Type 1 (NF1) and Tibial DysplasiaCompletedNCT003033681 country
- Resiliency Training for Adolescent Neurofibromatosis Patients Via Videoconferencing With SkypeCompletedNCT02387177Not applicable1 country
- R115777 to Treat Children With Neurofibromatosis Type 1 and Progressive Plexiform NeurofibromasCompletedNCT00021541Phase 22 countries
- Study of Sutent®/Sunitinib (SU11248) in Subjects With NF-1 Plexiform NeurofibromasTerminatedNCT01402817Phase 21 country
- Neurofibromatosis Type 1 Patient RegistryCompletedNCT014100061 country
- Pilot Study of Gleevec/Imatinib Mesylate (STI-571, NSC 716051) in Neurofibromatosis (NF1) Patient With Plexiform NeurofibromasCompletedNCT01140360Phase 1/21 country
- Study of Tasigna®/Nilotinib (AMN107) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasCompletedNCT01275586Early Phase 11 country
- Relationship Between Psychosocial Factors, Health Literacy, Quality of Life and Satisfaction With Medical Visits in Adults With NFCompletedNCT024356281 country
- Resiliency Training for Patients With Neurofibromatosis Via Videoconferencing With SkypeCompletedNCT02298270Not applicable1 country
- Phase II Study of Gleevec/Imatinib Mesylate (STI-571, NCS 716051) in Neurofibromatosis (NF1) Patients With Plexiform NeurofibromasCompletedNCT01673009Phase 21 country
- Phase II Trial of Sunitinib (SU011248) in Patients With Recurrent or Inoperable MeningiomaCompletedNCT00589784Phase 21 country