Neurofibromatosis Type 1 (NF1): Clinical Trials

6 trials for Neurofibromatosis Type 1 (NF1), newest first

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2 recruiting, 1 active not recruiting, 1 completed, 1 not yet recruiting. 2 Phase 2, 1 Phase 1.

By phase and status

PhaseTrialsStatus
Phase 22Active not recruiting 1, Terminated 1
Phase 11Recruiting 1

Follow Neurofibromatosis Type 1 (NF1): new trials by email Literature Review of Neurofibromatosis Type 1 (NF1) trials Research Report on Neurofibromatosis Type 1 (NF1)

Trials newest first · largest first

Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.

TrialPhaseStatusSponsorEnrollmentStartUpdated
Observational Study of the Real-world Effectiveness and Safety of Selumetinib Treating Adult Patients With Neurofibromatosis Type 1 (NF1) Who Have Symptomatic, Inoperable Plexiform Neurofibroma (PN)
Neurofibromatosis Type 1 (NF1)
RecruitingAstraZeneca8002026-09-282026-10-22
How Can the Experience of Hospital Outpatient Departments be Improved?
Qualitative data collection activities - Neurofibromatosis Type 1 (NF1), NF2-related Schwannomatosis
Not yet recruitingKing's College London302026-09-012026-09-14
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
PAS-004 Tablets - NF1 Mutation, Neurofibroma Plexiform
Phase 1RecruitingPasithea Therapeutics Corp.562025-05-302025-12-04
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
Mirdametinib (PD-0325901) oral capsule or dispersible tablet - Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
Phase 2Active not recruitingSpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany1142019-09-292026-10-06
Vitamin D Supplementation for Adults With Neurofibromatosis Type 1 (NF1)
Cholecalciferol - Neurofibromatosis Type 1 (NF1)
Phase 2TerminatedUniversity of Utah262017-08-162022-05-25
Precision Medicine for Neurocutaneous Syndromes in Western China
Neurocutaneous Syndromes, Neurofibromatosis Type 1 (NF1)
CompletedWest China Hospital12002000-01-012026-01-09

Narrower conditions

Neurofibromatosis 1 (6)  ·  Neurofibromatoses (3)  ·  Neurofibroma, Plexiform (2)  ·  Hearing Loss (1)  ·  Learning Disabilities (1)  ·  Neurocutaneous Syndromes (1)  ·  Neurofibroma (1)  ·  Sturge-Weber Syndrome (1)  ·  Tuberous Sclerosis (1)  ·  Vision Disorders (1)  ·  von Hippel-Lindau Disease (1)

Broader conditions

Congenital, Hereditary, and Neonatal Diseases and Abnormalities (6)  ·  Genetic Diseases, Inborn (6)  ·  Heredodegenerative Disorders, Nervous System (6)  ·  Neoplasms (6)  ·  Neoplasms by Histologic Type (6)  ·  Neoplasms, Nerve Tissue (6)  ·  Neoplastic Syndromes, Hereditary (6)  ·  Nerve Sheath Neoplasms (6)  ·  Nervous System Diseases (6)  ·  Neurodegenerative Diseases (6)  ·  Neuromuscular Diseases (6)  ·  Peripheral Nervous System Diseases (6)  ·  Neurocutaneous Syndromes (5)  ·  Neurofibroma (5)  ·  Neurofibromatoses (3)

Interventions

Cholecalciferol (1)  ·  Dietary Supplements (1)  ·  Vitamin D (1)  ·  mirdametinib (1)

Sponsors

AstraZeneca (1)  ·  King's College London (1)  ·  Pasithea Therapeutics Corp. (1)  ·  SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany (1)  ·  University of Utah (1)  ·  West China Hospital (1)

Countries

United States (3)  ·  China (2)  ·  Australia (1)  ·  Canada (1)  ·  South Korea (1)  ·  United Kingdom (1)

Top investigators

David H. Viskochil, MD, PhD (1)  ·  Pinan Liu (1)  ·  Sue Woodward, PhD (1)  ·  Tiago R Marques, MD (1)  ·  Zhichao Wang (1)

Results publications

PMID 39514826