Neurofibromatosis Type
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CLINICAL_TRIALS research · 2026-03-20Clinical Trial Update: Natural History Study of Patients With Neurofibromatosis Type I
The National Cancer Institute is conducting an observational natural history study of patients with Neurofibromatosis Type 1. The study is c…
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CLINICAL_TRIALS trial_result · 2026-03-20Clinical Trial Update: A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1 [Phase 2]
Phase 2 open-label trial evaluating safety and effects of HLX-1502 in patients with Neurofibromatosis Type 1 is currently recruiting.
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CLINICAL_TRIALS trial_result · 2026-03-13Clinical Trial Update: Phase 2 Clinical Trial of Crizotinib for Children and Adults With Neurofibromatosis Type 2 and Progressive Vestibular Schwannomas [Phase 2]
Phase 2 clinical trial of crizotinib in children and adults with Neurofibromatosis type 2 and progressive vestibular schwannomas has complet…
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CLINICAL_TRIALS trial_result · 2026-03-12Clinical Trial Update: Study of Cabozantinib With Selumetinib for Plexiform Neurofibromas [Phase 1]
A Phase 1 clinical trial is evaluating the combination of cabozantinib and selumetinib for plexiform neurofibromas in Neurofibromatosis type…
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: PASS of Paediatric Patients Initiating Selumetinib
A post-authorization safety study (PASS) update notes ongoing safety monitoring for pediatric patients initiating selumetinib in Neurofibrom…
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CLINICAL_TRIALS research · 2026-03-12Clinical Trial Update: Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1 [Phase 2]
A Phase 2 clinical trial is evaluating selumetinib (Koselugo) to prevent plexiform neurofibroma growth in Neurofibromatosis type 1; the stud…
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CLINICAL_TRIALS trial_result · 2026-03-05Clinical Trial Update: Assessing the Efficacy of Repeat, Monthly Treatments of Alexandrite Laser for NF1-associated Cutaneous Neurofibromas (cNFs) [NA]
A clinical trial evaluating repeat monthly Alexandrite laser treatment for neurofibromatosis type 1-associated cutaneous neurofibromas is cu…
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CLINICAL_TRIALS trial_result · 2026-03-05Clinical Trial Update: MEK 1/2 Inhibitor Selumetinib (AZD6244 Hydrogen Sulfate) in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas [Phase 2]
Phase 2 trial update for the MEK inhibitor selumetinib in adults with Neurofibromatosis Type 1 and inoperable plexiform neurofibromas; the s…
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CLINICAL_TRIALS research · 2026-02-27Clinical Trial Update: Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy [NA]
A clinical trial to tailor an online platform to promote evidence-based care for adults with Neurofibromatosis type 1 who have low health li…
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CLINICAL_TRIALS research · 2026-02-25Clinical Trial Update: A Study of Avutometinib for People With Solid Tumor Cancers [Phase 1]
A Phase 1 study of Avutometinib for people with solid tumors, including CNS tumors and NF1-associated tumors, is currently recruiting.
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CLINICAL_TRIALS research · 2026-02-23Clinical Trial Update: Phase I Study to Assess the Effect of Food on the PK and Gastrointestinal Tolerability of Selumetinib in Adolescent Children With Neurofibromatosis Type 1 Related Plexiform Neurofibromas [Phase 1]
This Phase I trial conducted by AstraZeneca evaluates how food affects the pharmacokinetics and gastrointestinal tolerability of selumetinib…
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CLINICAL_TRIALS research · 2026-02-19Clinical Trial Update: Acceptance and Commitment Therapy for Caregivers of Children With a RASopathy: An Internal Pilot Feasibility Study and Follow-up Randomized Controlled Trial [NA]
This clinical trial update describes an internal pilot feasibility study and follow-up randomized controlled trial of Acceptance and Commitm…
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CLINICAL_TRIALS trial_result · 2026-02-17Clinical Trial Update: NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF) [Phase 2]
Phase 2 trial of NFX-179 topical gel for adults with Neurofibromatosis Type 1 and cutaneous neurofibromas has completed, and results have be…
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CLINICAL_TRIALS research · 2026-02-17Clinical Trial Update: Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1 [Phase 1]
Phase 1 study evaluating the feasibility and tolerability of talimogene laherparepvec (IMLYGIC) for treating cutaneous neurofibromas in adul…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: FARD (RaDiCo Cohort) (RaDiCo-FARD)
The RaDiCo-FARD clinical trial sponsored by INSERM is currently recruiting, studying a cohort of patients with inherited skin and ectodermal…
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CLINICAL_TRIALS research · 2026-02-12Clinical Trial Update: Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs
An update on an observational study evaluating the effect and safety of selumetinib in pediatric patients with NF1-associated plexiform neur…
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CLINICAL_TRIALS research · 2026-02-11Clinical Trial Update: Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas [Phase 2]
A Phase 2 clinical trial evaluating a combination of selumetinib, a bromodomain inhibitor, and durvalumab for sarcomas, with recruitment not…
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CLINICAL_TRIALS research · 2026-02-03Clinical Trial Update: A Window of Opportunity Trial of Mirdametinib Plus Vorinostat for NF1 Associated, H3K27 Trimethylation Deficient Malignant Peripheral Nerve Sheath Tumor [MPNST] [EARLY_Phase 1]
Early-phase window-of-opportunity trial evaluating the combination of mirdametinib and vorinostat for NF1-associated, H3K27 trimethylation-d…
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CLINICAL_TRIALS research · 2026-01-30Clinical Trial Update: A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) [Phase 1]
A Phase 1 study of selumetinib in Chinese pediatric and adult subjects with Neurofibromatosis Type 1 and inoperable plexiform neurofibromas …
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EMA approval · 2026-01-28EMA Authorised: Koselugo (selumetinib sulfate) — AstraZeneca AB
The EMA authorized Koselugo (selumetinib) for Neurofibromatosis type 1 with conditional approval and orphan medicine designation. This repre…
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CLINICAL_TRIALS research · 2026-01-13Clinical Trial Update: NF-1, Nutraceutical Intervention [Phase 1]
This Phase 1 clinical trial update investigates a nutraceutical intervention for Neurofibromatosis type 1, sponsored by the Masonic Cancer C…
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CLINICAL_TRIALS research · 2026-01-12Clinical Trial Update: Multi-parametric Biomarker Development to Predict Malignant Conversion in Patients With Neurofibromatosis Type 1
This Johns Hopkins University study is developing multi-parameter biomarkers to predict malignant transformation in Neurofibromatosis type 1…
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CLINICAL_TRIALS research · 2026-01-12Clinical Trial Update: Innovative Trial for Understanding the Impact of Targeted Therapies in NF2-Related Schwannomatosis (INTUITT-NF2) [Phase 2]
This update covers an ongoing Phase 2 clinical trial (INTUITT-NF2) evaluating targeted therapies for NF2-related schwannomatosis. The study …
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CLINICAL_TRIALS research · 2026-01-09Clinical Trial Update: Neurofibromatosis Type 1 Tumor Early Detection Study
This clinical trial update announces a study for early detection of tumors in Neurofibromatosis Type 1, and the study is currently recruitin…
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CLINICAL_TRIALS trial_result · 2026-01-09Clinical Trial Update: Precision Medicine for Neurocutaneous Syndromes in Western China
A completed clinical trial from West China Hospital exploring precision medicine approaches for neurocutaneous syndromes, including NF1, TSC…