Clinical trials: Neurofibromatosis
Neurofibromatosis Type 1
PASS of Paediatric Patients Initiating Selumetinib
Neurofibromatosis type 1 (NF1) is a rare, autosomal dominant genetic disorder that is caused by germline mutations in the NF1 tumour suppressor gene, which encodes the tumour suppressor protein neurofibromin 1.
- Trial name
- NCT05388370
- Where
- 46 sites in Austria, France and 8 more
- Volunteers
- 124 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1
A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
The goal of this fully decentralized, randomized controlled trial is to compare the efficacy of two educational interventions for individuals with Neurofibromatosis 1 (NF1).
- Trial name
- NCT06262113
- Testing
- Letters about NF1 Care (Content Type 1), Letters about NF1 Care (Content Type 2)
- Where
- United States
- Volunteers
- 294 people
- Sponsor
- Massachusetts General Hospital
Neurofibromatosis Type 1
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
This study in adolescent participants with NF1 who have inoperable PN is designed to evaluate the effect of a low fat meal on steady state selumetinib exposure; to assess the effect on GI tolerability when selumetinib is dosed…
- Trial name
- NCT05101148
- Testing
- Selumetinib
- Where
- 9 sites in United States, Poland and 2 more
- Volunteers
- 24 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1, Neurofibroma Plexiform
A Study of Selumetinib in Chinese Paediatric and Adult Subjects With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN)
A Phase 1 Open Label Study to Assess the Safety, Tolerability, Pharmacokinetics and Clinical Efficacy of Selumetinib, a Selective Mitogen Activated Protein Kinase Kinase (MEK) 1 Inhibitor, in Chinese Paediatric and Adult Subjects…
- Trial name
- NCT04590235
- Testing
- Selumetinib
- Where
- 2 sites in China
- Volunteers
- 32 people
- Sponsor
- AstraZeneca
Neurofibromatosis Type 2
Efficacy and Safety of REC-2282 in Patients With Progressive Neurofibromatosis Type 2 (NF2) Mutated Meningiomas
This is a two-staged, Phase 2/3, randomized, multi-center study to investigate the efficacy and safety of REC-2282 in participants with progressive NF2 mutated meningiomas.
- Trial name
- POPLAR-NF2 (NCT05130866)
- Testing
- REC-2282
- Where
- 15 sites in United States, United Kingdom
- Volunteers
- 25 people
- Sponsor
- Recursion Pharmaceuticals Inc.
Low Grade Glioma, Neurofibromatosis Type 1
A Study of the Drugs Selumetinib Versus Carboplatin/Vincristine in Patients With Neurofibromatosis and Low-Grade Glioma
This phase III trial studies if selumetinib works just as well as the standard treatment with carboplatin/vincristine (CV) for subjects with NF1-associated low grade glioma (LGG), and to see if selumetinib is better than CV in…
- Conditions
- Low Grade Glioma, Neurofibromatosis Type 1, Visual Pathway Glioma
- Trial name
- NCT03871257
- Testing
- Carboplatin, Magnetic Resonance Imaging, Quality-of-Life Assessment
- Where
- 132 sites in United States, Canada and 1 more
- Volunteers
- 165 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 1
Pharmacokinetics, Safety and Efficacy of the Selumetinib Granule Formulation in Children Aged ≥1 to <7 Years With NF1-related Symptomatic, Inoperable PN
This study is designed to define a dosing regimen and assess the pharmacokinetics(PK) and safety of the granule formulation; the study will also include descriptive analyses of exploratory efficacy endpoints.
- Trial name
- SPRINKLE (NCT05309668)
- Testing
- Selumetinib granule formulation, Selumetinib capsule formulation
- Where
- 16 sites in United States, Germany and 5 more
- Volunteers
- 36 people
- Sponsor
- AstraZeneca
MPNST (Malignant Peripheral Nerve Sheath Tumor)
Combination of Avutometinib and Defactinib for Treatment of Relapsed, Refractory, Metastatic, or Unresectable Malignant Peripheral Nerve Sheath Tumor
The purpose of this study is to learn whether the study drugs avutometinib and defactinib can help stop or shrink malignant peripheral nerve sheath tumors (MPNST) that have returned or cannot be removed with surgery.
- Trial name
- NF120 (NCT07743554)
- Testing
- avutometinib capsule + defactinib tablet
- Volunteers
- 23 people
- Sponsor
- Girish Dhall, MD
Neurofibromatosis Type 1
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
The trial will be an open label, single arm, phase 2 study to assess the tolerability and efficacy of HLX-1502 in participants with NF1 that are 16 years or older in age with progressive and/or symptomatic PN.
- Trial name
- INSPIRE-NF1 (NCT06541847)
- Testing
- HLX-1502
- Where
- 14 sites in United States
- Volunteers
- 25 people
- Sponsor
- Healx Limited
Malignant Peripheral Nerve Sheath Tumor (MPNST), Neurofibromatosis 1 (NF1)
Early Phase Study Evaluating MEK and MDM2 Inhibition in Patients With NF1 and MPNST
This is a phase 0/1/2, multi-site study to evaluate the MEK inhibitor Selumetinib with the MDM2 Inhibitor APG-115 in patients with Neurofibromatosis Type 1 and pre-malignant and malignant peripheral nerve sheath tumors
- Conditions
- Malignant Peripheral Nerve Sheath Tumor (MPNST), Neurofibromatosis 1 (NF1), Atypical Neurofibroma
- Trial name
- MEKMDM2 (NCT06735820)
- Testing
- APG-115, Selumetinib
- Where
- 2 sites in United States
- Volunteers
- 45 people
- Sponsor
- AeRang Kim
Juvenile Myelomonocytic Leukemia, Neurofibromatosis Type 1
Trametinib in Treating Patients With Relapsed or Refractory Juvenile Myelomonocytic Leukemia
This phase II trial studies how well trametinib works in treating patients with juvenile myelomonocytic leukemia that has come back (relapsed) or does not respond to treatment (refractory).
- Trial name
- NCT03190915
- Testing
- Bone Marrow Aspiration and Biopsy, Trametinib
- Where
- 57 sites in United States
- Volunteers
- 10 people
- Sponsor
- National Cancer Institute (NCI)
Neurofibromatosis Type 2, Vestibular Schwannoma
Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2)
This is an adaptive platform-basket trial that aims to evaluate the safety and efficacy of multiple novel agents and combination therapies in patients with NF2-related schwannomatosis (NF2-SWN).
- Conditions
- Neurofibromatosis Type 2, Vestibular Schwannoma, Non-vestibular Schwannoma, Meningioma
- Trial name
- PRIME-NF2 (NCT07713745)
- Testing
- Selumetinib 25mg/m2, Luvometinib, Serplulimab
- Where
- 5 sites in China
- Volunteers
- 200 people
- Sponsor
- Beijing Tiantan Hospital
Neurofibromatosis Type 2, NF2
Selumetinib for NF2-Related Schwannomatosis
The goal of this clinical trial is to evaluate the efficacy and safety of the MEK1/2 inhibitor selumetinib in treating patients with neurofibromatosis type 2-related schwannomatosis (NF2-SWN), including both adults and children…
- Conditions
- Neurofibromatosis Type 2, NF2, NF2-related Schwannomatosis, Meningioma
- Trial name
- ASSIST (NCT07707947)
- Testing
- Selumetinib
- Where
- China
- Volunteers
- 20 people
- Sponsor
- Beijing Tiantan Hospital
NF2-related Schwannomatosis, NF2
Luvometinib in Combination With Serplulimab for NF2-Related Tumors
This is an investigator-initiated, exploratory, multicenter, open-label, single-arm clinical trial and a substudy of the Platform Research for Innovative Medicines in NF2-SWN (PRIME-NF2).
- Conditions
- NF2-related Schwannomatosis, NF2, Neurofibromatosis Type 2, Vestibular Schwannoma
- Trial name
- NCT07708285
- Testing
- Luvometinib, Serplulimab
- Where
- 5 sites in China
- Volunteers
- 30 people
- Sponsor
- Beijing Tiantan Hospital
Neurofibromatosis 1
Tailoring an Online Platform to Promote Evidence-Based Care for Adults With Neurofibromatosis 1 and Low Health Literacy
This decentralized, randomized study seeks to assess the feasibility, acceptability, and preliminary effectiveness of two approaches to assisting Neurofibromatosis 1 (NF1) patients with low health literacy improve their…
- Trial name
- NCT07233408
- Testing
- NF1 Educational Videos, Peer Navigator, Letters about NF1 Care
- Where
- United States
- Volunteers
- 50 people
- Sponsor
- Massachusetts General Hospital
NEUROFIBROMATOSIS 1
Photodynamic Therapy for Benign Dermal Neurofibromas- Phase II
The investigators wish to determine the time to disease progression for benign neurofibromas treated with Levulan Kerastick topical photosensitizer and red light photodynamic therapy (PDT) in patients with neurofibromatosis type…
- Trial name
- NCT02728388
- Testing
- aminolevulinic acid
- Where
- United States
- Volunteers
- 30 people
- Sponsor
- Donald Basel
Neurofibromatosis Type 1, Learning Disability
Interventions for Reading Disabilities in NF1
Neurofibromatosis Type 1 (NF1) is a common genetic disorder that is associated with a four times greater risk of learning disabilities, including reading disabilities, and a deficiency of neurofibromin - a protein important in a…
- Conditions
- Neurofibromatosis Type 1, Learning Disability, Reading Disability, NF1
- Trial name
- NCT02964884
- Testing
- Lovastatin, reading tutoring intervention
- Where
- United States
- Volunteers
- 120 people, healthy volunteers welcome
- Sponsor
- Vanderbilt University
Neurofibromatosis 2, Dermatology/Skin - Other
Cutaneous and Mucosal Manifestations of Neurofribromatosis Type 2 in Children Under 15
.Neurofibromatosis type 2 is an inherently autosomal dominant genetic disease, but cases of mosaicism or de novo mutation are not uncommon. the prevalence is estimated at 1 / 60,000.
- Trial name
- NCT03893643
- Testing
- no intervention
- Where
- France
- Volunteers
- 1,000 people
- Sponsor
- Centre Hospitalier Universitaire de Nice
Acute Leukemia, Adenomatous Polyposis
Familial Investigations of Childhood Cancer Predisposition
NOTE: This is a research study and is not meant to be a substitute for clinical genetic testing. Families may never receive results from the study or may receive results many years from the time they enroll.
- Conditions
- Acute Leukemia, Adenomatous Polyposis, Adrenocortical Carcinoma, AML
- Trial name
- SJFAMILY (NCT03050268)
- Where
- United States
- Volunteers
- 1,500 people
- Sponsor
- St. Jude Children's Research Hospital
Neurofibromatosis 1
Antioxidant Therapy With N-acetylcysteine for Children With Neurofibromatosis Type 1
Children with neurofibromatosis type 1 (NF1) commonly suffer from the effects of cognitive, behavioral, and motor impairments. At present, there is no specific treatment for this NF1 complication.
- Trial name
- DoDNAC (NCT04481048)
- Testing
- N-Acetyl cysteine
- Where
- United States
- Volunteers
- 25 people, healthy volunteers welcome
- Sponsor
- Children's Hospital Medical Center, Cincinnati