Clinical trials: Neurofibromatosis 1 (NF1), open to volunteers
Neurofibromatosis Type 1 (NF1)
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)
This is a prospective, multicentre, observational registry study with primary data collection designed to evaluate the real-world effectiveness and safety of selumetinib in adult patients with NF1 who have symptomatic, inoperable…
- Trial name
- PROCEED (NCT07888517)
- Where
- 26 sites in China
- Volunteers
- 800 people
- Sponsor
- AstraZeneca
Neurofibromatosis 1 (NF1), Executive Function (Cognition)
Cognitive Intervention for Children and Adolescents With NF1
Psychological interventions have been shown to be effective for managing cognitive and mental health challenges in different patient populations.
- Conditions
- Neurofibromatosis 1 (NF1), Executive Function (Cognition), Cognitive Intervention
- Trial name
- NCT07869914
- Testing
- Unstuck and On Target!
- Volunteers
- 60 people
- Sponsor
- The Hospital for Sick Children
Neurofibromatosis 1 (NF1)
Efficacy of Mirdametinib Alone or Combination With Radiotherapy for Germline and Sporadic NF1-Altered High-Grade Glioma
This is a phase 2, open label, parallel multi-arm study of mirdametinib in combination with radiation in participants with recurrent sporadic glioblastoma (GBM) harboring NF1 alterations (Cohort 1); participants with NF1 with a…
- Trial name
- NF118 (NCT07521657)
- Testing
- mirdametinib with radiation, mirdametinib
- Where
- United States
- Volunteers
- 55 people
- Sponsor
- University of Alabama at Birmingham
Neurofibromatosis Type 1 (NF1), NF2-related Schwannomatosis
How Can the Experience of Hospital Outpatient Departments be Improved?
This project aims to improve outpatient departments by working with patients and staff to make these spaces more inclusive for people with sensory challenges (it focuses on people with nerve tumour conditions as they are more…
- Trial name
- NCT07818213
- Testing
- Qualitative data collection activities
- Where
- United Kingdom
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- King's College London
Neurofibromatosis 1, NF1
Developing Biomarkers of Plexiform Tumor Burden in Patients With Neurofibromatosis-Type 1
The purpose of this study is to identify tumor biomarkers in individuals with Neurofibromatosis type 1 (NF1). Biomarkers are signals that the investigator can measure that tell us about a process such as progress of a disease or…
- Conditions
- Neurofibromatosis 1, NF1, Neurofibromatosis Type 1
- Trial name
- NCT05238909
- Where
- United States
- Volunteers
- 200 people
- Sponsor
- Ann & Robert H Lurie Children's Hospital of Chicago
MPNST, NF1
Trial of Selumetinib and Bromodomain Inhibitor With Durvalumab for Sarcomas
A multi-institutional open-label phase 1/2 trial of selumetinib in combination with ZEN-3694 and durvalumab in refractory/unresectable sarcomas including MPNST.
- Conditions
- MPNST, NF1, Sarcoma
- Trial name
- NCT05253131
- Testing
- Selumetinib + ZEN-3694 ± Durvalumab
- Where
- United States
- Volunteers
- 41 people
- Sponsor
- University of Alabama at Birmingham
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
NF1, Neurofibromatosis
Feasibility and Tolerability of IMLYGIC for the Treatment of Cutaneous Neurofibromas in Adults With NF1
This study is designed to establish the foundation for a new therapy for neurofibromatosis Type I (NF1)-related cutaneous neurofibromas (cNFs) by assessing the feasibility and efficacy of IMLYGIC in adults with NF1 and cNFs who…
- Conditions
- NF1, Neurofibromatosis, Cutaneous Neurofibroma, Neurofibroma
- Trial name
- NCT07102394
- Testing
- Imlygic
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Johns Hopkins University
Hematologic Malignancy, Acute Leukemia
MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
This is a phase II, open-label, prospective study of T cell receptor alpha/beta depletion (TCR α/β TCD) peripheral blood stem cell (PBSC) transplantation for children and adults with hematological malignancies.
- Conditions
- Hematologic Malignancy, Acute Leukemia, Remission, Acute Myeloid Leukemia
- Trial name
- NCT05735717
- Testing
- Fludarabine, Busulfan, Melphalan
- Where
- United States
- Volunteers
- 70 people
- Sponsor
- Masonic Cancer Center, University of Minnesota
NF1 Mutation, Neurofibroma Plexiform
PAS-004 in Adults Who Have Neurofibromatosis Type 1 With Plexiform Neurofibromas
The main purpose of this clinical trial is to test PAS-004 in people with at least one symptomatic plexiform neurofibroma due to Neurofibromatosis Type 1 (NF1).
- Conditions
- NF1 Mutation, Neurofibroma Plexiform, Neurofibroma, Plexiform, Neurofibromatosis Type 1 (NF1)-Related Plexiform Neurofibromas (PNs)
- Trial name
- NCT06961565
- Testing
- PAS-004 Tablets
- Where
- 5 sites in United States, Australia and 1 more
- Volunteers
- 56 people
- Sponsor
- Pasithea Therapeutics Corp.
Neurofibromatosis 1, Plexiform Neurofibroma
Follow-up Study to Evaluate the Safety and Efficacy of FCN-159 in Pediatric Participants With Neurofibromatosis Type 1
FCN-159 (Luvometinib Tablets), an orally available and highly potent selective inhibitor of MEK1/2,demonstrated good tolerability and exhibited notable anti-tumor activity in pediatric pts with NF1-related PN in study…
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT07024394
- Testing
- Luvometinib Tablets
- Where
- China
- Volunteers
- 65 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.