Neurofibromatosis 1 Clinical Trial
Official title:
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 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 with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN).
Paediatric and adult patients with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas (PN) will be evaluated in the screening visit to confirm eligibility. Approximately 16 paediatric and 16 adult qualified patients will receive oral selumetinib 25 mg/m^2 twice a day (approximately every 12 hours) continuously until disease progression or unacceptable drug-related toxicity, whichever occurs first. Once a patient has discontinued the study treatment then the patient will be followed for specified period for safety assessment ;
| Status | Clinical Trial | Phase | |
|---|---|---|---|
| Active, not recruiting |
NCT01362803 -
AZD6244 Hydrogen Sulfate for Children With Nervous System Tumors
|
Phase 1/Phase 2 | |
| Completed |
NCT00352599 -
Trial to Evaluate the Safety of Lovastatin in Individuals With Neurofibromatosis Type I (NF1)
|
Phase 1 | |
| Completed |
NCT05005845 -
NFX-179 Topical Gel Treatment for Adults With Neurofibromatosis 1 (NF1) and Cutaneous Neurofibromas (cNF)
|
Phase 2 | |
| Completed |
NCT03531814 -
Medication Adherence in Children, Adolescents and Adults With Neurofibromatosis Type 1(NF1) on Clinical Treatment Trials
|
N/A | |
| Completed |
NCT00846430 -
Medical Treatment of "High-Risk" Neurofibromas
|
Phase 2 | |
| Completed |
NCT03433183 -
SARC031: MEK Inhibitor Selumetinib (AZD6244) in Combination With the mTOR Inhibitor Sirolimus for Patients With Malignant Peripheral Nerve Sheath Tumors
|
Phase 2 | |
| Recruiting |
NCT05331105 -
HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
|
Phase 2 | |
| Completed |
NCT03310996 -
Non-invasive Stimulation in Neurofibromatosis Type 1
|
N/A | |
| Recruiting |
NCT03406208 -
Resiliency Training for Adults With Neurofibromatosis Via Live Videoconferencing
|
N/A | |
| Recruiting |
NCT04750928 -
Cyclin-Dependent Kinase (CDK)4/6 Inhibitor Abemaciclib for Neurofibromatosis Type I (NF1) Related Atypical Neurofibromas
|
Phase 1/Phase 2 | |
| Not yet recruiting |
NCT06262113 -
A Decentralized Clinical Trial to Promote Evidence-Based Care for Underserved Patients With Neurofibromatosis 1
|
N/A | |
| Active, not recruiting |
NCT04924608 -
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
|
Phase 3 | |
| Recruiting |
NCT02544022 -
Development and Validation of Patient Reported Outcome (PRO) Measures for Individuals With Neurofibromatosis 1 (NF1) and Plexiform Neurofibromas (pNFs)
|
||
| Recruiting |
NCT04941027 -
Evaluating Genetic Modifiers of Cutaneous Neurofibromas in Adults With Neurofibromatosis Type 1
|
||
| Completed |
NCT02153931 -
Internet Support Group for Parents of a Child With Neurofibromatosis Type 1
|
||
| Completed |
NCT05196854 -
Clinical Hypnosis and Home Blood Pressure Monitoring in Children With Neurofibromatosis Type 1
|
N/A | |
| Active, not recruiting |
NCT01650142 -
Modifying Genes in Neurofibromatosis 1
|
N/A | |
| Completed |
NCT05377008 -
Intervention Effectiveness on the Neurocognitive Functioning of Children and Adolescents With Neurofibromatosis Type 1
|
N/A | |
| Completed |
NCT04879160 -
Systematically Assessing Changes in Plexiform Neurofibroma Related Disfigurement From Photographs of Subjects With Neurofibromatosis Type 1 on a Phase 2 Clinical Trial
|
||
| Recruiting |
NCT05735717 -
MT2021-08T Cell Receptor Alpha/Beta Depletion PBSC Transplantation for Heme Malignancies
|
Phase 2 |