Today, we are looking into the latest clinical trials focused on neurofibromatosis. This series aims to offer a glimpse into upcoming innovations and research, and how the outcomes of these studies could potentially influence clinical guidelines related to neurofibromatosis.
The following list features ongoing Phase 3 trials for neurofibromatosis. Please note that the dates provided are approximate and subject to change.
Quick View Table of Neurofibromatosis Clinical Trials
| Study Title | Phase | Enrollment | Start Date | Last Update Posted |
|---|---|---|---|---|
| FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas | 3 | 167 | June 20, 2023 | March 4, 2025 |
| Efficacy and Safety of Selumetinib in Adults with NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET) | 3 | 146 | November 19, 2021 | March 4, 2025 |
Phase 3 Clinical Trials
FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
- Sponsor: Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
- Goal: Evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
- Interventions: Test group: FCN-159, 8mg orally once daily; Control group: placebo, orally, once daily.
- Primary Outcome Measures: Objective response rate evaluated by BIRC (Response evaluation in Nerufibromatosis and Schwannomatosis, REiNS criteria)
- Timeframe: Estimated 2 years
Efficacy and Safety of Selumetinib in Adults with NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas (KOMET)
- Sponsor: AstraZeneca
- Goal: Demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas
- Interventions: Selumetinib; Placebo.
- Primary Outcome Measures: Confirmed Objective Response Rate (ORR) for Arm A versus Arm B
- Timeframe: Estimated 3 years
Potential Guidelines That May Be Affected Include:
- Role of Imaging in the Diagnosis and Management of Patients with Vestibular Schwannomas
- Congress of Neurological Surgeons
- Latest Update: August 2023
- Emerging Therapies for the Treatment of Patients with Vestibular Schwannomas
- Congress of Neurological Surgeons
- Latest Update: August 2023
- Selection of Germline Genetic Testing Panels in Patients with Cancer
- American Society of Clinical Oncology
- Latest Update: February 2025
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