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Neurofibroma, Plexiform clinical trials

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NCT ID: NCT06188741 Not yet recruiting - Neurofibromatosis 1 Clinical Trials

Selumetinib for the Prevention of Plexiform Neurofibroma Growth in NF Type 1

NF114
Start date: June 15, 2024
Phase: Phase 2
Study type: Interventional

Plexiform neurofibromas (PN) are known to cause significant morbidity in children with NF1. The recent FDA approval for selumetinib in children 2 years and older with inoperable symptomatic PN was based on the finding that selumetinib shrinks the majority of PN in children with NF1 and results in clinically meaningful benefit such as improvement in pain or range of motion. However, many morbidities, such as blindness or nerve damage, cannot be fully reversed with PN shrinkage. Therefore, there remains a critical need in this patient population to determine if young participants with PN in high-risk locations may benefit from early medical intervention prior to the development of clinical problems. This study will determine whether participants with asymptomatic PN in high-risk locations can potentially benefit from early treatment with selumetinib.

NCT ID: NCT06175637 Recruiting - Clinical trials for Neurofibroma, Plexiform

Observational Study to Evaluate the Effect and Safety of Selumetinib in Pediatric Patients With NF1-PNs

PEDIA
Start date: December 20, 2023
Phase:
Study type: Observational

This is a prospective, multicenter, observational study of Chinese pediatric NF1-PN patients treated with selumetinib. The study will be conducted at approximately 12 centers in China and will include approximately 80-100 patients. Treatment centers that have PN diagnosis and/or selumetinib treatment experience will be targeted for recruitment. Patients/caregivers who are eligible and willing to participate will be enrolled into the study. Patients will start selumetinib treatment after enrollment. The study will have a 16-month enrollment period. Patients will be followed up until the end of a 24-month observation period after first dose of selumetinib, or patient death, lost to follow-up, withdrawal of consent, whichever occurs first. Patients will be followed within a 24-month period (starting after first dose received) in the study even if selumetinib is discontinued. The aims of this study are to expand understanding of disease characteristics and treatment pattern of NF1-PN in China in a real-world setting and to evaluate real-world effectiveness and safety of selumetinib for Chinese pediatric patients with NF1-PN.

NCT ID: NCT06104488 Recruiting - Solid Tumor Clinical Trials

A Study of Avutometinib for People With Solid Tumor Cancers

Start date: October 20, 2023
Phase: Phase 1
Study type: Interventional

The purpose of this study is to find out whether avutometinib is a safe treatment for advanced or recurrent solid tumor cancers in children and young adults. Researchers will look for the highest dose of avutometinib that is safe and cause few or mild side effects.

NCT ID: NCT05913037 Recruiting - Neurofibromatosis 1 Clinical Trials

FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas

Start date: June 20, 2023
Phase: Phase 3
Study type: Interventional

A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.

NCT ID: NCT05825365 Withdrawn - Clinical trials for Neurofibromatosis Type 1

Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, Phaseâ…¡, Double-Blinded, Placebo-Controlled Study

PROTECT
Start date: August 31, 2023
Phase: Phase 2
Study type: Interventional

This is a phase II, multicenter, randomised, parallel, double-blind, placebo-controlled study assessing the efficacy and safety of the MEKi selumetinib compared with placebo in Chinese paediatric participants with post-operative NF1-associated PNs.

NCT ID: NCT05683678 Recruiting - Clinical trials for Neurofibromatosis Type 1

US Selumetinib Registry

Start date: July 18, 2023
Phase:
Study type: Observational

The purpose of this study is to understand treatment patterns and assess long-term effectiveness and safety outcomes associated with selumetinib treatment as well as to explore clinical and non-clinical factors affecting those outcomes in participants with neurofibromatosis type 1 (NF1) and plexiform neurofibromas (PNs) who were aged 2 to 18 years at the time selumetinib was started in a real-world setting.

NCT ID: NCT05331105 Recruiting - Neurofibromatosis 1 Clinical Trials

HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

Start date: October 18, 2021
Phase: Phase 2
Study type: Interventional

This is a Multi-center, Open-label, Single-arm Phase II Study to Evaluate the Efficacy and Safety of HL-085 in the treatment of Adult Participants with Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas(PN)

NCT ID: NCT05199376 Recruiting - Clinical trials for Neurofibroma, Plexiform

Evaluation of Percutaneous Cryotherapy in the Treatment of Plexiform Neurofibromas and Unresectable Neurofibromas in Neurofibromatosis Type 1

CryoNF1
Start date: February 14, 2022
Phase: N/A
Study type: Interventional

Neurofibromatosis type 1 (NF1) is an autosomal dominant disease affecting chromosome 17. It is manifested by a neurogenic tumor proliferation that forms cutaneous, subcutaneous or deep neurofibromas. Neurofibromas can cause functional discomfort, neurogenic pain that is difficult to treat, and severe cosmetic disorders. Treatment is essentially surgical. It is sometimes a heavy invasive surgery with complicated postoperative follow-up and significant scarring on the aesthetic level. Currently, no systemic treatment has proven its effectiveness in this pathology. Percutaneous cryotherapy is a cold thermoablation procedure using fine 17 G needles introduced into the lesion after targeting by imaging. This technique is used in the treatment of soft tissue tumors and desmoid tumors. The treatment of neurofibromas with percutaneous cryotherapy is not well known. Encouraging results (unpublished) have been observed in patients with NF1 treated with cryotherapy at the Léon Bérard Center. The beneficial effect was observed in terms of quality of life (in particular, pain) as well as a decrease in tumor size. On the basis of this first experience, it appears important to corroborate these preliminary results by a prospective study allowing the use of this technique to treat patients with unresectable or resectable neurofibromas but with mutilating surgery in a NF1 context.

NCT ID: NCT05101148 Active, not recruiting - Clinical trials for 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

Start date: July 21, 2021
Phase: Phase 1
Study type: Interventional

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 under fed and fasted conditions; and potentially, to confirm an appropriate dosing recommendation of selumetinib with a low fat meal that maintains efficacy with acceptable safety. These results may support labelling statements with regard to posology and food.

NCT ID: NCT05028166 Available - Clinical trials for Histiocytic Neoplasm

Individual Patient Compassionate Use of Mirdametinib

Start date: n/a
Phase:
Study type: Expanded Access

This program is being offered on a patient by patient basis and will require company, Institutional Review Board/Independent Ethics Committee, and applicable competent authority approval.