Clinical trials: Neurofibroma
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
Central Nervous System (CNS) Lesions, Brain Metastases
Phase 2 Ascending Dose Safety and Efficacy Study of RVP-001, a Manganese-based MRI Contrast Agent
This Phase 2 clinical trial will study RVP-001, a new manganese-based MRI contrast agent, in people who are known to have gadolinium-enhancing central nervous system (CNS) lesions, for example brain tumors or multiple sclerosis.
- Conditions
- Central Nervous System (CNS) Lesions, Brain Metastases, Brain Neoplasms, Brain Neoplasms, Benign
- Trial name
- NCT06322342
- Testing
- RVP-001
- Where
- 5 sites in United States
- Volunteers
- 18 people
- Sponsor
- Reveal Pharmaceuticals Inc.
Leiomyosarcoma, Liposarcoma
MASCT-I Combined With Doxorubicin and Ifosfamide for First-line Treatment of Advanced Soft Tissue Sarcoma
This study will evaluate the safety and efficacy of MASCT-I combined with Doxorubicin and Ifosfamide for first-line treatment in patients with advanced soft tissue sarcoma.
- Conditions
- Leiomyosarcoma, Liposarcoma, Synovial Sarcoma, Angiosarcoma
- Trial name
- NCT06277154
- Testing
- MASCT-I, Doxorubicin, Ifosfamide
- Where
- China
- Volunteers
- 148 people
- Sponsor
- HRYZ Biotech Co.
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.
Solid Tumor
PF-07284892 in Participants With Advanced Solid Tumors
The purpose of this first-in-patient, open label study is to determine the maximum tolerated dose and/or recommended dose for further study of PF-07284892 as a single agent and in combination with lorlatinib, encorafenib and…
- Trial name
- NCT04800822
- Testing
- PF-07284892, lorlatinib, binimetinib
- Where
- 11 sites in United States
- Volunteers
- 53 people
- Sponsor
- Pfizer
Plexiform Neurofibroma
Evaluation of TQ-B3234 Capsules in Patients With Symptomatic, Non-Surgical Type 1 Neurofibromatosis-Associated Plexiform Neurofibromas
This study aims to demonstrate that in subjects with symptomatic, inoperable plexiform neurofibromas associated with neurofibromatosis type 1, TQ-B3234 capsules significantly improve the objective response rate at Week 24…
- Trial name
- NCT07407803
- Testing
- TQ-B3234 capsules
- Where
- 29 sites in China
- Volunteers
- 177 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Neurofibromatosis Type 1, Plexiform Neurofibromas
US Selumetinib Registry
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…
- Trial name
- NCT05683678
- Where
- 10 sites in United States
- Volunteers
- 37 people
- Sponsor
- Alexion Pharmaceuticals, Inc.
Neurofibromatosis 1, Plexiform Neurofibroma
FCN-159 in Adult Patients With Symptomatic, Inoperable Neurofibromatosis Type 1-Related Plexiform Neurofibromas
A study to evaluate the efficacy of FCN-159 in adult patients with symptomatic, inoperable neurofibromatosis type 1-related plexiform neurofibromas.
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibroma, NF1
- Trial name
- NCT05913037
- Testing
- Test group (Group A): FCN-159 8 mg, orally, once daily;
- Where
- 15 sites in China
- Volunteers
- 167 people
- Sponsor
- Shanghai Fosun Pharmaceutical Industrial Development Co. Ltd.
Neurofibromatosis 1, Plexiform Neurofibroma (PN)
Efficacy and Safety of Selumetinib in Adults With NF1 Who Have Symptomatic, Inoperable Plexiform Neurofibromas
A global study to demonstrate the effectiveness of selumetinib in participants with NF1 who have symptomatic, inoperable plexiform neurofibromas.
- Trial name
- KOMET (NCT04924608)
- Testing
- Selumetinib
- Where
- 34 sites in United States, Australia and 11 more
- Volunteers
- 145 people
- Sponsor
- AstraZeneca
Life Threatening Diseases
Phase II Study of Imatinib Mesylate in Patients With Life Threatening Malignant Rare Diseases
Exploratory study to examine the effect(s) of Imatinib mesylate treatment on life threatening rare diseases with known associations to one or more Imatinib mesylate -sensitive tyrosine kinases, and to identify the contribution of…
- Trial name
- NCT00154388
- Testing
- Imatinib mesylate
- Volunteers
- 185 people
- Sponsor
- Novartis Pharmaceuticals
NF type1 With Inoperable Plexiform Neurofibromas
An Intermediate Access Protocol for Selumetinib for Treatment of Neurofibromatosis Type 1
This will be an open-label, single-arm, multicenter intermediate access protocol which provides treatment access to selumetinib for eligible patients with neurofibromatosis type 1 (NF1) who have inoperable…
- Trial name
- NCT03259633
- Testing
- Selumetinib
- Where
- United States
- Sponsor
- AstraZeneca
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 and Malignant Peripheral Nerve Sheath Tumors
A Clinical Trial to Evaluate the Safety and Efficacy of AL2846 Capsules in Chinese Patients With Type I Neurofibromatosis
AL2846 is a multi-target receptor tyrosine kinase inhibitor. The purpose of this study is to evaluate the safety and efficacy of AL2846 capsules in Chinese patients with type I neurofibromatosis (NF1) (neurofibromas and malignant…
- Trial name
- NCT05011019
- Testing
- AL2846 capsules
- Where
- China
- Volunteers
- 22 people
- Sponsor
- Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Neurofibromatosis Type 1-Associated Plexiform Neurofibromas
A Study of Mirdametinib in Infants and Toddlers With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
The purpose of this study is to determine the safety, pharmacokinetic (PK), and tolerability of multiple treatment cycles of oral mirdametinib in infants and toddlers (0 to less than [<] 24 months of age) with measurable…
- Trial name
- NCT07850635
- Testing
- Mirdametinib
- Where
- Germany
- Volunteers
- 6 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Neurofibromatosis
A Long-term Study of NPC-12G Gel in Neurofibromatosis Type I
The purpose of this tria is to evaluate the safety and efficacy of long-term treatment with NPC-12G gel to patients with neurofibromatosis type I.
- Trial name
- NCT04461886
- Testing
- NPC-12G gel
- Where
- 4 sites in Japan
- Volunteers
- 100 people
- Sponsor
- Nobelpharma
Diffuse Astrocytoma, Anaplastic Astrocytoma
Pediatric Long-Term Follow-up and Rollover Study
A roll-over study to assess long-term effect in pediatric patients treated with dabrafenib and/or trametinib.
- Conditions
- Diffuse Astrocytoma, Anaplastic Astrocytoma, Astrocytoma, Oligodendroglioma, Childhood
- Trial name
- NCT03975829
- Testing
- dabrafenib, trametinib
- Where
- 54 sites in United States, Argentina and 17 more
- Volunteers
- 163 people
- Sponsor
- Novartis Pharmaceuticals
Malignant Neoplasms
A Study of DEG6498 in Participants With Solid Tumors
The goal of this first in human, Phase 1, multi-center, open-label, and 2-part study is to learn whether DEG6498 is safe and tolerable in participants with advanced solid tumors.
- Trial name
- NCT07244835
- Testing
- DEG6498
- Where
- 3 sites in China
- Volunteers
- 100 people
- Sponsor
- Degron Therapeutics Co.
Plexiform Neurofibroma, Neurofibromatosis Type 1 (NF1)
MEK Inhibitor Mirdametinib (PD-0325901) in Patients With Neurofibromatosis Type 1 Associated Plexiform Neurofibromas
This study evaluates mirdametinib (PD-0325901) in the treatment of symptomatic inoperable neurofibromatosis type-1 (NF1)-associated plexiform neurofibromas (PNs). All participants will receive mirdametinib (PD-0325901).
- Trial name
- ReNeu (NCT03962543)
- Testing
- Mirdametinib (PD-0325901) oral capsule or dispersible tablet
- Where
- 43 sites in United States
- Volunteers
- 114 people
- Sponsor
- SpringWorks Therapeutics, Inc., a healthcare company of Merck KGaA, Darmstadt, Germany
Neurofibroma
Non-interventional Study of Patients With PN NF1 Starting Selumetinib in Russia
The purpose of this study is to evaluate the effectiveness, safety of selumetinib and patient's parameters of symptomatic inoperable PN NF1 in real clinical practice in Russia
- Trial name
- NCT05891847
- Where
- 5 sites in Russia
- Volunteers
- 150 people
- Sponsor
- AstraZeneca
Neurofibromatosis Type 1, Plexiform Neurofibromas
Selumetinib in Chinese Paediatric With Post-operative NF1-PNs, PhaseⅡ, Double-Blinded, Placebo-Controlled Study
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…
- Conditions
- Neurofibromatosis Type 1, Plexiform Neurofibromas, Post-operative
- Trial name
- PROTECT (NCT05825365)
- Testing
- Selumetinib
- Sponsor
- AstraZeneca