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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)
Full description
The study includes 2 parts, phase IIa and IIb. Phase IIa is to evaluate the preliminary safety, pharmacokinetic characteristics and efficacy of HL-085, and to determine the recommended dose. To observe the 9mg dose level, approximately 15 patients will receive HL-085 at a dose of 9mg BID on a continuous dosing schedule(1 cycle=21 days). The investigator and sponsor will evaluate the safety and efficacy data to determine whether HL-085 9mg BID is appropriate. HL-085 12mg BID, 6mg BID, or other HL-085 dosing regimen will be observed as needed. A total of 15-35 patients will be enrolled in phase IIa. Phase IIb is to further evaluate the safety and efficacy of HL-085 in patients with NF1 and inoperable PN and is expected to enroll 35 patients.
Enrollment
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Inclusion criteria
Age: patients must be ≥18 years of age at the time of study entry.
Diagnosis: Patients must have inoperable and symptomatic plexiform neurofibromas(PN), and patients must have NF1 mutation or meet at least 1 of the following NF1 diagnostic criteria:
① ≥6 cafe-au-lait macules ;
② Axillary freckling or freckling in inguinal regions;
③ ≥2 Lisch nodules (iris hamartomas);
④ A distinctive bony lesion such as dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex);
⑤ An optic pathway glioma;
⑥ First-degree relative with NF1.
Patients must have a measurable lesion, defined as at least 3 cm in length, amenable to MRI for efficacy assessment.
Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.
Patients are able to understand and voluntarily sign a written informed consent form.
Patients must be willing and able to complete study procedures and follow-up examinations.
Exclusion criteria
Primary purpose
Allocation
Interventional model
Masking
70 participants in 1 patient group
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Central trial contact
Hongqi Tian, Ph.D; Zhimei Zhu, Master
Data sourced from clinicaltrials.gov
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