Brief Title
HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
Official Title
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
Brief Summary
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)
Detailed 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.
Study Phase
Phase 2
Study Type
Interventional
Primary Outcome
Objective Response Rate (ORR)
Secondary Outcome
Disease Control Rate(DCR)
Condition
Neurofibromatosis 1
Intervention
HL-085
Study Arms / Comparison Groups
HL-085
Description: HL-085 9mg BID
Publications
* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.
Recruitment Information
Recruitment Status
Drug
Estimated Enrollment
70
Start Date
October 18, 2021
Completion Date
October 31, 2028
Primary Completion Date
October 30, 2025
Eligibility Criteria
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: - Patients who are unable to undergo MRI scans (prosthesis, prosthesis, braces, etc.) or patients with lesions that cannot be evaluated by MRI. - Patients do not have adequate organ function. - Patients who are unable to take drugs orally, have difficulty swallowing or anything that may lead to inadequate drug absorption. - Prior treatment with MEK 1/2 inhibitors. - Patients known to be allergic to the ingredients or analogues of the study drug. - Patients with previous or current retinal diseases such as retinal vein occlusion (RVO), retinal pigment epithelium detachment (RPED), central serous retinopathy (CSR), etc. (except retinopathy caused by research diseases). - With infections or other uncontrolled disease. - Strong CYP2C9 inhibitors or inducers within 7 days before treatment of the study drug. - Patients who received surgery within 4 weeks or radiotherapy within 6 weeks before enrollment. - Patients who participated in any other clinical study treatment within 4 weeks before enrollment. - Patients treated with anti-NF1 treatment with unresolved chronic toxicity. - Clinical judgment by the investigator that the patient should not participate in the study.
Gender
All
Ages
18 Years - 80 Years
Accepts Healthy Volunteers
No
Contacts
Hongqi Tian, Ph.D, 86 215201345822, [email protected]
Location Countries
China
Location Countries
China
Administrative Informations
NCT ID
NCT05331105
Organization ID
HL-085-106-II
Responsible Party
Sponsor
Study Sponsor
Shanghai Kechow Pharma, Inc.
Study Sponsor
Hongqi Tian, Ph.D, Study Chair, Shanghai Kechow Pharma, Inc.
Verification Date
April 2022