Actively Recruiting
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
Led by Shanghai Kechow Pharma, Inc. · Updated on 2023-05-31
70
Participants Needed
1
Research Sites
156 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating HL-085 in adults with Neurofibromatosis Type 1 (NF1) who have inoperable and symptomatic plexiform neurofibromas (PN). This multi-center, open-label, single-arm Phase II study aims to assess the safety, efficacy, and pharmacokinetics of HL-085. The study includes two parts: phase IIa to determine a recommended dose and evaluate preliminary safety and efficacy, and phase IIb to further assess safety and efficacy in a larger group of patients. During phase IIa, approximately 15 patients will receive HL-085 orally at a dose of 9 mg twice daily in continuous 21-day cycles. Dosage adjustments to 12 mg or 6 mg twice daily may be made if needed. Phase IIb will enroll around 35 patients to receive HL-085 at the recommended dose determined from phase IIa. Treatment continues in cycles of 21 days. Participants will undergo regular assessments including MRI scans to measure tumor response at the end of cycles 4, 8, 12, 16, 20, 24, 28, and 32, then every 8 cycles thereafter. Researchers will monitor objective response rate, disease control rate, duration of response, progression-free survival up to 3 years, and pharmacokinetics during treatment. Patients must be able to complete follow-up exams and provide informed consent, with overall participation lasting through multiple treatment cycles and evaluations.
CONDITIONS
Brief Title
HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Patients must be 18 years of age or older at the time of study entry.
- Diagnosis of Neurofibromatosis Type 1 (NF1) with inoperable and symptomatic plexiform neurofibromas.
- Meet at least one of the following NF1 diagnostic criteria: 6 or more cafe-au-lait macules, axillary or inguinal freckling, 2 or more Lisch nodules, distinctive bony lesions, optic pathway glioma, or have a first-degree relative with NF1.
- Have at least one measurable lesion 3 cm or longer suitable for MRI assessment.
- Eastern Cooperative Oncology Group (ECOG) performance status of 0 to 2.
- Able to understand and voluntarily sign informed consent.
- Willing and able to complete study procedures and follow-up examinations.
You will not qualify if you...
- Unable to undergo MRI scans or have lesions that cannot be evaluated by MRI.
- Inadequate organ function.
- Unable to take drugs orally or have difficulty swallowing that may affect drug absorption.
- Prior treatment with MEK 1/2 inhibitors.
- Known allergy to the study drug or its ingredients.
- Previous or current retinal diseases such as retinal vein occlusion, retinal pigment epithelium detachment, or central serous retinopathy (except retinopathy caused by NF1).
- Uncontrolled infections or other diseases.
- Use of strong CYP2C9 inhibitors or inducers within 7 days before study drug treatment.
- Surgery within 4 weeks or radiotherapy within 6 weeks before enrollment.
- Participation in another clinical study within 4 weeks before enrollment.
- Treatment with anti-NF1 therapies with unresolved chronic toxicity.
- Investigator judgement that participation is not appropriate.
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Repeated 21-day cycles until disease progression or discontinuation, assessed up to 3 years
Participants receive HL-085 capsules orally twice daily in continuous 21-day treatment cycles. Dosage may be adjusted as needed.
Visits at the end of cycles 4, 8, 12, 16, 20, 24, 28, and 32, then every 8 cycles (each cycle is 21 days)
Trial Site Locations
Total: 1 location
1
Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, China, 200011
Actively Recruiting
Research Team
Z
Zhimei Zhu, Master
H
Hongqi Tian, Ph.D
How is the study designed?
Study Type
INTERVENTIONAL
Masking
NONE
Allocation
NA
Model
SINGLE_GROUP
Primary Purpose
TREATMENT
Number of Arms
1
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