Actively Recruiting
A Phase 2, Open-Label, Single Arm Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Plexiform Neurofibroma and Neurofibromatosis Type 1
Led by Healx Limited · Updated on 2026-05-19
25
Participants Needed
14
Research Sites
30 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating HLX-1502 in an open-label, single-arm phase 2 study focused on people aged 16 and older with Neurofibromatosis Type 1 (NF1) who have progressive or symptomatic plexiform neurofibromas (PN). The study also includes a smaller group of participants aged 12 to 15 to investigate safety and effects in this younger group. The goal is to assess how well participants tolerate HLX-1502 and how it affects their condition. Participants will take an oral dose of HLX-1502 three times daily, with or without food, in 28-day cycles. The initial treatment period includes 12 cycles. Those showing partial response, stable disease after prior progression, or clinical improvement may continue treatment for an additional 12 cycles, totaling 24 cycles. This approach aims to explore both short-term and extended treatment effects. During the study, participants will have measurable PNs evaluated by volumetric MRI to monitor tumor response. Researchers will assess the PN response rate at 12 and 24 months, safety, tolerability, pharmacokinetics, and duration and time to response. Participants will be monitored regularly for organ function, performance status, and overall health, with study visits and procedures conducted throughout the treatment period and follow-up. The total study duration varies according to treatment response and continuation criteria.
CONDITIONS
Brief Title
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
Who Can Participate
Eligibility Criteria
You may qualify if you...
- Diagnosis of Neurofibromatosis Type 1 based on 2021 revised consensus criteria
- Progressive or symptomatic plexiform neurofibromas causing significant problems
- Measurable plexiform neurofibromas suitable for volumetric MRI analysis
- Age 12 years or older at study entry
- Weight at least 42 kg
- Lansky (for ages 12-15) or Karnofsky (16+ years) performance score of 50% or higher
- Adequate bone marrow, renal, liver, and pancreatic function
- Blood pressure within normal limits, stable on antihypertensive medication if needed
- Effective contraception use for sexually active participants of childbearing potential
- No planned surgery or other treatments for target lesion during study
- Ability to understand and voluntarily sign informed consent
- Willingness and ability to comply with study procedures and drug administration
You will not qualify if you...
- Prior treatment with HLX-1502 for plexiform neurofibroma
- Recent use of MEK inhibitors, tyrosine kinase inhibitors, or other systemic anti-cancer therapies without full recovery
- Presence of other NF1-related tumors requiring active treatment
- Treatment for high-grade glioma, malignant peripheral nerve sheath tumor, or other malignancies in last 12 months (except certain skin cancers)
- Dental braces or prosthesis interfering with MRI analysis
- Major surgery within 12 weeks or minor surgery within 1 month before treatment without complete healing
- Cataracts detected on eye exam
- Cardiovascular disorders
- Active infection, HIV, immunodeficiency, or hepatitis B or C
- Use of certain medications with narrow therapeutic index during study
- Known severe allergies to HLX-1502 or related compounds, or to FD&C Yellow No. 5
- Therapeutic anticoagulation with vitamin K antagonists
- Iron deficiency or need for copper or zinc treatment at baseline
- Pregnancy or breastfeeding
- Inability or unwillingness to swallow tablets
- Gastrointestinal issues affecting drug absorption
- Other medical or social conditions that may impact safe study participation
AI-Screening
AI-Powered Screening
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Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
1 visit (in-person)
Duration - Up to 24 cycles of 28 days each
Participants take the oral drug HLX-1502 three times a day in 28-day cycles. Initial treatment lasts for 12 cycles. Participants with partial response or stable disease and/or clinical improvement may continue for an additional 12 cycles, up to 24 cycles total.
Regular visits during treatment cycles (schedule per protocol)
Trial Site Locations
Total: 14 locations
1
University of Alabama at Birmingham
Birmingham, Alabama, United States, 35294
Actively Recruiting
2
Children's Hospital Los Angeles
Los Angeles, California, United States, 90027
Actively Recruiting
3
Stanford University
Palo Alto, California, United States, 94304
Actively Recruiting
4
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
5
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
6
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
7
University of Chicago
Chicago, Illinois, United States, 60637
Actively Recruiting
8
Indiana University
Indianapolis, Indiana, United States, 46202
Actively Recruiting
9
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Actively Recruiting
10
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
11
New York University Langone Health
New York, New York, United States, 10016
Actively Recruiting
12
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
13
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
14
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
Research Team
K
Karen Cole-Plourde
L
Lauren Baldwin
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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