Actively Recruiting
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
Led by Healx Limited · Updated on 2026-03-20
25
Participants Needed
13
Research Sites
150 weeks
Total Duration
On this page
AI-Summary
What this Trial Is About
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. This study will also investigate the safety and efficacy of HLX-1502 in a small cohort of 12 to 15 year olds.
CONDITIONS
Official 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 (NF1) based on 2021 consensus criteria
- Presence of progressive or symptomatic plexiform neurofibromas causing significant morbidity
- Measurable plexiform neurofibromas suitable for volumetric MRI analysis, with tumors at least 3 mL in volume or meeting specific size criteria
- Age 12 years or older at study entry
- Weight of at least 42 kg
- Lansky score (for ages 12-15) or Karnofsky score (for age 16+) of 50% or higher
- Adequate bone marrow, kidney, liver, and pancreatic function; controlled blood pressure within normal limits on a stable regimen if needed
- Agreement to use effective contraception during the study and for 4 months after last dose, if applicable
- No planned surgery or other treatments for the target lesion during the study except the study drug
- Ability to understand and sign informed consent
- Willingness and ability to attend study visits, follow study procedures, and take study medication as directed
You will not qualify if you...
- Prior treatment with HLX-1502 for a plexiform neurofibroma
- Use of MEK inhibitors, tyrosine kinase inhibitors, HLX-1502, or other systemic anti-cancer therapies within specified times before enrollment
- Presence of NF1-related tumors requiring active treatment such as gliomas or malignant peripheral nerve sheath tumors
- Recent treatment for high-grade glioma, malignant peripheral nerve sheath tumor, or other malignancies within 12 months, except certain skin cancers treated curatively
- Dental braces or prostheses interfering with tumor MRI analysis
- Recent major or minor surgery without complete wound healing before first dose
- Presence of cataracts on eye exam
- Cardiovascular disorders
- Significant infections, known HIV, immunodeficiency, or hepatitis B or C
- Need for certain medications with a narrow therapeutic index during the study
- Known severe allergies to HLX-1502 or its components
- Allergy to FD&C Yellow No. 5
- Use of therapeutic anticoagulation with vitamin K antagonists
- Active iron deficiency or treatment with iron, copper, or zinc at baseline
- Pregnancy or breastfeeding
- Inability or unwillingness to swallow tablets
- Gastrointestinal conditions affecting absorption of the study drug
- Other medical, social, or concurrent issues that could interfere with safe participation
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Trial Site Locations
Total: 13 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
Children's Hospital Colorado
Aurora, Colorado, United States, 80045
Actively Recruiting
4
Children's National Hospital
Washington D.C., District of Columbia, United States, 20010
Actively Recruiting
5
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, United States, 60611
Actively Recruiting
6
University of Chicago
Chicago, Illinois, United States, 60637
Actively Recruiting
7
Indiana University
Indianapolis, Indiana, United States, 46202
Actively Recruiting
8
Johns Hopkins Hospital
Baltimore, Maryland, United States, 21287
Actively Recruiting
9
Washington University School of Medicine
St Louis, Missouri, United States, 63110
Actively Recruiting
10
New York University Langone Health
New York, New York, United States, 10016
Actively Recruiting
11
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, United States, 45229
Actively Recruiting
12
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, United States, 19104
Actively Recruiting
13
University of Texas Southwestern Medical Center
Dallas, Texas, United States, 75390
Actively Recruiting
Research Team
K
Karen Cole-Plourde
CONTACT
L
Lauren Baldwin
CONTACT
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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