A Phase 2, Open-Label, Single Arm, Non-Controlled, Single-Stage Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Plexiform Neurofibroma and Neurofibromatosis Type 1
Healx Limited
Summary
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.
Eligibility
- Age range
- 12+ years
- Sex
- All
- Healthy volunteers
- No
Inclusion Criteria: 1. All participants must have a diagnosis of NF1 based on the 2021 revised consensus criteria. 2. Participants must have PN(s) that are progressive OR are causing significant morbidity, such as (but not limited to) head and neck lesions that are compromising the airway or great vessels, brachial or lumbar plexus lesions that are causing nerve compression and loss of function, lesions causing significant disfigurement (e.g., orbital lesions), lesions of the extremity that cause limb hypertrophy or loss of function, and painful lesions. Participants with paraspinal PN will b…
Interventions
- DrugHLX-1502
Participants will take an oral dose three times a day (with or without food) for up to 24 cycles (a cycle is defined as 28 days).
Locations (13)
- University of Alabama at BirminghamBirmingham, Alabama
- Children's Hospital Los AngelesLos Angeles, California
- Children's Hospital ColoradoAurora, Colorado
- Children's National HospitalWashington D.C., District of Columbia
- Ann & Robert H. Lurie Children's Hospital of ChicagoChicago, Illinois
- University of ChicagoChicago, Illinois