Neurofibromatosis Type 1Phase 2INSPIRE-NF1NCT06541847
A Phase 2, Open-Label Study to Evaluate the Safety and Effects of HLX-1502 in Patients With Neurofibromatosis Type 1
A Phase 2 study testing HLX-1502 in people with Neurofibromatosis Type 1, run by Healx Limited at 14 sites in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 12 years and older
- Conditions the study is for
- Neurofibromatosis Type 1
- What is being tested?
- HLX-1502
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 25 participants
- Where
- 14 sitesUnited States
- Study dates
- 10 Feb 2025to Jan 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 12 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- All participants must have a diagnosis of NF1 based on the 2021 revised consensus criteria.
- 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 be eligible for this trial. Histologic confirmation of tumor is not necessary but should be considered if there are clinical or radiographic findings concerning for malignant transformation of a PN.
- Measurable Disease: Participants must have measurable PN(s) amenable to volumetric MRI analysis. For the purpose of this study, the target lesion must be seen on at least 3 consecutive MRI slices and the field of view must contain the entire tumor of interest. Tumors must be at least 3 mL in volume (most PN 3 cm in longest diameter will meet this criteria). If the tumor is < 3 cm in longest diameter, the participant may still be eligible. Central review of the MRI of the target PN is required prior to enrollment to ensure that the tumor is measurable and amenable to volumetric analysis.
- Age: Participants must be ≥ 12 years of age at the time of study entry. Note: Although prior MEKi therapy is not a requirement, patients should be counseled on the availability of FDA-approved MEKi therapies prior to enrollment.
- Weight ≥ 42 kg.
- Performance Level: Participants must have a Lansky (12-15 years of age) or Karnofsky (16+ years of age) score ≥ 50%.
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- Organ Function Requirements: Adequate Bone Marrow Function, Adequate Renal Function, Adequate Liver Function, Normal pancreatic function: amylase and lipase levels ≤ 1.5 x ULN. Blood pressure within upper limit of normal as defined below based on the average of the 2nd and 3rd of a total of 3 consecutive measurements, 5 minutes apart. Antihypertensives are permissible to achieve blood pressure within ULN, however must be on stable antihypertensive regimen with no adjustments within 14 days of enrollment.
- Sexually active women of childbearing potential and fertile male participants and their partners must agree to use effective methods of contraception e.g., hormonal oral contraception, injectables, intrauterine device, surgical sterilization including vasectomy, or hormonal implant with barrier methods (male condom, female condom, or diaphragm with spermicidal gel) during the course of the study and for 4 months after the last dose of study treatment. Barrier methods alone are insufficient. True sexual abstinence or evidence of surgical sterility (e.g. vasectomized partner, post-hysterectomy, menopause with last menstrual period >=12 months prior to screening visit) are acceptable method of birth control. Persons of childbearing potential will be given a pregnancy test within 14 days prior to first dose of study treatment and must have a negative urine or serum pregnancy test.
- The participant is not planning to undergo surgery or other interventions/ treatments for the target lesion, except protocol specified therapy, for the duration of the study.
- The participant or the participant's legal authorized representative is able to understand the informed consent form describing the risks of the study and voluntarily signs the informed consent document.
- In the opinion of the investigator, the participant is willing and able to attend study visits, comply with the study procedures as specified in the protocol, and comply with the administration of the study drug.
Who cannot join
- Prior treatment with HLX-1502 for a PN.
- The participant has used any of the following systemic medications/ therapies within the specified period prior to enrollment: MEK-inhibitors, other drugs in the TKI class, HLX-1502, Participants may have received treatment for a PN or other tumor/malignancy but must have fully recovered to baseline or CTCAE ≤ Grade 1 from acute toxicities from prior therapies except alopecia, Myelosuppressive chemotherapy, Hematopoietic growth factors, Biologic (anti-neoplastic agent), Investigational Drugs, Any other systemically administered anti-neoplastic agent and Radiation therapy.
- Evidence of an NF1-related tumor such as optic pathway or other low-grade glioma, high-grade glioma, malignant peripheral nerve sheath tumor, or other cancer/tumor requiring treatment with chemotherapy, biologic therapy, surgery or radiation therapy.
- Participants with high-grade glioma, malignant peripheral nerve sheath tumor, or other malignancy who received treatment in the last 12 months. Exceptions include basal cell carcinoma of the skin and squamous cell carcinoma of the skin that have undergone potentially curative therapy. If the investigator has any clinical concerns for ANNUBP/Atypical Neurofibroma or MPNST, a biopsy sample must be taken prior to study confirming eligibility.
- Dental braces or prosthesis that interfere with volumetric analysis of the neurofibroma(s).
- Surgery: Any major surgery within 12 weeks before starting the Treatment Period or foreseen during participation in the trial, any minor surgeries within 1 month before first dose of study treatment and Participants must have complete wound healing from major surgery or minor surgery before the first dose of study treatment. Participants with clinically relevant ongoing complications from prior surgery are not eligible.
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- Cataracts noted on ophthalmologic exam.
- Cardiovascular disorders.
- Other clinically significant disorders that would preclude safe study participation, including active infection, a known history of HIV seropositivity or known immunodeficiency or Known history of Hepatitis B or Hepatitis C.
- Participants who require treatment with a drug that is a substrate of CYP1A2, CYP2C8, UGT1A1, UGT1A3 with a narrow therapeutic index during protocol therapy.
- Known severe sensitivity to HLX-1502 or any excipient of HLX-1502 or history of allergic reactions attributed to compounds of similar chemical or biologic composition to HLX-1502.
- Known severe sensitivity to FD\&C Yellow No. 5.
- Participants receiving therapeutic anticoagulation with vitamin K antagonist.
- Participants presently with iron deficiency and/or actively receiving iron replacement or requiring treatment with copper or zinc for any indication at study baseline.
- Pregnant or breast-feeding women.
- Unable or unwilling to swallow tablets.
- Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter ingestion and/or the absorption of HLX-1502.
- Participants who have other medical, social or concurrent challenges that are likely to negatively impact their ability to meet all of the trial obligations and therefore may increase the risk of safe participation in the study.
Trial locations and contacts
14 sites in 1 country. Contact the study team or a site near you to ask about taking part.
Lauren Baldwin contact
14 sites in 1 country
United States14 sites, 14 recruiting
- Recruiting
Children's Hospital Colorado
Aurora, Colorado, 80045
Sarah Dickens, sarah.dickens@childrenscolorado.org
Nicholas Foreman
- Recruiting
Johns Hopkins Hospital
Baltimore, Maryland, 21287
Joshua Roberts, jrobe112@jhmi.edu
Jaishri Blakeley
- Recruiting
University of Alabama at Birmingham
Birmingham, Alabama, 35294
Brian Roberts, brianroberts@uabmc.edu
Girish Dhall
- Recruiting
Ann & Robert H. Lurie Children's Hospital of Chicago
Chicago, Illinois, 60611
Kelly Bruno, kbruno@luriechildrens.org
Miriam Bornhorst
- Recruiting
University of Chicago
Chicago, Illinois, 60637
Cynthia MacKenzie, cmackenzie@bsd.uchicago.edu
James Tonsgard
- Recruiting
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
Lori Backus, lori.backus@cchmc.org
Peter De Blank
- Recruiting
University of Texas Southwestern Medical Center
Dallas, Texas, 75390
Omar Raslan, omar.raslan@utsouthwestern.edu
Laura Klesse
- Recruiting
Indiana University
Indianapolis, Indiana, 46202
Sara Quetant, squetant@iu.edu
Steven Rhodes
- Recruiting
Children's Hospital Los Angeles
Los Angeles, California, 90027
Sandy Gutierrez, sagutierrez@chla.usc.edu
Tena Rosser
- Recruiting
New York University Langone Health
New York, New York, 10016
Anna Yaffe, Anna.Yaffe@nyulangone.org
Kaleb Yohay
- Recruiting
Stanford University
Palo Alto, California, 94304
Jenika Maala, jmaala@stanford.edu
Cynthia Campen
- Recruiting
Children's Hospital of Philadelphia
Philadelphia, Pennsylvania, 19104
Ratnakar Patti, PATTIR@chop.edu
Michael Fisher
- Recruiting
Washington University School of Medicine
St Louis, Missouri, 63110
Audrey Merz, amerz@wustl.edu
Amy Armstrong
- Recruiting
Children's National Hospital
Washington D.C., District of Columbia, 20010
NeuroFibromatosis Team, NF@childrensnational.org
Benjamin Siegel
About this study
Official title: 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
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
- Neurofibromatosis Type 1
- Being tested
- HLX-1502
- Keywords
- Neurofibromatosis Type 1, Plexiform Neurofibroma, pediatric
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HLX-1502Drug
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).
| Study group | Role | What they receive |
|---|---|---|
| HLX-1502 | Experimental | Participants will take an oral dose three times a day (with or without food) for 12 cycles. A cycle is defined as 28 days. Participants with a partial response or stable disease with a previously progressive tumor and/or clinical improvement can continue therapy for an additional 12 cycles for a total of 24 cycles. Receives: Drug: HLX-1502 |
What is being measured
The primary outcome is the main question the study is designed to answer.
PN response rate
Response will be determined by a blinded centralized review of volumetric MRI. A partial response (PR) is defined as ≥ 20% decrease in the volume of target PN.
Measured: 12 months
Secondary outcomes (5)
Duration of response (DOR) and Time to response (TOR)
Response will be determined by a blinded centralized review of volumetric MRI.
Measured: 12 months
Safety and tolerability of HLX-1502
Adverse events (AEs) will be assessed according to toxicities graded by National Cancer Institute (NCI) Common Terminology Criteria for Adverse Events (CTCAE) Version 5.0.
Measured: 12 months
PN response rate
Response will be determined by a blinded centralized review of volumetric MRI. A partial response (PR) is defined as ≥ 20% decrease in the volume of target PN.
Measured: 24 months
Pharmacokinetics of HLX-1502
Measured by plasma concentrations
Measured: 12 months
Confirmed PN response rate
Confirmed response will be determined by a blinded centralized review of volumetric MRI
Measured: 12 and 24 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted7 Aug 2024
- Study start10 Feb 2025
- Primary completionJun 2027 (estimated)
- Study completionJan 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis Type 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 25 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Healx Limited
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Miriam Bornhorst, Study chair, Lurie's Children's HospitalMichael Fisher, Study chair, Children's Hospital of PhiladelphiaKathryn Nevel, Study chair, Indiana University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06541847
- Sponsor's protocol ID
- HLX015-0201
- Acronym
- INSPIRE-NF1
- Status last verified
- Jul 2026
- First submitted
- 30 Jul 2024
- Last updated
- 29 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 29 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT06541847
- Acronym / sponsor ID
- INSPIRE-NF1 / HLX015-0201
- Conditions
- Neurofibromatosis Type 1
- Keywords
- Neurofibromatosis Type 1, Plexiform Neurofibroma, pediatric
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 25 participants (estimated)
- Lead sponsor
- Healx LimitedIndustry (pharma, biotech, devices)
- Study start
- 10 Feb 2025 (actual)
- Primary completion
- Jun 2027 (estimated)
- Study completion
- Jan 2028 (estimated)
- First posted
- 7 Aug 2024 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HLX-1502 | Experimental | Drug: HLX-1502Participants will take an oral dose three times a day (with or without food) for 12 cycles. A cycle is defined as 28 days. Participants with a partial response or stable disease with a previously progressive tumor and/or clinical improvement can continue therapy for an additional 12 cycles for a total of 24 cycles. |
Who may qualify
- Ages
- 12 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (11 listed)
- All participants must have a diagnosis of NF1 based on the 2021 revised consensus criteria.
- 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 be eligible for this trial. Histologic confirmation of tumor is not necessary but should be considered if there are clinical or radiographic findings concerning for malignant transformation of a PN.
- Measurable Disease: Participants must have measurable PN(s) amenable to volumetric MRI analysis. For the purpose of this study, the target lesion must be seen on at least 3 consecutive MRI slices and the field of view must contain the entire tumor of interest. Tumors must be at least 3 mL in volume (most PN 3 cm in longest diameter will meet this criteria). If the tumor is < 3 cm in longest diameter, the participant may still be eligible. Central review of the MRI of the target PN is required prior to enrollment to ensure that the tumor is measurable and amenable to volumetric analysis.
- Age: Participants must be ≥ 12 years of age at the time of study entry. Note: Although prior MEKi therapy is not a requirement, patients should be counseled on the availability of FDA-approved MEKi therapies prior to enrollment.
- Weight ≥ 42 kg.
Key exclusion criteria (18 listed)
- Prior treatment with HLX-1502 for a PN.
- The participant has used any of the following systemic medications/ therapies within the specified period prior to enrollment: MEK-inhibitors, other drugs in the TKI class, HLX-1502, Participants may have received treatment for a PN or other tumor/malignancy but must have fully recovered to baseline or CTCAE ≤ Grade 1 from acute toxicities from prior therapies except alopecia, Myelosuppressive chemotherapy, Hematopoietic growth factors, Biologic (anti-neoplastic agent), Investigational Drugs, Any other systemically administered anti-neoplastic agent and Radiation therapy.
- Evidence of an NF1-related tumor such as optic pathway or other low-grade glioma, high-grade glioma, malignant peripheral nerve sheath tumor, or other cancer/tumor requiring treatment with chemotherapy, biologic therapy, surgery or radiation therapy.
- Participants with high-grade glioma, malignant peripheral nerve sheath tumor, or other malignancy who received treatment in the last 12 months. Exceptions include basal cell carcinoma of the skin and squamous cell carcinoma of the skin that have undergone potentially curative therapy. If the investigator has any clinical concerns for ANNUBP/Atypical Neurofibroma or MPNST, a biopsy sample must be taken prior to study confirming eligibility.
- Dental braces or prosthesis that interfere with volumetric analysis of the neurofibroma(s).
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HLX-1502Participants 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). | Drug | HLX-1502 |
Primary outcome
PN response rate
Time frame: 12 months
Response will be determined by a blinded centralized review of volumetric MRI. A partial response (PR) is defined as ≥ 20% decrease in the volume of target PN.
5 secondary outcomes
Duration of response (DOR) and Time to response (TOR)
Time frame: 12 months
Safety and tolerability of HLX-1502
Time frame: 12 months
PN response rate
Time frame: 24 months
Pharmacokinetics of HLX-1502
Time frame: 12 months
Confirmed PN response rate
Time frame: 12 and 24 months
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 29 Jul 2026.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Lauren Baldwin contact
Miriam Bornhorst study chair
Lurie's Children's Hospital
Michael Fisher study chair
Children's Hospital of Philadelphia
Kathryn Nevel study chair
Indiana University
14 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HLX-1502 in people with Neurofibromatosis Type 1, run by Healx Limited at 14 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 11 inclusion and 18 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 29 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 14 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Karen Cole-Plourde ((205) 934-5140, kplourde@uab.edu), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
PN response rate (time frame: 12 months). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 29 Jul 2026.