HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas

  • STATUS
    Recruiting
  • End date
    Oct 31, 2028
  • participants needed
    70
  • sponsor
    Shanghai Kechow Pharma, Inc.
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Updated on 28 April 2022
See if I qualify
measurable disease
dysplasia
nf1 mutation

Summary

This is 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(PN)

Description

The study includes 2 parts, phase IIa and IIb. Phase IIa is to evaluate the preliminary safety, pharmacokinetic characteristics and efficacy of HL-085, and to determine the recommended dose. To observe the 9mg dose level, approximately 15 patients will receive HL-085 at a dose of 9mg BID on a continuous dosing schedule(1 cycle=21 days). The investigator and sponsor will evaluate the safety and efficacy data to determine whether HL-085 9mg BID is appropriate. HL-085 12mg BID, 6mg BID, or other HL-085 dosing regimen will be observed as needed. A total of 15-35 patients will be enrolled in phase IIa. Phase IIb is to further evaluate the safety and efficacy of HL-085 in patients with NF1 and inoperable PN and is expected to enroll 35 patients.

Details
Condition Neurofibromatosis 1, Plexiform Neurofibromas
Treatment HL-085
Clinical Study IdentifierNCT05331105
SponsorShanghai Kechow Pharma, Inc.
Last Modified on28 April 2022

Eligibility

 Yes No Not Sure

Inclusion Criteria

Age: patients must be ≥18 years of age at the time of study entry
Diagnosis: Patients must have inoperable and symptomatic plexiform neurofibromas(PN), and patients must have NF1 mutation or meet at least 1 of the following NF1 diagnostic
criteria
① ≥6 cafe-au-lait macules
② Axillary freckling or freckling in inguinal regions
③ ≥2 Lisch nodules (iris hamartomas)
④ A distinctive bony lesion such as dysplasia of the sphenoid bone or dysplasia or
thinning of long bone cortex)
⑤ An optic pathway glioma
⑥ First-degree relative with NF1
Patients must have a measurable lesion, defined as at least 3 cm in length, amenable
to MRI for efficacy assessment
Eastern Cooperative Oncology Group (ECOG) performance status of 0-2
Patients are able to understand and voluntarily sign a written informed consent form
Patients must be willing and able to complete study procedures and follow-up
examinations

Exclusion Criteria

Patients who are unable to undergo MRI scans (prosthesis, prosthesis, braces, etc.) or
patients with lesions that cannot be evaluated by MRI
Patients do not have adequate organ function
Patients who are unable to take drugs orally, have difficulty swallowing or anything
that may lead to inadequate drug absorption
Prior treatment with MEK 1/2 inhibitors
Patients known to be allergic to the ingredients or analogues of the study drug
Patients with previous or current retinal diseases such as retinal vein occlusion
(RVO), retinal pigment epithelium detachment (RPED), central serous retinopathy (CSR)
etc. (except retinopathy caused by research diseases)
With infections or other uncontrolled disease
Strong CYP2C9 inhibitors or inducers within 7 days before treatment of the study drug
Patients who received surgery within 4 weeks or radiotherapy within 6 weeks before
enrollment
Patients who participated in any other clinical study treatment within 4 weeks before
enrollment
Patients treated with anti-NF1 treatment with unresolved chronic toxicity
Clinical judgment by the investigator that the patient should not participate in the
study
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If you are confirmed eligible after full screening, you will be required to understand and sign the informed consent if you decide to enroll in the study. Once enrolled you may be asked to make scheduled visits over a period of time.

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Complete your scheduled study participation activities and then you are done. You may receive summary of study results if provided by the sponsor.

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