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

NCT ID: NCT05331105

Last Updated: 2023-05-31

Basic Information

• Recruitment Status: RECRUITING
• Clinical Phase: PHASE2
• Total Enrollment: 70 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-10-18
• Study Completion Date: 2028-10-31

Brief 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)

Detailed 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.

Conditions

Neurofibromatosis 1; Plexiform Neurofibromas

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

HL-085

HL-085 9mg BID

Group Type: EXPERIMENTAL

HL-085

Intervention Type: DRUG

IIa: HL-085 capsule 9mg administered orally twice daily in a continuous 21-day treatment cycle. If required, dosing schedule can be adjusted to 12mg BID, 6mg BID, or other dosage regimens.

IIb: HL-085 at the recommended dose or dosage regimen.

Interventions

HL-085

IIa: HL-085 capsule 9mg administered orally twice daily in a continuous 21-day treatment cycle. If required, dosing schedule can be adjusted to 12mg BID, 6mg BID, or other dosage regimens.

IIb: HL-085 at the recommended dose or dosage regimen.

Intervention Type: DRUG

Eligibility Criteria

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.

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 80 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Shanghai Kechow Pharma, Inc.

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Hongqi Tian, Ph.D

Role: STUDY_CHAIR

Shanghai Kechow Pharma, Inc.

Locations

Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine

Shanghai, Shanghai Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Zhimei Zhu, Master

Role: CONTACT

zhuzm@kechowpharma.com

86 215201345822

Hongqi Tian, Ph.D

Role: CONTACT

tianhq@kechowpharma.com

Facility Contacts

Qingfeng Li, M.D.

Role: primary

dr.liqingfeng@shsmu.edu.cn

13301990666

Other Identifiers

HL-085-106-II

Identifier Type: -

Identifier Source: org_study_id