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

NCT05331105 | Recruiting Phase 2

• 1 other identifier: HL-085-106-II
• Study Type: interventional
• Target: 70
• Locations: 1 country
• Sites: 1

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)

Conditions

Neurofibromatosis 1; Plexiform Neurofibromas

Outcome Measures

Primary Outcomes (1)

• Objective Response Rate (ORR)

  To assess the efficacy of HL-085 on the tumor volume (plexiform neurofibromas) using volumetric MRI per REiNS criteria. ORR is defined as the percentage of patients who have achieved a confirmed Partial Responses (PR) or Complete Responses (CR).

  At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days)

Secondary Outcomes (4)

• Disease Control Rate（DCR）

  At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days)

• Duration of Overall Response（DOR）

  At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days)

• Progression Free survival (PFS)

  From date of dosing until the date of first documented progression or date of death from any cause, whichever came first, assessed up to 3 years

• Pharmacokinetic characteristics

  During the intervention

Study Arms (1)

HL-085

EXPERIMENTAL

HL-085 9mg BID

Drug: HL-085

Interventions

HL-085 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.

HL-085

Eligibility Criteria

• Age: 18 Years - 80 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Shanghai Kechow Pharma, Inc.: lead

Study Sites (1)

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

Shanghai, Shanghai Municipality, 200011, China

RECRUITING

MeSH Terms

Conditions

Neurofibromatosis 1; Neurofibroma, Plexiform

Condition Hierarchy (Ancestors)

Neurofibromatoses; Neurofibroma; Nerve Sheath Neoplasms; Neoplasms, Nerve Tissue; Neoplasms by Histologic Type; Neoplasms; Neoplastic Syndromes, Hereditary; Neurocutaneous Syndromes; Nervous System Diseases; Heredodegenerative Disorders, Nervous System; Neurodegenerative Diseases; Peripheral Nervous System Diseases; Neuromuscular Diseases; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities; Peripheral Nervous System Neoplasms; Nervous System Neoplasms

Study Officials

• Hongqi Tian, Ph.D

  Shanghai Kechow Pharma, Inc.

  STUDY CHAIR

Central Study Contacts

Zhimei Zhu, Master

CONTACT

86 215201345822; zhuzm@kechowpharma.com

Hongqi Tian, Ph.D

CONTACT

tianhq@kechowpharma.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 15, 2022
• First Posted: April 15, 2022
• Study Start: October 18, 2021
• Primary Completion: October 30, 2025
• Study Completion (Estimated): October 31, 2028
• Last Updated: May 31, 2023

Record last verified: 2023-05

Locations

CN (1)