HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
Trial statusRecruiting
Trial phasePhase 2
Trial typeInterventional
Biological sexAll
Age18-80
SponsorShanghai Kechow Pharma, Inc.
About this trial
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)
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
Age: patients must be ≥18 years of age at the time of study entry.
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.
Disqualifiers
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.
Trial design
Design model
Single group
Treatments tested in this trial
HL-085
DrugIIa: 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.
Treatment groups
70 Participants
are divided into 1 treatment groupGroup A: HL-085Experimental treatment 1 intervention
Trial outcomes
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).
Time frame
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
1
Disease Control Rate（DCR）
Defined as the percentage of patients who have achieved a confirmed response of CR or PR or SD
Time frame
At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days)2
Duration of Overall Response（DOR）
Defined as the time from first achieved CR or PR to disease progression
Time frame
At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days)3
Progression Free survival (PFS)
Defined as the time from first dosing (C1D1) to date of first observed progression or death from any cause (whichever comes first)
Time frame
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 years4
Pharmacokinetic characteristics
AUC
Time frame
During the interventionSponsors and contacts
Click on the lead sponsor to view all of their trials.
Source ClinicalTrials.gov