A Phase 2 interventional study of HL-085 in Neurofibromatosis 1 and Plexiform Neurofibromas, sponsored by Shanghai Kechow Pharma, Inc.. Recruiting at 1 site in China. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2023-05-31.
Sponsored by Shanghai Kechow Pharma, Inc. · Phase 2, Interventional, and Treatment
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)
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.
176 studies on the registry are indexed under Neurofibromatosis 1; 43 are open to participants now.
This study's planned enrollment of 70 is above the median of 35 across 114 interventional studies indexed under Neurofibromatosis 1.
Browse Neurofibromatosis 1 studies →Shanghai Kechow Pharma, Inc. is the lead sponsor of 12 studies on the registry; 8 are open to participants now.
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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.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Measurable disease
The amount of disease can be tracked with a defined measurement, so the study can see whether it shrinks or grows. It might be a tumour measured on a scan or, in some blood cancers such as myeloma, a marker measured in blood or urine.
The criteria or the study team say which measurement counts — having no tumour visible on a scan does not by itself rule you out.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ECOG performance status
A scale of how much an illness limits everyday activity. For people being assessed to join a study, scores run from 0 (fully active) to 4; lower is more active.
Studies most often ask for 0–1 or 0–2. Your doctor gives the score; the WHO scale uses the same steps. The full scale also has 5, which records death.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HL-085 9mg BID
Drug: 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.
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)
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)
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)
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 years
Pharmacokinetic characteristics
AUC
Time frame: During the intervention
Plan to share: Undecided
No publications or documents are linked to this record.
Started Oct 2021 (actual). Primary completion is estimated for Oct 2025, 48 months after the start.
Of 24 phase 2 interventional studies under Neurofibromatosis 1, half reached primary completion within 62 months of starting (the median), counting those still running; a quarter within 40 and three quarters within 95. This study's planned 48 months is below that.
How these figures are worked out, and which studies are left out of them.
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Shanghai Kechow Pharma, Inc.