Neurofibromatosis 1Phase 2NCT05331105
HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
A Phase 2 study testing HL-085 in people with Neurofibromatosis 1 and Plexiform Neurofibromas, run by Shanghai Kechow Pharma, Inc. at 1 site in China.
Trial 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) Read more about this study
- Who is it for?
- Ages 18 to 80 years
- Conditions the study is for
- Neurofibromatosis 1, Plexiform Neurofibromas
- What is being tested?
- HL-085
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 70 participants
- Where
- 1 siteChina
- Study dates
- 18 Oct 2021to 31 Oct 2028
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 80 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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);
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- 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.
Who cannot join
- 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).
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- 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.
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
Hongqi Tian, Ph.D contact
China1 site, 1 recruiting
- Recruiting
Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine
Shanghai, Shanghai Municipality, 200011
Qingfeng Li, M.D., 13301990666, dr.liqingfeng@shsmu.edu.cn
About this study
Official title: 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
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)
Read the 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
- Being tested
- HL-085
- Medical subject headings
- Neurofibromatosis 1, Neurofibroma, Plexiform
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
HL-085Drug
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.
| Study group | Role | What they receive |
|---|---|---|
| HL-085 | Experimental | HL-085 9mg BID Receives: Drug: HL-085 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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).
Measured: 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）
Defined as the percentage of patients who have achieved a confirmed response of CR or PR or SD
Measured: 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
Measured: 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)
Measured: 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
Measured: During the intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted15 Apr 2022
- Study start18 Oct 2021
- Primary completion30 Oct 2025 (estimated)
- Study completion31 Oct 2028 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Neurofibromatosis 1Studies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Shanghai Kechow Pharma, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Hongqi Tian, Ph.D, Study chair, Shanghai Kechow Pharma, Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05331105
- Sponsor's protocol ID
- HL-085-106-II
- Status last verified
- May 2023
- First submitted
- 15 Mar 2022
- Last updated
- 31 May 2023
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- Undecided
Study at a glance
From the registry record, last updated by the sponsor on 31 May 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- 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
- ClinicalTrials.gov ID
- NCT05331105
- Acronym / sponsor ID
- HL-085-106-II
- Conditions
- Neurofibromatosis 1, Plexiform Neurofibromas
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- Shanghai Kechow Pharma, Inc.Industry (pharma, biotech, devices)
- Study start
- 18 Oct 2021 (actual)
- Primary completion
- 30 Oct 2025 (estimated)
- Study completion
- 31 Oct 2028 (estimated)
- First posted
- 15 Apr 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| HL-085 | Experimental | Drug: HL-085HL-085 9mg BID |
Who may qualify
- Ages
- 18 to 80 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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);
Key exclusion criteria (12 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| HL-085IIa: 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. | Drug | HL-085 |
Primary outcome
Objective Response Rate (ORR)
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)
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).
4 secondary outcomes
Disease Control Rate（DCR）
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）
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)
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
Time frame: During the intervention
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 31 May 2023.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
Hongqi Tian, Ph.D contact
Hongqi Tian, Ph.D study chair
Shanghai Kechow Pharma, Inc.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing HL-085 in people with Neurofibromatosis 1 and Plexiform Neurofibromas, run by Shanghai Kechow Pharma, Inc. at 1 site in China.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 12 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 31 May 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in China. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Zhimei Zhu, Master (86 215201345822, zhuzm@kechowpharma.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Objective Response Rate (ORR) (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)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 31 May 2023.