Ignite Creation Date:
2025-12-25 @ 3:24 AM
Ignite Modification Date:
2025-12-26 @ 2:04 AM
Study NCT ID:
NCT05331105
Status:
RECRUITING
Last Update Posted:
2023-05-31
First Post:
2022-03-15
Is NOT Gene Therapy:
False
Has Adverse Events:
False
Brief Title:
HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'D009456', 'term': 'Neurofibromatosis 1'}, {'id': 'D018318', 'term': 'Neurofibroma, Plexiform'}], 'ancestors': [{'id': 'D017253', 'term': 'Neurofibromatoses'}, {'id': 'D009455', 'term': 'Neurofibroma'}, {'id': 'D018317', 'term': 'Nerve Sheath Neoplasms'}, {'id': 'D009380', 'term': 'Neoplasms, Nerve Tissue'}, {'id': 'D009370', 'term': 'Neoplasms by Histologic Type'}, {'id': 'D009369', 'term': 'Neoplasms'}, {'id': 'D009386', 'term': 'Neoplastic Syndromes, Hereditary'}, {'id': 'D020752', 'term': 'Neurocutaneous Syndromes'}, {'id': 'D009422', 'term': 'Nervous System Diseases'}, {'id': 'D020271', 'term': 'Heredodegenerative Disorders, Nervous System'}, {'id': 'D019636', 'term': 'Neurodegenerative Diseases'}, {'id': 'D010523', 'term': 'Peripheral Nervous System Diseases'}, {'id': 'D009468', 'term': 'Neuromuscular Diseases'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D010524', 'term': 'Peripheral Nervous System Neoplasms'}, {'id': 'D009423', 'term': 'Nervous System Neoplasms'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'NA', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SINGLE_GROUP'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 70}}, 'statusModule': {'overallStatus': 'RECRUITING', 'startDateStruct': {'date': '2021-10-18', 'type': 'ACTUAL'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2023-05', 'completionDateStruct': {'date': '2028-10-31', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2023-05-29', 'studyFirstSubmitDate': '2022-03-15', 'studyFirstSubmitQcDate': '2022-04-10', 'lastUpdatePostDateStruct': {'date': '2023-05-31', 'type': 'ACTUAL'}, 'studyFirstPostDateStruct': {'date': '2022-04-15', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2025-10-30', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Objective Response Rate (ORR)', 'timeFrame': 'At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days)', 'description': '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).'}], 'secondaryOutcomes': [{'measure': 'Disease Control Rate（DCR）', 'timeFrame': 'At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days)', 'description': 'Defined as the percentage of patients who have achieved a confirmed response of CR or PR or SD'}, {'measure': 'Duration of Overall Response（DOR）', 'timeFrame': 'At the end of cycle 4,8,12,16,20,24,28,32.Then after every 8 cycles(each cycle is 21 days)', 'description': 'Defined as the time from first achieved CR or PR to disease progression'}, {'measure': 'Progression Free survival (PFS)', 'timeFrame': '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', 'description': 'Defined as the time from first dosing (C1D1) to date of first observed progression or death from any cause (whichever comes first)'}, {'measure': 'Pharmacokinetic characteristics', 'timeFrame': 'During the intervention', 'description': 'AUC'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'conditions': ['Neurofibromatosis 1', 'Plexiform Neurofibromas']}, 'descriptionModule': {'briefSummary': '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)', 'detailedDescription': '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.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['ADULT', 'OLDER_ADULT'], 'maximumAge': '80 Years', 'minimumAge': '18 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Inclusion Criteria:\n\n* Age: patients must be ≥18 years of age at the time of study entry.\n* 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:\n\n ① ≥6 cafe-au-lait macules ;\n\n ② Axillary freckling or freckling in inguinal regions;\n\n ③ ≥2 Lisch nodules (iris hamartomas);\n\n ④ A distinctive bony lesion such as dysplasia of the sphenoid bone or dysplasia or thinning of long bone cortex);\n\n ⑤ An optic pathway glioma;\n\n ⑥ First-degree relative with NF1.\n* Patients must have a measurable lesion, defined as at least 3 cm in length, amenable to MRI for efficacy assessment.\n* Eastern Cooperative Oncology Group (ECOG) performance status of 0-2.\n* Patients are able to understand and voluntarily sign a written informed consent form.\n* Patients must be willing and able to complete study procedures and follow-up examinations.\n\nExclusion Criteria:\n\n* Patients who are unable to undergo MRI scans (prosthesis, prosthesis, braces, etc.) or patients with lesions that cannot be evaluated by MRI.\n* Patients do not have adequate organ function.\n* Patients who are unable to take drugs orally, have difficulty swallowing or anything that may lead to inadequate drug absorption.\n* Prior treatment with MEK 1/2 inhibitors.\n* Patients known to be allergic to the ingredients or analogues of the study drug.\n* 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).\n* With infections or other uncontrolled disease.\n* Strong CYP2C9 inhibitors or inducers within 7 days before treatment of the study drug.\n* Patients who received surgery within 4 weeks or radiotherapy within 6 weeks before enrollment.\n* Patients who participated in any other clinical study treatment within 4 weeks before enrollment.\n* Patients treated with anti-NF1 treatment with unresolved chronic toxicity.\n* Clinical judgment by the investigator that the patient should not participate in the study.'}, 'identificationModule': {'nctId': 'NCT05331105', 'briefTitle': 'HL-085 in Adults With Neurofibromatosis Type 1 (NF1) and Inoperable Plexiform Neurofibromas', 'organization': {'class': 'INDUSTRY', 'fullName': 'Shanghai Kechow Pharma, Inc.'}, 'officialTitle': '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', 'orgStudyIdInfo': {'id': 'HL-085-106-II'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'HL-085', 'description': 'HL-085 9mg BID', 'interventionNames': ['Drug: HL-085']}], 'interventions': [{'name': 'HL-085', 'type': 'DRUG', 'description': '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.\n\nIIb: HL-085 at the recommended dose or dosage regimen.', 'armGroupLabels': ['HL-085']}]}, 'contactsLocationsModule': {'locations': [{'zip': '200011', 'city': 'Shanghai', 'state': 'Shanghai Municipality', 'status': 'RECRUITING', 'country': 'China', 'contacts': [{'name': 'Qingfeng Li, M.D.', 'role': 'CONTACT', 'email': 'dr.liqingfeng@shsmu.edu.cn', 'phone': '13301990666'}], 'facility': "Shanghai Ninth People's Hospital, Shanghai JiaoTong University School of Medicine", 'geoPoint': {'lat': 31.22222, 'lon': 121.45806}}], 'centralContacts': [{'name': 'Zhimei Zhu, Master', 'role': 'CONTACT', 'email': 'zhuzm@kechowpharma.com', 'phone': '86 215201345822'}, {'name': 'Hongqi Tian, Ph.D', 'role': 'CONTACT', 'email': 'tianhq@kechowpharma.com'}], 'overallOfficials': [{'name': 'Hongqi Tian, Ph.D', 'role': 'STUDY_CHAIR', 'affiliation': 'Shanghai Kechow Pharma, Inc.'}]}, 'ipdSharingStatementModule': {'ipdSharing': 'UNDECIDED'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Shanghai Kechow Pharma, Inc.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}