Ignite Creation Date:
2025-12-24 @ 2:03 PM
Ignite Modification Date:
2026-01-10 @ 2:53 PM
Study NCT ID:
NCT07264595
Status:
NOT_YET_RECRUITING
Last Update Posted:
2025-12-04
First Post:
2025-11-24
Is Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}}, 'protocolSection': {'designModule': {'phases': ['PHASE1', 'PHASE2'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'TRIPLE', 'whoMasked': ['PARTICIPANT', 'CARE_PROVIDER', 'INVESTIGATOR']}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'SEQUENTIAL'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 88}}, 'statusModule': {'overallStatus': 'NOT_YET_RECRUITING', 'startDateStruct': {'date': '2025-12', 'type': 'ESTIMATED'}, 'expandedAccessInfo': {'hasExpandedAccess': False}, 'statusVerifiedDate': '2025-11', 'completionDateStruct': {'date': '2030-11', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-11-24', 'studyFirstSubmitDate': '2025-11-24', 'studyFirstSubmitQcDate': '2025-11-24', 'lastUpdatePostDateStruct': {'date': '2025-12-04', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-12-04', 'type': 'ESTIMATED'}, 'primaryCompletionDateStruct': {'date': '2030-11', 'type': 'ESTIMATED'}}, 'outcomesModule': {'primaryOutcomes': [{'measure': 'Number of Participants With Treatment Emergent Adverse Events (TEAEs)', 'timeFrame': 'up to 21 days (Ib phase)'}, {'measure': 'Annualized height velocity (AHV) at Week 26 of treatment', 'timeFrame': '26 weeks (II phase)'}, {'measure': 'Number of Participants With TEAEs', 'timeFrame': 'up to 160 weeks (II phase extension)'}], 'secondaryOutcomes': [{'measure': 'PK concentrations of GS3-007a and metabolite GS3-017', 'timeFrame': 'up to 7 days (Ib phase)'}, {'measure': 'Concentrations of serum growth hormone (GH), insulin-like growth factor-1 (IGF-1), and insulin-like growth factor-binding protein 3 (IGFBP-3)', 'timeFrame': 'up to 14 days (Ib phase)'}, {'measure': 'AHVs at each evaluation point', 'timeFrame': '13 weeks, 39 weeks, 52 weeks (II phase)'}, {'measure': 'Changes from baseline (ΔIGF-1 SDS) in the standard deviation score of the PD indicator insulin-like growth factor-1 (IGF-1 SDS) at each evaluation point', 'timeFrame': '4 weeks, 13 weeks, 26 weeks, 39 weeks, 52 weeks (II phase)'}, {'measure': 'AHVs at each evaluation point', 'timeFrame': 'up to 156 weeks (II phase extension)'}, {'measure': 'Change from extension baseline in the height standard deviation score at each evaluation point (ΔHT SDS)', 'timeFrame': 'up to 156 weeks (II phase extension)'}]}, 'oversightModule': {'oversightHasDmc': False, 'isFdaRegulatedDrug': False, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['GS3-007a dry suspension', 'PGHD'], 'conditions': ['Pediatric Growth Hormone Deficiency']}, 'descriptionModule': {'briefSummary': 'This is a two-part clinical study for children with growth hormone deficiency. In the first part, participants will be randomly assigned to receive different doses of an oral treatment (GS3-007a dry suspension) or a placebo for 14 days. This part is double-blinded, meaning neither the participants nor the doctors will know who is receiving the treatment or placebo. The goal is to find a safe and well-tolerated dose.\n\nIn the second part, participants will be randomly assigned to receive either the selected dose of GS3-007a or another approved treatment for 52 weeks. This part is open-label, so everyone will know which treatment is being given. After that, all participants may continue taking GS3-007a for another 156 weeks in an extension phase to study long-term effects.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT', 'OLDER_ADULT'], 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': "Inclusion Criteria:\n\n* chronological age (CA) ≥3 years at Screening\n* Prepubertal girls or boys\n* Height at Screening lower than the reference height for normal children of the same chronological age and sex minus 2 standard deviations (-2 SD)\n* A confirmed diagnosis of growth hormone deficiency (GHD)\n* Having not been treated with any growth-promoting drugs\n* BA delayed by ≥6 months compared with the CA at Screening Subjects who meet all of the following criteria are eligible to enroll in the extension study (applicable for Part II extension period)\n* Subjects who have completed the 52-week treatment period of phase II\n* Subjects who do not permanently discontinue the investigational Medicinal Product (IMP) during the 52-week treatment period of phase II\n\nExclusion Criteria:\n\n* A highly allergic constitution\n* Suspected or confirmed total pituitary deficiency, including patients previously confirmed with deficiency of ≥2 pituitary hormones other than GH\n* Being confirmed with other chromosomal abnormalities or growth abnormalities affecting growth\n* Congenital skeletal dysplasia or serious spinal anomalies\n* Cognitive hypofunction, neurodevelopmental disorders, or psychiatric/psychological disorders that, in the investigator's opinion, may interfere with evaluation of study endpoints\n* Any clinically significant abnormality that may affect growth or evaluation of the IMP\n* Screening magnetic resonance imaging (MRI) scan of the sellar region confirming prior or current intracranial tumor growth\n* Concurrent use of any medications that may affect growth or response to growth hormone therapy\n* Epiphyseal closure\n* Electrocardiogram (ECG) QTcF interval abnormal, with a history of QT/QTc interval prolonged\n* Hepatic function indicators abnormal at Screening Patients meeting any of the following criteria may not be enrolled in this extension study (applicable for Part II extension period)\n* Subjects with closed epiphyses\n* Any clinically significant abnormality that may affect growth or evaluation of the IMP\n* Known or suspected allergy to the IMP\n* Women with positive blood human chorionic gonadotropin (hCG) at the pre-treatment visit"}, 'identificationModule': {'nctId': 'NCT07264595', 'briefTitle': 'A Study (Phase 1b/2) of GS3-007a Oral Treatment in Children With Growth Hormone Deficiency (PGHD)', 'organization': {'class': 'INDUSTRY', 'fullName': 'Changchun GeneScience Pharmaceutical Co., Ltd.'}, 'officialTitle': 'A Study to Evaluate the Safety and Tolerability of Multiple Ascending Doses of GS3-007a Dry Suspension in Patients With PGHD and to Evaluate the Efficacy and Safety of 52-week of Treatment With GS3-007a Dry Suspension: a Two-part, Multicenter, Randomized, Phase Ib/II Clinical Trial', 'orgStudyIdInfo': {'id': 'GenSci073-201'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'PLACEBO_COMPARATOR', 'label': 'Dose Level 1~ Dose Level 4', 'description': 'dose level 1、dose level 2、dose level 3、dose level 4', 'interventionNames': ['Drug: GS3-007a dry suspension', 'Drug: Placebo for GS3-007a dry suspension']}, {'type': 'ACTIVE_COMPARATOR', 'label': 'Cohort 1~ Cohort 3', 'description': 'GS3-007a low dose , GS3-007a high dose , rhGH 0.033mg/kg', 'interventionNames': ['Drug: GS3-007a dry suspension', 'Drug: rhGH injection']}, {'type': 'EXPERIMENTAL', 'label': 'GS3-007a cohort', 'description': 'GS3-007a optimal dose', 'interventionNames': ['Drug: GS3-007a dry suspension']}], 'interventions': [{'name': 'GS3-007a dry suspension', 'type': 'DRUG', 'otherNames': ['GS3-007a'], 'description': 'GS3-007a', 'armGroupLabels': ['Cohort 1~ Cohort 3', 'Dose Level 1~ Dose Level 4', 'GS3-007a cohort']}, {'name': 'Placebo for GS3-007a dry suspension', 'type': 'DRUG', 'otherNames': ['placebo'], 'description': 'Placebo for GS3-007a dry suspension', 'armGroupLabels': ['Dose Level 1~ Dose Level 4']}, {'name': 'rhGH injection', 'type': 'DRUG', 'otherNames': ['rhGH'], 'description': 'rhGH injection', 'armGroupLabels': ['Cohort 1~ Cohort 3']}]}, 'contactsLocationsModule': {'locations': [{'zip': '430000', 'city': 'Wuhan', 'state': 'Hubei', 'country': 'China', 'facility': 'Tongji Hospital, Tongji Medical College, Huazhong University of Science & Technology', 'geoPoint': {'lat': 30.58333, 'lon': 114.26667}}], 'centralContacts': [{'name': 'Lingli Sun, bachelor', 'role': 'CONTACT', 'email': 'sunlingli@genscigroup.com', 'phone': '+86 18006171657'}]}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'Changchun GeneScience Pharmaceutical Co., Ltd.', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}