Ignite Creation Date:
2025-12-24 @ 10:37 PM
Ignite Modification Date:
2026-01-01 @ 4:24 PM
Study NCT ID:
NCT06873035
Status:
ENROLLING_BY_INVITATION
Last Update Posted:
2025-12-15
First Post:
2025-02-18
Is NOT Gene Therapy:
True
Has Adverse Events:
False
Brief Title:
An Interventional Study of Infigratinib in Children With Hypochondroplasia
Sponsor:
Organization:
Raw JSON
{'hasResults': False, 'derivedSection': {'miscInfoModule': {'versionHolder': '2025-12-24'}, 'conditionBrowseModule': {'meshes': [{'id': 'C562937', 'term': 'Hypochondroplasia'}, {'id': 'D009085', 'term': 'Mucopolysaccharidosis IV'}, {'id': 'D004392', 'term': 'Dwarfism'}, {'id': 'D001847', 'term': 'Bone Diseases'}, {'id': 'D009140', 'term': 'Musculoskeletal Diseases'}, {'id': 'D010009', 'term': 'Osteochondrodysplasias'}, {'id': 'D030342', 'term': 'Genetic Diseases, Inborn'}], 'ancestors': [{'id': 'D009083', 'term': 'Mucopolysaccharidoses'}, {'id': 'D002239', 'term': 'Carbohydrate Metabolism, Inborn Errors'}, {'id': 'D008661', 'term': 'Metabolism, Inborn Errors'}, {'id': 'D009358', 'term': 'Congenital, Hereditary, and Neonatal Diseases and Abnormalities'}, {'id': 'D016464', 'term': 'Lysosomal Storage Diseases'}, {'id': 'D017520', 'term': 'Mucinoses'}, {'id': 'D003240', 'term': 'Connective Tissue Diseases'}, {'id': 'D017437', 'term': 'Skin and Connective Tissue Diseases'}, {'id': 'D008659', 'term': 'Metabolic Diseases'}, {'id': 'D009750', 'term': 'Nutritional and Metabolic Diseases'}, {'id': 'D001848', 'term': 'Bone Diseases, Developmental'}, {'id': 'D004700', 'term': 'Endocrine System Diseases'}]}, 'interventionBrowseModule': {'meshes': [{'id': 'C568950', 'term': 'infigratinib'}]}}, 'protocolSection': {'designModule': {'phases': ['PHASE2', 'PHASE3'], 'studyType': 'INTERVENTIONAL', 'designInfo': {'allocation': 'RANDOMIZED', 'maskingInfo': {'masking': 'NONE'}, 'primaryPurpose': 'TREATMENT', 'interventionModel': 'PARALLEL', 'interventionModelDescription': 'This study comprises an open-label Phase 2 portion to evaluate safety and efficacy in participants receiving infigratinib at one of two doses followed by a Phase 3 randomized, double-blind portion of the dose selected from the Phase 2 portion versus placebo.'}, 'enrollmentInfo': {'type': 'ESTIMATED', 'count': 24}}, 'statusModule': {'overallStatus': 'ENROLLING_BY_INVITATION', 'startDateStruct': {'date': '2025-04-22', 'type': 'ACTUAL'}, 'statusVerifiedDate': '2025-03', 'completionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}, 'lastUpdateSubmitDate': '2025-12-09', 'studyFirstSubmitDate': '2025-02-18', 'studyFirstSubmitQcDate': '2025-03-06', 'lastUpdatePostDateStruct': {'date': '2025-12-15', 'type': 'ESTIMATED'}, 'studyFirstPostDateStruct': {'date': '2025-03-12', 'type': 'ACTUAL'}, 'primaryCompletionDateStruct': {'date': '2027-01', 'type': 'ESTIMATED'}}, 'outcomesModule': {'otherOutcomes': [{'measure': 'Change from BL in comorbidities associated with HCH', 'timeFrame': '52 weeks'}, {'measure': 'Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Pediatric Quality of Life Inventory [PedsQL tool])', 'timeFrame': '52 weeks'}, {'measure': 'Evaluate treatment benefit as assessed by qualitative interviews', 'timeFrame': '52 weeks'}], 'primaryOutcomes': [{'measure': 'Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year)', 'timeFrame': '26 weeks'}, {'measure': 'Incidence, severity, and seriousness of adverse events (AEs) that require dose reduction or discontinuation', 'timeFrame': 'at least 26 weeks'}], 'secondaryOutcomes': [{'measure': 'Change from BL in height Z-score (in relation to both HCH and average height tables)', 'timeFrame': '52 weeks'}, {'measure': 'Change from BL in upper to lower body segment ratio (cm)', 'timeFrame': '52 weeks'}, {'measure': 'Changes in cognitive function (as assessed by age appropriate computerized tests)', 'timeFrame': '52 weeks'}, {'measure': 'Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)', 'timeFrame': '52 weeks'}, {'measure': 'Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)', 'timeFrame': '52 weeks'}, {'measure': 'Change from BL in collagen X marker (CXM) levels', 'timeFrame': '52 weeks'}]}, 'oversightModule': {'oversightHasDmc': True, 'isFdaRegulatedDrug': True, 'isFdaRegulatedDevice': False}, 'conditionsModule': {'keywords': ['skeletal dysplasia', 'endochondral ossification', 'hypochondroplasia', 'HCH', 'shortened proximal limbs', 'fibroblast growth factor receptor 3', 'FGFR3', 'endochondral bone formation', 'disproportionate short stature', 'quality of life', 'dwarfism', 'bone diseases', 'musculoskeletal diseases', 'osteochondrodysplasia', 'functional abilities', 'annualized growth velocity', 'annualized height velocity', 'growth', 'genetic diseases', 'congenital', 'AHV', 'AGV', 'Pathogenic variants'], 'conditions': ['Hypochondroplasia']}, 'descriptionModule': {'briefSummary': 'ACCEL2/3 is a Phase 2/3 study. The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety, of infigratinib in children with hypochondroplasia (HCH) receiving infigratinib, at one of two doses, of who have completed at least 26 weeks of participation in QED-sponsored ACCEL (QBGJ398-004).', 'detailedDescription': 'ACCEL 2/3 is a Phase 2/3 study that comprises of 2 portions. The Phase 2 portion is an open-label, portion in children with HCH aged 5 to 11 years old followed by a Phase 3 portion which is double-blind, placebo-controlled in children with HCH aged \\>3 years old to \\<18 years old.'}, 'eligibilityModule': {'sex': 'ALL', 'stdAges': ['CHILD', 'ADULT'], 'maximumAge': '18 Years', 'minimumAge': '3 Years', 'healthyVolunteers': False, 'eligibilityCriteria': 'Key Inclusion Criteria:\n\n* Participants must have completed at least 26 weeks and still be on the observational study (QBGJ398-004).\n* Phase 2 portion: Participants 5-11 years of age (inclusive).\n* Phase 3 portion: Participants 3 to \\<18 years of age at screening with growth potential\n* Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.\n* Participants are able to swallow oral medication.\n* Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.\n* Participants are ambulatory and able to stand without assistance. Sex and Contraceptive/Barrier Requirements\n* Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.\n* If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception, as relevant, while taking study drug and for 1 month after the last dose of study drug.\n* Signed informed consent.\n\nKey Exclusion Criteria:\n\n* Participants who have ACH or a short stature condition other than HCH.\n* Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.\n* Current evidence of clinically significant corneal or retinal disorder/keratopathy confirmed by ophthalmic examination.\n* Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations.\n* History and/or current evidence of extensive ectopic tissue calcification.\n* History of malignancy.\n* Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature.\n* Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid.\n* Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study.\n* Participants receiving medications which could increase serum phosphorus and/or calcium concentrations\n* Clinically significant abnormality in any laboratory test result at screening.\n* Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study.\n* Allergy to any components of the study drug.\n* Concurrent circumstance, disease, or condition that would interfere with study participation.'}, 'identificationModule': {'nctId': 'NCT06873035', 'acronym': 'HCH', 'briefTitle': 'An Interventional Study of Infigratinib in Children With Hypochondroplasia', 'organization': {'class': 'INDUSTRY', 'fullName': 'QED Therapeutics, a BridgeBio company'}, 'officialTitle': 'A Phase 2/3, Multicenter, Open-Label Phase Followed by a Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children With Hypochondroplasia: ACCEL 2/3', 'orgStudyIdInfo': {'id': 'QBGJ398-304'}}, 'armsInterventionsModule': {'armGroups': [{'type': 'EXPERIMENTAL', 'label': 'Phase 2 Cohort 1', 'description': 'infigratinib (0.128 mg/kg/day)', 'interventionNames': ['Drug: infigratinib 0.128 mg/kg/day']}, {'type': 'EXPERIMENTAL', 'label': 'Phase 2 Cohort 2', 'description': 'infigratinib (0.25 mg/kg/day)', 'interventionNames': ['Drug: infigratinib 0.25 mg/kg/day']}], 'interventions': [{'name': 'infigratinib 0.128 mg/kg/day', 'type': 'DRUG', 'description': 'Oral infigratinib 0.128 mg/kg/day', 'armGroupLabels': ['Phase 2 Cohort 1']}, {'name': 'infigratinib 0.25 mg/kg/day', 'type': 'DRUG', 'description': 'Oral infigratinib 0.25 mg/kg/day', 'armGroupLabels': ['Phase 2 Cohort 2']}]}, 'contactsLocationsModule': {'locations': [{'zip': '94609', 'city': 'Oakland', 'state': 'California', 'country': 'United States', 'facility': "UCSF Benioff Children's Hospital", 'geoPoint': {'lat': 37.80437, 'lon': -122.2708}}, {'zip': '80045', 'city': 'Aurora', 'state': 'Colorado', 'country': 'United States', 'facility': 'Childrens Hospital Colorado', 'geoPoint': {'lat': 39.72943, 'lon': -104.83192}}, {'zip': '20010', 'city': 'Washington D.C.', 'state': 'District of Columbia', 'country': 'United States', 'facility': "Children's National Hospital", 'geoPoint': {'lat': 38.89511, 'lon': -77.03637}}, {'zip': '21287', 'city': 'Baltimore', 'state': 'Maryland', 'country': 'United States', 'facility': 'Johns Hopkins School of Medicine', 'geoPoint': {'lat': 39.29038, 'lon': -76.61219}}, {'zip': '65201', 'city': 'Columbia', 'state': 'Missouri', 'country': 'United States', 'facility': 'University of Missouri', 'geoPoint': {'lat': 38.95171, 'lon': -92.33407}}, {'zip': '37232', 'city': 'Nashville', 'state': 'Tennessee', 'country': 'United States', 'facility': 'Vanderbilt University Medical Center', 'geoPoint': {'lat': 36.16589, 'lon': -86.78444}}, {'zip': '53705', 'city': 'Madison', 'state': 'Wisconsin', 'country': 'United States', 'facility': 'University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic', 'geoPoint': {'lat': 43.07305, 'lon': -89.40123}}, {'zip': '3052', 'city': 'Parkville', 'state': 'Victoria', 'country': 'Australia', 'facility': "Murdoch Children's Research Institute", 'geoPoint': {'lat': -37.78333, 'lon': 144.95}}, {'zip': 'N6C 2R5', 'city': 'London', 'state': 'Ontario', 'country': 'Canada', 'facility': "London Health Services Center - Children's Hospital of Western Ontario", 'geoPoint': {'lat': 42.98339, 'lon': -81.23304}}, {'zip': 'K1H 8L1', 'city': 'Ottawa', 'state': 'Ontario', 'country': 'Canada', 'facility': "Children's Hospital of Eastern Ontario Research Institute", 'geoPoint': {'lat': 45.41117, 'lon': -75.69812}}, {'zip': 'H3T 1C5', 'city': 'Montreal', 'state': 'Quebec', 'country': 'Canada', 'facility': 'Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine', 'geoPoint': {'lat': 45.50884, 'lon': -73.58781}}, {'zip': '69677', 'city': 'Bron', 'state': 'France', 'country': 'France', 'facility': 'Hôpital Femme Mère Enfant', 'geoPoint': {'lat': 45.73865, 'lon': 4.91303}}, {'zip': '75015', 'city': 'Paris', 'state': 'France', 'country': 'France', 'facility': 'Hôpital Universitaire Necker-Enfants Malades', 'geoPoint': {'lat': 48.85341, 'lon': 2.3488}}, {'zip': '31059', 'city': 'Toulouse', 'state': 'France', 'country': 'France', 'facility': 'Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants', 'geoPoint': {'lat': 43.60426, 'lon': 1.44367}}, {'zip': '5021', 'city': 'Bergen', 'state': 'Norway', 'country': 'Norway', 'facility': 'Haukeland University Hospital', 'geoPoint': {'lat': 60.39299, 'lon': 5.32415}}, {'zip': '0372', 'city': 'Oslo', 'state': 'Norway', 'country': 'Norway', 'facility': 'Paediatric Clinical Research Unit at Osla University Hospital', 'geoPoint': {'lat': 59.91273, 'lon': 10.74609}}, {'zip': '3000-602', 'city': 'Coimbra', 'state': 'Portugal', 'country': 'Portugal', 'facility': 'Hospital Pediátrico de Coimbra', 'geoPoint': {'lat': 40.20686, 'lon': -8.41996}}, {'zip': '229899', 'city': 'Singapore', 'state': 'Singapore', 'country': 'Singapore', 'facility': "KK Women's and Children's Hospital", 'geoPoint': {'lat': 1.28967, 'lon': 103.85007}}, {'zip': '01008', 'city': 'Vitoria-Gasteiz', 'state': 'Spain', 'country': 'Spain', 'facility': 'Hospital Vithas San Jose', 'geoPoint': {'lat': 42.84998, 'lon': -2.67268}}, {'zip': '17164', 'city': 'Solna', 'state': 'Sweden', 'country': 'Sweden', 'facility': "Astrid Lindgren Children's Hospital", 'geoPoint': {'lat': 59.36004, 'lon': 18.00086}}, {'zip': 'M13 9WL', 'city': 'Manchester', 'state': 'United Kingdom', 'country': 'United Kingdom', 'facility': 'Manchester University', 'geoPoint': {'lat': 53.48095, 'lon': -2.23743}}, {'zip': 'S10 2TH', 'city': 'Sheffield', 'state': 'United Kingdom', 'country': 'United Kingdom', 'facility': "Sheffield Children's Hospital", 'geoPoint': {'lat': 53.38297, 'lon': -1.4659}}]}, 'ipdSharingStatementModule': {'ipdSharing': 'NO'}, 'sponsorCollaboratorsModule': {'leadSponsor': {'name': 'QED Therapeutics, a BridgeBio company', 'class': 'INDUSTRY'}, 'responsibleParty': {'type': 'SPONSOR'}}}}