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This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm 1: Rollover subjects | Experimental | Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3) |
|
| Arm 2: Treatment naïve subjects | Experimental | Children naïve to infigratinib |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Infigratinib | Drug | Infigratinib minitablets or sprinkle capsules to be administered by mouth. In subjects that completed a prior study with infigratinib, the starting dose will be the same as the last dose received in the prior interventional study with infigratinib. Infigratinib dose may be adjusted to 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)). |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment emergent adverse events (TEAE) and serious TEAE | 10 years | |
| Changes over time in height Z-score in relation to ACH and non-ACH growth charts | 10 years |
| Measure | Description | Time Frame |
|---|---|---|
| Changes over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth charts | 10 years | |
| Changes over time in body proportions | 10 years | |
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Key Rollover Subjects Inclusion Criteria:
Key Rollover Subjects Exclusion Criteria:
Key Inclusion Criteria for Treatment Naïve Subjects
Key Exclusion Criteria for Treatment Naïve Subjects
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| Name | Affiliation | Role |
|---|---|---|
| QED Therapeutics SVP, Clinical Development | QED Therapeutics | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| USCF Benioff Children's Hospital, Oakland | Oakland | California | 94609 | United States | ||
| Children's Hospital Colorado |
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|
| Infigratinib | Drug | Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the subjects naïve to Infigratinib will be 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)). |
|
| Changes over time in weight z-score |
| 10 years |
| Changes overtime in BMI | 10 years |
| Age of puberty onset and time to Tanner stage ≥4 | 10 years |
| Changes over time in number of episodes of otitis media per year | 10 years |
| Changes over time in number of episodes and/or severity of sleep apnea | 10 years |
| Changes over time in range of motion (elbow) | 10 years |
| Changes over time in skeletal abnormalities of the lower extremities and spine | 10 years |
| Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL) | 10 years |
| Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY) | 10 years |
| Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS) | 10 years |
| Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM) | 10 years |
| Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S) | 10 years |
| Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C) | 10 years |
| Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview | 10 years |
| Changes in cognitive functions assessed by age-appropriate computerized tests | 10 years |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Nemours Alfred I. Dupont Hospital for Children | Wilmington | Delaware | 19803 | United States |
| Johns Hopkins University | Baltimore | Maryland | 21205 | United States |
| University of Missouri | Columbia | Missouri | 65212 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| University Hospital and UW Health Clinics | Madison | Wisconsin | 53705 | United States |
| Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan | Ciudad Autonoma Buenos Aires | Buenos Aires | C1245AAM | Argentina |
| Murdoch Children's Hospital | Parkville | Victoria | 3052 | Australia |
| Stollery Children's Hospital | Edmonton | Alberta | T6C 2B7 | Canada |
| Children's Hospital - London Health Sciences Centre | London | Ontario | N6A 5W9 | Canada |
| Children's Hospital of Eastern Ontario | Ottawa | Ontario | K1H 8L1 | Canada |
| Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | H3T 1C5 | Canada |
| Hopital Femme Mere Enfant | Lyon | 69500 | France |
| Hopital Necker-Enfants Malades | Paris | 75743 | France |
| Hopital des Enfants | Toulouse | 31300 | France |
| Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico | Milan | 20122 | Italy |
| Policlinico A. Gemelli IRCCS | Roma | 00168 | Italy |
| Haukeland University Hospital | Bergen | 5021 | Norway |
| Oslo University Hospital | Oslo | 0372 | Norway |
| KK Women's and Children's Hosptial | Singapore | 229899 | Singapore |
| Hospital Universitario La Paz | Madrid | 28046 | Spain |
| Hospital Universitario Virgen de la Victoria | Málaga | 29010 | Spain |
| Hospital Vithas San José | Vitoria-Gasteiz | Álava | 01012 | Spain |
| Birmingham Women's and Children's NHS Foundation Trust | Birmingham | B5 6NH | United Kingdom |
| University Hospitals Bristol and Weston NHS Foundation Trust | Bristol | BS2 8AE | United Kingdom |
| Queen Elizabeth University Hospital | Glasgow | G12 0XH | United Kingdom |
| St. Thomas' Hospital | London | SE1 7EH | United Kingdom |
| Manchester University Children's Hospital | Manchester | M13 9WL | United Kingdom |
| Sheffield Children's Hospital | Sheffield | S10 2TH | United Kingdom |
| ID | Term |
|---|---|
| D000130 | Achondroplasia |
| D009085 | Mucopolysaccharidosis IV |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D010009 | Osteochondrodysplasias |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D004392 | Dwarfism |
| D001848 | Bone Diseases, Developmental |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
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| ID | Term |
|---|---|
| C568950 | infigratinib |
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