Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Phase 2, multicenter, OLE study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR (fibroblast growth factor receptor) 1-3-selective tyrosine kinase inhibitor, in participants with Hypochondroplasia (HCH) who previously completed ACCEL 2/3, and potentially additional participants who completed ACCEL. Participants rolling over directly from the observational ACCEL study must have had at least a 6-month period of growth assessment in that study.
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Arm 1: Rollover subjects | Experimental | Children who have completed QED-sponsored interventional study with infigratinib |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Infigratinib | Drug | Infigratinib to be administered by mouth and initiated at the last dose level received in the ACCEL 2/3 study or at the dose selected to be further evaluated after proof-of-concept is established for Phase 2 portion of ACCEL 2/3. |
| Measure | Description | Time Frame |
|---|---|---|
| Incidence of treatment emergent adverse events (TEAE) and serious TEAE | 10 years | |
| Changes over time in standing height Z-score in relation to HCH and non-HCH growth charts | 10 years |
| Measure | Description | Time Frame |
|---|---|---|
| Changes over time in AHV Z-score | 10 years | |
| Changes over time in body proportions | 10 years | |
| Changes over time in weight Z-score |
Not provided
Inclusion Criteria:
Inclusion Criteria for Participants Rolling Over from ACCEL 2/3
Exclusion Criteria:
Exclusion Criteria for Participants Rolling Over from ACCEL 2/3
Not provided
Not provided
Not provided
Not provided
Not provided
| Name | Affiliation | Role |
|---|---|---|
| QED Therapeutics SVP, Clinical Development | QED Therapeutics, a BridgeBio company | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| UCSF Benioff Children's Hospital | Oakland | California | 94609 | United States | ||
| Childrens Hospital Colorado |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| 10 years |
| Changes overtime in BMI | 10 years |
| Age of puberty onset and time to Tanner stage ≥4 | 10 years |
| Changes over time in body composition as assessed by DXA (dual x-ray absorptiometry) scans | 10 years |
| Changes over time in bone morphology/density by x-ray and DXA | 10 years |
| Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test) | 10 years |
| Change in attention assessed by age-appropriate computerized tests (Identification Test) | 10 years |
| Change in visual learning assessed by age-appropriate computerized tests (One Card Learning Test) | 10 years |
| Change in working memory assessed by age-appropriate computerized tests (One Back Test) | 10 years |
| Changes over time in severity of epilepsy measured by frequency and adverse event grading | 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 |
| 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 |
| Aurora |
| Colorado |
| 80045 |
| United States |
| Children's National Hospital | Washington D.C. | District of Columbia | 20010 | United States |
| Johns Hopkins School of Medicine | Baltimore | Maryland | 21287 | United States |
| University of Missouri | Columbia | Missouri | 65201 | United States |
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
| Vanderbilt University Medical Center | Nashville | Tennessee | 37232 | United States |
| University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic | Madison | Wisconsin | 53705 | United States |
| Murdoch Children's Research Institute - The Royal Children's Hospital Melbourne | Parkville | Victoria | 3052 | Australia |
| London Health Sciences Centre - Children's Hospital of Western Ontario | London | Ontario | N6C 2R5 | Canada |
| Children's Hospital of Eastern Ontario Research Institute | Ottawa | Ontario | K1H 8L1 | Canada |
| Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine | Montreal | Quebec | H3T 1C5 | Canada |
| Hôpital Femme Mère Enfant | Bron | 69677 | France |
| Hôpital Universitaire Necker-Enfants Malades | Paris | 75015 | France |
| Centre Hospitalier Universitaire (CHU) de Toulouse - Hôpital des Enfants | Toulouse | 31059 | France |
| Haukeland University Hospital | Bergen | 5021 | Norway |
| Paediatric Clinical Research Unit at Oslo University Hospital | Oslo | 0372 | Norway |
| Hospital Pediátrico de Coimbra | Coimbra | 3000-602 | Portugal |
| KK Women's and Children's Hospital | Singapore | 229899 | Singapore |
| Unidad de Cirugía Artroscopica, Hopsital MIKS | Vitoria-Gasteiz | 01010 | Spain |
| Astrid Lindgren Children's Hospital | Solna | 17164 | Sweden |
| Manchester University | Manchester | England | M13 9WL | United Kingdom |
| Sheffield Children's Hospital | Sheffield | England | S10 2TH | United Kingdom |
| Glasgow Clinical Research Facility, Queen Elizabeth University Hospital | Glasgow | Scotland | G51 4TF | United Kingdom |
| ID | Term |
|---|---|
| C562937 | Hypochondroplasia |
| D009085 | Mucopolysaccharidosis IV |
| D004392 | Dwarfism |
| D001847 | Bone Diseases |
| D009140 | Musculoskeletal Diseases |
| D010009 | Osteochondrodysplasias |
| D030342 | Genetic Diseases, Inborn |
| ID | Term |
|---|---|
| D009083 | Mucopolysaccharidoses |
| D002239 | Carbohydrate Metabolism, Inborn Errors |
| D008661 | Metabolism, Inborn Errors |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D016464 | Lysosomal Storage Diseases |
| D017520 | Mucinoses |
| D003240 | Connective Tissue Diseases |
| D017437 | Skin and Connective Tissue Diseases |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D001848 | Bone Diseases, Developmental |
| D004700 | Endocrine System Diseases |
Not provided
Not provided
| ID | Term |
|---|---|
| C568950 | infigratinib |
Not provided
Not provided
Not provided