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Also known as BGJ398
Infigratinib · 6 trials · 2 indications
| Arm | Type | Description |
|---|---|---|
| Infigratinib 0.25 mg/kg/day | EXPERIMENTAL | Infigratinib at 2, 3.5, 5, 7, 10 mg |
| Placebo 0.25 mg/kg/day | PLACEBO_COMPARATOR | Placebo Comparator at 2, 3.5, 5, 7, 10 mg |
| Phase 2 Cohort 1 | EXPERIMENTAL | Oral infigratinib 0.128 mg/kg/day |
| Phase 2 Cohort 2 | EXPERIMENTAL | Oral infigratinib 0.25 mg/kg/day |
| Arm 1: Rollover subjects | EXPERIMENTAL | Children who have completed QED-sponsored interventional study with infigratinib |
| SAD Cohort 1 infigratinib (2 to less than 3 years old) | EXPERIMENTAL | Single Ascending Dose Escalation and PK Portion |
| SAD Cohort 2 infigratinib (1 to less than 2 years old) | EXPERIMENTAL | Single Ascending Dose Escalation and PK Portion |
| SAD Cohort 3 infigratinib (6 months to less than 1 year old) | EXPERIMENTAL | Single Ascending Dose Escalation and PK Portion |
| SAD Cohort 4 infigratinib (0 to less than 6 months old) | EXPERIMENTAL | Single Ascending Dose Escalation and PK Portion |
| Phase 2 Cohort 1 infigratinib (2 to less than 3 years old) | EXPERIMENTAL | Open-label Safety and PK Portion |
| Phase 2 Cohort 2 infigratinib (1 to less than 2 years old) | EXPERIMENTAL | Open-label Safety and PK Portion |
| Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old) | EXPERIMENTAL | Open-label Safety and PK Portion |
| Phase 2 Cohort 4 infigratinib (0 to less than 6 months) | EXPERIMENTAL | Open-label Safety and PK Portion |
| Phase 2B Cohort 1 infigratinib (2 to less than 3 years old) | EXPERIMENTAL | Randomized Safety and Efficacy Portion |
| Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old) | EXPERIMENTAL | Randomized Safety and Efficacy Portion |
| Phase 2B Cohort 3 infigratinib (0 to less than 6 months old) | EXPERIMENTAL | Randomized Safety and Efficacy Portion |
| Open- label Extension infigratinib (0 months to 3 years [+6 months old]) | EXPERIMENTAL | Open-label extension portion continuing to assess safety and efficacy in children until they reach 3 years old (+6 months) |
| Phase 2B Cohort 1 Placebo (2 to less than 3 years old) | PLACEBO_COMPARATOR | Randomized Safety and Efficacy study |
| Phase 2B Cohort 2 placebo (6 months to less than 2 years old) | PLACEBO_COMPARATOR | Randomized Safety and Efficacy study |
| Phase 2B Cohort 3 Placebo (0 to less than 6 months old) | PLACEBO_COMPARATOR | Randomized Safety and Efficacy study |
| Arm 1: Rollover participants | EXPERIMENTAL | Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3) |
| Arm 2: Treatment naïve participants | EXPERIMENTAL | Children naïve to infigratinib |
| Infigratinib 0.016 mg/kg | EXPERIMENTAL | Dose Escalation: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. |
| Infigratinib 0.032 mg/kg | EXPERIMENTAL | Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. |
| Infigratinib 0.064 mg/kg | EXPERIMENTAL | Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. |
| Infigratinib 0.128 mg/kg | EXPERIMENTAL | Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. |
| Infigratinib 0.25 mg/kg | EXPERIMENTAL | Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. Dose Expansion: Upon identification of the recommended dose from all cohorts analyzed, an expansion cohort of 20 subjects may begin enrollment to further determine safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of the selected dose. |
| Name | Type | Description |
|---|---|---|
| Infigratinib 0.25 mg/kg/day | DRUG | Daily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg |
| Placebo Comparator 0.25 mg/kg/day | DRUG | Daily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg |
| infigratinib 0.128 mg/kg/day | DRUG | Oral infigratinib 0.128 mg/kg/day |
| 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. |
| Infigratinib is provided as a single dose of minitablets for oral administration | DRUG | * The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria. * The dose and number of minitablets will be calculated based on individual participant age and weight. |
| Infigratinib is provided as sprinkle capsules for daily oral administration | DRUG | * The cohort dose of infigratinib will be the dose identified in the Single Ascending Dose portion for the age group. * The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months. |
| Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration | DRUG | * The cohort dose of infigratinib or placebo will be the dose confirmed in the Phase 2 portion for the age group. * The dose and number of sprinkle capsules/day will be calculated based on individual participant age and weight. Doses will be adjusted based on age and weight changes approximately every 3 months. |
| Infigratinib 0.016 mg/kg | DRUG | Initial cohort dose of infigratinib at the protocol-specified starting dose, with subsequent cohort escalations based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
| Infigratinib 0.032 mg/kg | DRUG | Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
| Infigratinib 0.064 mg/kg | DRUG | Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
| Infigratinib 0.128 mg/kg | DRUG | Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
| Infigratinib 0.25 mg/kg | DRUG | Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth. |
Inclusion Criteria: 1. Subject must be 3 to \<18 years of age at screening with growth potential defined as annualized height velocity of \>1.5 cm/year over a period of at least 6 months of participation in the PROPEL observational study (QBGJ398-001), pubertal Tanner stage ≤4, and bone age ≤13 yea...
Infigratinib is an investigational oral small molecule being developed for achondroplasia and hypochondroplasia, two rare genetic conditions affecting bone growth. It is formulated as minitablets or sprinkle capsules for daily oral administration in children. It is not approved for these uses and remains in clinical development.
Infigratinib is a small molecule inhibitor of the fibroblast growth factor receptors FGFR1, FGFR2, FGFR3, and FGFR4. FGFR3 is the receptor most directly tied to the skeletal growth conditions it is being studied in. By inhibiting these receptors, infigratinib is designed to act on signaling that regulates bone growth.
Infigratinib is being developed by BridgeBio Pharma, Inc., which trades under the ticker BBIO. BridgeBio is advancing the drug in achondroplasia and hypochondroplasia, with a pediatric-focused clinical program spanning Phase 2 and Phase 3 studies across multiple countries.
Infigratinib is at the NDA/BLA stage of development for achondroplasia and hypochondroplasia, meaning a marketing application has been filed. It has received FDA designations including Breakthrough Therapy, Orphan Drug, Fast Track, Rare Pediatric Disease, and Priority Review. It remains investigational and is not yet approved.
Infigratinib has been studied in a completed Phase 3 trial in children and adolescents with achondroplasia (NCT06164951) and is in Phase 2 trials including a long-term extension study in hypochondroplasia (NCT07393373), a study in children under 3 with achondroplasia (NCT07169279), and a hypochondroplasia study (NCT06873035).