Approval Probability 71%
TA Base Rate26%
Adjusted LOA41%
ML RiskLOW_RISK
| NCT ID | Title | Phase | Status | Enrollment | Velocity | Design | Start | Completion | Last Updated | Sites | Countries |
|---|---|---|---|---|---|---|---|---|---|---|---|
| NCT06164951 | A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia | PHASE3 | COMPLETED | 114 | — | — | Nov 10, 2023 | Dec 18, 2025 | Mar 17, 2026 | 27 | United States, Argentina +8 |
| NCT05145010 | Extension Study of Infigratinib in Children With Achondroplasia (ACH) | PHASE2 | ENROLLING BY_INVITATION | 300 | — | — | Dec 6, 2021 | Feb 1, 2032 | Oct 31, 2025 | 31 | United States, Argentina +8 |
| NCT04265651 | Study of Infigratinib in Children With Achondroplasia | PHASE2 | COMPLETED | 84 | — | — | Mar 10, 2020 | Oct 21, 2024 | Oct 22, 2025 | 19 | United States, Australia +4 |
| 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 |
| 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 |
| 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 | 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)). |
| 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...