Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
Phase 2
77
about 6.4 years
0–2.66666666666667
3 sites in CA, MD, WI
What this study is about
This trial is testing a treatment called infigratinib in children under 3 years old who have achondroplasia. The goal is to determine the best dose of this treatment for these young patients.
Simplified from trial records by PatientMatch.
What you may be asked to do
- 1.Take Infigratinib is provided as a single dose of minitablets for oral administration
- 2.Take Infigratinib is provided as sprinkle capsules for daily oral administration
- 3.Take Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
Participation Burden
What's physically and logistically required of participants.
Requires travel to a study site
How treatment is administered
You may get a placebo/standard care, and you won't know which.
Extracted study details
Pulled from the trial record to show what is being tested and what the study is measuring.
infigratinib, ANTINEOPLASTIC AND IMMUNOMODULATING AGENTS
oral (Oral Capsule), oral
Primary: Extension Portion: Evaluate the safety and efficacy of infigratinib in participants who completed the Phase 2 or Phase 2b portion of the study until they have reached 3 years old (+6 months) (by assessing AE's and SAE's), Phase 2 Portion: Confirm the doses to be used in each age cohort in the Phase 2b portion of the study (by assessing safety and PK of infigratinib and its active metabolites)., Phase 2b Portion: Evaluate the safety and efficacy of infigratinib in infants and children < 3 years old with ACH (by assessing AE's & SAE's), Single Ascending Dose Portion: Identify the dose of infigratinib to be used in each age cohort of the Phase 2 potion of the study (by assessing safety and PK of infigratinib and its active metabolites)
Secondary: Extension Phase: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs), Phase 2: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool), Phase 2: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs), Phase 2: Skull and brain morphology (as assessed using MRI), Phase 2b: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool), Phase 2b: Evaluate the pharmacokinetic (PK) profile of infigratinib and its metabolites (by assessing the PK of infigratinib and its active metabolites), Phase 2b: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs), Phase 2b: Skull and brain morphology (as assessed using MRI)