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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT07169279
Registration number
NCT07169279
Ethics application status
Date submitted
23/06/2025
Date registered
11/09/2025
Date last updated
11/09/2025
Titles & IDs
Public title
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
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Scientific title
Phase 2b, Randomized, Double-Blind, Placebo-Controlled Clinical Trial, Preceded by a Single Ascending Dose Portion and a Phase 2 Open-Label Portion, to Evaluate the Safety and Efficacy of Oral Infigratinib in Infants and Young Children With Achondroplasia
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Secondary ID [1]
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QBGJ398-204
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Achondroplasia
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Condition category
Condition code
Human Genetics and Inherited Disorders
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0
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Other human genetics and inherited disorders
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Musculoskeletal
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0
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Other muscular and skeletal disorders
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Metabolic and Endocrine
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0
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Other metabolic disorders
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Metabolic and Endocrine
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Other endocrine disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Infigratinib is provided as a single dose of minitablets for oral administration
Treatment: Drugs - Infigratinib is provided as sprinkle capsules for daily oral administration
Treatment: Drugs - Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
Treatment: Drugs - Infigratinib is provided as sprinkle capsules for daily oral administration
Experimental: SAD Cohort 1 infigratinib (2 to less than 3 years old) - Single Ascending Dose Escalation and PK Portion
Experimental: SAD Cohort 2 infigratinib (1 to less than 2 years old) - Single Ascending Dose Escalation and PK Portion
Experimental: SAD Cohort 3 infigratinib (6 months to less than 1 year old) - Single Ascending Dose Escalation and PK Portion
Experimental: SAD Cohort 4 infigratinib (0 to less than 6 months old) - Single Ascending Dose Escalation and PK Portion
Experimental: Phase 2 Cohort 1 infigratinib (2 to less than 3 years old) - Open-label Safety and PK Portion
Experimental: Phase 2 Cohort 2 infigratinib (1 to less than 2 years old) - Open-label Safety and PK Portion
Experimental: Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old) - Open-label Safety and PK Portion
Experimental: Phase 2 Cohort 4 infigratinib (0 to less than 6 months) - Open-label Safety and PK Portion
Experimental: Phase 2B Cohort 1 infigratinib (2 to less than 3 years old) - Randomized Safety and Efficacy Portion
Experimental: Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old) - Randomized Safety and Efficacy Portion
Experimental: Phase 2B Cohort 3 infigratinib (0 to less than 6 months old) - Randomized Safety and Efficacy Portion
Experimental: Open- label Extension infigratinib (0 months to 3 years [+6 months old]) - Open-label extension portion continuing to assess safety and efficacy in children until they reach 3 years old (+6 months)
Placebo comparator: Phase 2B Cohort 1 Placebo (2 to less than 3 years old) - Randomized Safety and Efficacy study
Placebo comparator: Phase 2B Cohort 2 placebo (6 months to less than 2 years old) - Randomized Safety and Efficacy study
Placebo comparator: Phase 2B Cohort 3 Placebo (0 to less than 6 months old) - Randomized Safety and Efficacy study
Treatment: Drugs: Infigratinib is provided as a single dose of minitablets for oral administration
* The initial cohort dose of infigratinib will begin at the protocol-specified starting dose, with subsequent cohort escalation based on protocol specific criteria.
* The target dose is the dose that will provide similar exposure to the dose of 0.25 mg/kg/day in participants 3 years old and older.
* The dose and number of minitablets will be calculated based on individual participant age and weight.
Treatment: Drugs: Infigratinib is provided as sprinkle capsules for daily oral administration
* 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.
Treatment: Drugs: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
* 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.
Treatment: Drugs: Infigratinib is provided as sprinkle capsules for daily oral administration
* The dose of infigratinib will be the dose confirmed in the Phase 2 portion and used in the Phase 2b 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 for the first year and every 6 months thereafter.
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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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)
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Assessment method [1]
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Timepoint [1]
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2 weeks
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Primary outcome [2]
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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).
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Assessment method [2]
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Timepoint [2]
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52 weeks
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Primary outcome [3]
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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)
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Assessment method [3]
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Timepoint [3]
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52 weeks
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Primary outcome [4]
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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)
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Assessment method [4]
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Timepoint [4]
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3 years and 6 months
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Secondary outcome [1]
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Phase 2: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
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Assessment method [1]
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0
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Timepoint [1]
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52 weeks
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Secondary outcome [2]
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Phase 2: Evaluate changes in indicators of growth (by assessing change from BL in body length z-score at Week 52 in relation to ACH tables)
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Assessment method [2]
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Timepoint [2]
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52 weeks
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Secondary outcome [3]
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Phase 2: Evaluate changes in indicators of body proportions (by assessing change from BL to Week 52 in upper-to-lower body segment ratio and head circumference/body length ratio)
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Assessment method [3]
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0
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Timepoint [3]
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52 weeks
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Secondary outcome [4]
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Phase 2: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool)
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Assessment method [4]
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0
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Timepoint [4]
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52 weeks
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Secondary outcome [5]
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Phase 2: Milestone Development of motor skills (assessed with development milestone charts)
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Assessment method [5]
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0
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Timepoint [5]
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52 weeks
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Secondary outcome [6]
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Phase 2: Milestone Development of language skills (assessed with development milestone charts)
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Assessment method [6]
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0
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Timepoint [6]
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52 weeks
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Secondary outcome [7]
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Phase 2: Milestone Development of personal-social skills (assessed with development milestone charts)
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Assessment method [7]
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0
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Timepoint [7]
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52 weeks
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Secondary outcome [8]
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0
Phase 2: Skull and brain morphology (as assessed using MRI)
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Assessment method [8]
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0
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Timepoint [8]
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52 weeks
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Secondary outcome [9]
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0
Phase 2: Age at closure of cranial structures (as assessed by physical examination)
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Assessment method [9]
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0
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Timepoint [9]
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52 weeks
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Secondary outcome [10]
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Phase 2: Age at closure of fontanelles (as assessed by physical examination)
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Assessment method [10]
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Timepoint [10]
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52 weeks
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Secondary outcome [11]
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Phase 2: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)
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Assessment method [11]
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Timepoint [11]
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52 weeks
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Secondary outcome [12]
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Phase 2: Incidence and severity of sleep apnea (as assessed by polysomnography)
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Assessment method [12]
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Timepoint [12]
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52 weeks
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Secondary outcome [13]
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Phase 2: Bone morphology (as assessed using x-rays)
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Assessment method [13]
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Timepoint [13]
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52 weeks
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Secondary outcome [14]
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Phase 2b: Evaluate the pharmacokinetic (PK) profile of infigratinib and its metabolites (by assessing the PK of infigratinib and its active metabolites)
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Assessment method [14]
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Timepoint [14]
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52 weeks
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Secondary outcome [15]
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Phase 2b: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool)
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Assessment method [15]
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0
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Timepoint [15]
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52 weeks
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Secondary outcome [16]
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Phase 2b: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
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Assessment method [16]
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0
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Timepoint [16]
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52 weeks
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Secondary outcome [17]
0
0
Phase 2b: Evaluate changes in indicators of growth (by assessing change from BL in body length z-score at Week 52 in relation to ACH tables)
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Assessment method [17]
0
0
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Timepoint [17]
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52 weeks
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Secondary outcome [18]
0
0
Phase 2b: Evaluate changes in indicators of body proportions (by assessing change from BL to Week 52 in upper-to-lower body segment ratio and head circumference/body length ratio)
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Assessment method [18]
0
0
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Timepoint [18]
0
0
52 weeks
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Secondary outcome [19]
0
0
Phase 2b: Milestone Development of motor skills (assessed with development milestone charts)
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Assessment method [19]
0
0
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Timepoint [19]
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52 weeks
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Secondary outcome [20]
0
0
Phase 2b: Milestone development of language skills (assessed with development milestone charts)
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Assessment method [20]
0
0
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Timepoint [20]
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52 weeks
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Secondary outcome [21]
0
0
Phase 2b: Milestone development of personal-social skills (assessed with development milestone charts)
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Assessment method [21]
0
0
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Timepoint [21]
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0
52 weeks
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Secondary outcome [22]
0
0
Phase 2b: Skull and brain morphology (as assessed using MRI)
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Assessment method [22]
0
0
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Timepoint [22]
0
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52 weeks
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Secondary outcome [23]
0
0
Phase 2b: Age at closure of cranial structures (as assessed by physical examination)
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Assessment method [23]
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0
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Timepoint [23]
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52 weeks
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Secondary outcome [24]
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0
Phase 2b: Age at closure of fontanelles (as assessed by physical examination)
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Assessment method [24]
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0
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Timepoint [24]
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52 weeks
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Secondary outcome [25]
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Phase 2b: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)
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Assessment method [25]
0
0
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Timepoint [25]
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52 weeks
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Secondary outcome [26]
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Phase 2b: Incidence and severity of sleep apnea (as assessed by polysomnography)
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Assessment method [26]
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Timepoint [26]
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52 weeks
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Secondary outcome [27]
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Phase 2b: Bone morphology (as assessed using x-rays)
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Assessment method [27]
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Timepoint [27]
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52 weeks
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Secondary outcome [28]
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Extension Phase: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
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Assessment method [28]
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0
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Timepoint [28]
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Until they have reached 3 years old (+6 months)
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Secondary outcome [29]
0
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Extension Phase: Change from BL in body length Z-score
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Assessment method [29]
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0
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Timepoint [29]
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Until they have reached 3 years old (+6 months)
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Secondary outcome [30]
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Extension Phase: Change from BL in upper to lower body segment ratio (cm)
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Assessment method [30]
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0
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Timepoint [30]
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Until they have reached 3 years old (+6 months)
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Secondary outcome [31]
0
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Extension Phase: Change from BL in head circumference body length ratio
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Assessment method [31]
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0
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Timepoint [31]
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Until they have reached 3 years old (+6 months)
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Secondary outcome [32]
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Extension Phase: Milestone development of social/emotional skills (assessed with development milestone charts)
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Assessment method [32]
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0
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Timepoint [32]
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Until they have reached 3 years old (+6 months)
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Secondary outcome [33]
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Extension Phase: Milestone development of language communication skills (assessed with development milestone charts)
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Assessment method [33]
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0
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Timepoint [33]
0
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Until they have reached 3 years old (+6 months)
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Secondary outcome [34]
0
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Extension Phase: Milestone development of cognitive skills (assessed with development milestone charts)
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Assessment method [34]
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0
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Timepoint [34]
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Until they have reached 3 years old (+6 months)
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Secondary outcome [35]
0
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Extension Phase: Milestone development of movement/physical development skills (assessed with development milestone charts)
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Assessment method [35]
0
0
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Timepoint [35]
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Until they have reached 3 years old (+6 months)
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Secondary outcome [36]
0
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Extension Phase: Age at closure of cranial structures (as assessed by physical examination)
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Assessment method [36]
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0
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Timepoint [36]
0
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Until they have reached 3 years old (+6 months)
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Secondary outcome [37]
0
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Extension Phase: Age at closure of fontanelles (as assessed by physical examination)
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Assessment method [37]
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0
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Timepoint [37]
0
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Until they have reached 3 years old (+6 months)
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Secondary outcome [38]
0
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Extension Phase: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)
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Assessment method [38]
0
0
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Timepoint [38]
0
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Until they have reached 3 years old (+6 months)
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Secondary outcome [39]
0
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Extension Phase: Bone morphology (as assessed using x-rays)
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Assessment method [39]
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0
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Timepoint [39]
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Until they have reached 3 years old (+6 months)
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Eligibility
Key inclusion criteria
* Diagnosis of ACH confirmed by genetic testing. If prospective participants had prior genetic testing, the diagnosis must be confirmed by a report from a certified laboratory, documenting the specific mutation.
* Age 0 to 32 months (2 years and 8 months) at screening.
* Signed informed consent, which must be obtained from each participant's parent(s) or legal guardian.
* Parent(s)/Guardian(s) willing and able to attend all study visits and comply with all study requirements.
* Parent(s)/Guardian(s) willing and able to comply with the routine care of the study participants according to local guidance for the management of infants and young children with ACH.
* Able to swallow age-appropriate oral medication.
* In participants <1 year old, be compliant with recommended vitamin D supplementation of 5 10 µg/day or higher (or as recommended by country specific guidelines).
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Minimum age
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Years
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Maximum age
32
Months
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
* Participants who have hypochondroplasia or diagnosis of genetic condition other than ACH, or any clinical condition that can affect growth.
* Gestational age at birth <37 weeks and/or birth weight <2500 grams.
* Gastroesophageal reflux disease requiring prolonged treatment (>1 week) with prohibited medications.
* Evidence of cervicomedullary compression, as defined by an Achondroplasia Foramen Magnum Score (AFMS) 4, symptomatic or asymptomatic, diagnosed during MRI done at screening or a previous MRI done at any time if the participant had not undergone decompression surgery.
* History of fracture of a long bone or spine within 6 months prior to screening.
* Any other significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib and/or would require treatment with a prohibited medication (per protocol), and/or would place the participant at high risk for poor treatment compliance or for failure to complete the study.
* Regular long-term (>3 weeks; more than twice/year) treatment with supraphysiologic doses of glucocorticoid therapy (ie, >15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (for over 3 weeks within 6 months of the screening visit. NOTE: Low-dose topical, inhaled, or intranasal corticosteroids are acceptable.
* Significant abnormality in screening laboratory results,
* Allergy or hypersensitivity to any components of the study drug.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Blinded (masking used)
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Who is / are masked / blinded?
The people receiving the treatment/s
The people analysing the results/data
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Intervention assignment
Parallel
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Not yet recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
1/10/2025
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Actual
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/03/2032
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Actual
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Sample size
Target
77
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Children's Research Institute - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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0
United States of America
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State/province [1]
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0
California
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Country [2]
0
0
United States of America
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State/province [2]
0
0
Maryland
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Country [3]
0
0
United States of America
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State/province [3]
0
0
Wisconsin
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Country [4]
0
0
Canada
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State/province [4]
0
0
Ontario
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Country [5]
0
0
Canada
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State/province [5]
0
0
Quebec
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Country [6]
0
0
Norway
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State/province [6]
0
0
Oslo
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Country [7]
0
0
Singapore
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State/province [7]
0
0
Singapore
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Country [8]
0
0
Spain
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State/province [8]
0
0
Vitoria-Gasteiz
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Country [9]
0
0
United Kingdom
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State/province [9]
0
0
Glasgow
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Country [10]
0
0
United Kingdom
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State/province [10]
0
0
London
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Country [11]
0
0
United Kingdom
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State/province [11]
0
0
Manchester
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Country [12]
0
0
United Kingdom
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State/province [12]
0
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Sheffield
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
QED Therapeutics, Inc., a Bridgebio company
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Address
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Country
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Ethics approval
Ethics application status
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Summary
Brief summary
This is a Phase 2, multicenter, randomized, placebo-controlled study to evaluate the safety and efficacy of infigratinib in participants \< 3 years old with ACH. The purposes of the SAD and Phase 2 portions are to identify and confirm the dose of infigratinib to be used in the Phase 2b portion, based on safety and PK. The purpose of the Phase 2b, placebo-controlled portion is to evaluate the safety and efficacy of infigratinib in children \< 3 years old with ACH at the selected dose.
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Trial website
https://clinicaltrials.gov/study/NCT07169279
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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Address
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Country
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0
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Phone
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0
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Fax
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0
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Email
0
0
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Contact person for public queries
Name
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QED Therapeutics Inc.
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Address
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0
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Country
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0
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Phone
0
0
18772805655
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Fax
0
0
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Email
0
0
[email protected]
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Contact person for scientific queries
Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
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No/undecided IPD sharing reason/comment
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What supporting documents are/will be available?
No Supporting Document Provided
Results publications and other study-related documents
No documents have been uploaded by study researchers.
Results not provided in
https://clinicaltrials.gov/study/NCT07169279
Download to PDF