AchondroplasiaPhase 2NCT07169279
Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)
A Phase 2 study testing Infigratinib is provided as a single dose of minitablets for oral administration and Infigratinib is provided as sprinkle capsules for daily oral administration in people with Achondroplasia, run by QED Therapeutics, a BridgeBio company at 12 sites in 7 countries.
Trial 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. Read more about this study
- Who is it for?
- Ages 0 years to 32 months
- Conditions the study is for
- Achondroplasia
- What is being tested?
- Infigratinib is provided as a single dose of minitablets for oral administration and Infigratinib is provided as sprinkle capsules for daily oral administration
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 77 participants
- Where
- 12 sitesin 7 countries
- Study dates
- 19 Nov 2025to Mar 2032
- Phase
- Phase 2
- Are there results?
- Not yet: the study is under way
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 0 years to 32 months
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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).
Who cannot join
- 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.
Show all 10Show fewer
- Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature, including (but not limited to) r-hGH, IGF-1, CNP analog, FGF ligand trap, or treatment targeting FGFR inhibition at any time.
- 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.
Trial locations and contacts
12 sites in 7 countries. Contact the study team or a site near you to ask about taking part.
12 sites in 7 countries
United States3 sites, 3 recruiting
- Recruiting
Johns Hopkins University
Baltimore, Maryland, 21218
- Recruiting
University of Wisconsin Madison - Waisman Center Bone Dysplasia Clinic
Madison, Wisconsin, 53705
- Recruiting
UCSF Benioff Children's Hospital
Oakland, California, 94609
United Kingdom3 sites, 3 recruiting
- Recruiting
NHS Greater Glasgow and Clyde
Glasgow, G3 8SJ
- Recruiting
Manchester University NHS Foundation Trust
Manchester, M13 9WL
- Recruiting
Sheffield Children's NHS Foundation Trust
Sheffield, S10 2TH
Canada2 sites, 2 recruiting
- Recruiting
Université de Montréal - Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
- Recruiting
Children's Hospital of Eastern Ontario Research Institute
Ottawa, Ontario, K1H 8L1
Australia1 site, 1 recruiting
- Recruiting
Murdoch Children's Research Institute
Parkville, Victoria, 3052
Norway1 site, 1 recruiting
- Recruiting
Paediatric Clinical Research Unit at Oslo University Hospital
Oslo, 0372
Singapore1 site, 1 recruiting
- Recruiting
KK Women's and Children's Hospital
Singapore, 2298899
Spain1 site, 1 recruiting
- Recruiting
Unidad de Cirugía Artroscópica (UCA)
Vitoria-Gasteiz, 01010
About this study
Official 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
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.
Read the detailed description
PROPEL Infant \& Toddler (I\&T) is a Phase 2, multicenter, randomized, placebo-controlled study that comprises 4 portions: the single ascending dose (SAD) portion (open-label), the Phase 2 portion (open-label), the Phase 2b portion (placebo-controlled), and an Extension Portion (open-label). The study will evaluate children with ACH < 3 years old being administered oral infigratinib.
- Conditions
- Achondroplasia
- Being tested
- Infigratinib is provided as a single dose of minitablets for oral administration, Infigratinib is provided as sprinkle capsules for daily oral administration, Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration
- Keywords
- skeletal dysplasia, endochondral ossification, achondroplasia, ACH, Shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, disproportionate short stature, quality of life, dwarfism, bone diseases, musculoskeletal diseases, osteochondrodysplasia, functional abilities, annualized growth velocity, annualized height velocity, growth, growth disorder, congenital, AHV
- Medical subject headings
- Achondroplasia, Mucopolysaccharidosis IV, Dwarfism, Bone Diseases, Musculoskeletal Diseases, Osteochondrodysplasias, Growth Disorders
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Infigratinib is provided as a single dose of minitablets for oral administrationDrug
- 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 administrationDrug
- 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 administrationDrug
- 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 is provided as sprinkle capsules for daily oral administrationDrug
- 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.
| Study group | Role | What they receive |
|---|---|---|
| SAD Cohort 1 infigratinib (2 to less than 3 years old) | Experimental | Single Ascending Dose Escalation and PK Portion Receives: Drug: Infigratinib is provided as a single dose of minitablets for oral administration |
| SAD Cohort 2 infigratinib (1 to less than 2 years old) | Experimental | Single Ascending Dose Escalation and PK Portion Receives: Drug: Infigratinib is provided as a single dose of minitablets for oral administration |
| SAD Cohort 3 infigratinib (6 months to less than 1 year old) | Experimental | Single Ascending Dose Escalation and PK Portion Receives: Drug: Infigratinib is provided as a single dose of minitablets for oral administration |
| SAD Cohort 4 infigratinib (0 to less than 6 months old) | Experimental | Single Ascending Dose Escalation and PK Portion Receives: Drug: Infigratinib is provided as a single dose of minitablets for oral administration |
| Phase 2 Cohort 1 infigratinib (2 to less than 3 years old) | Experimental | Open-label Safety and PK Portion Receives: Drug: Infigratinib is provided as sprinkle capsules for daily oral administration |
| Phase 2 Cohort 2 infigratinib (1 to less than 2 years old) | Experimental | Open-label Safety and PK Portion Receives: Drug: Infigratinib is provided as sprinkle capsules for daily oral administration |
| Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old) | Experimental | Open-label Safety and PK Portion Receives: Drug: Infigratinib is provided as sprinkle capsules for daily oral administration |
| Phase 2 Cohort 4 infigratinib (0 to less than 6 months) | Experimental | Open-label Safety and PK Portion Receives: Drug: Infigratinib is provided as sprinkle capsules for daily oral administration |
| Phase 2B Cohort 1 infigratinib (2 to less than 3 years old) | Experimental | Randomized Safety and Efficacy Portion Receives: Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration |
| Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old) | Experimental | Randomized Safety and Efficacy Portion Receives: Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration |
| Phase 2B Cohort 3 infigratinib (0 to less than 6 months old) | Experimental | Randomized Safety and Efficacy Portion Receives: Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration |
| 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) Receives: Drug: Infigratinib is provided as sprinkle capsules for daily oral administration |
| Phase 2B Cohort 1 Placebo (2 to less than 3 years old) | Placebo comparator | Randomized Safety and Efficacy study Receives: Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration |
| Phase 2B Cohort 2 placebo (6 months to less than 2 years old) | Placebo comparator | Randomized Safety and Efficacy study Receives: Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration |
| Phase 2B Cohort 3 Placebo (0 to less than 6 months old) | Placebo comparator | Randomized Safety and Efficacy study Receives: Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration |
What is being measured
The primary outcome is the main question the study is designed to answer.
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)
Measured: 2 weeks
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).
Measured: 52 weeks
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)
Measured: 52 weeks
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)
Measured: 3 years and 6 months
Secondary outcomes (42)
Phase 2: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
Measured: 52 weeks
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)
Measured: 52 weeks
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)
Measured: 52 weeks
Phase 2: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool)
Measured: 52 weeks
Phase 2: Milestone Development of motor skills (assessed with development milestone charts)
Measured: 52 weeks
Phase 2: Milestone Development of language skills (assessed with development milestone charts)
Measured: 52 weeks
Phase 2: Milestone Development of personal-social skills (assessed with development milestone charts)
Measured: 52 weeks
Phase 2: Skull and brain morphology (as assessed using MRI)
Measured: 52 weeks
Phase 2: Age at closure of cranial structures (as assessed by physical examination)
Measured: 52 weeks
Phase 2: Age at closure of fontanelles (as assessed by physical examination)
Measured: 52 weeks
Phase 2: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)
Measured: 52 weeks
Phase 2: Incidence and severity of sleep apnea (as assessed by polysomnography)
Measured: 52 weeks
Phase 2: Bone morphology (as assessed using x-rays)
Measured: 52 weeks
Phase 2b: Evaluate the pharmacokinetic (PK) profile of infigratinib and its metabolites (by assessing the PK of infigratinib and its active metabolites)
Measured: 52 weeks
Phase 2b: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool)
Measured: 52 weeks
Phase 2b: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
Measured: 52 weeks
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)
Measured: 52 weeks
Phase 2b: Evaluate changes in indicators of growth (by assessing AHV at Week 52 in relation to ACH tables)
Measured: 52 weeks
Phase 2b: Evaluate changes in indicators of growth (by assessing AHV Z-score at Week 52 in relation to ACH tables)
Measured: 52 weeks
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)
Measured: 52 weeks
Phase 2b: Evaluate changes in indicators of growth (by assessing change from BL in Weight-for-Length Z-score at Week 52)
Measured: 52 weeks
Phase 2b: Milestone Development of motor skills (assessed with development milestone charts)
Measured: 52 weeks
Phase 2b: Milestone development of language skills (assessed with development milestone charts)
Measured: 52 weeks
Phase 2b: Milestone development of personal-social skills (assessed with development milestone charts)
Measured: 52 weeks
Phase 2b: Skull and brain morphology (as assessed using MRI)
Measured: 52 weeks
Phase 2b: Age at closure of cranial structures (as assessed by physical examination)
Measured: 52 weeks
Phase 2b: Age at closure of fontanelles (as assessed by physical examination)
Measured: 52 weeks
Phase 2b: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)
Measured: 52 weeks
Phase 2b: Incidence and severity of sleep apnea (as assessed by polysomnography)
Measured: 52 weeks
Phase 2b: Bone morphology (as assessed using x-rays)
Measured: 52 weeks
Extension Phase: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Change from BL in body length Z-score
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Change from BL in upper to lower body segment ratio (cm)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Change from BL in head circumference body length ratio
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Milestone development of social/emotional skills (assessed with development milestone charts)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Milestone development of language communication skills (assessed with development milestone charts)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Milestone development of cognitive skills (assessed with development milestone charts)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Milestone development of movement/physical development skills (assessed with development milestone charts)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Age at closure of cranial structures (as assessed by physical examination)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Age at closure of fontanelles (as assessed by physical examination)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)
Measured: Until they have reached 3 years old (+6 months)
Extension Phase: Bone morphology (as assessed using x-rays)
Measured: Until they have reached 3 years old (+6 months)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Sep 2025
- Study start19 Nov 2025
- Primary completionMar 2030 (estimated)
- Study completionMar 2032 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for AchondroplasiaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Model description
- This study comprises 4 portions: a Single Ascending Dose (SAD) portion and open-label Phase 2 portion to evaluate safety and PK and select a dose level for Phase 2b; a placebo-controlled Phase 2b portion, to assess safety and efficacy at the selected dose level; and an open-label extension portion to evaluate safety and efficacy in children \< 3 years old who have completed the Phase 2 or Phase 2b portion of the study.
- Masking
- Double blind
- Who is masked
- Participant, Investigator
- Enrolment
- 77 participants (estimated)
Sponsor and investigators
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT07169279
- Sponsor's protocol ID
- QBGJ398-204
- Status last verified
- Jul 2026
- First submitted
- 23 Jun 2025
- Last updated
- 13 Jul 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 13 Jul 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official 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
- ClinicalTrials.gov ID
- NCT07169279
- Acronym / sponsor ID
- QBGJ398-204
- Conditions
- Achondroplasia
- Keywords
- skeletal dysplasia, endochondral ossification, achondroplasia, ACH, Shortened proximal limbs, fibroblast growth factor receptor 3, FGFR3, disproportionate short stature
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Recruiting
- Enrolment
- 77 participants (estimated)
- Lead sponsor
- QED Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Study start
- 19 Nov 2025 (actual)
- Primary completion
- Mar 2030 (estimated)
- Study completion
- Mar 2032 (estimated)
- First posted
- 11 Sep 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
PROPEL Infant \& Toddler (I\&T) is a Phase 2, multicenter, randomized, placebo-controlled study that comprises 4 portions: the single ascending dose (SAD) portion (open-label), the Phase 2 portion (open-label), the Phase 2b portion (placebo-controlled), and an Extension Portion (open-label). The study will evaluate children with ACH < 3 years old being administered oral infigratinib.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Double blind (participant, investigator)
- Number of arms
- 15
- Model description
- This study comprises 4 portions: a Single Ascending Dose (SAD) portion and open-label Phase 2 portion to evaluate safety and PK and select a dose level for Phase 2b; a placebo-controlled Phase 2b portion, to assess safety and efficacy at the selected dose level; and an open-label extension portion to evaluate safety and efficacy in children < 3 years old who have completed the Phase 2 or Phase 2b portion of the study.
| Arm | Type | Receives |
|---|---|---|
| SAD Cohort 1 infigratinib (2 to less than 3 years old) | Experimental | Drug: Infigratinib is provided as a single dose of minitablets for oral administrationSingle Ascending Dose Escalation and PK Portion |
| SAD Cohort 2 infigratinib (1 to less than 2 years old) | Experimental | Drug: Infigratinib is provided as a single dose of minitablets for oral administrationSingle Ascending Dose Escalation and PK Portion |
| SAD Cohort 3 infigratinib (6 months to less than 1 year old) | Experimental | Drug: Infigratinib is provided as a single dose of minitablets for oral administrationSingle Ascending Dose Escalation and PK Portion |
| SAD Cohort 4 infigratinib (0 to less than 6 months old) | Experimental | Drug: Infigratinib is provided as a single dose of minitablets for oral administrationSingle Ascending Dose Escalation and PK Portion |
| Phase 2 Cohort 1 infigratinib (2 to less than 3 years old) | Experimental | Drug: Infigratinib is provided as sprinkle capsules for daily oral administrationOpen-label Safety and PK Portion |
| Phase 2 Cohort 2 infigratinib (1 to less than 2 years old) | Experimental | Drug: Infigratinib is provided as sprinkle capsules for daily oral administrationOpen-label Safety and PK Portion |
| Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old) | Experimental | Drug: Infigratinib is provided as sprinkle capsules for daily oral administrationOpen-label Safety and PK Portion |
| Phase 2 Cohort 4 infigratinib (0 to less than 6 months) | Experimental | Drug: Infigratinib is provided as sprinkle capsules for daily oral administrationOpen-label Safety and PK Portion |
| Phase 2B Cohort 1 infigratinib (2 to less than 3 years old) | Experimental | Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administrationRandomized Safety and Efficacy Portion |
| Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old) | Experimental | Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administrationRandomized Safety and Efficacy Portion |
| Phase 2B Cohort 3 infigratinib (0 to less than 6 months old) | Experimental | Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administrationRandomized Safety and Efficacy Portion |
| Open- label Extension infigratinib (0 months to 3 years [+6 months old]) | Experimental | Drug: Infigratinib is provided as sprinkle capsules for daily oral administrationOpen-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 | Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administrationRandomized Safety and Efficacy study |
| Phase 2B Cohort 2 placebo (6 months to less than 2 years old) | Placebo comparator | Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administrationRandomized Safety and Efficacy study |
| Phase 2B Cohort 3 Placebo (0 to less than 6 months old) | Placebo comparator | Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administrationRandomized Safety and Efficacy study |
Who may qualify
- Ages
- 0 years to 32 months
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (7 listed)
- 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.
Key exclusion criteria (10 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| 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 dose and number of minitablets will be calculated based on individual participant age and weight. | Drug | SAD Cohort 1 infigratinib (2 to less than 3 years old), SAD Cohort 2 infigratinib (1 to less than 2 years old), SAD Cohort 3 infigratinib (6 months to less than 1 year old), SAD Cohort 4 infigratinib (0 to less than 6 months old) |
| 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. | Drug | Phase 2 Cohort 1 infigratinib (2 to less than 3 years old), Phase 2 Cohort 2 infigratinib (1 to less than 2 years old), Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old), Phase 2 Cohort 4 infigratinib (0 to less than 6 months) |
| 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. | Drug | Phase 2B Cohort 1 Placebo (2 to less than 3 years old), Phase 2B Cohort 1 infigratinib (2 to less than 3 years old), Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old), Phase 2B Cohort 2 placebo (6 months to less than 2 years old), Phase 2B Cohort 3 Placebo (0 to less than 6 months old), Phase 2B Cohort 3 infigratinib (0 to less than 6 months old) |
| 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. | Drug | Open- label Extension infigratinib (0 months to 3 years [+6 months old]) |
Primary outcome
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)
Time frame: 2 weeks
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).
Time frame: 52 weeks
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)
Time frame: 52 weeks
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)
Time frame: 3 years and 6 months
42 secondary outcomes
Phase 2: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
Time frame: 52 weeks
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)
Time frame: 52 weeks
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)
Time frame: 52 weeks
Phase 2: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool)
Time frame: 52 weeks
Phase 2: Milestone Development of motor skills (assessed with development milestone charts)
Time frame: 52 weeks
Phase 2: Milestone Development of language skills (assessed with development milestone charts)
Time frame: 52 weeks
Phase 2: Milestone Development of personal-social skills (assessed with development milestone charts)
Time frame: 52 weeks
Phase 2: Skull and brain morphology (as assessed using MRI)
Time frame: 52 weeks
Phase 2: Age at closure of cranial structures (as assessed by physical examination)
Time frame: 52 weeks
Phase 2: Age at closure of fontanelles (as assessed by physical examination)
Time frame: 52 weeks
Phase 2: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)
Time frame: 52 weeks
Phase 2: Incidence and severity of sleep apnea (as assessed by polysomnography)
Time frame: 52 weeks
Phase 2: Bone morphology (as assessed using x-rays)
Time frame: 52 weeks
Phase 2b: Evaluate the pharmacokinetic (PK) profile of infigratinib and its metabolites (by assessing the PK of infigratinib and its active metabolites)
Time frame: 52 weeks
Phase 2b: Change from BL in Health-related Quality of Life (HRQoL) (as assessed by Infant and Toddler Quality of Life Inventory [ITQoL] tool)
Time frame: 52 weeks
Phase 2b: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
Time frame: 52 weeks
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)
Time frame: 52 weeks
Phase 2b: Evaluate changes in indicators of growth (by assessing AHV at Week 52 in relation to ACH tables)
Time frame: 52 weeks
Phase 2b: Evaluate changes in indicators of growth (by assessing AHV Z-score at Week 52 in relation to ACH tables)
Time frame: 52 weeks
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)
Time frame: 52 weeks
Phase 2b: Evaluate changes in indicators of growth (by assessing change from BL in Weight-for-Length Z-score at Week 52)
Time frame: 52 weeks
Phase 2b: Milestone Development of motor skills (assessed with development milestone charts)
Time frame: 52 weeks
Phase 2b: Milestone development of language skills (assessed with development milestone charts)
Time frame: 52 weeks
Phase 2b: Milestone development of personal-social skills (assessed with development milestone charts)
Time frame: 52 weeks
Phase 2b: Skull and brain morphology (as assessed using MRI)
Time frame: 52 weeks
Phase 2b: Age at closure of cranial structures (as assessed by physical examination)
Time frame: 52 weeks
Phase 2b: Age at closure of fontanelles (as assessed by physical examination)
Time frame: 52 weeks
Phase 2b: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)
Time frame: 52 weeks
Phase 2b: Incidence and severity of sleep apnea (as assessed by polysomnography)
Time frame: 52 weeks
Phase 2b: Bone morphology (as assessed using x-rays)
Time frame: 52 weeks
Extension Phase: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Change from BL in body length Z-score
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Change from BL in upper to lower body segment ratio (cm)
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Change from BL in head circumference body length ratio
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Milestone development of social/emotional skills (assessed with development milestone charts)
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Milestone development of language communication skills (assessed with development milestone charts)
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Milestone development of cognitive skills (assessed with development milestone charts)
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Milestone development of movement/physical development skills (assessed with development milestone charts)
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Age at closure of cranial structures (as assessed by physical examination)
Time frame: Until they have reached 3 years old (+6 months)
Extension Phase: Age at closure of fontanelles (as assessed by physical examination)
Time frame: Until they have reached 3 years old (+6 months)
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 13 Jul 2026.
- Individual participant dataSharing planned: No
Related trials for Achondroplasia
Enrolling by invitationPhase 2
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
Achondroplasia
RecruitingPhase 2/3
Study to Evaluate the Efficacy and Safety of BMN 333 Versus Vosoritide in Children With Achondroplasia
Achondroplasia
Active, not recruitingPhase 2
A Clinical Trial to Evaluate Efficacy and Safety of Navepegritide in Adolescents (12 - 18 Years of Age) With Achondroplasia.
Achondroplasia
Active, not recruitingPhase 3
Evaluate the Efficacy and Safety of KK8398 in Patients With Achondroplasia（AOBA Study）
Achondroplasia
RecruitingPhase 2
A Study of TYRA-300 in Children With Achondroplasia: BEACH301
Achondroplasia
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part.
12 sites in 7 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Infigratinib is provided as a single dose of minitablets for oral administration and Infigratinib is provided as sprinkle capsules for daily oral administration in people with Achondroplasia, run by QED Therapeutics, a BridgeBio company at 12 sites in 7 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: looking for participants now, as of the sponsor's last update on 13 Jul 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 12 sites in United States, United Kingdom, Canada, Australia, Norway and 2 more countries. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact QED Therapeutics Inc. (18772805655, medinfo@qedtx.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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) (time frame: 2 weeks). The study lists 4 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 13 Jul 2026.