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RecruitingNCT07169279Updated Jul 13, 2026

Interventional Study of Infigratinib in Children < 3 Years Old With Achondroplasia (ACH)

A Phase 2 interventional study of Infigratinib is provided as a single dose of minitablets for oral administration and Infigratinib is provided as sprinkle capsules for daily oral administration in Achondroplasia, sponsored by QED Therapeutics, a BridgeBio company. Recruiting at 12 sites in 7 countries. Open to participants aged 0 Years to 32 Months. Per ClinicalTrials.gov, last updated 2026-07-13.

Sponsored by QED Therapeutics, a BridgeBio company (part of BridgeBio) · Phase 2, Interventional, and Treatment

From the registry’s dates

  • Started Nov 2025; still recruiting 11 months later.
Phase
Phase 2
Study type
Interventional
Enrollment
77
Allocation
Randomized
Ages
0 Years to 32 Months
Sex
All
01

Study 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.

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.

02

Conditions studied

  • Achondroplasia

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
  • AGV
  • infant and toddler
  • endochondral bone formation
03

In context

Achondroplasia

47 studies on the registry are indexed under Achondroplasia; 13 are open to participants now.

This study's planned enrollment of 77 is close to the median of 74 across 28 interventional studies indexed under Achondroplasia.

Browse Achondroplasia studies →

Lead sponsor

QED Therapeutics, a BridgeBio company is the lead sponsor of 8 studies on the registry; 5 are open to participants now.

Browse QED Therapeutics studies →All BridgeBio studies →

Counted across the registry records on this site, refreshed daily.

04

Who can participate

Ages eligible
0 Years to 32 Months
Sexes eligible
All
Accepts healthy volunteers
No

Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.

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 , 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).

Exclusion criteria

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.
  • Having received or planning to receive treatment with any other 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 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 to any components of the study drug.
Terms in these criteria, in plain language(4)

Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.

Informed consent

Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.

You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.

Investigational drug or device

A drug or device being studied in a clinical trial.

It may be new, or already approved and being studied for another use, dose or form. Criteria often leave out people who took part in another study of one within a recent period, commonly 30 days or five half-lives.

Corticosteroids (steroids)

Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.

Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.

Hypersensitivity

An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Double (Participant, Investigator)
Enrollment
77 participants (estimated)

Study arms

  • Experimental
    SAD Cohort 1 infigratinib (2 to less than 3 years old)

    Single Ascending Dose Escalation and PK Portion

    Drug: Infigratinib is provided as a single dose of minitablets for oral administration

  • Experimental
    SAD Cohort 2 infigratinib (1 to less than 2 years old)

    Single Ascending Dose Escalation and PK Portion

    Drug: Infigratinib is provided as a single dose of minitablets for oral administration

  • Experimental
    SAD Cohort 3 infigratinib (6 months to less than 1 year old)

    Single Ascending Dose Escalation and PK Portion

    Drug: Infigratinib is provided as a single dose of minitablets for oral administration

  • Experimental
    SAD Cohort 4 infigratinib (0 to less than 6 months old)

    Single Ascending Dose Escalation and PK Portion

    Drug: Infigratinib is provided as a single dose of minitablets for oral administration

  • Experimental
    Phase 2 Cohort 1 infigratinib (2 to less than 3 years old)

    Open-label Safety and PK Portion

    Drug: Infigratinib is provided as sprinkle capsules for daily oral administration

  • Experimental
    Phase 2 Cohort 2 infigratinib (1 to less than 2 years old)

    Open-label Safety and PK Portion

    Drug: Infigratinib is provided as sprinkle capsules for daily oral administration

  • Experimental
    Phase 2 Cohort 3 infigratinib (6 months to less than 1 year old)

    Open-label Safety and PK Portion

    Drug: Infigratinib is provided as sprinkle capsules for daily oral administration

  • Experimental
    Phase 2 Cohort 4 infigratinib (0 to less than 6 months)

    Open-label Safety and PK Portion

    Drug: Infigratinib is provided as sprinkle capsules for daily oral administration

  • Experimental
    Phase 2B Cohort 1 infigratinib (2 to less than 3 years old)

    Randomized Safety and Efficacy Portion

    Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration

  • Experimental
    Phase 2B Cohort 2 infigratinib (6 months to less than 2 years old)

    Randomized Safety and Efficacy Portion

    Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration

  • Experimental
    Phase 2B Cohort 3 infigratinib (0 to less than 6 months old)

    Randomized Safety and Efficacy Portion

    Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration

  • 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)

    Drug: Infigratinib is provided as sprinkle capsules for daily oral administration

  • Placebo comparator
    Phase 2B Cohort 1 Placebo (2 to less than 3 years old)

    Randomized Safety and Efficacy study

    Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration

  • Placebo comparator
    Phase 2B Cohort 2 placebo (6 months to less than 2 years old)

    Randomized Safety and Efficacy study

    Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration

  • Placebo comparator
    Phase 2B Cohort 3 Placebo (0 to less than 6 months old)

    Randomized Safety and Efficacy study

    Drug: Infigratinib or placebo comparator is provided as sprinkle capsules for daily oral administration

Interventions

  • DrugInfigratinib 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.

  • DrugInfigratinib 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.

  • DrugInfigratinib 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.

  • DrugInfigratinib 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.

06

What researchers measure

Primary outcomes

  1. 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

  2. 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

  3. 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

  4. 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

Secondary outcomes

  1. Phase 2: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)

    Time frame: 52 weeks

  2. 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

  3. 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

  4. 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

  5. Phase 2: Milestone Development of motor skills (assessed with development milestone charts)

    Time frame: 52 weeks

  6. Phase 2: Milestone Development of language skills (assessed with development milestone charts)

    Time frame: 52 weeks

  7. Phase 2: Milestone Development of personal-social skills (assessed with development milestone charts)

    Time frame: 52 weeks

  8. Phase 2: Skull and brain morphology (as assessed using MRI)

    Time frame: 52 weeks

  9. Phase 2: Age at closure of cranial structures (as assessed by physical examination)

    Time frame: 52 weeks

  10. Phase 2: Age at closure of fontanelles (as assessed by physical examination)

    Time frame: 52 weeks

  11. Phase 2: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)

    Time frame: 52 weeks

  12. Phase 2: Incidence and severity of sleep apnea (as assessed by polysomnography)

    Time frame: 52 weeks

  13. Phase 2: Bone morphology (as assessed using x-rays)

    Time frame: 52 weeks

  14. 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

  15. 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

  16. Phase 2b: Evaluate the safety of oral daily doses of infigratinib (by assessing AEs and SAEs)

    Time frame: 52 weeks

  17. 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

  18. Phase 2b: Evaluate changes in indicators of growth (by assessing AHV at Week 52 in relation to ACH tables)

    Time frame: 52 weeks

  19. 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

  20. 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

  21. 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

  22. Phase 2b: Milestone Development of motor skills (assessed with development milestone charts)

    Time frame: 52 weeks

  23. Phase 2b: Milestone development of language skills (assessed with development milestone charts)

    Time frame: 52 weeks

  24. Phase 2b: Milestone development of personal-social skills (assessed with development milestone charts)

    Time frame: 52 weeks

  25. Phase 2b: Skull and brain morphology (as assessed using MRI)

    Time frame: 52 weeks

  26. Phase 2b: Age at closure of cranial structures (as assessed by physical examination)

    Time frame: 52 weeks

  27. Phase 2b: Age at closure of fontanelles (as assessed by physical examination)

    Time frame: 52 weeks

  28. Phase 2b: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)

    Time frame: 52 weeks

  29. Phase 2b: Incidence and severity of sleep apnea (as assessed by polysomnography)

    Time frame: 52 weeks

  30. Phase 2b: Bone morphology (as assessed using x-rays)

    Time frame: 52 weeks

  31. 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)

  32. Extension Phase: Change from BL in body length Z-score

    Time frame: Until they have reached 3 years old (+6 months)

  33. Extension Phase: Change from BL in upper to lower body segment ratio (cm)

    Time frame: Until they have reached 3 years old (+6 months)

  34. Extension Phase: Change from BL in head circumference body length ratio

    Time frame: Until they have reached 3 years old (+6 months)

  35. Extension Phase: Milestone development of social/emotional skills (assessed with development milestone charts)

    Time frame: Until they have reached 3 years old (+6 months)

  36. Extension Phase: Milestone development of language communication skills (assessed with development milestone charts)

    Time frame: Until they have reached 3 years old (+6 months)

  37. Extension Phase: Milestone development of cognitive skills (assessed with development milestone charts)

    Time frame: Until they have reached 3 years old (+6 months)

  38. 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)

  39. Extension Phase: Age at closure of cranial structures (as assessed by physical examination)

    Time frame: Until they have reached 3 years old (+6 months)

  40. Extension Phase: Age at closure of fontanelles (as assessed by physical examination)

    Time frame: Until they have reached 3 years old (+6 months)

  41. Extension Phase: Incidence of surgical interventions (i.e., cervical decompression, adenotonsillectomy etc)

    Time frame: Until they have reached 3 years old (+6 months)

  42. Extension Phase: Bone morphology (as assessed using x-rays)

    Time frame: Until they have reached 3 years old (+6 months)

07

Study locations

12 of 12 sites recruiting
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Updates

Tracking since Sep 25, 2026
No changes since tracking began. The registry record was last updated on Jul 13, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT07169279
Lead sponsor
QED Therapeutics, a BridgeBio company
Responsible party
Sponsor
First posted
Sep 11, 2025
Start date
Nov 19, 2025
Primary completion
Mar 2030 (estimated)
Completion
Mar 2032 (estimated)
Last update
Jul 13, 2026

Study contacts

QED Therapeutics Inc.
Contact
medinfo@qedtx.com
18772805655

Oversight

Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
View the source record on ClinicalTrials.gov ↗

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