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CompletedNCT06164951PROPEL3Updated Mar 17, 2026

A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia

A Phase 3 interventional study of Infigratinib 0.25 mg/kg/day and Placebo Comparator 0.25 mg/kg/day in Achondroplasia, sponsored by QED Therapeutics, a BridgeBio company. Completed at 27 sites in 10 countries. Open to participants aged 3 Years to 17 Years. Per ClinicalTrials.gov, last updated 2026-03-17.

Sponsored by QED Therapeutics, a BridgeBio company (part of BridgeBio) · Phase 3, Interventional, and Treatment

Phase
Phase 3
Study type
Interventional
Enrollment
114
Allocation
Randomized
Ages
3 Years to 17 Years
Sex
All
01

Study summary

This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and adolescents with achondroplasia (ACH) who have completed at least 26 weeks of participation in the QED-sponsored study PROPEL (QBGJ398-001).

02

Conditions studied

  • Achondroplasia

Keywords

  • Skeletal dysplasia
  • Endochondral ossification
  • Quality of life in achondroplasia
  • Fibroblast growth factor receptor 3
  • FGFR3
  • Endochondral bone formation
  • Short-limb disproportionate dwarfism
  • Dwarfism
  • Bone disease
  • Functionality in achondroplasia
  • Musculoskeletal diseases
  • Osteochondrodysplasia
  • Genetic diseases
  • Long-term treatment
  • Growth
  • Annualized height velocity
03

In context

Achondroplasia

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

This study's enrollment of 114 is above 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.

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

04

Who can participate

Ages eligible
3 Years to 17 Years
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

  1. Subject must be 3 to \<18 years of age at screening with growth potential defined as annualized height velocity of >1.5 cm/year over a period of at least 6 months of participation in the PROPEL observational study (QBGJ398-001), pubertal Tanner stage ≤4, and bone age ≤13 years in females and ≤15 years in males.

    Type of Subject and Disease Characteristics

  2. Subjects who have a diagnosis of ACH that has been documented clinically and confirmed by genetic testing.
  3. Subjects must have completed at least 26 weeks in the PROPEL (QBGJ398-001) study before screening.
  4. Subjects are able to swallow oral medication.
  5. Subjects and parent(s), legal guardian(s), or caregivers are willing and able to comply with study visits and study procedures.
  6. Subjects are ambulatory and able to stand without assistance.

    Sex and /Barrier Requirements

  7. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
  8. If sexually active, subjects, whether male or female, must be willing to use a highly effective method of contraception while taking study drug and for 3 months after the last dose of study drug.

  9. Signed informed consent, which includes compliance with the requirements and restrictions listed in the informed consent form and in this protocol, must be obtained for each subject from their parent(s) or legal guardian and signed informed consent/assent must be obtained from the subject (when applicable)

Exclusion criteria

Exclusion Criteria:

Medical Conditions

  1. Subjects who have hypochondroplasia or short stature condition other than ACH.
  2. Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
  3. Current evidence of corneal or retinal disorder/keratopathy -confirmed by ophthalmic examination.
  4. Concurrent circumstance, disease or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations and/or would require treatment with a prohibited medication, and/or would place the subject at high risk for poor treatment compliance or for not completing the study.
  5. History and/or current evidence of extensive ectopic tissue calcification.
  6. History of .

    Prior/Concomitant Therapy

  7. Having received or planning to receive treatment with any other or approved product for the treatment of ACH or short stature.
  8. Regular long-term treatment (≥3 weeks) with supraphysiologic doses of therapy (ie, >15 mg/m2/day of hydrocortisone or equivalent) or treatment with glucocorticoids at anti-inflammatory doses (ie, 2.5-10 mg/kg/day of hydrocortisone or equivalent) for over 3 weeks within 6 months of the screening visit (low-dose local preparations including inhaled steroid for asthma, intranasal sprays for allergies, and topical steroids are allowed).
  9. Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening surgery or guided growth surgery during the study period. Guided growth surgery with plates removed at least 12 months prior to screening is allowed.
  10. Currently receiving treatment with agents that are known strong inducers or inhibitors of or prolonged treatment (>1 week) with medications that alter the pH of the gastrointestinal tract including antacids, H2 antagonists (eg, ranitidine, famotidine), and proton-pump inhibitors (eg, omeprazole).
  11. Current evidence of endocrine alterations of calcium/phosphorus homeostasis.

    Diagnostic assessments

  12. Subjects who have significant abnormality in screening laboratory results.

    Other Exclusions

  13. Having had a fracture of the long bones (ie, extremities) or spine within 12 months prior to screening.
  14. Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study.
  15. Allergy or to any components of the study drug.
Terms in these criteria, in plain language(8)

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.

Contraception

Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.

Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.

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.

Clinically significant

Judged by the study doctor to matter for your health or for the study. It is a doctor’s decision, so ask the study team if you are unsure whether something counts.

Cancer (malignancy)

Malignancy means cancer. Criteria about another or earlier malignancy ask whether you have had a different cancer, usually within a set number of years.

Some are commonly allowed, such as treated skin cancers other than melanoma and early cancers removed completely — check the exceptions this study lists.

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.

CYP3A (CYP3A4) inhibitors and inducers

CYP3A is a group of enzymes, mainly CYP3A4, in the liver and gut that break down many medicines. Some medicines, supplements and foods (grapefruit, St John’s wort) slow them down or speed them up, which changes how much of a study drug stays in the body.

Studies list them so they can be avoided or switched; bring a list of everything you take, including supplements.

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 3
Primary purpose
Treatment
Allocation
Randomized
Intervention model
Parallel assignment
Masking
Quadruple (Participant, Care provider, Investigator, Outcomes assessor)
Enrollment
114 participants (actual)

Study arms

  • Experimental
    Infigratinib 0.25 mg/kg/day

    Infigratinib at 2, 3.5, 5, 7, 10 mg

    Drug: Infigratinib 0.25 mg/kg/day

  • Placebo comparator
    Placebo 0.25 mg/kg/day

    Placebo Comparator at 2, 3.5, 5, 7, 10 mg

    Drug: Placebo Comparator 0.25 mg/kg/day

Interventions

  • DrugInfigratinib 0.25 mg/kg/day

    Drug page: Infigratinib

    Daily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg

  • DrugPlacebo Comparator 0.25 mg/kg/day

    Daily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg

06

What researchers measure

Primary outcomes

  1. Change from baseline (BL) in annualized height velocity (cm/year)

    Time frame: Week 52

Secondary outcomes

  1. Change from BL in height Z-score (in relation to ACH tables)

    Time frame: Week 52

  2. Change from BL in upper to lower body segment ratio

    Time frame: Week 52

  3. Change from BL in height Z-score (in relation to non-ACH tables)

    Time frame: Week 52

  4. Annualized height velocity (cm/year)

    Time frame: Week 52

  5. Absolute and change from baseline in upper arm to forearm length ratio (cm)

    Time frame: Week 52

  6. Absolute and change from baseline in upper leg to lower leg length ratio (cm)

    Time frame: Week 52

  7. Absolute and change from baseline in arm span (cm) to standing height ratio

    Time frame: Week 52

  8. Absolute and change from baseline in head circumference (cm) to standing height ratio

    Time frame: Week 52

  9. Absolute value and change in body mass index

    Time frame: Week 52

  10. Incidence of adverse events

    Time frame: Week 52

  11. Change from BL in annualized height velocity (cm/year) in children 5 years old and older, compared to placebo

    Time frame: Week 52

  12. Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form

    Scale scores 0-100. Higher score=better Health-Related Quality of Life

    Time frame: Week 52

  13. Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test), compared to placebo

    Lower score=better performance. Range values=2-6

    Time frame: Week 52

  14. Change from BL in attention assessed by age-appropriate computerized tests (Identification Test)

    Lower score=better performance. Range values=2-6

    Time frame: Week 52

  15. Change from BL in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)

    Higher score=better performance. Range values=0-1.6

    Time frame: Week 52

  16. Change from BL in working memory assessed by age-appropriate computerized tests (One Back Test)

    Lower score=better performance. Range values=2-6

    Time frame: Week 52

  17. Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)

    Time frame: Week 52

  18. Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)

    Time frame: Week 52

  19. Change from BL in collagen X marker concentration (ug/L)

    Time frame: Week 52

  20. Evaluate the acceptability and palatability of infigratinib using a 5-point hedonic scale

    Time frame: Week 13

07

Study locations

27 sites
  • QED Investigative Site
    San Francisco, California 94609, United States
  • QED Investigative Site
    Aurora, Colorado 80045, United States
  • QED Investigative Site
    Baltimore, Maryland 21287, United States
  • QED Investigative Site
    Columbia, Missouri 65212, United States
  • QED Investigative Site
    Cincinnati, Ohio 45229, United States
  • QED Investigative Site
    Nashville, Tennessee 37232, United States
  • QED Investigative Site
    Madison, Wisconsin 53792, United States
  • QED Investigative Site
    Buenos Aires, Buenos Aires F.D. C1245AAM, Argentina
  • QED Investigative Site
    Parkville, Victoria 3052, Australia
  • QED Investigative Site
    Edmonton, Alberta T6G 2B7, Canada
  • QED Investigative Site
    London, Ontario N6A 5W9, Canada
  • QED Investigative Site
    Ottawa, Ontario K1H 8L1, Canada
  • QED Investigative Site
    Montreal, Quebec H3T 1C5, Canada
  • QED Investigative Site
    Bron, 69677, France
  • QED Investigative Site
    Paris, 75015, France
  • QED Investigative Site
    Toulouse, 31059, France
  • QED Investigative Site
    Rome, 00168, Italy
  • QED Investigative Site
    Bergen, 5009, Norway
  • QED Investigative Site
    Oslo, 0372, Norway
  • QED Investigative Site
    Singapore, 229899, Singapore
  • QED Investigative Site
    Málaga, 29010, Spain
  • QED Investigative Site
    Vitoria-Gasteiz, 01008, Spain
  • QED Investigative Site
    Bristol, BS2 8BJ, United Kingdom
  • QED Investigative Site
    Glasgow, G51 4TF, United Kingdom
  • QED Investigative Site
    London, SE1 7EH, United Kingdom
  • QED Investigative Site
    Manchester, M13 9WL, United Kingdom
  • QED Investigative Site
    Sheffield, S10 2TH, United Kingdom
08

References and documents

Publications

  • Savarirayan R, Hoover-Fong J, Irving M, Arundel P, de Bergua JM, Campeau PM, Candler T, Cocanougher BT, Cormier-Daire V, Edouard T, Fredwall SO, Harmatz P, Hoernschemeyer D, Irgens HU, Jamuar S, Kannu P, Legare JM, Leiva-Gea A, McDevitt H, Onesimo R, Phillips J 3rd, Del Pino M, Robinson ME, Rossi M, Skae M, Ward LM, White KK, Schmidt J, Lystig T, Salvatici A, Bai Y, Butler PW, van Veenhuyzen D, Rogoff D. Phase 3 Trial of Oral Infigratinib in Children with Achondroplasia. N Engl J Med. 2026 Jun 28. doi: 10.1056/NEJMoa2604565. Online ahead of print. PubMed 42370681 ↗

Individual participant data

Plan to share: No

09

Timeline

Tracking since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Ran from Nov 2023 to primary completion in Dec 2025, 25 months.

How these figures are worked out, and which studies are left out of them.

Registered
Nov 29, 2023
First posted
Dec 11, 2023
StartActual
Nov 10, 2023
Primary completionActual
Dec 18, 2025
CompletionActual
Dec 18, 2025
Last update
Mar 17, 2026
What these dates mean
Start.
When the first participant enrolled; while estimated, when the study expects to open to recruitment.
Primary completion.
When the last participant was examined or treated to collect data for the study's main question, its primary outcome. A study that stopped early gives the date its data collection stopped.
Completion.
When the last participant's last visit took place, for every outcome, secondary ones and side effects included: the end of the study. Often later than primary completion, sometimes the same day.
Actual and Est.
Actual means the registry says the date has happened. Est. (estimated) is a date the sponsor plans or expects. It can still move, and the sponsor is expected to change it to actual once it has passed.
No changes since tracking began. The registry record was last updated on Mar 17, 2026, before this site started recording changes on Sep 25, 2026. Its history is on ClinicalTrials.gov ↗
10

Registry details

Key details

Study ID
NCT06164951
Responsible party
Sponsor
First posted
Dec 11, 2023
Start date
Nov 10, 2023
Primary completion
Dec 18, 2025
Completion
Dec 18, 2025
Last update
Mar 17, 2026

Study contacts

QED Therapeutics, Inc. Medical Director, Clinical Development
study director · QED Therapeutics

Oversight

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

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Studies of the drug tested here: Infigratinib

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