AchondroplasiaPhase 3PROPEL3NCT06164951
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
A Phase 3 study testing Infigratinib 0.25 mg/kg/day in people with Achondroplasia, run by QED Therapeutics, a BridgeBio company at 27 sites in 10 countries.
Trial 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). Read more about this study
- Who is it for?
- Ages 3 to 17 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- Infigratinib 0.25 mg/kg/day
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 114 participants
- Where
- 27 sitesin 10 countries
- Study dates
- 10 Nov 2023to 18 Dec 2025
- Phase
- Phase 3
- Are there results?
- None posted in the registry
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 3 to 17 years
- All sexes
- No healthy volunteers
- Child
Who may be able to join
- 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
- Subjects who have a diagnosis of ACH that has been documented clinically and confirmed by genetic testing.
- Subjects must have completed at least 26 weeks in the PROPEL (QBGJ398-001) study before screening.
- Subjects are able to swallow oral medication.
- Subjects and parent(s), legal guardian(s), or caregivers are willing and able to comply with study visits and study procedures.
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- Subjects are ambulatory and able to stand without assistance.
- Sex and Contraceptive/Barrier Requirements
- Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
- 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.
- Informed Consent
- 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)
Who cannot join
- Medical Conditions
- Subjects who have hypochondroplasia or short stature condition other than ACH.
- Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
- Current evidence of clinically significant corneal or retinal disorder/keratopathy -confirmed by ophthalmic examination.
- 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.
- History and/or current evidence of extensive ectopic tissue calcification.
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- History of malignancy.
- Prior/Concomitant Therapy
- Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH or short stature.
- Regular long-term treatment (≥3 weeks) with supraphysiologic doses of glucocorticoid 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).
- 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.
- Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 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).
- Current evidence of endocrine alterations of calcium/phosphorus homeostasis.
- Diagnostic assessments
- Subjects who have significant abnormality in screening laboratory results.
- Other Exclusions
- Having had a fracture of the long bones (ie, extremities) or spine within 12 months prior to screening.
- Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study.
- Allergy or hypersensitivity to any components of the study drug.
Trial locations and contacts
27 sites in 10 countries.
27 sites in 10 countries
United States7 sites
QED Investigative Site
Aurora, Colorado, 80045
QED Investigative Site
Baltimore, Maryland, 21287
QED Investigative Site
Cincinnati, Ohio, 45229
QED Investigative Site
Columbia, Missouri, 65212
QED Investigative Site
Madison, Wisconsin, 53792
QED Investigative Site
Nashville, Tennessee, 37232
QED Investigative Site
San Francisco, California, 94609
United Kingdom5 sites
QED Investigative Site
Bristol, BS2 8BJ
QED Investigative Site
Glasgow, G51 4TF
QED Investigative Site
London, SE1 7EH
QED Investigative Site
Manchester, M13 9WL
QED Investigative Site
Sheffield, S10 2TH
Canada4 sites
QED Investigative Site
Edmonton, Alberta, T6G 2B7
QED Investigative Site
London, Ontario, N6A 5W9
QED Investigative Site
Montreal, Quebec, H3T 1C5
QED Investigative Site
Ottawa, Ontario, K1H 8L1
France3 sites
QED Investigative Site
Bron, 69677
QED Investigative Site
Paris, 75015
QED Investigative Site
Toulouse, 31059
Norway2 sites
QED Investigative Site
Bergen, 5009
QED Investigative Site
Oslo, 0372
Spain2 sites
QED Investigative Site
Málaga, 29010
QED Investigative Site
Vitoria-Gasteiz, 01008
Argentina1 site
QED Investigative Site
Buenos Aires, Buenos Aires F.D., C1245AAM
About this study
Official title: A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age With Achondroplasia: PROPEL 3
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).
- Conditions
- Achondroplasia
- Being tested
- Infigratinib 0.25 mg/kg/day, Placebo Comparator 0.25 mg/kg/day
- 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
- Medical subject headings
- Achondroplasia, Mucopolysaccharidosis IV, Dwarfism, Bone Diseases, Musculoskeletal Diseases, Osteochondrodysplasias, Genetic Diseases, Inborn
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Infigratinib 0.25 mg/kg/dayDrug
Daily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
Placebo Comparator 0.25 mg/kg/dayDrug
Daily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
| Study group | Role | What they receive |
|---|---|---|
| Infigratinib 0.25 mg/kg/day | Experimental | Infigratinib at 2, 3.5, 5, 7, 10 mg Receives: Drug: Infigratinib 0.25 mg/kg/day |
| Placebo 0.25 mg/kg/day | Placebo comparator | Placebo Comparator at 2, 3.5, 5, 7, 10 mg Receives: Drug: Placebo Comparator 0.25 mg/kg/day |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change from baseline (BL) in annualized height velocity (cm/year)
Measured: Week 52
Secondary outcomes (20)
Change from BL in height Z-score (in relation to ACH tables)
Measured: Week 52
Change from BL in upper to lower body segment ratio
Measured: Week 52
Change from BL in height Z-score (in relation to non-ACH tables)
Measured: Week 52
Annualized height velocity (cm/year)
Measured: Week 52
Absolute and change from baseline in upper arm to forearm length ratio (cm)
Measured: Week 52
Absolute and change from baseline in upper leg to lower leg length ratio (cm)
Measured: Week 52
Absolute and change from baseline in arm span (cm) to standing height ratio
Measured: Week 52
Absolute and change from baseline in head circumference (cm) to standing height ratio
Measured: Week 52
Absolute value and change in body mass index
Measured: Week 52
Incidence of adverse events
Measured: Week 52
Change from BL in annualized height velocity (cm/year) in children 5 years old and older, compared to placebo
Measured: Week 52
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
Measured: Week 52
Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test), compared to placebo
Lower score=better performance. Range values=2-6
Measured: Week 52
Change from BL in attention assessed by age-appropriate computerized tests (Identification Test)
Lower score=better performance. Range values=2-6
Measured: Week 52
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
Measured: Week 52
Change from BL in working memory assessed by age-appropriate computerized tests (One Back Test)
Lower score=better performance. Range values=2-6
Measured: Week 52
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Measured: Week 52
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Measured: Week 52
Change from BL in collagen X marker concentration (ug/L)
Measured: Week 52
Evaluate the acceptability and palatability of infigratinib using a 5-point hedonic scale
Measured: Week 13
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted11 Dec 2023
- Study start10 Nov 2023
- Primary completion18 Dec 2025
- Study completion18 Dec 2025
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 114 participants (actual)
Sponsor and investigators
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- QED Therapeutics, Inc. Medical Director, Clinical Development, Study director, QED Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06164951
- Sponsor's protocol ID
- QBGJ398-303
- Acronym
- PROPEL3
- Status last verified
- Mar 2026
- First submitted
- 29 Nov 2023
- Last updated
- 17 Mar 2026
- Results posted
- No
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
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
Study at a glance
From the registry record, last updated by the sponsor on 17 Mar 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age With Achondroplasia: PROPEL 3
- ClinicalTrials.gov ID
- NCT06164951
- Acronym / sponsor ID
- PROPEL3 / QBGJ398-303
- Conditions
- 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
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 114 participants (actual)
- Lead sponsor
- QED Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Study start
- 10 Nov 2023 (actual)
- Primary completion
- 18 Dec 2025 (actual)
- Study completion
- 18 Dec 2025 (actual)
- First posted
- 11 Dec 2023 (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 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).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Infigratinib 0.25 mg/kg/day | Experimental | Drug: Infigratinib 0.25 mg/kg/dayInfigratinib at 2, 3.5, 5, 7, 10 mg |
| Placebo 0.25 mg/kg/day | Placebo comparator | Drug: Placebo Comparator 0.25 mg/kg/dayPlacebo Comparator at 2, 3.5, 5, 7, 10 mg |
Who may qualify
- Ages
- 3 to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
Key inclusion criteria (12 listed)
- 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
- Subjects who have a diagnosis of ACH that has been documented clinically and confirmed by genetic testing.
- Subjects must have completed at least 26 weeks in the PROPEL (QBGJ398-001) study before screening.
- Subjects are able to swallow oral medication.
Key exclusion criteria (19 listed)
- Medical Conditions
- Subjects who have hypochondroplasia or short stature condition other than ACH.
- Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
- Current evidence of clinically significant corneal or retinal disorder/keratopathy -confirmed by ophthalmic examination.
- 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.
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 0.25 mg/kg/dayDaily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg | Drug | Infigratinib 0.25 mg/kg/day |
| Placebo Comparator 0.25 mg/kg/dayDaily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg | Drug | Placebo 0.25 mg/kg/day |
Primary outcome
Change from baseline (BL) in annualized height velocity (cm/year)
Time frame: Week 52
20 secondary outcomes
Change from BL in height Z-score (in relation to ACH tables)
Time frame: Week 52
Change from BL in upper to lower body segment ratio
Time frame: Week 52
Change from BL in height Z-score (in relation to non-ACH tables)
Time frame: Week 52
Annualized height velocity (cm/year)
Time frame: Week 52
Absolute and change from baseline in upper arm to forearm length ratio (cm)
Time frame: Week 52
Absolute and change from baseline in upper leg to lower leg length ratio (cm)
Time frame: Week 52
Absolute and change from baseline in arm span (cm) to standing height ratio
Time frame: Week 52
Absolute and change from baseline in head circumference (cm) to standing height ratio
Time frame: Week 52
Absolute value and change in body mass index
Time frame: Week 52
Incidence of adverse events
Time frame: Week 52
Change from BL in annualized height velocity (cm/year) in children 5 years old and older, compared to placebo
Time frame: Week 52
Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form
Time frame: Week 52
Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test), compared to placebo
Time frame: Week 52
Change from BL in attention assessed by age-appropriate computerized tests (Identification Test)
Time frame: Week 52
Change from BL in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)
Time frame: Week 52
Change from BL in working memory assessed by age-appropriate computerized tests (One Back Test)
Time frame: Week 52
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Time frame: Week 52
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Time frame: Week 52
Change from BL in collagen X marker concentration (ug/L)
Time frame: Week 52
Evaluate the acceptability and palatability of infigratinib using a 5-point hedonic scale
Time frame: Week 13
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 17 Mar 2026.
- Derived from this study: PubMed 42370681Savarirayan 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.
- Individual participant dataSharing planned: No
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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. This study is not recruiting at the moment.
QED Therapeutics, Inc. Medical Director, Clinical Development study director
QED Therapeutics
27 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Infigratinib 0.25 mg/kg/day in people with Achondroplasia, run by QED Therapeutics, a BridgeBio company at 27 sites in 10 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 12 inclusion and 19 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: finished, as of the sponsor's last update on 17 Mar 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 27 sites in United States, United Kingdom, Canada, France, Norway and 5 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change from baseline (BL) in annualized height velocity (cm/year) (time frame: Week 52). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT06164951, which also lists 1 linked publication.
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 17 Mar 2026.