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Enrolling by invitationNCT05145010Updated Oct 26, 2026

Extension Study of Infigratinib in Children With Achondroplasia (ACH)

A Phase 2 interventional study of Infigratinib and Infigratinib in Achondroplasia, sponsored by QED Therapeutics, a BridgeBio company. Enrolling by invitation at 31 sites in 10 countries. Open to participants aged 3 Years to 18 Years. Per ClinicalTrials.gov, last updated 2026-10-26.

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

Updated Oct 26, 2026Enrollment updatedEligibility revised+4 moreGo to Updates ↓
Phase
Phase 2
Study type
Interventional
Enrollment
325
Allocation
Non-randomized
Ages
3 Years to 18 Years
Sex
All
01

Study summary

This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in participants with ACH who previously completed a QED-sponsored interventional study, and in additional participants who are naïve to infigratinib treatment. Quality of Life assessments for this participant population will also be evaluated. Treatment-naïve participants must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study if the sponsor deems additional long-term safety and/or efficacy data are necessary, and participants are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.

02

Conditions studied

  • Achondroplasia

Keywords

  • Skeletal dysplasia
  • Endochondral ossification
  • Achondroplasia (ACH)
  • Quality of life in achondroplasia
  • Fibroblast growth factor receptor 3
  • FGFR3
  • Endochondral bone formation
  • Short-limb disproportionate stature
  • Bone disease
  • Functionality in achondroplasia
  • Musculoskeletal diseases
  • Osteochondrodysplasia
  • Genetic diseases
  • Long - term treatment
  • Growth
  • Annualized height velocity
  • Treatment option
  • Tyrosine kinase inhibitor
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 325 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.

Browse QED Therapeutics studies →All BridgeBio studies →

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

04

Who can participate

The criteria were revised on the registry on Oct 26, 2026. See what changed

Ages eligible
3 Years to 18 Years
Sexes eligible
All
Accepts healthy volunteers
No

Eligibility criteria

Key Rollover Participants Inclusion Criteria:

  1. Pediatric participants ≥3 years old with ACH who have completed a previous QED-sponsored interventional study with infigratinib.
  2. Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
  3. Participants are able to swallow oral medication.
  4. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
  5. If sexually active, participant must be willing to use a highly effective method of while taking study drug and for 1 month after the last dose of study drug.
  6. The PI, or a person designated by the PI, will obtain written from each participant's parent(s), legal guardian(s), or caregiver(s) and the participant's assent, when applicable, before any study-specific activity is performed.

Key Rollover Participants Exclusion Criteria:

  1. Participant has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.
  2. Participants who developed a medical condition that will require the initiation of treatment with a prohibited medication.
  3. Participants prematurely discontinued a prior QED-sponsored interventional study with infigratinib
  4. Current participation in an ongoing clinical study with a sponsor other than QED
  5. Participants that have reached final height or near final height.

Key Inclusion Criteria for Treatment Naïve Participants:

  1. Participants must be 3 to \<18 years of age at screening and have growth potential.
  2. Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
  3. Participants are able to swallow oral medication.
  4. Participants who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
  5. Participants from PROPEL Study (Protocol QBGJ398-001) only: Must have completed at least a 6-month period of growth assessment in PROPEL prior to study entry.
  6. Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
  7. If sexually active, participant must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
  8. The PI, or a person designated by the PI, will obtain written informed consent from each participant's parent(s), legal guardian(s), or caregiver(s) and the participant's assent, when applicable, before any study-specific activity is performed.

Key Exclusion Criteria for Treatment Naïve Participants:

  1. Participants who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature).
  2. Participants who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the participant or would interfere with study participation or safety evaluations.
  3. Participants who have a history of .
  4. Participants who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4.
  5. Participants who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible.
  6. Participants who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any or approved drug for the treatment of ACH in the previous 6 months.
  7. Participants who have significant abnormality in screening laboratory results.
  8. Participants who have had a fracture within 12 months of screening.
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.

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.

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.

05

Study design

Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomized
Intervention model
Single group
Masking
None (open label)
Enrollment
325 participants (estimated)

Study arms

  • Experimental
    Arm 1: Rollover participants

    Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)

    Drug: Infigratinib

  • Experimental
    Arm 2: Treatment naïve participants

    Children naïve to infigratinib

    Drug: Infigratinib

Interventions

  • DrugInfigratinib

    Infigratinib minitablets or sprinkle capsules to be administered by mouth. In participants that completed a prior study with infigratinib, the starting dose will be the same as the last dose received in the prior interventional study with infigratinib. Infigratinib dose may be adjusted to 0.25 mg/kg/day.

  • DrugInfigratinib

    Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the participants naïve to Infigratinib will be 0.25 mg/kg/day.

06

What researchers measure

Primary outcomes

  1. Incidence of treatment emergent adverse events (TEAE) and serious TEAE

    Time frame: 10 years

  2. Changes over time in height Z-score in relation to ACH and general pediatric population tables

    Time frame: 10 years

Secondary outcomes

  1. Changes over time in height velocity Z-score in relation to ACH and general pediatric population tables

    Time frame: 10 years

  2. Changes over time in body proportions

    Time frame: 10 years

  3. Changes over time in weight Z-score

    Time frame: 10 years

  4. Changes over time in BMI in relation to ACH and general pediatric population tables

    Time frame: 10 years

  5. Age of puberty onset and time to Tanner stage ≥4

    Time frame: 10 years

  6. Changes over time in number of episodes of otitis media per year

    Time frame: 10 years

  7. Changes over time in number of episodes and/or severity of sleep apnea

    Time frame: 10 years

  8. Changes over time in range of motion (elbow)

    Time frame: 10 years

  9. Changes over time in skeletal abnormalities of the lower extremities and spine

    Time frame: 10 years

  10. Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)

    Time frame: 10 years

  11. Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)

    Time frame: 10 years

  12. Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)

    Time frame: 10 years

  13. Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)

    Time frame: 10 years

  14. Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)

    Time frame: 10 years

  15. Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)

    Time frame: 10 years

  16. Participant and caregiver evaluation of treatment benefit as assessed by a qualitative interview

    Time frame: 10 years

  17. Changes in cognitive functions assessed by age-appropriate computerized tests

    Time frame: 10 years

07

Study locations

31 sites
08

References and documents

Individual participant data

Plan to share: No

No publications or documents are linked to this record.

09

Timeline

1 registry update since Sep 25, 2026
ElapsedEstimatedEstimated, not yet reached

Started Dec 2021 (actual). Primary completion is estimated for Dec 2031, 119 months after the start.

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

Registered
Nov 22, 2021
First posted
Dec 6, 2021
StartActual
Dec 6, 2021
Primary completionEst.
Dec 1, 2031
CompletionEst.
Feb 1, 2032
Last update
Oct 26, 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.
Sites
2 sites added, 2 sites removed
Show 2 added (1 United States, 1 Singapore)
  • UCSF Benioff Children's Hospital, Oakland · Oakland, United States
  • KK Women's and Children's Hospital · Singapore, Singapore
Show 2 removed
  • USCF Benioff Children's Hospital, Oakland · Oakland, United States
  • KK Women's and Children's Hosptial · Singapore, Singapore
Oct 26, 2026
Enrollment
300→325
Oct 26, 2026
Eligibility
28 criteria edited
What changed in the criteria
  • EditedKey Rollover SubjectsParticipants Inclusion Criteria:
  • EditedPediatric subjectsparticipants ≥3 years old with ACH who have completed a previous QED-sponsored interventional study with infigratinib.
  • EditedSubjectsParticipants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
  • EditedSubjectsParticipants are able to swallow oral medication.
  • EditedIf sexually active, subjectparticipant must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
  • EditedThe PI, or a person designated by the PI, will obtain written informed consent from each subjectparticipant's parent(s), legal guardian(s), or caregiver(s) and the subjectparticipant's assent, when applicable, before any study-specific activity is performed.
  • EditedKey Rollover SubjectsParticipants Exclusion Criteria:
  • EditedSubjectParticipant has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.
  • EditedSubjectsParticipants who developed a medical condition that will require the initiation of treatment with a prohibited medication.
  • EditedSubjectsParticipants prematurely discontinued a prior QED-sponsored interventional study with infigratinib
  • EditedSubjectsParticipants that have reached final height or near final height.
  • EditedKey Inclusion Criteria for Treatment Naïve SubjectsParticipants:
  • EditedSubjectParticipants must be 3 to <18 years of age at screening and have growth potential.
  • EditedSubjectsParticipants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
  • EditedSubjectsParticipants are able to swallow oral medication.
  • EditedSubjectsParticipants who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
  • EditedSubjectsParticipants from PROPEL Study (Protocol QBGJ398-001) only: Must have completed at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) beforeprior to study entry.
  • EditedIf sexually active, subjectparticipant must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
  • EditedThe PI, or a person designated by the PI, will obtain written informed consent from each subjectparticipant's parent(s), legal guardian(s), or caregiver(s) and the subjectparticipant's assent, when applicable, before any study-specific activity is performed.
  • EditedKey Exclusion Criteria for Treatment Naïve SubjectsParticipants:
  • EditedSubjectsParticipants who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature).
  • EditedSubjectsParticipants who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the subjectparticipant or would interfere with study participation or safety evaluations.
  • EditedSubjectsParticipants who have a history of malignancy.
  • EditedSubjectsParticipants who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4.
  • EditedSubjectsParticipants who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible.
  • EditedSubjectsParticipants who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months.
  • EditedSubjectsParticipants who have significant abnormality in screening laboratory results.
  • EditedSubjectsParticipants who have had a fracture within 12 months of screening.
Oct 26, 2026
Interventions
Arm changed Arm 1: Rollover subjects→Arm 1: Rollover participants; Arm 2: Treatment naïve subjects→Arm 2: Treatment naïve participants
Oct 26, 2026
Primary outcomes
1 edited
What changed in the outcomes
  • EditedChanges over time in height Z-score in relation to ACH and non-ACH growth chartsgeneral pediatric population tablesTime frame: 10 years
Oct 26, 2026
Secondary outcomes
4 edited
What changed in the outcomes
  • EditedChanges over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth chartsgeneral pediatric population tablesTime frame: 10 years
  • EditedChanges over time in weight zZ-scoreTime frame: 10 years
  • EditedSubjectParticipant and caregiver evaluation of treatment benefit as assessed by a qualitative interviewTime frame: 10 years
  • ChangedBefore Changes overtime in BMINow Changes over time in BMI in relation to ACH and general pediatric population tablesTime frame: 10 years
Oct 26, 2026
Show all 1 update
  1. Oct 26, 2026
    2 sites added, 2 sites removed
    Show 2 added (1 United States, 1 Singapore)
    • UCSF Benioff Children's Hospital, Oakland · Oakland, United States
    • KK Women's and Children's Hospital · Singapore, Singapore
    Show 2 removed
    • USCF Benioff Children's Hospital, Oakland · Oakland, United States
    • KK Women's and Children's Hosptial · Singapore, Singapore
    Enrollment 300→325
    Eligibility 28 criteria edited
    What changed in the criteria
    • EditedKey Rollover SubjectsParticipants Inclusion Criteria:
    • EditedPediatric subjectsparticipants ≥3 years old with ACH who have completed a previous QED-sponsored interventional study with infigratinib.
    • EditedSubjectsParticipants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
    • EditedSubjectsParticipants are able to swallow oral medication.
    • EditedIf sexually active, subjectparticipant must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
    • EditedThe PI, or a person designated by the PI, will obtain written informed consent from each subjectparticipant's parent(s), legal guardian(s), or caregiver(s) and the subjectparticipant's assent, when applicable, before any study-specific activity is performed.
    • EditedKey Rollover SubjectsParticipants Exclusion Criteria:
    • EditedSubjectParticipant has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.
    • EditedSubjectsParticipants who developed a medical condition that will require the initiation of treatment with a prohibited medication.
    • EditedSubjectsParticipants prematurely discontinued a prior QED-sponsored interventional study with infigratinib
    • EditedSubjectsParticipants that have reached final height or near final height.
    • EditedKey Inclusion Criteria for Treatment Naïve SubjectsParticipants:
    • EditedSubjectParticipants must be 3 to <18 years of age at screening and have growth potential.
    • EditedSubjectsParticipants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
    • EditedSubjectsParticipants are able to swallow oral medication.
    • EditedSubjectsParticipants who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
    • EditedSubjectsParticipants from PROPEL Study (Protocol QBGJ398-001) only: Must have completed at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) beforeprior to study entry.
    • EditedIf sexually active, subjectparticipant must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
    • EditedThe PI, or a person designated by the PI, will obtain written informed consent from each subjectparticipant's parent(s), legal guardian(s), or caregiver(s) and the subjectparticipant's assent, when applicable, before any study-specific activity is performed.
    • EditedKey Exclusion Criteria for Treatment Naïve SubjectsParticipants:
    • EditedSubjectsParticipants who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature).
    • EditedSubjectsParticipants who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the subjectparticipant or would interfere with study participation or safety evaluations.
    • EditedSubjectsParticipants who have a history of malignancy.
    • EditedSubjectsParticipants who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4.
    • EditedSubjectsParticipants who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible.
    • EditedSubjectsParticipants who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months.
    • EditedSubjectsParticipants who have significant abnormality in screening laboratory results.
    • EditedSubjectsParticipants who have had a fracture within 12 months of screening.
    Primary outcomes 1 edited
    What changed in the outcomes
    • EditedChanges over time in height Z-score in relation to ACH and non-ACH growth chartsgeneral pediatric population tablesTime frame: 10 years
    Secondary outcomes 4 edited
    What changed in the outcomes
    • EditedChanges over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth chartsgeneral pediatric population tablesTime frame: 10 years
    • EditedChanges over time in weight zZ-scoreTime frame: 10 years
    • EditedSubjectParticipant and caregiver evaluation of treatment benefit as assessed by a qualitative interviewTime frame: 10 years
    • ChangedBefore Changes overtime in BMINow Changes over time in BMI in relation to ACH and general pediatric population tablesTime frame: 10 years
    Interventions Arm changed Arm 1: Rollover subjects→Arm 1: Rollover participants; Arm 2: Treatment naïve subjects→Arm 2: Treatment naïve participants
    + 2 other changes: verification date and description

From the registry record's own update history. This site started tracking changes on Sep 25, 2026; for anything earlier, see the record history on ClinicalTrials.gov ↗

10

Registry details

Key details

Study ID
NCT05145010
Lead sponsor
QED Therapeutics, a BridgeBio company
Responsible party
Sponsor
First posted
Dec 6, 2021
Start date
Dec 6, 2021
Primary completion
Dec 1, 2031 (estimated)
Completion
Feb 1, 2032 (estimated)
Last update
Oct 26, 2026

Study contacts

QED Therapeutics SVP, 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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