AchondroplasiaPhase 2NCT05145010
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
A Phase 2 study testing Infigratinib in people with Achondroplasia, run by QED Therapeutics, a BridgeBio company at 31 sites in 10 countries.
Trial 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… Read more about this study
- Who is it for?
- Ages 3 to 18 years
- Conditions the study is for
- Achondroplasia
- What is being tested?
- Infigratinib
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 325 participants
- Where
- 31 sitesin 10 countries
- Study dates
- 6 Dec 2021to 1 Feb 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 3 to 18 years
- All sexes
- No healthy volunteers
- Child, Adult
Who may be able to join
- Participants must be 3 to <18 years of age at screening and have growth potential.
- Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
- Participants are able to swallow oral medication.
- Participants who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
- 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.
- Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
- 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.
- 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.
Who cannot join
- Participants who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature).
- 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.
- Participants who have a history of malignancy.
- Participants who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4.
- Participants who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible.
- Participants 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.
- Participants who have significant abnormality in screening laboratory results.
- Participants who have had a fracture within 12 months of screening.
Trial locations and contacts
31 sites in 10 countries. Contact the study team or a site near you to ask about taking part.
31 sites in 10 countries
United States8 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Johns Hopkins University
Baltimore, Maryland, 21205
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
University of Missouri
Columbia, Missouri, 65212
University Hospital and UW Health Clinics
Madison, Wisconsin, 53705
Vanderbilt University Medical Center
Nashville, Tennessee, 37232
UCSF Benioff Children's Hospital, Oakland
Oakland, California, 94609
Nemours Alfred I. Dupont Hospital for Children
Wilmington, Delaware, 19803
United Kingdom6 sites
Birmingham Women's and Children's NHS Foundation Trust
Birmingham, B5 6NH
University Hospitals Bristol and Weston NHS Foundation Trust
Bristol, BS2 8AE
Queen Elizabeth University Hospital
Glasgow, G12 0XH
St. Thomas' Hospital
London, SE1 7EH
Manchester University Children's Hospital
Manchester, M13 9WL
Sheffield Children's Hospital
Sheffield, S10 2TH
Canada4 sites
Stollery Children's Hospital
Edmonton, Alberta, T6C 2B7
Children's Hospital - London Health Sciences Centre
London, Ontario, N6A 5W9
Centre Hospitalier Universitaire Sainte-Justine
Montreal, Quebec, H3T 1C5
Children's Hospital of Eastern Ontario
Ottawa, Ontario, K1H 8L1
France3 sites
Hopital Femme Mere Enfant
Lyon, 69500
Hopital Necker-Enfants Malades
Paris, 75743
Hopital des Enfants
Toulouse, 31300
Spain3 sites
Hospital Universitario La Paz
Madrid, 28046
Hospital Universitario Virgen de la Victoria
Málaga, 29010
Hospital Vithas San José
Vitoria-Gasteiz, Álava, 01012
Italy2 sites
Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico
Milan, 20122
Policlinico A. Gemelli IRCCS
Roma, 00168
Norway2 sites
Haukeland University Hospital
Bergen, 5021
Oslo University Hospital
Oslo, 0372
Argentina1 site
Hospital de Pediatría SAMIC Prof. Dr. Juan P. Garrahan
Ciudad Autonoma Buenos Aires, Buenos Aires, C1245AAM
About this study
Official title: Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL OLE
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.
- Conditions
- Achondroplasia
- Being tested
- Infigratinib
- 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
- Medical subject headings
- Achondroplasia, Mucopolysaccharidosis IV, 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.
InfigratinibDrug
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.
InfigratinibDrug
Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the participants naïve to Infigratinib will be 0.25 mg/kg/day.
| Study group | Role | What they receive |
|---|---|---|
| Arm 1: Rollover participants | Experimental | Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3) Receives: Drug: Infigratinib |
| Arm 2: Treatment naïve participants | Experimental | Children naïve to infigratinib Receives: Drug: Infigratinib |
What is being measured
The primary outcome is the main question the study is designed to answer.
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Measured: 10 years
Changes over time in height Z-score in relation to ACH and general pediatric population tables
Measured: 10 years
Secondary outcomes (17)
Changes over time in height velocity Z-score in relation to ACH and general pediatric population tables
Measured: 10 years
Changes over time in body proportions
Measured: 10 years
Changes over time in weight Z-score
Measured: 10 years
Changes over time in BMI in relation to ACH and general pediatric population tables
Measured: 10 years
Age of puberty onset and time to Tanner stage ≥4
Measured: 10 years
Changes over time in number of episodes of otitis media per year
Measured: 10 years
Changes over time in number of episodes and/or severity of sleep apnea
Measured: 10 years
Changes over time in range of motion (elbow)
Measured: 10 years
Changes over time in skeletal abnormalities of the lower extremities and spine
Measured: 10 years
Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)
Measured: 10 years
Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)
Measured: 10 years
Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)
Measured: 10 years
Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)
Measured: 10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)
Measured: 10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)
Measured: 10 years
Participant and caregiver evaluation of treatment benefit as assessed by a qualitative interview
Measured: 10 years
Changes in cognitive functions assessed by age-appropriate computerized tests
Measured: 10 years
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted6 Dec 2021
- Study start6 Dec 2021
- Primary completion1 Dec 2031 (estimated)
- Study completion1 Feb 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
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 325 participants (estimated)
Sponsor and investigators
- Lead sponsor
- QED Therapeutics, a BridgeBio company
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- QED Therapeutics SVP, Clinical Development, Study director, QED Therapeutics
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05145010
- Sponsor's protocol ID
- QBGJ398-203
- Status last verified
- Oct 2026
- First submitted
- 22 Nov 2021
- Last updated
- 26 Oct 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 26 Oct 2026. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL OLE
- ClinicalTrials.gov ID
- NCT05145010
- Acronym / sponsor ID
- QBGJ398-203
- Conditions
- 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
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Enrolling by invitation
- Enrolment
- 325 participants (estimated)
- Lead sponsor
- QED Therapeutics, a BridgeBio companyIndustry (pharma, biotech, devices)
- Study start
- 6 Dec 2021 (actual)
- Primary completion
- 1 Dec 2031 (estimated)
- Study completion
- 1 Feb 2032 (estimated)
- First posted
- 6 Dec 2021 (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, 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.
Study design
- Primary purpose
- Treatment
- Allocation
- Non-randomised
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Arm 1: Rollover participants | Experimental | Drug: InfigratinibChildren who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3) |
| Arm 2: Treatment naïve participants | Experimental | Drug: InfigratinibChildren naïve to infigratinib |
Who may qualify
- Ages
- 3 to 18 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult
Key inclusion criteria (8 listed)
- Participants must be 3 to <18 years of age at screening and have growth potential.
- Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
- Participants are able to swallow oral medication.
- Participants who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.
- 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.
Key exclusion criteria (8 listed)
- Participants who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature).
- 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.
- Participants who have a history of malignancy.
- Participants who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4.
- Participants who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible.
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 |
|---|---|---|
| InfigratinibInfigratinib 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. | Drug | Arm 1: Rollover participants |
| InfigratinibInfigratinib sprinkle capsules to be administered by mouth. Starting dose for the participants naïve to Infigratinib will be 0.25 mg/kg/day. | Drug | Arm 2: Treatment naïve participants |
Primary outcome
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Time frame: 10 years
Changes over time in height Z-score in relation to ACH and general pediatric population tables
Time frame: 10 years
17 secondary outcomes
Changes over time in height velocity Z-score in relation to ACH and general pediatric population tables
Time frame: 10 years
Changes over time in body proportions
Time frame: 10 years
Changes over time in weight Z-score
Time frame: 10 years
Changes over time in BMI in relation to ACH and general pediatric population tables
Time frame: 10 years
Age of puberty onset and time to Tanner stage ≥4
Time frame: 10 years
Changes over time in number of episodes of otitis media per year
Time frame: 10 years
Changes over time in number of episodes and/or severity of sleep apnea
Time frame: 10 years
Changes over time in range of motion (elbow)
Time frame: 10 years
Changes over time in skeletal abnormalities of the lower extremities and spine
Time frame: 10 years
Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)
Time frame: 10 years
Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)
Time frame: 10 years
Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)
Time frame: 10 years
Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)
Time frame: 10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)
Time frame: 10 years
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)
Time frame: 10 years
Participant and caregiver evaluation of treatment benefit as assessed by a qualitative interview
Time frame: 10 years
Changes in cognitive functions assessed by age-appropriate computerized tests
Time frame: 10 years
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 26 Oct 2026.
- 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.
QED Therapeutics SVP, Clinical Development study director
QED Therapeutics
31 sites in 10 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Infigratinib in people with Achondroplasia, run by QED Therapeutics, a BridgeBio company at 31 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 8 inclusion and 8 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: enrolling by invitation, as of the sponsor's last update on 26 Oct 2026. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 31 sites in United States, United Kingdom, Canada, France, Spain 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. 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?
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?
Incidence of treatment emergent adverse events (TEAE) and serious TEAE (time frame: 10 years). The study lists 2 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 26 Oct 2026; AzTrials last refreshed its copy on 26 Oct 2026.