A Phase 2 interventional study of Infigratinib 0.016 mg/kg and Infigratinib 0.032 mg/kg in Achondroplasia, sponsored by QED Therapeutics, a BridgeBio company. Completed at 19 sites in 6 countries. Open to participants aged 3 Years to 11 Years. Per ClinicalTrials.gov, last updated 2025-10-22.
Sponsored by QED Therapeutics, a BridgeBio company (part of BridgeBio) · Phase 2, Interventional, and Treatment
This is a Phase 2, multicenter, open-label, dose-escalation and dose-expansion study to evaluate the safety, tolerability, and efficacy of infigratinib, a fibroblast growth factor receptor (FGFR) 1-3-selective tyrosine kinase inhibitor, in children 3 to 11 years of age with Achondroplasia (ACH) who previously participated in the PROPEL study (Protocol QBGJ398-001) for at least 6 months. The study includes dose escalation with extended treatment, and dose expansion. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.
47 studies on the registry are indexed under Achondroplasia; 13 are open to participants now.
This study's enrollment of 84 is above the median of 74 across 28 interventional studies indexed under Achondroplasia.
Browse Achondroplasia studies →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.
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.
Exclusion Criteria:
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.
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.
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.
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.
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.
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.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
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.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Dose Escalation: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Drug: Infigratinib 0.016 mg/kg
Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Drug: Infigratinib 0.032 mg/kg
Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Drug: Infigratinib 0.064 mg/kg
Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months.
Drug: Infigratinib 0.128 mg/kg
Dose Escalation and PK substudy: Infigratinib is provided as minitablets in 2 strengths: 0.1 mg and 1 mg for daily oral administration. The dose and number of minitablets/day will be calculated based on individual participant weight. Doses will be adjusted based on weight changes approximately every 3 months. Dose Expansion: Upon identification of the recommended dose from all cohorts analyzed, an expansion cohort of 20 subjects may begin enrollment to further determine safety, tolerability, efficacy, pharmacokinetics (PK) and pharmacodynamics (PD) of the selected dose.
Drug: Infigratinib 0.25 mg/kg
Drug page: Infigratinib
Initial cohort dose of infigratinib at the protocol-specified starting dose, with subsequent cohort escalations based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Drug page: Infigratinib
Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Drug page: Infigratinib
Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Drug page: Infigratinib
Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Drug page: Infigratinib
Subsequent cohort dose escalation based on protocol-specific criteria. Infigratinib tablets to be administered by mouth.
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Time frame: Up to 18 months
Change from baseline in annualized height velocity
Time frame: Up to 18 months
PK parameters of infigratinib (Cmax- PK substudy only)
Time frame: 21 days
PK parameters of infigratinib (Clast- PK substudy only)
Time frame: 21 days
PK parameters of infigratinib (Tmax- PK substudy only)
Time frame: 21 days
PK parameters of infigratinib (AUC24- PK substudy only)
Time frame: 21 days
PK parameters of infigratinib (T1/2- PK substudy only)
Time frame: 21 days
PK parameters of infigratinib (AUCinf- PK substudy only)
Time frame: 21 days
PK parameters of infigratinib (CL/F- PK substudy only)
Time frame: 21 days
PK parameters of infigratinib (Vz/F- PK substudy only)
Time frame: 21 days
PK parameters of infigratinib (Racc- PK substudy only)
Time frame: 21 days
Incidence of adverse events (AEs) and serious adverse events (SAEs) as a measure of safety and tolerability
Time frame: Up to 18 months
Absolute height velocity (annualized to cm/year), expressed numerically and as Z-score in relation to ACH and non-ACH tables
Time frame: Up to 18 months
Absolute and change from baseline in weight (kg)
Time frame: Up to 18 months
Absolute and change from baseline in sitting height (cm)
Time frame: Up to 18 months
Absolute and change from baseline in head circumference (cm)
Time frame: Up to 18 months
Absolute and change from baseline in upper and lower arm length (cm)
Time frame: Up to 18 months
Absolute and change from baseline in thigh length (cm)
Time frame: Up to 18 months
Absolute and change from baseline in knee height (cm)
Time frame: Up to 18 months
Absolute and change from baseline in arm span (cm)
Time frame: Up to 18 months
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Time frame: Up to 18 months
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Time frame: Up to 18 months
Changes in pharmacodynamic parameters by assessing collagen X marker
Time frame: Up to 18 months
This study is completed, as verified in Oct 2025. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Infigratinib
QED Therapeutics, a BridgeBio company →