CompletedPhase 2

Study of Infigratinib in Children With Achondroplasia

United States, Australia, Canada84 participantsStarted 2020-03-10

Plain-language summary

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.

Who can participate

Age range

3 Years – 11 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable).
. Diagnosis of ACH, documented clinically and confirmed by genetic testing.
. At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.
. Ambulatory and able to stand without assistance
. Able to swallow oral medication.

Exclusion criteria

. Hypochondroplasia or short stature condition other than ACH.

Questions worth asking your doctor

Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.

1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?

Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.

Questions for the trial coordinator

The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.

1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?

A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.

What they're measuring

Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation

Timeframe: Up to 18 months

Change from baseline in annualized height velocity

Timeframe: Up to 18 months

PK parameters of infigratinib (Cmax- PK substudy only)

Timeframe: 21 days

PK parameters of infigratinib (Clast- PK substudy only)

Timeframe: 21 days

PK parameters of infigratinib (Tmax- PK substudy only)

Timeframe: 21 days

PK parameters of infigratinib (AUC24- PK substudy only)

Timeframe: 21 days

PK parameters of infigratinib (T1/2- PK substudy only)

Timeframe: 21 days

Trial details

NCT IDNCT04265651

SponsorQED Therapeutics, a BridgeBio company

Sponsor typeINDUSTRY

Study typeINTERVENTIONAL

Primary completion2024-10-21

View on ClinicalTrials.gov More Achondroplasia trials

Plain-language summary

Who can participate

Age range

3 Years – 11 Years

Sex

ALL

See this in plain English?

AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.

Inclusion criteria

. Signed informed consent by participant or parent(s) or legally authorized representative (LAR) and signed informed assent by the participant (when applicable).
. Diagnosis of ACH, documented clinically and confirmed by genetic testing.
. At least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398-001) before study entry.
. Ambulatory and able to stand without assistance
. Able to swallow oral medication.

Exclusion criteria

. Hypochondroplasia or short stature condition other than ACH.

What they're measuring

Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation

Timeframe: Up to 18 months

Change from baseline in annualized height velocity

Timeframe: Up to 18 months

PK parameters of infigratinib (Cmax- PK substudy only)

Timeframe: 21 days

PK parameters of infigratinib (Clast- PK substudy only)

Timeframe: 21 days

PK parameters of infigratinib (Tmax- PK substudy only)

Timeframe: 21 days

PK parameters of infigratinib (AUC24- PK substudy only)

Timeframe: 21 days

PK parameters of infigratinib (T1/2- PK substudy only)

Timeframe: 21 days