Infigratinib for Hypochondroplasia

Enrolling by invitation at 24 trial locations

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: QED Therapeutics, a BridgeBio company

Must not be taking: Prohibited medications

Disqualifiers: Final height, Other clinical study, others

No Placebo Group All trial participants will receive the active study treatment (no placebo)

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: Unknown

Duration: Long-term

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial tests a treatment called infigratinib for children with Hypochondroplasia, a condition that affects bone growth. The main goal is to assess the long-term safety and effectiveness of this treatment. It targets children who have already participated in a previous study with infigratinib and have been monitored for growth. A suitable candidate would be a child with Hypochondroplasia who has participated in the ACCEL study and has not yet reached their final height. As a Phase 2 trial, this research measures the treatment's effectiveness in an initial, smaller group, providing an opportunity to contribute to important findings.

Do I need to stop my current medications for the trial?

The trial does not specify if you need to stop your current medications, but it mentions that participants who need to start a prohibited medication cannot join. It's best to discuss your current medications with the trial team.

Is there any evidence suggesting that infigratinib is likely to be safe for humans?

Research shows that infigratinib, a drug targeting specific proteins, has undergone safety testing in people. Studies have found it can improve bone growth in conditions like achondroplasia, which is somewhat similar to hypochondroplasia. In these studies, the main safety concern was whether side effects would lead participants to lower their dose or stop taking the drug. So far, these side effects appear manageable, with no major issues reported that led to stopping the treatment entirely.

Researchers are also studying infigratinib over a longer period to ensure its continued safety. This ongoing research helps build confidence in its safety, but discussing any concerns with the trial team or a healthcare provider is always advisable. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising?

Infigratinib is unique because it targets the fibroblast growth factor receptor (FGFR), specifically FGFR3, which plays a crucial role in bone development. Unlike current treatments for hypochondroplasia, which primarily focus on managing symptoms, infigratinib works at a molecular level to address the underlying cause of abnormal bone growth. Researchers are excited about this treatment because it has the potential to improve bone growth and overall health outcomes for children with hypochondroplasia, offering a more targeted and effective approach than symptomatic management alone.

What evidence suggests that infigratinib might be an effective treatment for Hypochondroplasia?

Research has shown that infigratinib may help treat hypochondroplasia, a condition affecting bone growth. In studies using cells and mice with hypochondroplasia, infigratinib significantly improved bone growth, suggesting it might also aid human bone growth. Although most research has focused on achondroplasia, a similar condition, these findings offer hope for treating hypochondroplasia. Studies on related conditions have demonstrated improvements in body shape and growth rates, indicating potential benefits for hypochondroplasia. ^{1 2 5 6 7}

Who Is on the Research Team?

QED Therapeutics SVP, Clinical Development

Principal Investigator

QED Therapeutics, a BridgeBio company

Are You a Good Fit for This Trial?

This trial is for children with Hypochondroplasia who finished the ACCEL studies. It's open to those who've been assessed for growth over at least 6 months in these studies. Girls aged ≥10 or those who have started menstruating must test negative for pregnancy to join.

Inclusion Criteria

Pediatric participants with HCH who have completed ACCEL 2/3

I am not pregnant or I am a girl under 10 who hasn't had her period.

Exclusion Criteria

Current participation in an ongoing clinical study with a sponsor other than QED

Participant has concurrent medical condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations

Participants who prematurely discontinued ACCEL 2/3

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment

Participants receive infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, to evaluate long-term safety, tolerability, and efficacy

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

6 months

Open-label extension

Participants continue to receive infigratinib as part of the long-term extension study

Long-term

What Are the Treatments Tested in This Trial?

Interventions

• Infigratinib

Trial Overview

The study tests Infigratinib, a drug targeting specific enzymes (FGFR1-3) involved in bone development and growth. This Phase 2 trial aims to understand its long-term safety and effectiveness in kids with Hypochondroplasia after previous trials.

How Is the Trial Designed?

Group I: Arm 1: Rollover subjects Experimental Treatment 1 Intervention

Children who have completed QED-sponsored interventional study with infigratinib

Who Is Running the Clinical Trial?

QED Therapeutics, a BridgeBio company

Lead Sponsor

Citations

1.

pubmed.ncbi.nlm.nih.gov

pubmed.ncbi.nlm.nih.gov/40581757/

Infigratinib low dose therapy is an effective strategy to treat ...

Infigratinib showed potent inhibitory effect in cellular model and significantly improved skeletal growth in a mouse model of hypochondroplasia.

2.

investor.bridgebio.com

investor.bridgebio.com/news/news-details/2026/BridgeBio-Reports-Positive-Phase-3-Topline-Results-for-Oral-Infigratinib-with-the-First-Statistically-Significant-Improvements-in-Body-Proportionality-in-Achondroplasia/

News Details

PROPEL 3 successfully met the primary endpoint of change from baseline in AHV at Week 52 (p<0.0001). - Change from baseline in AHV was ...

3.

nejm.org

nejm.org/doi/abs/10.1056/NEJMoa2411790

Oral Infigratinib Therapy in Children with Achondroplasia

The primary safety outcome was the incidence of adverse events that led to a decrease in the dose or discontinuation of infigratinib. The primary efficacy ...

4.

clinicaltrials.gov

clinicaltrials.gov/study/NCT06873035

NCT06873035 - Infigratinib in Hypochondroplasia Study

The purpose of the Phase 2 portion of the study (ACCEL2/3) is to evaluate the efficacy and safety, of infigratinib in children with hypochondroplasia (HCH) ...

5.

fundacionalpe.org

fundacionalpe.org/media/Biblioteca/Investigacion_cientifica/BridgeBio%20Announces%20Durable%20Month%2012%20and%2018%20Phase%202%20Cohort%205%20Results%20of%20Oral%20Infigratinib%20in%20Achondroplasia.pdf

BridgeBio Announces Durable Month 12 and 18 Phase 2 ...

BridgeBio is committed to exploring the potential of infigratinib on wider medical and functional impacts of achondroplasia, hypochondroplasia ...

6.

pmc.ncbi.nlm.nih.gov

pmc.ncbi.nlm.nih.gov/articles/PMC12578284/

Infigratinib low dose therapy is an effective strategy to treat ...

Results from a phase 2 study support the concept that infigratinib has a potential to improve bone growth in achondroplasia. We report results of a step-wise ...

7.

academic.oup.com

academic.oup.com/jbmr/advance-article/doi/10.1093/jbmr/zjag018/8442825

Response to letter to the editor for “Infigratinib low dose ...

The objective of our recently published study was to evaluate the therapeutic potential of infigratinib for hypochondroplasia (HCH) using models ...