Infigratinib for Achondroplasia

Enrolling by invitation at 31 trial locations

Overseen By QED Therapeutics SVP, Clinical Development

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: QED Therapeutics, Inc., a Bridgebio company

Must not be taking: CYP3A4 inducers/inhibitors, Growth hormone

Disqualifiers: Hypochondroplasia, Malignancy, Fracture, 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

What You Need to Know Before You Apply

What is the purpose of this trial?

This trial explores the long-term safety and effectiveness of infigratinib, a medication for treating achondroplasia, a condition marked by dwarfism due to abnormal bone growth. The study includes two groups: children who have previously used infigratinib in past trials and those who haven't tried it yet but have been monitored for growth in a related study. Ideal participants are children diagnosed with achondroplasia who can take oral medication and are willing to follow study procedures. The research also examines how the treatment might improve the quality of life for participants. As a Phase 2 trial, this research measures how well the treatment works in an initial, smaller group, offering participants a chance to contribute to important advancements in achondroplasia treatment.

Will I have to stop taking my current medications?

The trial does not specify if you must stop taking your current medications, but you cannot be on medications that strongly affect a specific liver enzyme (CYP3A4). If you are on such medications, you must stop them for a certain period before joining the trial.

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

Research has shown that infigratinib is safe for children with achondroplasia for up to 18 months. One study found that a daily dose of 0.25 mg/kg significantly helped children grow without causing serious safety issues. Although some side effects occurred, they rarely led to stopping the treatment. This suggests that infigratinib is generally safe for children with this condition. ^{1 2 3 4 5}

Why do researchers think this study treatment might be promising for achondroplasia?

Infigratinib is unique because it specifically targets the fibroblast growth factor receptor 3 (FGFR3), which plays a crucial role in bone growth. Unlike the current standard treatments for achondroplasia that often involve surgical interventions or growth hormone therapy, infigratinib offers a non-invasive option with a targeted approach. Researchers are excited about infigratinib because it has the potential to directly address the underlying genetic cause of achondroplasia, potentially improving growth outcomes in children with this condition.

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

Research has shown that infigratinib offers promising results for treating children with achondroplasia, a condition affecting bone growth. In earlier studies, children taking infigratinib grew faster, with most experiencing over a 25% increase in their growth rate compared to before treatment. Importantly, researchers reported no major safety issues, which is encouraging for its use in children. This trial includes two groups: one for treatment-naïve children and another for those who previously participated in a QED-sponsored interventional study with infigratinib. This suggests that infigratinib might improve growth and overall quality of life for those with achondroplasia. ^{1 2 3 5 6}

Who Is on the Research Team?

QED Therapeutics SVP, Clinical Development

Principal Investigator

QED Therapeutics

Are You a Good Fit for This Trial?

This trial is for children with Achondroplasia (ACH) aged 3 to <18 who have growth potential and completed a previous infigratinib study or have been assessed for growth over 6 months. They must be able to swallow pills, comply with the study plan, and use contraception if applicable. Those with recent fractures, other short stature conditions, or using certain drugs can't join.

Inclusion Criteria

I can take pills by mouth.

I agree to use effective birth control during and for 1 month after the study.

Pediatric subjects with ACH who have completed study activities in a previous QED-sponsored interventional study with infigratinib

Exclusion Criteria

I am not taking medication that strongly affects liver enzyme CYP3A4.

I have reached my full adult height.

I need to start a medication that is not allowed in the trial.

Timeline for a Trial Participant

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Growth Assessment

Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL)

6 months

Open-label Extension

Long-term safety, tolerability, and efficacy of infigratinib are evaluated in subjects with ACH

Long-term

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

What Are the Treatments Tested in This Trial?

Interventions

• Infigratinib

Trial Overview The trial tests the long-term safety and effectiveness of Infigratinib, an FGFR inhibitor, in kids who've taken it before or are new to it after a specific dose has been identified. It's an open-label extension study where all participants receive the drug and their quality of life is monitored.

How Is the Trial Designed?

Group I: Arm 2: Treatment naïve subjects Experimental Treatment 1 Intervention

Children naïve to infigratinib

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

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

Who Is Running the Clinical Trial?

QED Therapeutics, Inc., a Bridgebio company

Lead Sponsor

QED Therapeutics, a BridgeBio company

Lead Sponsor

QED Therapeutics, Inc.

Lead Sponsor

Trials

11

Recruited

1,200+

Published Research Related to This Trial

The pan-FGFR tyrosine kinase inhibitor NVP-BGJ398 effectively reduces FGFR3 hyperactivity, leading to improvements in bone growth and structure in a mouse model of achondroplasia after just 10 days of treatment.

NVP-BGJ398 not only corrects abnormalities in the growth plate and skeleton but also inhibits key signaling pathways associated with FGFR3, suggesting it could be a promising therapeutic option for treating achondroplasia.

NCBI (Pubmed)

pubmed.ncbi.nlm.nih.gov

Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model. Komla-Ebri, D., Dambroise, E., Kramer, I., et al.[2020]

Citations

1. pubmed.ncbi.nlm.nih.gov pubmed.ncbi.nlm.nih.gov/39555818/

Oral Infigratinib Therapy in Children with Achondroplasia Conclusions: The administration of oral infigratinib did not result in any apparent major safety signal and increased the annualized height ...

2. sciencedirect.com sciencedirect.com/science/article/pii/S2949774425001281

Oral infigratinib for children with achondroplasia: Month 18 ... Of 10/11 children with positive AHV changes from baseline at Month 18, 73% experienced a >25% AHV increase. Mean (SD) change from baseline in height z-score was ...

3. nejm.org nejm.org/doi/abs/10.1056/NEJMoa2411790

Oral Infigratinib Therapy in Children with Achondroplasia The primary efficacy outcome was the change from baseline in the annualized height velocity. Results. During treatment, all the children had at least one ...

4. bridgebio.com bridgebio.com/news/bridgebio-announces-first-child-dosed-with-infigratinib-in-propel-3-its-phase-3-clinical-trial-in-children-with-achondroplasia/

BridgeBio Announces First Child Dosed in PROPEL 3, its ... “The Phase 2 data for infigratinib has been very promising and suggests potential to increase growth, improve functionality and reduce the ...

5. clinicaltrials.gov clinicaltrials.gov/study/NCT06164951

NCT06164951 | A Study to Evaluate the Efficacy and ... This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to evaluate the efficacy and safety of infigratinib in children and ...

6. bridgebio.com bridgebio.com/news/bridgebio-pharma-announces-publication-in-the-new-england-journal-of-medicine-of-phase-2-propel-2-study-of-infigratinib-for-children-living-with-achondroplasia/

BridgeBio Pharma Announces Publication in the New ... Positive 18-month results from PROPEL 2, a Phase 2 trial of the investigational therapy infigratinib in children with achondroplasia, were published as an ...