Infigratinib for Achondroplasia

Recruiting at 23 trial locations

Overseen By QED Therapeutics SVP, Clinical Development

Age: < 65

Sex: Any

Trial Phase: Phase 2

Sponsor: QED Therapeutics, Inc.

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

300 Participants Needed

Trial Summary

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.

What data supports the effectiveness of the drug Infigratinib for treating achondroplasia?

Research shows that Infigratinib, a drug that targets specific growth factor receptors, has improved bone growth in animal models of achondroplasia, suggesting it may help counteract the effects of the condition in humans. ^{1 2 3 4 5}

How does the drug Infigratinib work for achondroplasia?

Infigratinib is unique because it directly targets the overactive FGFR3 gene, which is responsible for achondroplasia, by inhibiting its activity. Unlike other treatments that mainly address symptoms, Infigratinib aims to counteract the root cause of the condition. ^{2 3 4 6 7}

What is the purpose of this trial?

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 subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.

Research Team

QED Therapeutics SVP, Clinical Development

Principal Investigator

QED Therapeutics

Eligibility Criteria

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

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

Treatment Details

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.

Participant Groups

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.

Lead Sponsor

Trials

11

Recruited

1,200+

Findings from Research

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]

References

1. England pubmed.ncbi.nlm.nih.gov

Medical complications in children with achondroplasia. [2022]

2. United States pubmed.ncbi.nlm.nih.gov

Tyrosine kinase inhibitor NVP-BGJ398 functionally improves FGFR3-related dwarfism in mouse model. [2020]

3. England pubmed.ncbi.nlm.nih.gov

Infigratinib in children with achondroplasia: the PROPEL and PROPEL 2 studies. [2022]

4. United States pubmed.ncbi.nlm.nih.gov

Infigratinib, a selective FGFR1-3 tyrosine kinase inhibitor, alters dentoalveolar development at high doses. [2023]

5. United States pubmed.ncbi.nlm.nih.gov

A height-for-age growth reference for children with achondroplasia: Expanded applications and comparison with original reference data. [2020]

6. United States pubmed.ncbi.nlm.nih.gov

In vitro and in vivo characterization of Recifercept, a soluble fibroblast growth factor receptor 3, as treatment for achondroplasia. [2021]

7. United States pubmed.ncbi.nlm.nih.gov

Evaluation of the therapeutic potential of a CNP analog in a Fgfr3 mouse model recapitulating achondroplasia. [2021]