What side effects are associated with this type of intervention?

FGFR inhibitors, such as Infigratinib, commonly cause side effects including diarrhea, rash, and decreased appetite. These adverse events are frequently reported and can vary in severity. Other potential side effects may include fatigue, dry skin, and elevated liver enzymes. It is important for patients to discuss these potential side effects with their healthcare provider to manage them effectively.

What prior FDA approvals exist?

As of now, there have been limited FDA approvals specifically for the treatment of Achondroplasia. The focus has been on novel therapeutics targeting the fibroblast growth factor receptor (FGFR) pathway, similar to Infigratinib. One notable FDA-approved treatment is Vosoritide, a C-type natriuretic peptide analog, which addresses the underlying genetic cause of Achondroplasia by targeting the FGFR3 pathway. Infigratinib, currently under investigation, represents another promising approach by selectively inhibiting FGFR 1-3, potentially offering a new therapeutic option for children with Achondroplasia.

What other types of research exist?

Promising alternatives to Infigratinib for Achondroplasia patients include Vosoritide, a C-type natriuretic peptide (CNP) analog that has shown positive results in increasing growth velocity in children with Achondroplasia. Additionally, TransCon CNP, a long-acting prodrug of CNP, is under investigation and may offer another effective treatment option. Gene therapy approaches targeting the FGFR3 mutation are also being explored and could provide future therapeutic alternatives.

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Tyrosine Kinase Inhibitor

Infigratinib for Achondroplasia

Phase 2

Waitlist Available

Research Sponsored by QED Therapeutics, Inc.

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Ambulatory and able to stand without assistance

Diagnosis of ACH, documented clinically and confirmed by genetic testing.

Must not have

Hypochondroplasia or short stature condition other than ACH.

History of malignancy.

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is testing a medicine called infigratinib in children aged 3 to 11 years with Achondroplasia. The medicine works by blocking proteins that cause abnormal bone growth. The children have already participated in an earlier study.

Who is the study for?

This trial is for children aged 3-11 with Achondroplasia who were part of the PROPEL study. They must be able to swallow pills, stand unassisted, and not have started puberty. Kids can't join if they've had certain treatments like growth hormones recently or have eye disorders, a history of cancer, or been on long-term steroids.

What is being tested?

The trial tests different doses of Infigratinib (a drug that targets specific growth receptors) in kids with Achondroplasia to see which dose is safe and works best. It's an open-label study where everyone knows what treatment they're getting, involving both increasing the dose over time and expanding it to more patients.

What are the potential side effects?

Infigratinib may cause side effects such as liver issues, high phosphate levels in blood leading to muscle problems or rash, finger or toe pain/swelling due to increased blood flow there; also possible are gastrointestinal symptoms like diarrhea.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

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I can walk and stand up on my own without help.

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I have been diagnosed with ACH through clinical assessment and genetic testing.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

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I have a condition causing short stature, but it's not achondroplasia.

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I have had cancer before.

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I have never been treated with CNP analogs, FGF ligand traps, or FGFR inhibitors.

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I have started my menstrual periods.

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I am on medication that affects liver enzymes or changes calcium/phosphorus levels.

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My height is much shorter or taller than average for my age and sex.

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I have a current eye condition affecting my cornea or retina.

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I have had surgery to lengthen my limbs or correct their growth.

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I haven't taken growth hormone, IGF-1, or steroids in the last 6 months.

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I have not used any experimental drugs or devices for ACH or growth issues.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

5Treatment groups

Experimental Treatment

Group I: Infigratinib 0.25 mg/kgExperimental Treatment1 Intervention

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.

Group II: Infigratinib 0.128 mg/kgExperimental Treatment1 Intervention

Group III: Infigratinib 0.064 mg/kgExperimental Treatment1 Intervention

Group IV: Infigratinib 0.032 mg/kgExperimental Treatment1 Intervention

Group V: Infigratinib 0.016 mg/kgExperimental Treatment1 Intervention

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.

0 / 12Eligibility criteria met

Research Highlights

Information in this section is not a recommendation. We encourage patients to speak with their healthcare team when evaluating any treatment decision.

Mechanism Of Action

Side Effect Profile

Prior Approvals

Other Research

Achondroplasia is caused by mutations in the FGFR3 gene, leading to abnormal bone growth. FGFR inhibitors, such as infigratinib, target and inhibit FGFR1-3 tyrosine kinases, which helps normalize the overactive signaling pathways caused by the FGFR3 mutation. This targeted approach can potentially improve bone growth and reduce complications, directly addressing the genetic cause of Achondroplasia.