Study of Infigratinib in Children With Achondroplasia

  • STATUS
    Recruiting
  • End date
    Feb 11, 2023
  • participants needed
    60
  • sponsor
    QED Therapeutics, Inc.
Updated on 11 August 2021
tyrosine kinase inhibitor

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.

Details
Condition Dwarfism, Achondroplasia, Idiopathic Short Stature, Severe Short Stature
Treatment Infigratinib 0.016 mg/kg, Infigratinib 0.032 mg/kg, Infigratinib 0.064 mg/kg, Infigratinib 0.128 mg/kg
Clinical Study IdentifierNCT04265651
SponsorQED Therapeutics, Inc.
Last Modified on11 August 2021

Eligibility

Yes No Not Sure

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
In females, having had their menarche
Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH
Significant concurrent disease or condition that, in the view of the Investigator and/or Sponsor, would confound assessment of efficacy or safety of infigratinib
Current evidence of corneal or retinal disorder/keratopathy
History of malignancy
Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 and medications which increase serum phosphorus and/or calcium concentration
Treatment with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time
Treatment with a C-type natriuretic peptide (CNP) analog, fibroblast growth factor (FGF) ligand trap, or treatment targeting FGFR inhibition at any time
Regular long-term treatment (>3 weeks) with oral corticosteroids (low-dose ongoing inhaled steroid for asthma is acceptable)
Treatment with any other investigational product or investigational medical device for the treatment of ACH or short stature
Previous limb-lengthening surgery or guided growth surgery
Fracture within 6 months of screening
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