Study of Infigratinib in Children With Achondroplasia (NCT04265651) | Clinical Trial Compass
CompletedPhase 2
Study of Infigratinib in Children With Achondroplasia
United States84 participantsStarted 2020-03-10
Plain-language 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. The study also includes a PK Substudy to fully characterize the pharmacokinetics of infigratinib in children with ACH.
Who can participate
Age range3 Years – 11 Years
SexALL
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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.
What they're measuring
1
Incidence of treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation
Timeframe: Up to 18 months
2
Change from baseline in annualized height velocity
Timeframe: Up to 18 months
3
PK parameters of infigratinib (Cmax- PK substudy only)
Timeframe: 21 days
4
PK parameters of infigratinib (Clast- PK substudy only)
Timeframe: 21 days
5
PK parameters of infigratinib (Tmax- PK substudy only)
Timeframe: 21 days
6
PK parameters of infigratinib (AUC24- PK substudy only)
Timeframe: 21 days
7
PK parameters of infigratinib (T1/2- PK substudy only)
Timeframe: 21 days
8
PK parameters of infigratinib (AUCinf- PK substudy only)
. 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.
PK parameters of infigratinib (CL/F- PK substudy only)
Timeframe: 21 days
10
PK parameters of infigratinib (Vz/F- PK substudy only)
Timeframe: 21 days
11
PK parameters of infigratinib (Racc- PK substudy only)