EUCTR2019-002954-21-GBActive, not recruitingPhase 1
Phase 2, Open-Label, Dose-Escalation and Dose-Expansion Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children with Achondroplasia: PROPEL 2 - PROPEL 2
Conditions
Trial Snapshot
- Phase
- Phase 1
- Status
- Active, not recruiting
- Sponsor
- QED Therapeutics, Inc.
- Enrollment
- 78
Study Overview
Brief Summary
No summary available.
Study Design
- Study Type
- Interventional clinical trial of medicinal product
Eligibility Criteria
- Sex
- All
Inclusion Criteria
- •- Signed informed consent by subject or parent(s) or legally authorized representative (LAR) and signed informed assent by the subject (when applicable).
- •- 3 to 11 years of age (inclusive) at screening. The PK Substudy will only enroll subjects =8 years old.
- •- Diagnosis of ACH, documented clinically and confirmed by genetic testing.
- •- If a girl =10 years of age, negative pregnancy test.
- •-.If sexually active, willing to use a highly effective method of contraception while taking study drug and for 3 months after the last dose of study drug.
- •- Subjects and parent(s) or LAR are willing and able to comply with study visits and study procedures.
- •- Able to swallow oral medication.
- •- Willing to stop consumption of grapefruit, grapefruit juice, grapefruit hybrids, pomegranates, star fruits, pomelos, Seville oranges, or products containing juice of these fruits while participating in the study; and have not consumed these within 7 days before the first dose of study drug.
- •Are the trial subjects under 18? yes
- •Number of subjects for this age range: 78
- •F.1.2 Adults (18-64 years) no
- •F.1.2.1 Number of subjects for this age range
- •F.1.3 Elderly (>=65 years) no
- •F.1.3.1 Number of subjects for this age range
Exclusion Criteria
- •1. Hypochondroplasia or short stature condition other than ACH (eg, trisomy 21, pseudoachondroplasia, psychosocial short stature).
- •2. In females, having had their menarche.
- •3. Height < -2 or > +2 standard deviations for age and sex based on reference tables on growth in children with ACH (Horton 1978).
- •4. Annualized height velocity =1.5 cm/year over a period =6 months prior to screening.
- •5. Significant concurrent disease or condition that, in the view of the Investigator and/or Sponsor, would confound assessment of efficacy or safety of infigratinib, including but not limited to cardiac or vascular disease; hyperthyroidism; abnormal thyroid levels or recently diagnosed hypothyroidism that has not been stable on therapy for at least 3 months; insulin-requiring diabetes mellitus; adrenal insufficiency; autoimmune inflammatory disease; inflammatory bowel disease; presence of a functioning ventriculoperitoneal shunt; or diagnosis of severe sleep apnea (pre existing or done at screening based on the sleep
- •study) requiring surgery or continuous positive airway pressure (CPAP) machine.
- •6. Significant abnormality in screening laboratory results, including but not limited to the following:
- •a. Hemoglobin <10.0 g/dL.
- •b. Total bilirubin >1.5× upper limit of normal (ULN).
- •c. AST/SGOT or ALT/SGPT >2× ULN.
- •d. Calculated or measured creatinine clearance of <60 mL/min.
- •7. Current evidence of corneal or retinal disorder/keratopathy including, but not limited to, bullous/band keratopathy, corneal abrasion, inflammation/ulceration, or keratoconjunctivitis, confirmed by ophthalmic examination.
- •8. History and/or current evidence of extensive ectopic tissue calcification.
- •9. History of malignancy.
- •10. Currently receiving treatment with agents that are known strong inducers or inhibitors of CYP3A4 and medications which increase serum phosphorus and/or calcium concentration. Subjects are not permitted to receive vitamin D analogues for the treatment of vitamin D deficiency or to initiate multivitamin supplementation containing any form of vitamin D (Over-the-counter [OTC] multivitamin supplements containing vitamin D that were initiated =3 months before screening are allowed at the same dose). Subjects receiving medications that alter the pH of the gastrointestinal tract, including antacids, H2 antagonists (eg, ranitidine), and proton-pump inhibitors (eg, omeprazole); or enzyme-inducing antiepileptic drugs, including carbamazepine, phenytoin, phenobarbital, and primidone will be excluded.
- •11. Current evidence of endocrine alterations of calcium/phosphorus homeostasis:
- •a. Inorganic phosphorus outside of normal limits.
- •b. Total serum calcium (can be corrected) outside of normal limits.
- •12. Allergy to any components of the study drug.
- •13. Treatment with growth hormone, insulin-like growth factor 1 (IGF1), or anabolic steroids in the previous 6 months or long-term treatment (>3 months) at any time.
- •14. Treatment with a C-type natriuretic peptide (CNP) analog, fibroblast growth factor (FGF) ligand trap, or treatment targeting FGFR inhibition at any time.
- •15. Regular long-term treatment (=3 weeks) with supraphysiologic doses of glucocorticoid therapy (ie, >15 mg/m2/day of hydrocortisone or equivalence) or treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within 6 months of the screening visit (low-dose ongoing inhaled steroid for asthma is acceptable).
- •16. Treatment with any other investigational product or investigatio
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