Achondroplasia in Children MedDRA version: 20.0 Level: LLT Classification code 10000452 Term: Achondroplasia System Organ Class: 100000004850
Conditions
Sponsors
QED Therapeutics, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
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. - 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. - Subjects and parent(s) or LAR are willing and able to comply with study visits and study procedures. - Able to swallow oral medication. Are the trial subjects under 18? yes Number of subjects for this age range: 60 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
Exclusion criteria: -Hypochondroplasia or short stature condition other than 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, including but not limited to cardiac or vascular disease; severe sleep apnea, among others. -Evidence of corneal or retinal disorder. -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 (including vitamin D analogues); medications that alter the pH of the gastrointestinal tract, including antacids, H2 antagonists (eg, ranitidine), and proton-pump inhibitors (eg, omeprazole); or enzyme-inducing anti-epileptic drugs, including carbamazepine, phenytoin, phenobarbital, and primidone. - Current evidence of endocrine alterations of calcium/phosphorus homeostasis - 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 any other investigational product or investigational medical device for the treatment of ACH or short stature.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To identify a dose of oral infigratinib, based on safety and efficacy evaluations, for children with achondroplasia (ACH) to be used for further study;Secondary Objective: To evaluate the safety and tolerability of oral infigratinib in children with ACH To evaluate changes from baseline in anthropometric parameters after administration of oral infigratinib To evaluate the pharmacokinetic and pharmacodynamic (PK/PD) profile of infigratinib in children with ACH after administration of oral infigratinib To evaluate changes in ACH disease burden;Primary end point(s): - Treatment-emergent adverse events (TEAEs) that lead to dose decrease or discontinuation. - Change from baseline in height velocity (annualized to cm/year). (Baseline is defined as the annualized height velocity obtained from a minimum of 6 months of observation in the PROPEL study.) ;Timepoint(s) of evaluation of this end point: The evaluation is scheduled at 6, and 18 months for dose escalation, and 12 months for dose expansion. | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Safety evaluations by incidence, type, severity, and causality of adverse events (AEs), serious adverse events (SAEs), laboratory test results (urinalysis, chemistry, hematology), clinically significant changes in vital signs, physical examination (including ophthalmic and dental evaluation), electrocardiograms, and imaging. - Absolute height velocity (annualized to cm/year), expressed numerically and as Z-score in relation to non-ACH tables. - Absolute (expressed as absolute value and Z-score in relation to ACH and non-ACH standardized pediatric growth curves) and change from baseline in anthropometric parameters, including body proportions. - PK parameters (eg, Cmax and tmax) - Changes in PD parameters (biomarkers of bone turnover that may include type X collagen degradation fragment, collagen X marker [CXM]). - Changes in disease-specific complications, such as changes in mobility (assessed by elbow, hip, and knee range of motion), changes in the number of episodes of otitis media per year, changes in number of episodes and/or severity of sleep apnea, and changes in quality of life [QoL] as assessed by PedsQL (Exploratory endpoint);Timepoint(s) of evaluation of this end point: The evaluation is scheduled at 6, and 18 months for dose escalation, and 12 months for dose expansion. For the complete list please refers to the protocol | — |
Countries
Australia, Canada, France, Spain, United Kingdom, United States
Contacts
Public ContactDaniela Rogoff
QED Therapeutics
Outcome results
None listed