Phase 2, Open-Label, Long-Term, Extension (OLE) Study of Infigratinib, an FGFR 1-3-Selective Tyrosine Kinase Inhibitor, in Children With Achondroplasia: PROPEL OLE

QED Therapeutics, a BridgeBio company31 sites in 10 countries300 target enrollmentDecember 6, 2021

ConditionsAchondroplasia

InterventionsInfigratinib

DrugsInfigratinib

Overview

Phase: Phase 2
Intervention: Infigratinib
Conditions: Achondroplasia
Sponsor: QED Therapeutics, a BridgeBio company
Enrollment: 300
Locations: 31
Primary Endpoint: Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Status: Enrolling By Invitation
Last Updated: 6 months ago

Overview Investigators Eligibility Criteria Arms & Interventions Outcomes Sites Similar Trials

Overview

Brief Summary

This is a Phase 2, multicenter, open-label, extension (OLE) study to evaluate the long-term safety, tolerability, and efficacy of infigratinib, an FGFR 1-3-selective tyrosine kinase inhibitor, in subjects with ACH who previously completed a QED-sponsored interventional study, and potentially in additional subjects who are naïve to infigratinib treatment. Quality of Life assessments for this subject population will also be evaluated. Treatment-naïve subjects must have at least a 6-month period of growth assessment in study QBGJ398-001 (PROPEL) and will be enrolled in this OLE study only after a dose to be explored further is identified in Phase 2 Study QBGJ398-201 and subjects are not otherwise eligible to enroll in another QED-sponsored Phase 2 or Phase 3 ACH study.

Registry

clinicaltrials.gov

Start Date

December 6, 2021

End Date

February 1, 2032

Last Updated

6 months ago

Study Type

Interventional

Study Design

Single Group

Sex

All

Investigators

Sponsor

QED Therapeutics, a BridgeBio company

Responsible Party

Sponsor

Eligibility Criteria

Inclusion Criteria

•Pediatric subjects with ACH who have completed a previous QED-sponsored interventional study with infigratinib.
•Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
•Subjects are able to swallow oral medication.
•Negative pregnancy test in girls ≥10 years of age or girls of any age who have experienced menarche.
•If sexually active, subject must be willing to use a highly effective method of contraception while taking study drug and for 1 month after the last dose of study drug.
•The PI, or a person designated by the PI, will obtain written informed consent from each subject's parent(s), legal guardian(s), or caregiver(s) and the subject's assent, when applicable, before any study-specific activity is performed.
•Key Rollover Subjects

Exclusion Criteria

•Subject has concurrent circumstance, disease, or condition that, in the view of the PI and/or sponsor, would interfere with study participation or safety evaluations.
•Subjects who developed a medical condition that will require the initiation of treatment with a prohibited medication.
•Subjects prematurely discontinued a prior QED-sponsored interventional study with infigratinib
•Current participation in an ongoing clinical study with a sponsor other than QED
•Subjects that have reached final height or near final height.
•Key Inclusion Criteria for Treatment Naïve Subjects
•Subject must be 3 to \<18 years of age at screening and have growth potential.
•Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
•Subjects are able to swallow oral medication.
•Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic testing.

Arms & Interventions

Arm 1: Rollover subjects

Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)

Intervention: Infigratinib

Arm 2: Treatment naïve subjects

Children naïve to infigratinib

Intervention: Infigratinib

Outcomes

Primary Outcomes

Incidence of treatment emergent adverse events (TEAE) and serious TEAE

Time Frame: 10 years

Changes over time in height Z-score in relation to ACH and non-ACH growth charts

Time Frame: 10 years

Secondary Outcomes

Changes over time in range of motion (elbow)(10 years)
Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview(10 years)
Changes over time in weight z-score(10 years)
Age of puberty onset and time to Tanner stage ≥4(10 years)
Changes over time in number of episodes of otitis media per year(10 years)
Changes over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth charts(10 years)
Changes over time in body proportions(10 years)
Changes overtime in BMI(10 years)
Changes over time in number of episodes and/or severity of sleep apnea(10 years)
Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)(10 years)
Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)(10 years)
Changes in cognitive functions assessed by age-appropriate computerized tests(10 years)
Changes over time in skeletal abnormalities of the lower extremities and spine(10 years)
Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)(10 years)
Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)(10 years)
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)(10 years)
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)(10 years)