Extension Study of Infigratinib in Children With Achondroplasia (ACH)
a study on Achondroplasia FGFR Gene Alteration
Summary
- Eligibility
- for people ages 3-18 (full criteria)
- Location
- at Oakland, California and other locations
- Dates
- study startedcompletion around
Description
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.
Official Title
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
Keywords
Achondroplasia, Skeletal dysplasia, Endochondral ossification, Achondroplasia (ACH), Quality of life in achondroplasia, Fibroblast growth factor receptor 3, FGFR3, Endochondral bone formation, Short-limb disproportionate stature, Bone disease, Functionality in achondroplasia, Musculoskeletal diseases, Osteochondrodysplasia, Genetic diseases, Long - term treatment, Growth, Annualized height velocity, Treatment option, Tyrosine kinase inhibitor, Infigratinib
Eligibility
For people ages 3-18
Key Rollover Subjects 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.
- Subjects have at least a 6-month period of growth assessment in the PROPEL study (Protocol QBGJ398 001) before study entry.
- 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 Exclusion Criteria for Treatment Naïve Subjects
- Subjects who have hypochondroplasia or short stature condition other than ACH (e.g., trisomy 21, pseudoachondroplasia, psychosocial short stature).
- Subjects who have significant concurrent disease or condition that, in the view of the PI and/or sponsor, would represent an increased risk to the subject or would interfere with study participation or safety evaluations.
- Subjects who have a history of malignancy.
- Subjects who are currently receiving treatment with agents that are known strong inducers or inhibitors of cytochrome P450 (CYP) 3A4.
- Subjects who discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible.
- Subjects who have received treatment with growth hormone, insulin-like growth factor 1 (IGF 1), anabolic steroids or any investigational or approved drug for the treatment of ACH in the previous 6 months.
- Subjects who have significant abnormality in screening laboratory results.
- Subjects who have had a fracture within 12 months of screening.
Locations
- USCF Benioff Children's Hospital, Oakland
Oakland California 94609 United States - Stollery Children's Hospital
Edmonton Alberta T6C 2B7 Canada
Details
- Status
- accepting new patients by invitation only
- Start Date
- Completion Date
- (estimated)
- Sponsor
- QED Therapeutics, Inc.
- ID
- NCT05145010
- Phase
- Phase 2 research study
- Study Type
- Interventional
- Participants
- Expecting 300 study participants
- Last Updated