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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT05145010
Registration number
NCT05145010
Ethics application status
Date submitted
22/11/2021
Date registered
6/12/2021
Date last updated
22/05/2024
Titles & IDs
Public title
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
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Scientific 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
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Secondary ID [1]
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QBGJ398-203
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Universal Trial Number (UTN)
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Trial acronym
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Linked study record
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Health condition
Health condition(s) or problem(s) studied:
Achondroplasia
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Condition category
Condition code
Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Musculoskeletal
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Other muscular and skeletal disorders
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Infigratinib
Treatment: Drugs - Infigratinib
Experimental: Arm 1: Rollover subjects - Children who have completed QED-sponsored interventional study with infigratinib (Phase 2 or Phase 3)
Experimental: Arm 2: Treatment naïve subjects - Children naïve to infigratinib
Treatment: Drugs: Infigratinib
Infigratinib minitablets or sprinkle capsules to be administered by mouth. In subjects that completed a prior study with infigratinib, the starting dose will be the same as the last dose received in the prior interventional study with infigratinib. Infigratinib dose may be adjusted to the dose identified from the dose escalation portion of the Phase 2 study (PROPEL 2).
Treatment: Drugs: Infigratinib
Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the subjects naive to infigratinib will be the one identified during QBGJ398-201 (PROPEL 2).
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Intervention code [1]
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Treatment: Drugs
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Comparator / control treatment
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Control group
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Outcomes
Primary outcome [1]
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Incidence of treatment emergent adverse events (TEAE) and serious TEAE
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Assessment method [1]
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Timepoint [1]
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10 years
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Primary outcome [2]
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Changes over time in height Z-score in relation to ACH and non-ACH growth charts
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Assessment method [2]
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Timepoint [2]
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10 years
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Secondary outcome [1]
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Changes over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth charts
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Assessment method [1]
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Timepoint [1]
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10 years
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Secondary outcome [2]
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Changes over time in body proportions
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Assessment method [2]
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Timepoint [2]
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10 years
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Secondary outcome [3]
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Changes over time in weight z-score
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Assessment method [3]
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Timepoint [3]
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10 years
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Secondary outcome [4]
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Changes overtime in BMI
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Assessment method [4]
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Timepoint [4]
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10 years
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Secondary outcome [5]
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Age of puberty onset and time to Tanner stage =4
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Assessment method [5]
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Timepoint [5]
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10 years
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Secondary outcome [6]
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Changes over time in number of episodes of otitis media per year
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Assessment method [6]
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Timepoint [6]
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10 years
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Secondary outcome [7]
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Changes over time in number of episodes and/or severity of sleep apnea
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Assessment method [7]
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Timepoint [7]
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10 years
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Secondary outcome [8]
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Changes over time in range of motion (elbow)
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Assessment method [8]
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Timepoint [8]
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10 years
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Secondary outcome [9]
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Changes over time in skeletal abnormalities of the lower extremities and spine
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Assessment method [9]
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Timepoint [9]
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10 years
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Secondary outcome [10]
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Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)
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Assessment method [10]
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Timepoint [10]
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10 years
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Secondary outcome [11]
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Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)
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Assessment method [11]
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Timepoint [11]
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10 years
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Secondary outcome [12]
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Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)
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Assessment method [12]
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Timepoint [12]
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10 years
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Secondary outcome [13]
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Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)
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Assessment method [13]
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Timepoint [13]
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10 years
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Secondary outcome [14]
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Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)
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Assessment method [14]
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Timepoint [14]
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10 years
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Secondary outcome [15]
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Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)
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Assessment method [15]
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Timepoint [15]
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10 years
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Secondary outcome [16]
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Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview
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Assessment method [16]
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Timepoint [16]
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10 years
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Secondary outcome [17]
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Changes in cognitive functions assessed by age-appropriate computerized tests
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Assessment method [17]
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Timepoint [17]
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10 years
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Eligibility
Key inclusion criteria
Key Rollover Subjects
1. Pediatric subjects with ACH who have completed a previous QED-sponsored interventional
study with infigratinib.
2. Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to
comply with study visits and study procedures.
3. Subjects are able to swallow oral medication.
4. Negative pregnancy test in girls =10 years of age or girls of any age who have
experienced menarche.
5. 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.
6. 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
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Minimum age
3
Years
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Maximum age
18
Years
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Sex
Both males and females
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Can healthy volunteers participate?
No
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Key exclusion criteria
1. 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.
2. Subjects who developed a medical condition that will require the initiation of
treatment with a prohibited medication.
3. Subjects prematurely discontinued a prior QED-sponsored interventional study with
infigratinib
4. Subjects that have reached final height or near final height.
Key Inclusion Criteria for Treatment Naïve Subjects
1. Subject must be 3 to <18 years of age at screening and have growth potential.
2. Subjects and parent(s), legal guardian(s), or caregiver(s) are willing and able to
comply with study visits and study procedures.
3. Subjects are able to swallow oral medication.
4. Subjects who have a diagnosis of ACH, documented clinically and confirmed by genetic
testing.
5. Subjects have at least a 6-month period of growth assessment in the PROPEL study
(Protocol QBGJ398 001) before study entry.
6. Negative pregnancy test in girls =10 years of age or girls of any age who have
experienced menarche.
7. 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.
8. 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
1. Subjects who have hypochondroplasia or short stature condition other than ACH (e.g.,
trisomy 21, pseudoachondroplasia, psychosocial short stature).
2. 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.
3. Subjects who have a history of malignancy.
4. Subjects who are currently receiving treatment with agents that are known strong
inducers or inhibitors of cytochrome P450 (CYP) 3A4.
5. 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.
6. Subjects who have significant abnormality in screening laboratory results.
7. Subjects who have had a fracture within 12 months of screening.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Non-randomised trial
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Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
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Methods used to generate the sequence in which subjects will be randomised (sequence generation)
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Masking / blinding
Open (masking not used)
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Who is / are masked / blinded?
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Intervention assignment
Single group
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Other design features
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Phase
Phase 2
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Type of endpoint/s
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Statistical methods / analysis
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Recruitment
Recruitment status
Recruiting
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Data analysis
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Reason for early stopping/withdrawal
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Other reasons
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Date of first participant enrolment
Anticipated
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Actual
6/12/2021
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Date of last participant enrolment
Anticipated
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Actual
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Date of last data collection
Anticipated
1/02/2032
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Actual
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Sample size
Target
280
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Accrual to date
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Final
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Recruitment in Australia
Recruitment state(s)
VIC
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Recruitment hospital [1]
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Murdoch Children's Hospital - Parkville
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Recruitment postcode(s) [1]
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3052 - Parkville
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Recruitment outside Australia
Country [1]
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United States of America
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State/province [1]
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California
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Country [2]
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United States of America
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State/province [2]
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Delaware
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Country [3]
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United States of America
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State/province [3]
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Ohio
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Country [4]
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United States of America
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State/province [4]
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Tennessee
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Country [5]
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Canada
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State/province [5]
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Alberta
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Country [6]
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France
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State/province [6]
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Lyon
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Country [7]
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France
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State/province [7]
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Paris
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Country [8]
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France
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State/province [8]
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Toulouse
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Country [9]
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Spain
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State/province [9]
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Madrid
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Country [10]
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Spain
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State/province [10]
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Málaga
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Country [11]
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Spain
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State/province [11]
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Álava
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Country [12]
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United Kingdom
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State/province [12]
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Birmingham
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Country [13]
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United Kingdom
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State/province [13]
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Glasgow
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Country [14]
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United Kingdom
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State/province [14]
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London
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Country [15]
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United Kingdom
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State/province [15]
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Manchester
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Country [16]
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United Kingdom
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State/province [16]
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Sheffield
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Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
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Name
QED Therapeutics, Inc.
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Address
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Country
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Ethics approval
Ethics application status
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Summary
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 the
PROPEL study (Protocol QBGJ398 001) and will be enrolled in this OLE study only after a dose
to be explored further is identified in Phase 2 Study QBGJ398-201.
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Trial website
https://clinicaltrials.gov/ct2/show/NCT05145010
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Trial related presentations / publications
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Public notes
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Contacts
Principal investigator
Name
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QED Therapeutics SVP, Clinical Development
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Address
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QED Therapeutics
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Country
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Phone
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Fax
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Email
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Contact person for public queries
Name
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Address
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Country
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Phone
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Fax
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Email
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Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT05145010
Download to PDF