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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05145010




Registration number
NCT05145010
Ethics application status
Date submitted
22/11/2021
Date registered
6/12/2021

Titles & IDs
Public title
Extension Study of Infigratinib in Children With Achondroplasia (ACH)
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
Secondary ID [1] 0 0
QBGJ398-203
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Achondroplasia 0 0
Condition category
Condition code
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders

Intervention/exposure
Study type
Interventional
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 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)).

Treatment: Drugs: Infigratinib
Infigratinib sprinkle capsules to be administered by mouth. Starting dose for the subjects naïve to Infigratinib will be 0.25 mg/kg/day (the dose selected to be explored further in the dose escalation portion of Phase 2 study QBGJ398-201 (PROPEL 2)).
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence of treatment emergent adverse events (TEAE) and serious TEAE
Timepoint [1] 0 0
10 years
Primary outcome [2] 0 0
Changes over time in height Z-score in relation to ACH and non-ACH growth charts
Timepoint [2] 0 0
10 years
Secondary outcome [1] 0 0
Changes over time in absolute height velocity, expressed as height velocity Z-score in relation to ACH and non ACH growth charts
Timepoint [1] 0 0
10 years
Secondary outcome [2] 0 0
Changes over time in body proportions
Timepoint [2] 0 0
10 years
Secondary outcome [3] 0 0
Changes over time in weight z-score
Timepoint [3] 0 0
10 years
Secondary outcome [4] 0 0
Changes overtime in BMI
Timepoint [4] 0 0
10 years
Secondary outcome [5] 0 0
Age of puberty onset and time to Tanner stage =4
Timepoint [5] 0 0
10 years
Secondary outcome [6] 0 0
Changes over time in number of episodes of otitis media per year
Timepoint [6] 0 0
10 years
Secondary outcome [7] 0 0
Changes over time in number of episodes and/or severity of sleep apnea
Timepoint [7] 0 0
10 years
Secondary outcome [8] 0 0
Changes over time in range of motion (elbow)
Timepoint [8] 0 0
10 years
Secondary outcome [9] 0 0
Changes over time in skeletal abnormalities of the lower extremities and spine
Timepoint [9] 0 0
10 years
Secondary outcome [10] 0 0
Changes in health-related Quality of life [HRQoL] as assessed by Pediatric Quality of Life Inventory (PedsQL)
Timepoint [10] 0 0
10 years
Secondary outcome [11] 0 0
Changes in health-related Quality of life [HRQoL] as assessed by Quality of Life in Short Stature Youth questionnaire (QoLISSY)
Timepoint [11] 0 0
10 years
Secondary outcome [12] 0 0
Overall pain as assessed by Numeric Rating Scale for pain (Pain-NRS)
Timepoint [12] 0 0
10 years
Secondary outcome [13] 0 0
Changes in functional abilities as evaluated by Functional Independence Measure for Children (WeeFIM)
Timepoint [13] 0 0
10 years
Secondary outcome [14] 0 0
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Severity (PGI-S)
Timepoint [14] 0 0
10 years
Secondary outcome [15] 0 0
Severity of the physical functioning challenges as assessed by Patient/Parent Global Impression of Change (PGI-C)
Timepoint [15] 0 0
10 years
Secondary outcome [16] 0 0
Subject and caregiver evaluation of treatment benefit as assessed by a qualitative interview
Timepoint [16] 0 0
10 years
Secondary outcome [17] 0 0
Changes in cognitive functions assessed by age-appropriate computerized tests
Timepoint [17] 0 0
10 years

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
Minimum age
3 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
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. Current participation in an ongoing clinical study with a sponsor other than QED
5. 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 discontinued treatment with prohibited medications for at least 5 half-lives before screening are eligible.
6. 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.
7. Subjects who have significant abnormality in screening laboratory results.
8. Subjects who have had a fracture within 12 months of screening.

Study design
Purpose of the study
Treatment
Allocation to intervention
Non-randomised trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Single group
Other design features
Phase
Phase 2
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
6/12/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/02/2032
Actual
Sample size
Target
300
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Hospital - Parkville
Recruitment postcode(s) [1] 0 0
3052 - Parkville
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Delaware
Country [3] 0 0
United States of America
State/province [3] 0 0
Maryland
Country [4] 0 0
United States of America
State/province [4] 0 0
Ohio
Country [5] 0 0
United States of America
State/province [5] 0 0
Tennessee
Country [6] 0 0
Canada
State/province [6] 0 0
Alberta
Country [7] 0 0
France
State/province [7] 0 0
Lyon
Country [8] 0 0
France
State/province [8] 0 0
Paris
Country [9] 0 0
France
State/province [9] 0 0
Toulouse
Country [10] 0 0
Spain
State/province [10] 0 0
Madrid
Country [11] 0 0
Spain
State/province [11] 0 0
Málaga
Country [12] 0 0
Spain
State/province [12] 0 0
Álava
Country [13] 0 0
United Kingdom
State/province [13] 0 0
Birmingham
Country [14] 0 0
United Kingdom
State/province [14] 0 0
Glasgow
Country [15] 0 0
United Kingdom
State/province [15] 0 0
London
Country [16] 0 0
United Kingdom
State/province [16] 0 0
Manchester
Country [17] 0 0
United Kingdom
State/province [17] 0 0
Sheffield

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
QED Therapeutics, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
QED Therapeutics SVP, Clinical Development
Address 0 0
QED Therapeutics
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05145010