Please note that the copy function is not enabled for this field.
If you wish to
modify
existing outcomes, please copy and paste the current outcome text into the Update field.
LOGIN
CREATE ACCOUNT
LOGIN
CREATE ACCOUNT
MY TRIALS
REGISTER TRIAL
FAQs
HINTS AND TIPS
DEFINITIONS
Trial Review
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been endorsed by the ANZCTR. Before participating in a study, talk to your health care provider and refer to this
information for consumers
Download to PDF
Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/ct2/show/NCT06164951
Registration number
NCT06164951
Ethics application status
Date submitted
29/11/2023
Date registered
11/12/2023
Date last updated
10/05/2024
Titles & IDs
Public title
A Study to Evaluate the Efficacy and Safety of Infigratinib in Children and Adolescents With Achondroplasia
Query!
Scientific title
A Phase 3, Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children 3 to <18 Years of Age With Achondroplasia: PROPEL 3
Query!
Secondary ID [1]
0
0
QBGJ398-303
Query!
Universal Trial Number (UTN)
Query!
Trial acronym
PROPEL3
Query!
Linked study record
Query!
Health condition
Health condition(s) or problem(s) studied:
Achondroplasia
0
0
Query!
Condition category
Condition code
Human Genetics and Inherited Disorders
0
0
0
0
Query!
Other human genetics and inherited disorders
Query!
Musculoskeletal
0
0
0
0
Query!
Other muscular and skeletal disorders
Query!
Intervention/exposure
Study type
Interventional
Query!
Description of intervention(s) / exposure
Treatment: Drugs - Infigratinib 0.25 mg/kg/day
Treatment: Drugs - Placebo Comparator 0.25 mg/kg/day
Experimental: Infigratinib 0.25 mg/kg/day - Infigratinib at 2, 3.5, 5, 7, 10 mg
Placebo Comparator: Placebo 0.25 mg/kg/day - Placebo Comparator at 2, 3.5, 5, 7, 10 mg
Treatment: Drugs: Infigratinib 0.25 mg/kg/day
Daily doses of oral Infigratinib (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
Treatment: Drugs: Placebo Comparator 0.25 mg/kg/day
Daily doses of oral Placebo Comparator (sprinkle capsules) at 2, 3.5, 5, 7, 10 mg
Query!
Intervention code [1]
0
0
Treatment: Drugs
Query!
Comparator / control treatment
Query!
Control group
Query!
Outcomes
Primary outcome [1]
0
0
Change from baseline (BL) in annualized height velocity (cm/year)
Query!
Assessment method [1]
0
0
Query!
Timepoint [1]
0
0
Week 52
Query!
Secondary outcome [1]
0
0
Change from BL in height Z-score (in relation to ACH tables)
Query!
Assessment method [1]
0
0
Query!
Timepoint [1]
0
0
Week 52
Query!
Secondary outcome [2]
0
0
Change from BL in upper to lower body segment ratio
Query!
Assessment method [2]
0
0
Query!
Timepoint [2]
0
0
Week 52
Query!
Secondary outcome [3]
0
0
Change from BL in height Z-score (in relation to non-ACH tables)
Query!
Assessment method [3]
0
0
Query!
Timepoint [3]
0
0
Week 52
Query!
Secondary outcome [4]
0
0
Annualized height velocity (cm/year)
Query!
Assessment method [4]
0
0
Query!
Timepoint [4]
0
0
Week 52
Query!
Secondary outcome [5]
0
0
Absolute and change from baseline in upper arm to forearm length ratio (cm)
Query!
Assessment method [5]
0
0
Query!
Timepoint [5]
0
0
Week 52
Query!
Secondary outcome [6]
0
0
Absolute and change from baseline in upper leg to lower leg length ratio (cm)
Query!
Assessment method [6]
0
0
Query!
Timepoint [6]
0
0
Week 52
Query!
Secondary outcome [7]
0
0
Absolute and change from baseline in arm span (cm) to standing height ratio
Query!
Assessment method [7]
0
0
Query!
Timepoint [7]
0
0
Week 52
Query!
Secondary outcome [8]
0
0
Absolute and change from baseline in head circumference (cm) to standing height ratio
Query!
Assessment method [8]
0
0
Query!
Timepoint [8]
0
0
Week 52
Query!
Secondary outcome [9]
0
0
Absolute value and change in body mass index
Query!
Assessment method [9]
0
0
Query!
Timepoint [9]
0
0
Week 52
Query!
Secondary outcome [10]
0
0
Incidence of adverse events
Query!
Assessment method [10]
0
0
Query!
Timepoint [10]
0
0
Week 52
Query!
Secondary outcome [11]
0
0
Change from BL in annualized height velocity (cm/year) in children 5 years old and older, compared to placebo
Query!
Assessment method [11]
0
0
Query!
Timepoint [11]
0
0
Week 52
Query!
Secondary outcome [12]
0
0
Change from BL in the Physical Functioning dimension of the Pediatric Quality of Life Generic Core Scale Short Form
Query!
Assessment method [12]
0
0
Scale scores 0-100. Higher score=better Health-Related Quality of Life
Query!
Timepoint [12]
0
0
Week 52
Query!
Secondary outcome [13]
0
0
Change in psychomotor function assessed by age-appropriate computerized tests (Detection Test), compared to placebo
Query!
Assessment method [13]
0
0
Lower score=better performance. Range values=2-6
Query!
Timepoint [13]
0
0
Week 52
Query!
Secondary outcome [14]
0
0
Change from BL in attention assessed by age-appropriate computerized tests (Identification Test)
Query!
Assessment method [14]
0
0
Lower score=better performance. Range values=2-6
Query!
Timepoint [14]
0
0
Week 52
Query!
Secondary outcome [15]
0
0
Change from BL in visual learning assessed by age-appropriate computerized tests (One Card Learning Test)
Query!
Assessment method [15]
0
0
Higher score=better performance. Range values=0-1.6
Query!
Timepoint [15]
0
0
Week 52
Query!
Secondary outcome [16]
0
0
Change from BL in working memory assessed by age-appropriate computerized tests (One Back Test)
Query!
Assessment method [16]
0
0
Lower score=better performance. Range values=2-6
Query!
Timepoint [16]
0
0
Week 52
Query!
Secondary outcome [17]
0
0
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Query!
Assessment method [17]
0
0
Query!
Timepoint [17]
0
0
Week 52
Query!
Secondary outcome [18]
0
0
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Query!
Assessment method [18]
0
0
Query!
Timepoint [18]
0
0
Week 52
Query!
Secondary outcome [19]
0
0
Change from BL in collagen X marker concentration (ug/L)
Query!
Assessment method [19]
0
0
Query!
Timepoint [19]
0
0
Week 52
Query!
Secondary outcome [20]
0
0
Evaluate the acceptability and palatability of infigratinib using a 5-point hedonic scale
Query!
Assessment method [20]
0
0
Query!
Timepoint [20]
0
0
Week 13
Query!
Eligibility
Key inclusion criteria
-
1. Subject must be 3 to <18 years of age at screening with growth potential defined
as annualized height velocity of >1.5 cm/year over a period of at least 6 months,
pubertal Tanner stage =4, and bone age =13 years in females and =15 years in
males.
Type of Subject and Disease Characteristics
2. Subjects who have a diagnosis of ACH that has been documented clinically and
confirmed by genetic testing.
3. Subjects must have completed at least 26 weeks in the PROPEL (QBGJ398-001) study
before study entry.
4. Subjects are able to swallow oral medication.
5. Subjects and parent(s), legal guardian(s), or caregivers are willing and able to
comply with study visits and study procedures.
6. Subjects are ambulatory and able to stand without assistance. Sex and
Contraceptive/Barrier Requirements
7. Negative pregnancy test in girls =10 years of age or girls of any age who have
experienced menarche.
8. If sexually active, subjects must be willing to use a highly effective method of
contraception while taking study drug and for 3 months after the last dose of
study drug.
Informed Consent
9. Signed informed consent, which includes compliance with the requirements and
restrictions listed in the informed consent form and in this protocol, must be
obtained for each subject from their parent(s) or legal guardian and signed
informed consent/assent must be obtained from the subject (when applicable)
Query!
Minimum age
3
Years
Query!
Query!
Maximum age
17
Years
Query!
Query!
Sex
Both males and females
Query!
Can healthy volunteers participate?
No
Query!
Key exclusion criteria
- Exclusion Criteria Medical Conditions
1. Subjects who have hypochondroplasia or short stature condition other than ACH.
2. Significant concurrent disease or condition that, in the view of the investigator
and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
3. Current evidence of clinically significant corneal or retinal
disorder/keratopathy confirmed by ophthalmic examination.
4. Concurrent circumstance, disease or condition that, in the view of the
investigator and/or sponsor, would interfere with study participation or safety
evaluations and/or would require treatment with a prohibited medication, and/or
would place the subject at high risk for poor treatment compliance or for not
completing the study.
5. History and/or current evidence of extensive ectopic tissue calcification.
6. History of malignancy.
Prior/Concomitant Therapy
7. Having received or planning to receive treatment with any other investigational
or approved product for the treatment of ACH or short stature.
8. Regular long-term treatment (=3 weeks) with supraphysiologic doses of
glucocorticoid therapy (ie, >15 mg/m2/day of hydrocortisone or equivalence) or
treatment with glucocorticoids at anti-inflammatory doses for over 3 weeks within
6 months of the screening visit (low-dose ongoing inhaled steroid for asthma is
acceptable).
9. Previous limb-lengthening surgery at any time or planned/expected to have
limb-lengthening surgery or guided growth surgery during the study period. Guided
growth surgery with plates removed at least 12 months prior to screening is
allowed.
10. Currently receiving treatment with agents that are known strong inducers or
inhibitors of CYP3A4 or prolonged treatment (>1 week) with medications that alter
the pH of the gastrointestinal tract or antiepileptic drugs that are CYP3A4
and/or P-gp inducers, including carbamazepine, phenytoin, phenobarbital, and
primidone.
11. Current evidence of endocrine alterations of calcium/phosphorus homeostasis.
Diagnostic assessments
12. Subjects who have significant abnormality in screening laboratory results.
Other Exclusions
13. Having had a fracture of the long bones (ie, extremities) or spine within 12
months prior to screening.
14. Pregnant or breastfeeding at the screening visit or planning to become pregnant
(self or partner) at any time during the study.
15. Allergy or hypersensitivity to any components of the study drug.
Query!
Study design
Purpose of the study
Treatment
Query!
Allocation to intervention
Randomised controlled trial
Query!
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Query!
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Query!
Masking / blinding
Blinded (masking used)
Query!
Who is / are masked / blinded?
The people receiving the treatment/s
The people administering the treatment/s
The people assessing the outcomes
The people analysing the results/data
Query!
Query!
Query!
Query!
Intervention assignment
Parallel
Query!
Other design features
Query!
Phase
Phase 3
Query!
Type of endpoint/s
Query!
Statistical methods / analysis
Query!
Recruitment
Recruitment status
Recruiting
Query!
Data analysis
Query!
Reason for early stopping/withdrawal
Query!
Other reasons
Query!
Date of first participant enrolment
Anticipated
Query!
Actual
10/11/2023
Query!
Date of last participant enrolment
Anticipated
Query!
Actual
Query!
Date of last data collection
Anticipated
30/04/2026
Query!
Actual
Query!
Sample size
Target
110
Query!
Accrual to date
Query!
Final
Query!
Recruitment in Australia
Recruitment state(s)
VIC
Query!
Recruitment hospital [1]
0
0
QED Investigative Site - Parkville
Query!
Recruitment postcode(s) [1]
0
0
3052 - Parkville
Query!
Recruitment outside Australia
Country [1]
0
0
United States of America
Query!
State/province [1]
0
0
California
Query!
Country [2]
0
0
United States of America
Query!
State/province [2]
0
0
Colorado
Query!
Country [3]
0
0
United States of America
Query!
State/province [3]
0
0
Maryland
Query!
Country [4]
0
0
United States of America
Query!
State/province [4]
0
0
Missouri
Query!
Country [5]
0
0
United States of America
Query!
State/province [5]
0
0
Ohio
Query!
Country [6]
0
0
United States of America
Query!
State/province [6]
0
0
Tennessee
Query!
Country [7]
0
0
United States of America
Query!
State/province [7]
0
0
Wisconsin
Query!
Country [8]
0
0
Argentina
Query!
State/province [8]
0
0
Capital Federal
Query!
Country [9]
0
0
Canada
Query!
State/province [9]
0
0
Alberta
Query!
Country [10]
0
0
Canada
Query!
State/province [10]
0
0
Ontario
Query!
Country [11]
0
0
Canada
Query!
State/province [11]
0
0
Quebec
Query!
Country [12]
0
0
France
Query!
State/province [12]
0
0
Bron
Query!
Country [13]
0
0
France
Query!
State/province [13]
0
0
Paris
Query!
Country [14]
0
0
France
Query!
State/province [14]
0
0
Toulouse
Query!
Country [15]
0
0
Germany
Query!
State/province [15]
0
0
Sachsen-Anhalt
Query!
Country [16]
0
0
Italy
Query!
State/province [16]
0
0
Milano
Query!
Country [17]
0
0
Italy
Query!
State/province [17]
0
0
Rome
Query!
Country [18]
0
0
Norway
Query!
State/province [18]
0
0
Bergen
Query!
Country [19]
0
0
Norway
Query!
State/province [19]
0
0
Oslo
Query!
Country [20]
0
0
Singapore
Query!
State/province [20]
0
0
Singapore
Query!
Country [21]
0
0
Spain
Query!
State/province [21]
0
0
Madrid
Query!
Country [22]
0
0
Spain
Query!
State/province [22]
0
0
Málaga
Query!
Country [23]
0
0
Spain
Query!
State/province [23]
0
0
Vitoria-Gasteiz
Query!
Country [24]
0
0
United Kingdom
Query!
State/province [24]
0
0
Birmingham
Query!
Country [25]
0
0
United Kingdom
Query!
State/province [25]
0
0
Bristol
Query!
Country [26]
0
0
United Kingdom
Query!
State/province [26]
0
0
Glasgow
Query!
Country [27]
0
0
United Kingdom
Query!
State/province [27]
0
0
London
Query!
Country [28]
0
0
United Kingdom
Query!
State/province [28]
0
0
Manchester
Query!
Country [29]
0
0
United Kingdom
Query!
State/province [29]
0
0
Sheffield
Query!
Funding & Sponsors
Primary sponsor type
Commercial sector/Industry
Query!
Name
QED Therapeutics, Inc.
Query!
Address
Query!
Country
Query!
Ethics approval
Ethics application status
Query!
Summary
Brief summary
This is a Phase 3, multicenter, double-blind, randomized, placebo-controlled study to
evaluate the efficacy and safety of infigratinib in children and adolescents with
achondroplasia (ACH) who have completed at least 26 weeks of participation in the
QED-sponsored study PROPEL (QBGJ398-001).
Query!
Trial website
https://clinicaltrials.gov/ct2/show/NCT06164951
Query!
Trial related presentations / publications
Query!
Public notes
Query!
Contacts
Principal investigator
Name
0
0
QED Therapeutics, Inc. Medical Director, Clinical Development
Query!
Address
0
0
QED Therapeutics
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for public queries
Name
0
0
Query!
Address
0
0
Query!
Country
0
0
Query!
Phone
0
0
Query!
Fax
0
0
Query!
Email
0
0
Query!
Contact person for scientific queries
Summary Results
For IPD and results data, please see
https://clinicaltrials.gov/ct2/show/NCT06164951
Download to PDF