Register a trial

The ANZCTR website is back online for trial registration and updates. We apologise for any inconvenience caused while the site was inactive.

Please note that the ANZCTR will be unattended from Friday 18th April until Tuesday 22nd April due to the Easter long weekend. Submissions and updates will not be processed during that time.



Reset your password and enable multi-factor authentication (MFA)


For ANZCTR account holders: to help ensure the cyber safety of your account, you’ll need to reset your password and set-up multi-factor authentication (MFA) as per the instructions below.


  1. Go to the Login page, click ‘reset password’ and follow the instructions.
  2. Check your email for the link to set a new password.
  3. Create a new password that meets requirements. New passwords must include at least one lowercase letter, one uppercase letter, one number and one special character (e.g. !#$%&@).
  4. Return to the Login page and enter your new password. A verification code will be sent to your email.
  5. Check your email for the code and enter it on the Login page. If the code is entered incorrectly, you can re-enter the correct one or request a new one.

Learn more about MFA and its importance on the Australian Signals Directorate website.

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
Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06873035




Registration number
NCT06873035
Ethics application status
Date submitted
18/02/2025
Date registered
12/03/2025

Titles & IDs
Public title
An Interventional Study of Infigratinib in Children With Hypochondroplasia
Scientific title
A Phase 2/3, Multicenter, Open-Label Phase Followed by a Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Infigratinib in Children With Hypochondroplasia: ACCEL 2/3
Secondary ID [1] 0 0
QBGJ398-304
Universal Trial Number (UTN)
Trial acronym
HCH
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Hypochondroplasia 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Metabolic and Endocrine 0 0 0 0
Other endocrine disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Musculoskeletal 0 0 0 0
Normal musculoskeletal and cartilage development and function
Reproductive Health and Childbirth 0 0 0 0
Complications of newborn

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - infigratinib 0.128 mg/kg/day
Treatment: Drugs - infigratinib 0.25 mg/kg/day

Experimental: Phase 2 Cohort 1 - infigratinib (0.128 mg/kg/day)

Experimental: Phase 2 Cohort 2 - infigratinib (0.25 mg/kg/day)


Treatment: Drugs: infigratinib 0.128 mg/kg/day
Oral infigratinib 0.128 mg/kg/day

Treatment: Drugs: infigratinib 0.25 mg/kg/day
Oral infigratinib 0.25 mg/kg/day
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Change from baseline (BL) in Annualized Height Velocity (AHV; cm/year)
Timepoint [1] 0 0
52 weeks
Primary outcome [2] 0 0
Incidence, severity, and seriousness of adverse events (AEs) that require dose reduction or discontinuation
Timepoint [2] 0 0
52 weeks
Secondary outcome [1] 0 0
Change from BL in height Z-score
Timepoint [1] 0 0
52 weeks
Secondary outcome [2] 0 0
Change from BL in height z-score [ethnic-specific growth charts may be used if/when applicable and available] of growth and body proportions
Timepoint [2] 0 0
52 weeks
Secondary outcome [3] 0 0
Changes in upper to lower body segment ratio (cm)
Timepoint [3] 0 0
52 weeks
Secondary outcome [4] 0 0
Changes in cognitive function (as assessed by age appropriate computerized tests)
Timepoint [4] 0 0
52 weeks
Secondary outcome [5] 0 0
Pharmacokinetic profile of infigratinib by assessment of maximum concentration (Cmax)
Timepoint [5] 0 0
52 weeks
Secondary outcome [6] 0 0
Pharmacokinetic profile of infigratinib by assessment of time-to-maximum concentration (Tmax)
Timepoint [6] 0 0
52 weeks
Secondary outcome [7] 0 0
Change from BL in collagen X marker (CXM) levels
Timepoint [7] 0 0
52 weeks

Eligibility
Key inclusion criteria
Key

* Participants must have completed at least 26 weeks and still be on the observational study (QBGJ398-004).
* Phase 2 portion: Participants 5-11 years of age (inclusive).
* Phase 3 portion: Participants 3 to <18 years of age at screening with growth potential
* Diagnosis of HCH documented clinically by the presence of disproportionate short stature and confirmed with a molecular test.
* Participants are able to swallow oral medication.
* Participants and parent(s), legal guardian(s), or caregiver(s) are willing and able to comply with study visits and study procedures.
* Participants are ambulatory and able to stand without assistance. Sex and Contraceptive/Barrier Requirements
* Negative pregnancy test in girls =10 years of age or girls of any age who have experienced menarche.
* If sexually active, participants whether male or female, must be willing to use a highly effective method of contraception, as relevant, while taking study drug and for 1 month after the last dose of study drug.
* Signed informed consent.

Key
Minimum age
3 Years
Maximum age
18 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Participants who have ACH or a short stature condition other than HCH.
* Significant concurrent disease or condition that, in the view of the investigator and/or sponsor, would confound assessment of efficacy or safety of infigratinib.
* Current evidence of clinically significant corneal or retinal disorder/keratopathy confirmed by ophthalmic examination.
* Concurrent circumstance, disease, or condition that, in the view of the investigator and/or sponsor, would interfere with study participation or safety evaluations.
* History and/or current evidence of extensive ectopic tissue calcification.
* History of malignancy.
* Having received or planning to receive treatment with any other investigational or approved product for the treatment of ACH, HCH, or short stature.
* Regular long-term treatment (=3 weeks) with supraphysiologic doses of glucocorticoid.
* Previous limb-lengthening surgery at any time or planned/expected to have limb-lengthening or guided growth surgery while participating in the study.
* Participants receiving medications which could increase serum phosphorus and/or calcium concentrations
* Clinically significant abnormality in any laboratory test result at screening.
* Pregnant or breastfeeding at the screening visit or planning to become pregnant (self or partner) at any time during the study.
* Allergy to any components of the study drug.
* Concurrent circumstance, disease, or condition that would interfere with study participation.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled 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
Parallel
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
1/05/2025
Actual
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/01/2027
Actual
Sample size
Target
24
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 0 0
Murdoch Children's Research Institute - 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
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
District of Columbia
Country [4] 0 0
United States of America
State/province [4] 0 0
Maryland
Country [5] 0 0
United States of America
State/province [5] 0 0
Missouri
Country [6] 0 0
United States of America
State/province [6] 0 0
Tennessee
Country [7] 0 0
United States of America
State/province [7] 0 0
Wisconsin
Country [8] 0 0
Canada
State/province [8] 0 0
Ontario
Country [9] 0 0
Canada
State/province [9] 0 0
Quebec
Country [10] 0 0
France
State/province [10] 0 0
Bron
Country [11] 0 0
France
State/province [11] 0 0
Paris
Country [12] 0 0
France
State/province [12] 0 0
Toulouse
Country [13] 0 0
Norway
State/province [13] 0 0
Bergen
Country [14] 0 0
Norway
State/province [14] 0 0
Oslo
Country [15] 0 0
Portugal
State/province [15] 0 0
Coimbra
Country [16] 0 0
Singapore
State/province [16] 0 0
Singapore
Country [17] 0 0
Spain
State/province [17] 0 0
Vitoria-gasteiz
Country [18] 0 0
Sweden
State/province [18] 0 0
Solna
Country [19] 0 0
United Kingdom
State/province [19] 0 0
Manchester
Country [20] 0 0
United Kingdom
State/province [20] 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
Address 0 0
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


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/NCT06873035