Trial Review

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Trial registered on ANZCTR


Registration number
ACTRN12624001288516
Ethics application status
Approved
Date submitted
1/10/2024
Date registered
23/10/2024
Date last updated
23/10/2024
Date data sharing statement initially provided
23/10/2024
Type of registration
Retrospectively registered

Titles & IDs
Public title
Clinical outcomes following prescription of Vosoritide in children with Achondroplasia 0-2 years of age
Scientific title
Clinical outcomes following prescription of Vosoritide in children with Achondroplasia 0-2 years of age
Secondary ID [1] 313086 0
Nil known
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
achondroplasia 335325 0
Condition category
Condition code
Human Genetics and Inherited Disorders 331897 331897 0 0
Other human genetics and inherited disorders

Intervention/exposure
Study type
Observational
Patient registry
False
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
This study will prospectively observe the growth and developmental outcomes of children who are given a daily subcutaneous injection of vosoritide prescribed under the Australian prescribing guidelines, in comparison to age-matched historical controls who did not receive any therapeutic intervention. Outcomes measured will be achondroplasia-related comorbidities, age meeting developmental milestones, functional/quality of life status, skull and brain morphology, growth and body proportions. Assessments completed for prospective participants receiving vosoritide will be those routinely collected as part of standard of care for patients receiving vosoritide therapy. These assessments include: physical examination, weight, growth measurements, vital signs, achondroplasia developmental checklist, adverse events, MRI or sleep studies as clinically indicated, review of any interventions related to achondroplasia management, review of vosoritide administration and management. The only addition to routine assessment is the WeeFIM functional and quality of life measure which will be administered for participants >3 years. The total time to complete all assessments will be approximately one hour. The overall duration of observation will be five years from time of enrolment.
Intervention code [1] 329641 0
Not applicable
Comparator / control treatment
The control group will be children with achondroplasia who have not had any therapeutic intervention for achondroplasia. Data will be retrospectively collected from medical records for patients seen between Jan 2000 and Dec 2023 in the Bone Dysplasia Clinic at RCH/MCRI.
Control group
Historical

Outcomes
Primary outcome [1] 339514 0
Any achondroplasia associated comorbidities or surgeries (composite outcome) such as foramen magnum decompression, sleep disordered breathing and ear nose and throat surgeries.
Timepoint [1] 339514 0
Baseline then 6 monthly visits until five years from consenting
Secondary outcome [1] 440260 0
Skull and brain morphology (composite outcome) including Achondroplasia Foramen Magnum Scores (AFMS).
Timepoint [1] 440260 0
Baseline then 6 monthly visits until five years from consenting
Secondary outcome [2] 440261 0
Length/height
Timepoint [2] 440261 0
Baseline then 6 monthly visits until five years from consenting
Secondary outcome [3] 440570 0
Sitting heights or crown to rump length
Timepoint [3] 440570 0
Baseline then 6 monthly visits until five years from consenting
Secondary outcome [4] 440888 0
Head circumference
Timepoint [4] 440888 0
Baseline then 6 monthly visits until five years from consenting
Secondary outcome [5] 440889 0
Weight
Timepoint [5] 440889 0
Baseline then 6 monthly visits until five years from consenting

Eligibility
Key inclusion criteria
Prospective observational study:
Children with achondroplasia aged 0 to 2 years who attend the Bone Dysplasia Clinic at the Royal Children's Hospital/Murdoch Children's Research Institute who are prescribed vosoritide and for whom the parent/guardian signs informed consent.

Retrospective chart review (historical controls):
All children with achondroplasia aged 0 to 7 years who attended the Bone Dysplasia Clinic at the Royal Children's Hospital/Murdoch Children's Research Institute between 2000 and 2023 and who have not had any therapeutic drug for achondroplasia.
Minimum age
0 Years
Maximum age
2 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
None

Study design
Purpose
Duration
Longitudinal
Selection
Defined population
Timing
Both
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Date of first participant enrolment
Anticipated
Actual
2/10/2024
Date of last participant enrolment
Anticipated
2/10/2028
Actual
Date of last data collection
Anticipated
3/10/2033
Actual
Sample size
Target
35
Accrual to date
1
Final
Recruitment in Australia
Recruitment state(s)
VIC
Recruitment hospital [1] 27173 0
The Royal Childrens Hospital - Parkville
Recruitment postcode(s) [1] 43255 0
3052 - Parkville

Funding & Sponsors
Funding source category [1] 317530 0
Commercial sector/Industry
Name [1] 317530 0
BioMarin
Country [1] 317530 0
Australia
Primary sponsor type
Other
Name
Murdoch Children's Research Institute
Address
Country
Australia
Secondary sponsor category [1] 319832 0
None
Name [1] 319832 0
Address [1] 319832 0
Country [1] 319832 0
Other collaborator category [1] 283244 0
Commercial sector/Industry
Name [1] 283244 0
BioMarin
Address [1] 283244 0
Country [1] 283244 0
Australia

Ethics approval
Ethics application status
Approved
Ethics committee name [1] 316242 0
The Royal Children’s Hospital Human Research Ethics Committee
Ethics committee address [1] 316242 0
Ethics committee country [1] 316242 0
Australia
Date submitted for ethics approval [1] 316242 0
18/07/2024
Approval date [1] 316242 0
13/09/2024
Ethics approval number [1] 316242 0

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

Contacts
Principal investigator
Name 137258 0
Prof Ravi Savarirayan
Address 137258 0
Murdoch Children's Research Institute 50 Flemington Rd, Parkville, Victoria, 3052
Country 137258 0
Australia
Phone 137258 0
+61 399366541
Fax 137258 0
Email 137258 0
ravi.savarirayan@mcri.edu.au
Contact person for public queries
Name 137259 0
Natassja Billich
Address 137259 0
Murdoch Children's Research Institute 50 Flemington Rd, Parkville, Victoria, 3052
Country 137259 0
Australia
Phone 137259 0
+61 399366532
Fax 137259 0
Email 137259 0
tash.billich@mcri.edu.au
Contact person for scientific queries
Name 137260 0
Natassja Billich
Address 137260 0
Murdoch Children's Research Institute 50 Flemington Rd, Parkville, Victoria, 3052
Country 137260 0
Australia
Phone 137260 0
+61 399366532
Fax 137260 0
Email 137260 0
tash.billich@mcri.edu.au

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

Documents added manually
No documents have been uploaded by study researchers.

Documents added automatically
No additional documents have been identified.