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Trial details imported from ClinicalTrials.gov
For full trial details, please see the original record at
https://clinicaltrials.gov/study/NCT06455059
Registration number
NCT06455059
Ethics application status
Date submitted
7/06/2024
Date registered
12/06/2024
Date last updated
12/06/2024
Titles & IDs
Public title
Interventional Study of Vosoritide for the Treatment of Children With Hypochondroplasia
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Scientific title
A Phase 3, Randomized, Double-Blind, Placebo-Controlled, Multicenter Study to Evaluate the Efficacy and Safety of Vosoritide in Children With Hypochondroplasia
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Secondary ID [1]
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111-303
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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:
Hypochondroplasia
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Condition category
Condition code
Musculoskeletal
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Other muscular and skeletal disorders
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Metabolic and Endocrine
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Other endocrine disorders
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Human Genetics and Inherited Disorders
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Other human genetics and inherited disorders
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Musculoskeletal
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Normal musculoskeletal and cartilage development and function
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Reproductive Health and Childbirth
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Complications of newborn
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Intervention/exposure
Study type
Interventional
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Description of intervention(s) / exposure
Treatment: Drugs - Vosoritide
Treatment: Drugs - Placebo
Experimental: vosoritide injection with vial and syringe -
Placebo comparator: Placebo injection with vial and syringe -
Treatment: Drugs: Vosoritide
Subcutaneous injection of recommended dose of vosoritide based on weight-band dosing once daily.
Treatment: Drugs: Placebo
Subcutaneous injection of recommended dose of placebo
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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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Change from baseline in annualized growth velocity (AGV) at Week 52 versus placebo
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Assessment method [1]
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Timepoint [1]
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At week 52
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Secondary outcome [1]
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Change from baseline in standing height at Week 52 versus placebo
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Assessment method [1]
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Timepoint [1]
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At week 52
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Secondary outcome [2]
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Change from baseline in height Z-score at Week 52 versus placebo
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Assessment method [2]
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Timepoint [2]
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At week 52
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Eligibility
Key inclusion criteria
1. Participants must be = 3 to < 18 years of age at enrollment
2. A confirmed genetic diagnosis of HCH
3. A height Z score of = - 2.0 standard deviations (SDs) in reference to the general population of the same age and sex, as calculated using the Center for Disease Control and Prevention (CDC) growth charts
4. Males and females are eligible to participate in this clinical study.
5. Females = 10 years old or who have begun menses must have a negative pregnancy test at the Screening Visit and be willing to have additional pregnancy tests during the study.
6. If sexually active, participants must be willing to use a highly effective method of contraception while participating in the study.
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Minimum age
3
Years
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Maximum age
17
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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. Short stature condition other than HCH
2. Have an unstable condition likely to require surgical intervention during the study.
3. Evidence of decreased growth velocity and/or growth plate closure
4. Taking any of the prohibited medications
5. Treated with growth hormone, insulin-like growth factor 1 (IGF-1), or anabolic steroids
6. Planned or expected to have limb-lengthening surgery during the study period.
7. Planned or expected bone-related surgery (ie, surgery involving disruption of bone cortex, excluding tooth extraction), during the study period
8. Require any investigational agent prior to completion of study period.
9. Received vosoritide or another investigational product or investigational medical device in the past
10. Have used any investigational product or investigational medical device for the treatment of HCH or short stature at any time, including vosoritide
11. Have current malignancy, history of malignancy, or currently under work-up for suspected malignancy.
12. Have known hypersensitivity to vosoritide or its excipients.
13. Concurrent disease or condition that, in the view of the investigator, would interfere with study participation or safety evaluations, for any reason.
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Study design
Purpose of the study
Treatment
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Allocation to intervention
Randomised controlled 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
Blinded (masking used)
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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
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Intervention assignment
Single group
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Other design features
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Phase
Phase 3
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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
1/06/2024
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Actual
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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
1/08/2026
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Actual
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Sample size
Target
80
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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 Research Institute - 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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District of Columbia
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Funding & Sponsors
Primary sponsor type
Commercial sector/industry
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Name
BioMarin Pharmaceutical
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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
The intent and design of this Phase 3 study is to assess vosoritide as a therapeutic option for the treatment of children with hypochondroplasia (HCH).
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Trial website
https://clinicaltrials.gov/study/NCT06455059
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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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Medical Director, MD
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Address
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BioMarin Pharmaceutical
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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
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/NCT06455059
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