Trial Review

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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT04652726




Registration number
NCT04652726
Ethics application status
Date submitted
2/12/2020
Date registered
3/12/2020
Date last updated
12/08/2024

Titles & IDs
Public title
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia
Scientific title
Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-16)
Secondary ID [1] 0 0
2020-002757-18
Secondary ID [2] 0 0
CKJX839C12301
Universal Trial Number (UTN)
Trial acronym
ORION-16
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Condition category
Condition code

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Comparator / control treatment
Control group

Outcomes

Eligibility
Key inclusion criteria
* Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria
* Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening
* Fasting triglycerides <400 mg/dL (4.5 mmol/L) at screening
* On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for = 30 days before screening
* Estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2 at screening
Minimum age
12 Years
Maximum age
17 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Homozygous familial hypercholesterolemia (HoFH)
* Active liver disease
* Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome
* Major adverse cardiovascular events within 3 months prior to randomization
* Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening)
* Recent and/or planned use of other investigational medicinal products or devices

Other protocol-defined inclusion/exclusion criteria may apply

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
Blinded (masking used)
Who is / are masked / blinded?
The people receiving the treatment/s


The people analysing the results/data
Intervention assignment
Parallel
Other design features
Phase
Phase 3
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Active, not recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
27/01/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
2/12/2024
Actual
Sample size
Target
Accrual to date
Final
141
Recruitment in Australia
Recruitment state(s)

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Novartis Pharmaceuticals
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C).
Trial website
https://clinicaltrials.gov/study/NCT04652726
Trial related presentations / publications
Public notes
This record is viewable in the ANZCTR as it had previously listed Australia and/or New Zealand as a recruitment site, however these sites have since been removed

Contacts
Principal investigator
Name 0 0
Novartis Pharmaceuticals
Address 0 0
Novartis Pharmaceuticals
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



Summary Results

For IPD and results data, please see https://clinicaltrials.gov/study/NCT04652726