Study to evaluate efficacy and safety of inclisiran in adolescents with homozygous familial hypercholesterolemia
- Conditions
- Homozygous Familial HypercholesterolemiaMedDRA version: 20.0Level: LLTClassification code 10057080Term: Homozygous familial hypercholesterolemiaSystem Organ Class: 100000004850Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]
- Registration Number
- EUCTR2020-002755-38-NL
- Lead Sponsor
- ovartis Pharma AG
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 12
Homozygous Familial Hypercholesterolemia (HoFH) diagnosed by genetic confirmation.
Fasting LDL-C >130 mg/dL (3.4 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.
Are the trial subjects under 18? yes
Number of subjects for this age range: 12
F.1.2 Adults (18-64 years) no
F.1.2.1 Number of subjects for this age range
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range
Documented evidence of a null (negative) mutation in both LDLR alleles.
Heterozygous familial hypercholesterolemia (HeFH).
Active liver disease.
Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome.
Major adverse cardiovascular events within 1 month prior to randomization.
Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening).
Treatment with mipomersen or lomitapide (within 5 months of screening).
Recent and/or planned use of other investigational medicinal products or devices.
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method