Study to evaluate efficacy and safety of inclisiran in adolescents with heterozygous familial hypercholesterolemia

Phase 1

• Conditions: Heterozygous Familial Hypercholesterolemia; MedDRA version: 20.0Level: LLTClassification code 10057079Term: Heterozygous familial hypercholesterolemiaSystem Organ Class: 100000004850; Therapeutic area: Diseases [C] - Cardiovascular Diseases [C14]

• Registration Number: EUCTR2020-002757-18-IT
• Lead Sponsor: OVARTIS PHARMA AG

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2021-05-24
• Last Update Posted: 30 August 2021

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 150

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
Other inclusion criteria are listed in the clinical study protocol
Are the trial subjects under 18? yes
Number of subjects for this age range:
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

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 exclusion criteria are listed in the clinical study protocol

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
Main Objective: Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [percent change] at Day 330 (Year 1);Secondary Objective: - Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [time-adjusted percent change] over Year 1<br>- Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [absolute change] at Day 330 (Year 1)<br>- Demonstrate superiority of inclisiran compared to placebo in reducing Apo B, lipoprotein (a) [Lp(a)], non-high density lipoprotein cholesterol (non-HDL-C), and total cholesterol [percent change] at Day 330 (Year 1)<br>- Hierarchical testing<br>Other secondary objectives are stated in the clinical study protocol;Primary end point(s): Percentage (%) change in low-density lipoprotein cholesterol (LDL-C);Timepoint(s) of evaluation of this end point: baseline to Day 330

Secondary Outcome Measures

Name	Time	Method
Secondary end point(s): - Time-adjusted % change in LDL-C<br>- Absolute change in LDL-C<br>- % change in apolipoprotein B (Apo B), lipoprotein (a) [Lp (a)], nonhigh density lipoprotein cholesterol (non-HDL-C), and total cholesterol<br>Other secondary endpoint can be found in the clinical study protocol;Timepoint(s) of evaluation of this end point: - Baseline, after Day 90 up to Day 330<br>- Baseline and Day 330<br>- Baseline and Day 330