Inclisiran for Hypercholesterolemia

(ORION-20 Trial)

Recruiting at 49 trial locations

Overseen By Novartis Pharmaceuticals

Age: < 18

Sex: Any

Trial Phase: Phase 3

Sponsor: Novartis Pharmaceuticals

Must be taking: Statins, Ezetimibe

Must not be taking: PCSK9 inhibitors

Disqualifiers: Secondary hypercholesterolemia, HoFH, Active liver disease, others

Pivotal Trial (Near Approval) This treatment is in the last trial phase before FDA approval

Prior Safety Data This treatment has passed at least one previous human trial

Compensation: Varies

Number of Visits: 6

51 Participants Needed

Trial Summary

What is the purpose of this trial?

This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in children (aged 6 to \<12 years) with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDLC).

Research Team

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

This trial is for children aged 6 to under 12 with heterozygous familial hypercholesterolemia (HeFH) and high LDL cholesterol levels. They must be on a stable dose of lipid-lowering treatments like statins or ezetimibe for at least 30 days before the study, with no changes expected during the trial.

Inclusion Criteria

I am between 6 and 11 years old.

Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening

I have been diagnosed with HeFH through genetic tests or based on symptoms.

Exclusion Criteria

I do not have active liver disease or unexplained high liver enzyme levels.

I am not pregnant or nursing.

I have not taken PCSK9 inhibitor drugs in the last 90 days.

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment (Year 1)

Participants receive inclisiran or placebo subcutaneous injections at Days 1, 90, and 270

12 months

3 visits (in-person)

Open-label Treatment (Year 2)

All participants receive inclisiran subcutaneous injections at Days 360, 450, and 630

12 months

3 visits (in-person)

Follow-up

Participants are monitored for safety and effectiveness after treatment

4 weeks

Treatment Details

Interventions

• Inclisiran

Trial Overview The study tests Inclisiran's safety and effectiveness in lowering bad cholesterol compared to a placebo in kids with HeFH. It's a phase III trial where participants are randomly assigned to receive either Inclisiran or an inactive substance.

Participant Groups

Group I: Inclisiran Experimental Treatment 1 Intervention

Year 1 - inclisiran sodium subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium subcutaneous injection (given at Days 450 and 630)

Group II: Placebo Placebo Group 1 Intervention

Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium subcutaneous injection (given at Days 360, 450, and 630)

Who Is Running the Clinical Trial?

Novartis Pharmaceuticals

Lead Sponsor

Trials

2,963

Recruited

4,275,000+

Founded

1996

Headquarters

Basel, Switzerland

Known For

Precision medicine

Top Products

Gleevec, Cosentyx, Entresto, Kisqali

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD