13 Participants Needed

Inclisiran for High Cholesterol

(ORION-13 Trial)

Recruiting at 17 trial locations
NP
Overseen ByNovartis Pharmaceuticals
Age: < 18
Sex: Any
Trial Phase: Phase 3
Sponsor: Novartis Pharmaceuticals
Must be taking: Statins
Pivotal Trial (Near Approval)This treatment is in the last trial phase before FDA approval
Prior Safety DataThis treatment has passed at least one previous human trial
Approved in 3 JurisdictionsThis treatment is already approved in other countries

Trial Summary

What is the purpose of this trial?

This trial tests inclisiran, a medication that lowers bad cholesterol, in adolescents with a genetic condition causing very high cholesterol. Inclisiran works by blocking a protein that increases cholesterol levels, leading to significant reductions in bad cholesterol.

Will I have to stop taking my current medications?

The trial does not specify if you need to stop your current medications, but you must be on a stable dose of statins or other lipid-lowering therapy for at least 30 days before joining. If you are taking certain medications like mipomersen or lomitapide, you need to stop them 5 months before screening.

How is the drug Inclisiran different from other treatments for high cholesterol?

Inclisiran is unique because it uses a small interfering RNA (siRNA) mechanism to lower cholesterol by targeting the PCSK9 protein, which is different from traditional statins that inhibit cholesterol production in the liver. It is administered as an injection every six months, offering a less frequent dosing schedule compared to daily oral medications.12345

Research Team

NP

Novartis Pharmaceuticals

Principal Investigator

Novartis Pharmaceuticals

Eligibility Criteria

This trial is for adolescents with a genetic condition called homozygous familial hypercholesterolemia (HoFH) and high LDL cholesterol levels, despite being on the highest dose of statins they can tolerate. They should not have used certain other cholesterol drugs recently and must have decent kidney function.

Inclusion Criteria

I have been genetically diagnosed with Homozygous Familial Hypercholesterolemia.
Your cholesterol level is too high when you haven't eaten for a while.
I have been on the highest dose of statin I can tolerate for at least 30 days.
See 1 more

Exclusion Criteria

My tests show a negative mutation in both LDLR genes.
I have not taken mipomersen or lomitapide in the last 5 months.
Recent and/or planned use of other investigational medicinal products or devices
See 5 more

Timeline

Screening

Participants are screened for eligibility to participate in the trial

2-4 weeks

Treatment (Year 1)

Participants receive either 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
  • Placebo
Trial OverviewThe study tests Inclisiran, a new medication aimed at lowering LDL cholesterol, against a placebo in young people with severe inherited high cholesterol. It's designed to see if it's safe and effective in reducing their cholesterol levels.
Participant Groups
2Treatment groups
Experimental Treatment
Placebo Group
Group I: InclisiranExperimental Treatment1 Intervention
Year 1 - inclisiran sodium 300 mg subcutaneous injection (given at Days 1, 90, and 270) Day 360 only - placebo subcutaneous injection Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 450 and 630)
Group II: PlaceboPlacebo Group1 Intervention
Year 1 - placebo subcutaneous injection (given at Days 1, 90 and 270) Year 2 - inclisiran sodium 300 mg subcutaneous injection (given at Days 360, 450, and 630)

Inclisiran is already approved in European Union, United States, China for the following indications:

🇪🇺
Approved in European Union as Leqvio for:
  • Primary hypercholesterolemia (heterozygous familial and non-familial)
  • Mixed dyslipidemia
🇺🇸
Approved in United States as Leqvio for:
  • Heterozygous familial hypercholesterolemia (HeFH)
  • Clinical atherosclerotic cardiovascular disease (ASCVD)
  • Primary hypercholesterolemia
🇨🇳
Approved in China as Leqvio for:
  • Primary hypercholesterolemia (heterozygous familial and non-familial)
  • Mixed dyslipidemia

Find a Clinic Near You

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 profile image

Dr. Vas Narasimhan

Novartis Pharmaceuticals

Chief Executive Officer since 2018

MD from Harvard Medical School

Dr. Shreeram Aradhye profile image

Dr. Shreeram Aradhye

Novartis Pharmaceuticals

Chief Medical Officer since 2021

MD

Findings from Research

Acitretin is a valuable treatment option for psoriasis, effective both alone and in combination with other therapies, due to its long-term efficacy and lack of direct immunosuppressive effects.
The consensus guidelines from the Spanish Academy of Dermatology provide comprehensive information on the safe use of acitretin, including its pharmacological properties, indications, contraindications, and treatment strategies for clinical practice.
Guidelines for the use of acitretin in psoriasis. Psoriasis Group of the Spanish Academy of Dermatology and Venereology.Carretero, G., Ribera, M., Belinchón, I., et al.[2022]
Acitretin is a non-immunosuppressive oral medication that has been used for treating psoriasis, indicating it may have a safer profile compared to immunosuppressive treatments.
Tofacitinib, an oral Janus kinase inhibitor, also shows efficacy in psoriasis treatment but comes with significant safety risks, highlighting the need for careful patient selection and monitoring.
Small Molecules for Psoriasis.Leonardi, CL.[2019]
Acitretin is a non-immunosuppressive oral medication that has been used for treating psoriasis, providing efficacy without the risks associated with immunosuppressive therapies.
Tofacitinib, an oral Janus kinase inhibitor, also shows effectiveness in psoriasis treatment but comes with significant safety concerns, highlighting the need for careful patient selection.
Small Molecules for Psoriasis.Leonardi, CL.[2016]

References

Guidelines for the use of acitretin in psoriasis. Psoriasis Group of the Spanish Academy of Dermatology and Venereology. [2022]
Small Molecules for Psoriasis. [2019]
Small Molecules for Psoriasis. [2016]
Open-label exploratory study of acitretin for the treatment of severe chronic hand dermatitis. [2018]
Effects of gemfibrozil (Lopid) on hyperlipidemia in acitretin-treated patients. Results of a double-blind cross-over study. [2019]