Inclisiran for Familial Hypercholesterolemia

Phase-Based Progress Estimates
Novartis Investigative Site, Ioannina, GreeceFamilial HypercholesterolemiaInclisiran - Drug
12 - 17
All Sexes
What conditions do you have?

Study Summary

This trial will test a new drug to see if it's safe and effective in reducing cholesterol in teens with a genetic condition that causes high cholesterol.

Eligible Conditions
  • Familial Hypercholesterolemia

Treatment Effectiveness

Effectiveness Progress

2 of 3
This is further along than 85% of similar trials

Study Objectives

1 Primary · 4 Secondary · Reporting Duration: Baseline, after Day 90 up to Day 330

Baseline and Day 330
Low-Density Lipoproteins
Day 330
Time-adjusted percent change in LDL-C from baseline after Day 90 and up to Day 330
Day 720
% change and absolute change in LDL-C from baseline up to Day 720
% change and absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9) from baseline up to Day 720
Day 720
% change and absolute change in other lipoprotein and lipid parameters from baseline up to Day 720

Trial Safety

Safety Progress

3 of 3
This is further along than 85% of similar trials

Side Effects for

9%Injection site reaction
7%Back pain
7%Upper respiratory tract infection
1%Aortic valve stenosis
1%Acute myocardial infarction
This histogram enumerates side effects from a completed 2019 Phase 3 trial (NCT03397121) in the Inclisiran ARM group. Side effects include: Nasopharyngitis with 12%, Injection site reaction with 9%, Back pain with 7%, Upper respiratory tract infection with 7%, Influenza with 5%.

Trial Design

2 Treatment Groups

1 of 2
1 of 2

Experimental Treatment

Non-Treatment Group

13 Total Participants · 2 Treatment Groups

Primary Treatment: Inclisiran · Has Placebo Group · Phase 3

Experimental Group · 1 Intervention: Inclisiran · Intervention Types: Drug
PlaceboComparator Group · 1 Intervention: Placebo · Intervention Types: Drug
First Studied
Drug Approval Stage
How many patients have taken this drug
FDA approved

Trial Logistics

Trial Timeline

Screening: ~3 weeks
Treatment: Varies
Reporting: baseline, after day 90 up to day 330

Who is running the clinical trial?

Novartis PharmaceuticalsLead Sponsor
2,735 Previous Clinical Trials
3,544,030 Total Patients Enrolled

Eligibility Criteria

Age 12 - 17 · All Participants · 4 Total Inclusion Criteria

Mark “Yes” if the following statements are true for you:
You are taking the highest safe amount of statin medication for your high cholesterol and have been on a stable dose for at least 30 days prior to being screened for the trial.

Frequently Asked Questions

Who meets the basic requirements to take part in this research?

"The target patients for this study are those who suffer from hyperlipoproteinemia type ii and are between the ages of 12 to 17. So far, the study has recruited 12 individuals." - Anonymous Online Contributor

Unverified Answer

What conditions has Inclisiran been shown to be effective in treating?

"Inclisiran is used to lower cholesterol levels in patients with primary hypercholesterolemia. Additionally, this medication can be employed to prevent atherosclerotic cardiovascular disease in those struggling with conditions like diet-induced hypercholesterolemia." - Anonymous Online Contributor

Unverified Answer

What are the unique aspects of this research?

"Inclisiran's journey from initial research to Phase 3 approval spanned 2018. The first clinical trial, sponsored by Novartis Pharmaceuticals, involved 15000 patients. Post-trial, Inclisiran was approved for Phase 3 drug trials. Currently, there are 11 ongoing trials in 588 different cities and 52 countries worldwide." - Anonymous Online Contributor

Unverified Answer

How many people are participating in this clinical trial?

"That is correct. Our research shows that this study, which was first advertised on February 16th 2021, is still looking for participants. They need 12 people in total and have 4 different sites where the trial is taking place." - Anonymous Online Contributor

Unverified Answer

What is the FDA's current stance on Inclisiran?

"Inclisiran has received a 3 on our safety scale at Power. This is because it is currently in Phase 3 clinical trials, which suggests that while there is some data affirming its efficacy, there are multiple rounds of data supporting its safety." - Anonymous Online Contributor

Unverified Answer

Can you tell us about any other research that has been conducted using Inclisiran?

"There are currently 11 clinical trials underway for Inclisiran. 10 of those studies are in the final Phase 3 stage. Most of the research locations for Inclisiran are based in Ciudad de Mexico, but there are a total of 1414 sites running trials for this treatment worldwide." - Anonymous Online Contributor

Unverified Answer

Are there still positions open in this clinical trial for volunteers?

"Yes, this information is available on The trial was posted on February 16th, 2021 and has been actively recruiting since then with the most recent edit being made on July 29th, 2022." - Anonymous Online Contributor

Unverified Answer

Is this research only interested in participants who are older than 20?

"The age limit for this particular clinical trial is 12-17. Out of the 81 total trials, 14 are geared towards children and 67 are for adults over the age of 65." - Anonymous Online Contributor

Unverified Answer
Please Note: These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.