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Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (V-PEDS-OLE)

Primary Purpose

Heterozygous or Homozygous Familial Hypercholesterolemia

Status

Recruiting

Phase

Phase 3

Locations

International

Study Type

Interventional

Intervention

Inclisiran

About this trial

This is an interventional treatment trial for Heterozygous or Homozygous Familial Hypercholesterolemia focused on measuring KJX839, heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, familial hypercholesterolemia, FH, inclisiran, pediatric

Eligibility Criteria

12 Years - undefined (Child, Adult, Older Adult)All SexesDoes not accept healthy volunteers

Key inclusion: Male and female participants with a diagnosis of HeFH or HoFH who completed the ORION-16 or ORION-13 studies Per investigator's clinical judgment, participant derived benefit from treatment with inclisiran in the ORION-16 or ORION-13 studies Key exclusion: Participants who in the feeder inclisiran ORION-16 and ORION-13 studies either screen failed or permanently discontinued from the treatment/study for any reason or had serious safety or tolerability issues related to inclisiran treatment Any uncontrolled or serious disease, or any medical, physical, or surgical condition, that may either interfere with participation in the clinical study or interpretation of clinical study results, and/or put the participant at significant risk

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Inclisiran

Arm Description

Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution

Outcomes

Primary Outcome Measures

Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs)

Safety and tolerability: TEAEs, TESAEs (incidence, severity, relationship to study drug and discontinuation due to TEAEs)

Secondary Outcome Measures

Percentage and absolute change in LDL-C from baseline in the feeder study to end of study

Evaluate the long-term effect of inclisiran (from baseline of feeder study to end of study) in lowering LDL-C

Full Information

NCT ID

NCT05682378

First Posted

December 27, 2022

Last Updated

October 23, 2023

Sponsor

Novartis Pharmaceuticals

1. Study Identification

Unique Protocol Identification Number

NCT05682378

Brief Title

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies

Acronym

V-PEDS-OLE

Official Title

An Open-label, Single Arm, Multicenter Extension Study to Evaluate Long-term Safety and Tolerability of Inclisiran in Participants With Heterozygous or Homozygous Familial Hypercholesterolemia Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (VICTORION-PEDS-OLE)

Study Type

Interventional

2. Study Status

Record Verification Date

October 2023

Overall Recruitment Status

Recruiting

Study Start Date

February 10, 2023 (Actual)

Primary Completion Date

December 1, 2027 (Anticipated)

Study Completion Date

December 1, 2027 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Sponsor

Name of the Sponsor

Novartis Pharmaceuticals

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

The purpose of this open-label, single arm, multicenter extension study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH who have completed the ORION-16 or ORION-13 studies.

Detailed Description

This is an open-label, single arm, multicenter study designed to evaluate long-term safety and tolerability of inclisiran. In addition, the study will provide participants the opportunity to have continued access to treatment with inclisiran.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Heterozygous or Homozygous Familial Hypercholesterolemia

Keywords

KJX839, heterozygous familial hypercholesterolemia, homozygous familial hypercholesterolemia, familial hypercholesterolemia, FH, inclisiran, pediatric

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 3

Interventional Study Model

Single Group Assignment

Masking

None (Open Label)

Allocation

N/A

Enrollment

165 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Inclisiran

Arm Type

Experimental

Arm Description

Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution

Intervention Type

Drug

Intervention Name(s)

Inclisiran

Other Intervention Name(s)

KJX839

Intervention Description

Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution administered subcutaneously in pre-filled syringe

Primary Outcome Measure Information:

Title

Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs)

Description

Safety and tolerability: TEAEs, TESAEs (incidence, severity, relationship to study drug and discontinuation due to TEAEs)

Time Frame

From Day 1 in the study up to the end of study visit; up to 1080 days

Secondary Outcome Measure Information:

Title

Percentage and absolute change in LDL-C from baseline in the feeder study to end of study

Description

Evaluate the long-term effect of inclisiran (from baseline of feeder study to end of study) in lowering LDL-C

Time Frame

Baseline (of feeder study) and Day 1080

10. Eligibility

Sex

All

Minimum Age & Unit of Time

12 Years

Accepts Healthy Volunteers

Eligibility Criteria

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Novartis Pharmaceuticals

Phone

1-888-669-6682

novartis.email@novartis.com

First Name & Middle Initial & Last Name or Official Title & Degree

Novartis Pharmaceuticals

Phone

+41613241111

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Novartis Pharmaceuticals

Organizational Affiliation

Novartis Pharmaceuticals

Official's Role

Study Director

Facility Information:

Facility Name

Novartis Investigative Site

City

Boca Raton

State/Province

Florida

ZIP/Postal Code

33434

Country

United States

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

New York

State/Province

New York

ZIP/Postal Code

10029

Country

United States

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Fortaleza

State/Province

Ceara

ZIP/Postal Code

60430275

Country

Brazil

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Sao Paulo

State/Province

ZIP/Postal Code

04023-900

Country

Brazil

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Sao Paulo

State/Province

ZIP/Postal Code

05403 000

Country

Brazil

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Quebec

ZIP/Postal Code

G1V 4W2

Country

Canada

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Toulouse Cedex

ZIP/Postal Code

31059

Country

France

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Frankfurt

ZIP/Postal Code

60590

Country

Germany

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Freiburg

ZIP/Postal Code

79100

Country

Germany

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Pecs

ZIP/Postal Code

7623

Country

Hungary

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Jerusalem

ZIP/Postal Code

9112001

Country

Israel

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Ramat Gan

ZIP/Postal Code

52621

Country

Israel

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Milano

State/Province

ZIP/Postal Code

20162

Country

Italy

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Ashrafieh

ZIP/Postal Code

166830

Country

Lebanon

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Rotterdam

State/Province

Zuid Holland

ZIP/Postal Code

3015 GD

Country

Netherlands

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Amsterdam

ZIP/Postal Code

1105 AZ

Country

Netherlands

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Oslo

ZIP/Postal Code

0514

Country

Norway

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Kemerovo

ZIP/Postal Code

650002

Country

Russian Federation

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Moscow

ZIP/Postal Code

127412

Country

Russian Federation

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Ljubljana

ZIP/Postal Code

1000

Country

Slovenia

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Bloemfontein

State/Province

Free State

ZIP/Postal Code

9301

Country

South Africa

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Pamplona

State/Province

Navarra

ZIP/Postal Code

31008

Country

Spain

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

A Coruna

ZIP/Postal Code

15001

Country

Spain

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Geneve 14

ZIP/Postal Code

1211

Country

Switzerland

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Taipei

ZIP/Postal Code

103616

Country

Taiwan

Individual Site Status

Recruiting

Facility Name

Novartis Investigative Site

City

Istanbul

State/Province

TUR

ZIP/Postal Code

34098

Country

Turkey

Individual Site Status

Recruiting

12. IPD Sharing Statement

Plan to Share IPD

Yes

IPD Sharing Plan Description

Novartis is committed to sharing with qualified external researchers, access to patient-level data and supporting clinical documents from eligible studies. These requests are reviewed and approved by an independent review panel on the basis of scientific merit. All data provided is anonymized to respect the privacy of patients who have participated in the trial in line with applicable laws and regulations. This trial data availability is according to the criteria and process described on www.clinicalstudydatarequest.com

Learn more about this trial

Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies

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Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies (V-PEDS-OLE)

Heterozygous or Homozygous Familial Hypercholesterolemia

About this trial

Eligibility Criteria

Sites / Locations

Arms of the Study

Arm 1

Outcomes

Primary Outcome Measures

Secondary Outcome Measures

Full Information

1. Study Identification

2. Study Status

3. Sponsor/Collaborators

4. Oversight

5. Study Description

6. Conditions and Keywords

7. Study Design

8. Arms, Groups, and Interventions

10. Eligibility

12. IPD Sharing Statement

Learn more about this trial