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)

December 27, 2023 updated by: Novartis Pharmaceuticals

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)

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.

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

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.

Study Type

Interventional

Enrollment (Estimated)

165

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Contact Backup

  • Name: Novartis Pharmaceuticals
  • Phone Number: +41613241111

Study Locations

  • Brazil
    • Ceara
      • Fortaleza, Ceara, Brazil, 60430275
        • Recruiting
        • Novartis Investigative Site
    • SP
      • Sao Paulo, SP, Brazil, 05403 000
        • Recruiting
        • Novartis Investigative Site
      • Sao Paulo, SP, Brazil, 04023-900
        • Recruiting
        • Novartis Investigative Site
  • Canada
      • Quebec, Canada, G1V 4W2
        • Recruiting
        • Novartis Investigative Site
  • France
      • Toulouse Cedex, France, 31059
        • Recruiting
        • Novartis Investigative Site
  • Germany
      • Frankfurt, Germany, 60590
        • Recruiting
        • Novartis Investigative Site
      • Freiburg, Germany, 79100
        • Recruiting
        • Novartis Investigative Site
  • Hungary
      • Pecs, Hungary, 7623
        • Recruiting
        • Novartis Investigative Site
  • Israel
      • Jerusalem, Israel, 9112001
        • Recruiting
        • Novartis Investigative Site
      • Ramat Gan, Israel, 52621
        • Recruiting
        • Novartis Investigative Site
  • Italy
    • MI
      • Milano, MI, Italy, 20162
        • Recruiting
        • Novartis Investigative Site
    • RM
      • Roma, RM, Italy, 00100
        • Recruiting
        • Novartis Investigative Site
  • Lebanon
      • Ashrafieh, Lebanon, 166830
        • Recruiting
        • Novartis Investigative Site
  • Netherlands
      • Amsterdam, Netherlands, 1105 AZ
        • Recruiting
        • Novartis Investigative Site
    • Zuid Holland
      • Rotterdam, Zuid Holland, Netherlands, 3015 GD
        • Recruiting
        • Novartis Investigative Site
  • Norway
      • Oslo, Norway, 0514
        • Recruiting
        • Novartis Investigative Site
  • Russian Federation
      • Kemerovo, Russian Federation, 650002
        • Recruiting
        • Novartis Investigative Site
      • Moscow, Russian Federation, 127412
        • Recruiting
        • Novartis Investigative Site
  • Slovenia
      • Ljubljana, Slovenia, 1000
        • Recruiting
        • Novartis Investigative Site
  • South Africa
    • Free State
      • Bloemfontein, Free State, South Africa, 9301
        • Recruiting
        • Novartis Investigative Site
    • Western Cape
      • Cape Town, Western Cape, South Africa, 7130
        • Recruiting
        • Novartis Investigative Site
  • Spain
      • A Coruna, Spain, 15001
        • Recruiting
        • Novartis Investigative Site
    • Asturias
      • Oviedo, Asturias, Spain, 33011
        • Recruiting
        • Novartis Investigative Site
    • Navarra
      • Pamplona, Navarra, Spain, 31008
        • Recruiting
        • Novartis Investigative Site
  • Switzerland
      • Geneve 14, Switzerland, 1211
        • Recruiting
        • Novartis Investigative Site
  • Taiwan
      • Taipei, Taiwan, 103616
        • Recruiting
        • Novartis Investigative Site
  • Turkey
      • Izmir, Turkey, 35040
        • Recruiting
        • Novartis Investigative Site
    • TUR
      • Istanbul, TUR, Turkey, 34098
        • Recruiting
        • Novartis Investigative Site
  • United Kingdom
      • Middlesex, United Kingdom, UB9 6JH
        • Recruiting
        • Novartis Investigative Site
  • United States
    • Florida
      • Boca Raton, Florida, United States, 33434
        • Recruiting
        • Novartis Investigative Site
    • New York
      • New York, New York, United States, 10029
        • Recruiting
        • Novartis Investigative Site

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

12 years and older (Child, Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

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

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Inclisiran
Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution
Drug: Inclisiran
Inclisiran sodium 300mg (equivalent to 284mg inclisiran*) in 1.5mL solution administered subcutaneously in pre-filled syringe
Other Names:
  • KJX839

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs)
Time Frame: From Day 1 in the study up to the end of study visit; up to 1080 days
Safety and tolerability: TEAEs, TESAEs (incidence, severity, relationship to study drug and discontinuation due to TEAEs)
From Day 1 in the study up to the end of study visit; up to 1080 days

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Percentage and absolute change in LDL-C from baseline in the feeder study to end of study
Time Frame: Baseline (of feeder study) and Day 1080
Evaluate the long-term effect of inclisiran (from baseline of feeder study to end of study) in lowering LDL-C
Baseline (of feeder study) and Day 1080

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Novartis Pharmaceuticals

Investigators

  • Study Director: Novartis Pharmaceuticals, Novartis Pharmaceuticals

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

February 10, 2023

Primary Completion (Estimated)

December 1, 2027

Study Completion (Estimated)

December 1, 2027

Study Registration Dates

First Submitted

December 27, 2022

First Submitted That Met QC Criteria

December 27, 2022

First Posted (Actual)

January 12, 2023

Study Record Updates

Last Update Posted (Actual)

December 28, 2023

Last Update Submitted That Met QC Criteria

December 27, 2023

Last Verified

December 1, 2023

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • CKJX839C12001B
  • 2022-002316-23 (EudraCT Number)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

YES

IPD 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

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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