Clinical trial NCT05682378: Long-term Safety and Tolerability of Inclisiran in Participants With HeFH or HoFH Who Have Completed the Adolescent ORION-16 or ORION-13 Studies
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
Overall status: RECRUITING
Phase: PHASE3
Conditions: Heterozygous or Homozygous Familial Hypercholesterolemia
Keywords: KJX839
heterozygous familial hypercholesterolemia
homozygous familial hypercholesterolemia
familial hypercholesterolemia
FH
inclisiran
pediatric
Lead sponsor: Novartis
Sponsor class: INDUSTRY
First posted: 2023-01-12
Enrollment: 154
Sex: ALL
Minimum age (years): 12
Maximum age (years): 100
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

Eligibility criteria: 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

Primary outcomes:
measure: Number of participants with treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs)
timeFrame: From Day 1 in the study up to the end of study visit; up to 1080 days
Secondary outcomes:
measure: Percentage and absolute change in LDL-C from baseline in the feeder study to end of study
timeFrame: Baseline (of feeder study) and Day 1080
Other outcomes:
Locations:
city: Boca Raton
country: United States
facility: Excel Medical Clinical Trials LLC
state: Florida
city: New York
country: United States
facility: Icahn School of Med at Mt Sinai
state: New York
city: Cincinnati
country: United States
facility: Cincinnati Childrens Hospital MC
state: Ohio
city: Pittsburgh
country: United States
facility: Childrens Hosp Pittsburgh UPMC
state: Pennsylvania
city: Salt Lake City
country: United States
facility: Primary Childrens Medical Center
state: Utah
city: Ciudad de Formosa
country: Argentina
facility: Novartis Investigative Site
state: Formosa
city: Fortaleza
country: Brazil
facility: Novartis Investigative Site
state: Ceara
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Sao Paulo
country: Brazil
facility: Novartis Investigative Site
state: SP
city: Quebec
country: Canada
facility: Novartis Investigative Site
city: Praha 5
country: Czechia
facility: Novartis Investigative Site
city: Praha
country: Czechia
facility: Novartis Investigative Site
city: Besancon Cedex
country: France
facility: Novartis Investigative Site
city: Toulouse Cedex
country: France
facility: Novartis Investigative Site
city: Frankfurt am Main
country: Germany
facility: Novartis Investigative Site
state: Hessen
city: Freiburg
country: Germany
facility: Novartis Investigative Site
city: Athens
country: Greece
facility: Novartis Investigative Site
city: Athens
country: Greece
facility: Novartis Investigative Site
city: Pecs
country: Hungary
facility: Novartis Investigative Site
city: Jerusalem
country: Israel
facility: Novartis Investigative Site
city: Ramat Gan
country: Israel
facility: Novartis Investigative Site
city: Milano
country: Italy
facility: Novartis Investigative Site
state: MI
city: Modena
country: Italy
facility: Novartis Investigative Site
state: MO
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Roma
country: Italy
facility: Novartis Investigative Site
state: RM
city: Irbid
country: Jordan
facility: Novartis Investigative Site
city: Ashrafieh
country: Lebanon
facility: Novartis Investigative Site
city: Sungai Buloh
country: Malaysia
facility: Novartis Investigative Site
state: Selangor Darul Ehsan
city: Kuala Lumpur
country: Malaysia
facility: Novartis Investigative Site
city: Rotterdam
country: Netherlands
facility: Novartis Investigative Site
state: Zuid Holland
city: Amsterdam
country: Netherlands
facility: Novartis Investigative Site
city: Oslo
country: Norway
facility: Novartis Investigative Site
city: Gdansk
country: Poland
facility: Novartis Investigative Site
city: Lodz
country: Poland
facility: Novartis Investigative Site
city: Kemerovo
country: Russian Federation
facility: Novartis Investigative Site
city: Moscow
country: Russian Federation
facility: Novartis Investigative Site
city: Poprad
country: Slovakia
facility: Novartis Investigative Site
city: Ljubljana
country: Slovenia
facility: Novartis Investigative Site
city: Bloemfontein
country: South Africa
facility: Novartis Investigative Site
state: Free State
city: Cape Town
country: South Africa
facility: Novartis Investigative Site
state: Western Cape
city: Cordoba
country: Spain
facility: Novartis Investigative Site
state: Andalucia
city: Oviedo
country: Spain
facility: Novartis Investigative Site
state: Asturias
city: Pamplona
country: Spain
facility: Novartis Investigative Site
state: Navarra
city: A Coruna
country: Spain
facility: Novartis Investigative Site
city: Geneve 14
country: Switzerland
facility: Novartis Investigative Site
city: Taipei
country: Taiwan
facility: Novartis Investigative Site
city: Istanbul
country: Turkey
facility: Novartis Investigative Site
state: TUR
city: Adana
country: Turkey
facility: Novartis Investigative Site
city: Ankara
country: Turkey
facility: Novartis Investigative Site
city: Izmir
country: Turkey
facility: Novartis Investigative Site
city: Middlesex
country: United Kingdom
facility: Novartis Investigative Site
Study officials:
affiliation: Novartis Pharmaceuticals
name: Novartis Pharmaceuticals
role: STUDY_DIRECTOR
References: