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Trial details
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia
Current status:
Approved
|
Date registered:
30/12/2021
Trial version(s)
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
History: 25/03/2021
Current: 25/03/2021
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Main Information
Primary registry identifying number
LBCTR2021034776
Protocol number
CKJX839C12301
MOH registration number
Trial already registered with the MoPH
Study registered at the country of origin
Type of registration
Prospective
Date of registration in national regulatory agency
Primary sponsor
Novartis Pharma Services inc.
Primary sponsor: Country of origin
Novartis Pharmaceuticals
Public title
Study to Evaluate Efficacy and Safety of Inclisiran in Adolescents With Heterozygous Familial Hypercholesterolemia
Acronym
Scientific title
Two Part (Double-blind Inclisiran Versus Placebo [Year 1] Followed by Open-label Inclisiran [Year 2]) Randomized Multicenter Study to Evaluate Safety, Tolerability, and Efficacy of Inclisiran in Adolescents (12 to Less Than 18 Years) With Heterozygous Familial Hypercholesterolemia and Elevated LDL-cholesterol (ORION-16)
Acronym
Brief summary of the study: English
This is a pivotal phase III study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C). This is a two-part (1 year double-blind inclisiran versus placebo / 1 year open-label inclisiran) multicenter study designed to evaluate safety, tolerability, and efficacy of inclisiran in adolescents with heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C) on stable standard of care background lipid-lowering therapy. The primary objective is to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330.
Brief summary of the study: Arabic
دراسة متعددة المراكز، عشوائيّة التوزيع من قسمين (إنكليسيران مزدوج التعمية مقابل الدواء الوهمي [السنة 1] يتبعه إنكليسيران مفتوح اللصاقة [السنة 2]) لتقييم سلامة إنكليسيران وقدرة تحمّله وفعاليّته لدى المراهقين (من 12 إلى أقل من 18 سنة) المصابين بفرط كوليسترول الدم العائلي متغاير الزيجوت وبارتفاع الكوليسترول الضار (أوريون-16) (ORION-16)
Health conditions/problem studied: Specify
Heterozygous Familial Hypercholesterolemia
Interventions: Specify
Drug: Inclisiran Drug: Placebo
Key inclusion and exclusion criteria: Inclusion criteria
Heterozygous Familial Hypercholesterolemia (HeFH) diagnosed either by genetic testing or on phenotypic criteria Fasting LDL-C >130 mg/dL (3.4 mmol/L) at screening Fasting triglycerides <400 mg/dL (4.5 mmol/L) at screening On maximally tolerated dose of statin (investigator's discretion) with or without other lipid-lowering therapy; stable for ≥ 30 days before screening Estimated glomerular filtration rate (eGFR) >30 mL/min/1.73 m2 at screening
Key inclusion and exclusion criteria: Gender
Both
Key inclusion and exclusion criteria: Age minimum
12
Key inclusion and exclusion criteria: Age maximum
17
Key inclusion and exclusion criteria: Exclusion criteria
Homozygous familial hypercholesterolemia (HoFH) Active liver disease Secondary hypercholesterolemia, e.g. hypothyroidism or nephrotic syndrome Major adverse cardiovascular events within 3 months prior to randomization Previous treatment with monoclonal antibodies directed towards PCSK9 (within 90 days of screening) Recent and/or planned use of other investigational medicinal products or devices Other protocol-defined inclusion/exclusion criteria may apply
Type of Study
Type
Interventional
Type of intervention
Pharmaceutical
Trial scope
Therapy
Trial scope: Specify scope
Study design: Allocation
Randomized controlled trial
Study design: Masking
Blinded (masking used)
Study design: Control
Placebo
Study phase
3
Study design: Purpose
Treatment
Study design: Assignment
Parallel
IMP has market authorization
Yes, Worldwide
IMP has market authorization: Specify the countries
European Union, United Arab Emirates, Great Britain
Name of IMP
inclisiran
Type of IMP
Others
Type of IMP: Specify
chemically synthesized double-stranded oligonucleotide
Pharmaceutical class
cholesterol-lowering small interfering ribonucleic acid (siRNA) that inhibits the production of proprotein convertase subtilisin/kexin type 9
Therapeutic indication
heterozygous familial hypercholesterolemia (HeFH) and elevated low density lipoprotein cholesterol (LDL-C)
Therapeutic benefit
to demonstrate superiority of inclisiran compared to placebo in reducing LDL-C (percent change) at Day 330 in adolescents (12 to less than 18 years) with heterozygous familial hypercholesterolemia and elevated LDL-cholesterol
Biospecimen retention
Samples with DNA**
Biospecimen description
Blood samples collected will be analyzed at Medpace Laboratories, central lab
Target sample size
4
Actual enrollment target size
1
Date of first enrollment: Type
Anticipated
Date of first enrollment: Date
31/08/2021
Date of study closure: Type
Anticipated
Date of study closure: Date
16/01/2024
Recruitment status
Recruiting
Date of completion
28/06/2022
IPD sharing statement plan
Yes
IPD sharing statement 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.
Additional data URL
https://clinicaltrials.gov/ct2/show/NCT04652726?cond=heterozygous+familial+hypercholesterolemia&draw=2&rank=2
Summary Results
Secondary Identifying Numbers
Full name of issuing authority
Secondary identifying number
NCT04652726
Clinical trials.gov
Sources of Monetary or Material Support
Name
Novartis Pharma Services inc.
Secondary Sponsors
Name
NA
Contact for Public/Scientific Queries
Contact type
First name
Last name
Address
Country
Telephone
Email
Affiliation
Public
Selim
Jambart
Beirut
Lebanon
961 3 406 001
sjambart@dm.net.lb
Hotel Dieu De France
Scientific
Hind
Khairallah
Sinelfil
Lebanon
01512002#271
Hind.khairallah@fattal.com.lb
Khalil Fattal et Fils s.a.l.
Public
Hala
Tfayli
Beirut
Lebanon
71729759
HT31@AUB .EDU .LB
American University of Beirut Medical Center
Centers/Hospitals Involved in the Study
Center/Hospital name
Name of principles investigator
Principles investigator speciality
Ethical approval
Hotel Dieu De France
Selim Jambart
Endocrinology
Approved
American University of Beirut Medical Center
Hala Tfayli
Pediatric Endocrinology
Approved
Ethics Review
Ethics approval obtained
Approval date
Contact name
Contact email
Contact phone
Hotel Dieu de France
21/12/2020
Sami Richa
cue@usj.edu.lb
961421229
American University of Beirut Medical Center
28/06/2021
Fuad Ziyadeh
irb@aub.edu.lb
00961 -1-350000 or 1 374374, ext: 5445
Countries of Recruitment
Name
Lebanon
Australia
Germany
Hungary
Norway
Spain
United States of America
Health Conditions or Problems Studied
Condition
Code
Keyword
heterozygous familial hypercholesterolemia
Hyperlipidaemia, unspecified (E78.5)
heterozygous familial hypercholesterolemia
Interventions
Intervention
Description
Keyword
Informed Consent/assent form discussion; Inclusion/exclusion assessment; physical examination; neurological examination; blood and urine samples collection; IMP dispensation
Informed Consent/assent form discussion; Inclusion/exclusion assessment; physical examination; neurological examination; blood and urine samples collection; IMP dispensation
ICF, IMP , Lab tests, physical exams
Primary Outcomes
Name
Time points
Measure
Percentage (%) change in low-density lipoprotein cholesterol (LDL-C)
baseline to Day 330
baseline to Day 330
Demonstrate superiority of inclisiran compared to placebo in reducing LDL-C [percent change]
Day 330 (Year 1)
Day 330 (Year 1)
Key Secondary Outcomes
Name
Time points
Measure
Time-adjusted % change in LDL-C from baseline
Baseline, after Day 90 up to Day 330
Baseline, after Day 90 up to Day 330
Absolute change in LDL-C from baseline to Day 330
Baseline and Day 330
Baseline and Day 330
% change in apolipoprotein B (Apo B), lipoprotein (a) [Lp(a)], non-high density lipoprotein cholesterol (non-HDL-C), and total cholesterol from baseline to Day 330
Baseline and Day 330
Baseline and Day 330
% change and absolute change in LDL-C from baseline up to Day 720
Baseline, up to Day 720
Baseline, up to Day 720
% change and absolute change in other lipoproteins and lipid parameters
Baseline, up to Day 720
Baseline, up to Day 720
% change and absolute change in proprotein convertase subtilisin/kexin type 9 (PCSK9)
Baseline, up to Day 720
Baseline, up to Day 720
Trial Results
Summary results in Lebanon
Study results globally
Date of posting of results summaries
Date of first journal publication of results
Results URL link
Baseline characteristics
Participant flow
Adverse events
Outcome measures
URL to protocol files
Link(s) to publications related to the study
Changes History
Change
Date
Annual submission 2021
23/12/2021
Download as PDF
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