Long-term safety and tolerability of inclisiran in participants with heterozygous familial hypercholesterolaemia or homozygous familial hypercholesterolaemia who have completed the adolescent ORION-16 or ORION-13 studies

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Pediatric, Patients

Age range

0-17 years, 18-64 years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

10 Feb 2023 → ongoing

Decision date (initial)

2024-11-28

Transition trial

Yes

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2023-507278-41-00

EudraCT number

2022-002316-23

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Others, Safety, Efficacy

The primary objective of the study is to evaluate the long-term safety and tolerability of inclisiran in participants with HeFH or HoFH.

Secondary objectives 1

1. To evaluate the long-term effect of inclisiran treatment on LDL-C levels

Conditions and MedDRA coding

Familial hypercholsterolemia

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Male and female participants with a diagnosis of HeFH or HoFH who completed the ORION-16 or ORION-13 studies, meaning the participant received the last dose of study drug and completed the final study visit as per applicable protocol
2. Per investigator`s clinical judgment, participant derived benefit from treatment with inclisiran in the ORION-16 or ORION-13 studies
3. Continuing current lipid-lowering therapies (such as e.g. statin and/or ezetimibe) from the feeder study with no planned medication or dose change during study participation

Exclusion criteria 3

1. Participants who in the preceding 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
2. 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
3. Active liver disease defined as any known current infectious, neoplastic, or metabolic pathology of the liver

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Treatment-emergent adverse events (TEAEs) and serious adverse events (TESAEs) (incidence, severity, relationship to study drug and discontinuation due to TEAEs); vital signs; growth; laboratory parameters

Secondary endpoints 1

1. Percentage change and absolute change in LDL-C from baseline in the feeder study to end of study (EoS)

Investigational products

Investigational medicinal products (IMPs), comparators, placebo, auxiliary

Test 1

Auxiliary 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel Town

Postcode

4056

Country

Switzerland

Scientific contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Public contact point

Organisation

Novartis Pharma AG

Contact name

Novartis Pharma Arzneimittel GmbH

Third parties 14

Locations

12 EU/EEA countries · 22 investigational sites

By country

Investigational sites

Country notifications

Trial-start, recruitment-start, end and early-termination notifications submitted per Member State

Results and documents

Annex IV summary of results, Annex V layperson summary, and all documents registered in CTIS for this trial

Documents 99 files

Public protocol annexes, IB summaries, regulatory submissions and post-authorisation documents registered in CTIS.

Application history

20 submissions — initial application plus substantial / non-substantial modifications