An open-label extension (OLE) trial to evaluate long-term safety and tolerability of pelacarsen (TQJ230)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Novartis Pharma AG

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Cardiovascular Diseases [C14]

Trial duration

11 May 2026 → ongoing

Decision date (initial)

2025-08-22

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

External identifiers

EU CT number

2024-519870-38-00

WHO UTN

U1111-1319-9494

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Safety, Therapy

To evaluate the long-term safety and tolerability of pelacarsen (TQJ230) in participants with elevated Lp(a) and established CVD who have completed the parent Lp(a)HORIZON study

Secondary objectives 2

1. To evaluate the long-term efficacy of pelacarsen (TQJ230) on 4P-MACE (cardiovascular death, nonfatal myocardial infarction, non-fatal stroke, and urgent coronary revascularization requiring hospitalization)
2. To evaluate the long-term efficacy of pelacarsen (TQJ230) in lowering Lp(a) levels

Conditions and MedDRA coding

Atherosclerotic cardiovascular disease (ASCVD)

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Participants who have provided informed consent prior to initiation of any study-specific activities/procedures.
2. Participants who have completed the parent study EOS visit while still on assigned investigational product

Exclusion criteria 5

1. Participants who for any reason permanently discontinued or have interrupted the investigational product for continuous 6 months at EOS during the parent study
2. Participants who have a history or evidence of any clinically significant disorder, condition, or disease that in the opinion of the investigator or Novartis physician (if consulted), would put the participant at risk or interfere with the study participation, including, but not restricted to conditions outlined in Table 6-3 and Table 6-5
3. Participants are receiving another investigational drug or device before the open-label treatment period
4. Pregnant or nursing (breastfeeding) women
5. Women of childbearing potential unless they are using highly effective methods of contraception

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. ● Occurrence of the followings: • Treatment Emergent Adverse Events (TEAEs) • Treatment Emergent Serious Adverse Events (TESAEs) • Treatment discontinuations due to TEAEs/TESAEs
2. ● TEAEs of special interest Observed value or change/shift from baseline in: • Safety laboratory measures • Vital signs

Secondary endpoints 6

1. Time to the first occurrence of 4P-MACE from parent study baseline
2. Time to the first occurrence of 4P-MACE from OLE study baseline
3. Cumulative number of 4P-MACE over time from parent study baseline
4. Cumulative number of 4P-MACE over time from OLE study baseline
5. Observed value and change in Lp(a) from the parent study baseline
6. Observed value and change in Lp(a) from the OLE study baseline

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

Sponsor organisation

Novartis Pharma AG

Address

Lichtstrasse 35

City

Basel

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 12

Locations

20 EU/EEA countries · 253 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 121 files

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

Application history

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