A multicenter trial assessing the impact of lipoprotein(a) lowering with pelacarsen (TQJ230) on the rate of weekly lipoprotein apheresis sessions in patients with hyperlipoproteinemia(a) and established cardiovascular disease in Germany.

2023-508324-35-00 Protocol CTQJ230A12302 Therapeutic confirmatory (Phase III) Ended

Start 19 Aug 2022 · End 29 Jan 2025 · Status Ended · 1 EU/EEA countries · 10 sites · Protocol CTQJ230A12302

Overview

Sponsor-declared trial summary

Phase Therapeutic confirmatory (Phase III)
Status Ended
Participants planned 51
Countries 1
Sites 10

Apheresis

Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the rate of lipoprotein apheresis sessions in patients with hyperlipoproteinemia(a) and established CVD during 52 weeks of treatment

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
19 Aug 2022 → 29 Jan 2025
Decision date (initial)
2024-04-24
Transition trial
Yes
Low-intervention
No
Rare-disease indication
No
Vulnerable population
Yes
Funding sources
Novartis Pharma AG

External identifiers

EU CT number
2023-508324-35-00
EudraCT number
2021-003059-41
ClinicalTrials.gov
NCT05305664

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Therapy, Safety, Efficacy, Pharmacogenetic

Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the rate of lipoprotein apheresis sessions in patients with hyperlipoproteinemia(a) and established CVD during 52 weeks of treatment

Secondary objectives 3

  1. Demonstrate superiority of pelacarsen (TQJ230) compared to placebo in reducing the time to lipoprotein apheresis avoidance
  2. Demonstrate superiority of pelacarsen (TQJ230) vs placebo in avoiding the performance of any lipoprotein apheresis in the last 40 weeks of the study
  3. Demonstrate superiority of pelacarsen (TQJ230) vs placebo in lowering Lp(a) after 52 weeks

Conditions and MedDRA coding

Apheresis

VersionLevelCodeTermSystem organ class
21.1 LLT 10051614 Arteriosclerotic cardiovascular disease 10047065
21.1 PT 10061653 Apheresis 100000004865

Regulatory references

Plan to share IPD
Yes
EU CT numberTitleSponsor
2023-503611-16-00 A single arm, open label extension (OLE), multicenter study to evaluate long-term safety and tolerability of pelacarsen (TQJ230) in patients with cardiovascular disease who have successfully completed the Frontiers Apheresis parent study Novartis Pharma AG

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

  1. Patients currently undergoing lipoprotein apheresis for isolated Lp(a) on a weekly schedule in Germany for ≥ 12 months prior to screening with at least 35 sessions within the past 52 weeks prior to randomization
  2. Lipoprotein(a) (Lp(a))> 60 mg/dL at screening
  3. Spontaneous prior myocardial infarction (MI): ≥ 3 months prior to the screening visit, and/or Ischemic stroke: ≥ 3 months prior to the screening visit, and/or Clinically significant symptomatic peripheral artery disease (PAD) and/or Clinically significant symptomatic coronary artery disease (CAD)

Exclusion criteria 8

  1. Uncontrolled hypertension
  2. Heart failure New York Heart Association (NYHA) class IV
  3. History of malignancy of any organ system
  4. History of hemorrhagic stroke or other major bleeding
  5. Platelet count <140,000 per mm3 at screening
  6. Active liver disease or hepatic dysfunction
  7. Significant kidney disease
  8. Pregnant or nursing women

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

  1. Rate of lipoprotein apheresis sessions performed over 52 weeks normalized to the weekly lipoprotein apheresis schedule

Secondary endpoints 3

  1. Time to lipoprotein apheresis avoidance (where lipoprotein apheresis avoidance is defined as at least 24 weeks of no lipoprotein apheresis until end of study)
  2. Total avoidance of lipoprotein apheresis from week 12 to week 52
  3. Change from baseline to week 52 in the log-transformed Lp(a) (measured prior to planned lipoprotein apheresis)

Investigational products

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

Test 1

TQJ230

PRD10213050 · Product

Active substance
Pelacarsen
Pharmaceutical form
SOLUTION FOR INJECTION IN PRE-FILLED SYRINGE
Route of administration
SUBCUTANEOUS USE
Max daily dose
80 mg milligram(s)
Max total dose
1120 mg milligram(s)
Max treatment duration
52 Week(s)
Authorisation status
Not Authorised
MA holder
NOVARTIS PHARMA AG
Paediatric formulation
No
Orphan designation
No

Placebo 1

Placebo to TQJ230 80 mg/0.8 mL Solution for injection in pre-filled syringe with needle safety device

N/A · Product

Other product name
N/A
Pharmaceutical form
N/A
ATC code
N/A — N/A
Marketing authorisation
N/A
MA holder
N/A
MA country
Iceland
Paediatric formulation
No

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Novartis Pharma AG

220 Total trials 69 Recruiting 130 Ended
Commercial
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 9

OrganisationCity, countryDuties
Icon Clinical Research Limited
ORG-100008322
 Dublin 18, Ireland On site monitoring
IQVIA Limited
ORG-100008655
 Reading, United Kingdom On site monitoring
RWS Life Sciences Inc.
ORG-100042348
 East Hartford, United States Other
MEDPACE LABORATORIES
ORG-100042942
 Leuven, Belgium Laboratory analysis
Medidata Solutions Inc.
ORG-100016256
 New York, United States E-data capture, Code 8
Syneos Health Inc.
ORG-100008382
 Morrisville, United States On site monitoring
Parexel International (IRL) Limited
ORG-100022780
 Dublin 2, Ireland Code 12
Iqvia Inc.
ORG-100010622
 Durham, United States Other, Interactive response technologies (IRT)
Kayentis
ORG-100037894
 Meylan, France Other, E-data capture

Locations

1 EU/EEA country · 10 investigational sites

By country

CountryMS statusPlanned subjectsSites
Germany Ended 51 10
Rest of world — 0 —

Investigational sites

Germany

10 sites · Ended
Universitaetsmedizin der Johannes Gutenberg-Universitaet Mainz KöR
#1006: Medizinische Klinik und Poliklinik, Langenbeckstrasse 1, Oberstadt, Mainz
Universitaetsklinikum Ulm AöR
#1008: Nephrologie, Albert-Einstein-Allee 23, Eselsberg, Ulm
DaVita Clinical Research Deutschland GmbH
#1015: Clinical Research, Bismarckstrasse 101, Stadtmitte, Duesseldorf
Universitaetsklinikum Erlangen AöR
#1017: Nephrologie und Bluthochdruck, Ulmenweg 18, Innenstadt, Erlangen
Universitaetsklinikum Carl Gustav Carus Dresden an der Technischen Universitaet Dresden AöR
#1003: Technische Universitaet, Fetscherstrasse 74, Johannstadt-Nord, Dresden
Agaplesion Frankfurter Diakonie Kliniken gGmbH
#1009: Medizinische Klinik II, Wilhelm-Epstein-Strasse 4, Bockenheim, Frankfurt Am Main
Klinikum der Universitaet Muenchen AöR
#1001: Medizinische Klinik und Poliklinik, Marchioninistrasse 15, Hadern, Munich
St. Josefs-Hospital Cloppenburg gGmbH
#1013: Lipid-Ambulanz, Krankenhausstrasse 13, 49661, Cloppenburg
Universitaetsklinikum Wuerzburg AöR
#1016: Transplantationszentrum, Oberduerrbacher Strasse 6, Grombuehl, Wuerzburg
Charite Universitaetsmedizin Berlin KöR
#1002: Universitaetsmedizin, Augustenburger Platz 1, Wedding, Berlin

Country notifications

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

Country Trial startTrial end Recruitment startRecruitment end Early termination
Germany 2022-08-19 2025-01-28 2022-08-19 2024-01-29

Results and documents

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

Summary of results Art. 37(4) CTR

TitleSubmission dateStatusType
CTQJ230A12302 summary of results
SUM-111467
 2025-12-16T18:06:26 Submitted Summary of Results

Layperson summary Annex V

TitleSubmission dateStatusType
CTQJ230A12302_PatientSummary_English-US 2026-01-16T14:15:53 Submitted Laypersons Summary of Results
CTQJ230A12302 - Patient Summary - German-Germany 2026-03-02T15:31:06 Submitted Laypersons Summary of Results

Documents 7 files

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

TypeTitleVersion
Laypersons summary of results (for publication) CTQJ230A12302_PatientSummary_English-US_15Jan2026 1
Laypersons summary of results (for publication) CTQJ230A12302_PatientSummary_German-Germany 1
Protocol (for publication) D1_Protocol - Signature Page_1_English_Red 21Mar2024
Protocol (for publication) D1_Protocol_2023-508324-35-00_1_English_Red 03
Protocol (for publication) D4_Patient-facing document - PRO_1_German_NonRed 1.1
Summary of results (for publication) CTQJ230A12302 EU CTIS 1
Synopsis of the protocol (for publication) D1_Protocol Summary in Lay Language_2023-508324-35-00_1_English_NonRed 01

Application history

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

#TypeCodeSubmittedReference MSConclusionDecision date
1 INITIAL IN 2024-04-03 Germany Acceptable
2024-04-19
 2024-04-24
2 SUBSTANTIAL MODIFICATION SM-1 2024-06-13 Germany Acceptable
2024-06-19
 2024-07-25
3 NON SUBSTANTIAL MODIFICATION NSM-1 2024-12-04 Germany Acceptable
2024-06-19
 2024-12-04

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