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

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

29 Sep 2023 → ongoing

Decision date (initial)

2023-09-13

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

Novartis Pharma AG

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Efficacy, Therapy, Safety

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

Secondary objectives 2

1. To evaluate the long-term effect of pelacarsen on the need for lipoprotein apheresis
2. To evaluate the long-term effect of pelacarsen on Lp(a) change

Conditions and MedDRA coding

Apheresis

Regulatory references

Plan to share IPD

Yes

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 2

1. Signed informed consent must be obtained prior to participation in the OLE study and before any study’s assessment is performed
2. Participant has successfully completed the parent study and is considered safe to participate by Investigator's clinical judgement [In case of a premature study treatment permanent discontinuation from the parent study for medical/safety reasons prior to week 52, participant is not considered as having successfully completed the parent study]

Exclusion criteria 3

1. Any medical condition(s) that may put the participant at risk in the investigator’s opinion, or that the investigator deems unsuitable for the study
2. Pregnant or nursing (lactating) women
3. Women of child-bearing potential, defined as all women physiologically capable of becoming pregnant, unless they are using highly-effective methods of contraception during dosing and for 16 weeks (= 5 times the terminal half-life) after stopping of study medication. Such highly-effective contraception methods include: • Total abstinence (when this is in line with the preferred and usual lifestyle of the participant). Periodic abstinence (e.g., calendar, ovulation, symptothermal, post-ovulation methods) and withdrawal are not acceptable methods of contraception • Female sterilization (have had surgical bilateral oophorectomy with or without hysterectomy) total hysterectomy or bilateral tubal ligation at least six weeks before taking investigational drug. In case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment • Male sterilization (at least 6 months prior to screening). For female participants on the study, the vasectomized male partner should be the sole partner for that participant. • Use of oral, (estrogen and progesterone), injected or implanted hormonal methods of contraception or placement of an intrauterine device (IUD) or intrauterine system (IUS) or other forms of hormonal contraception that have comparable efficacy (failure rate <1%), for example hormone vaginal ring or transdermal hormone contraception In case of use of oral contraception women should have been stable on the same pill for a minimum of 3 months before taking the investigational drug. Women are considered post-menopausal and not of childbearing potential if they have had 12 months of natural (spontaneous) amenorrhea with an appropriate clinical profile (e.g. age appropriate, history of vasomotor symptoms). Women are considered not of child-bearing potential if they are post-menopausal or have had surgical bilateral oophorectomy (with or without hysterectomy), total hysterectomy or bilateral tubal ligation at least six weeks prior to enrollment on study. In the case of oophorectomy alone, only when the reproductive status of the woman has been confirmed by follow up hormone level assessment is she considered not of childbearing potential

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Adverse events (AEs) or serious adverse events (SAEs), laboratory evaluations, vital signs and duration of drug exposure

Secondary endpoints 2

1. Number of lipoprotein apheresis sessions performed yearly for year 1, 2, 3, 4 and 5
2. Absolute and percentage change in Lp(a) on month 1, month 3, month 6, month 9, month 12, then every 6 months during OLE compared to baseline of the parent study

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 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 8

Locations

1 EU/EEA country · 9 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 9 files

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

Application history

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