Open-Label Extension (OLE) Study to Evaluate the Safety of Efgartigimod in Adult Patients With Primary Sjögren’s Syndrome (pSS)

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Argenx

Participant type

Patients

Age range

18-64 years

Gender

Male and Female

Therapeutic area

Phenomena and Processes [G] - Immune system processes [G12]

Trial duration

15 Nov 2023 → 3 Feb 2025

Decision date (initial)

2023-09-25

Transition trial

No

Low-intervention

No

Rare-disease indication

No

Vulnerable population

Yes

Funding sources

argenx BV

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Safety, Pharmacokinetic, Pharmacodynamic

To evaluate the long-term safety of efgartigimod in patients with pSS

Secondary objectives 6

1. To evaluate effect and assess long-term data on durability of CRESS response
2. To evaluate the effect of efgartigimod on clinical efficacy parameters
3. To evaluate the effect of efgartigimod on STAR
4. To assess the PD effect of efgartigimod
5. To assess the exposure to efgartigimod
6. To assess the immunogenicity of efgartigimod

Conditions and MedDRA coding

Primary Sjögren's Syndrome

Study design 1 period

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 4

1. The Participant is at least the legal age of consent for clinical trials when signing the ICF
2. The Participant is capable of providing signed informed consent, and complying with protocol requirements
3. The Participant agrees to use contraceptive measures consistent with local regulations and the following: a. WOCBP must have a negative urine pregnancy test at baseline before receiving IMP
4. The Participant has completed the qualifying efgartigimod pSS studies and agrees to continue study drug treatment without interruption in the extension study

Exclusion criteria 3

1. The Participant has clinically significant disease (including newly diagnosed malignancy or cardiovascular disease) or intention to have surgery during the study; or any other medical condition that, in the investigator’s opinion, would confound the results of the study or put the participant at undue risk
2. The Participant is pregnant or intention to become pregnant during the study
3. The Participant has any severe systemic pSS manifestation that may put the participant at undue risk based on the investigator’s opinion

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 1

1. Incidence and severity of AEs and AESIs, incidence SAEs, changes in laboratory test results, vital signs, and ECG results

Secondary endpoints 14

1. Proportion of CRESS responders on ≥ 3 of 5 items at weeks 24 and 48
2. Proportion of participants with minimal clinically important improvement from baseline in ESSDAI: improvement of ≥ 3 points in ESSDAI score at weeks 24 and 48
3. Proportion of participants with low disease activity: ESSDAI score of < 5 at weeks 24 and 48
4. Proportion of participants with minimal clinically important improvement from baseline in clinESSDAI: improvement of ≥ 3 points in clinESSDAI score at weeks 24 and 48
5. Proportion of participants with low disease activity: clinESSDAI score of < 5 at weeks 24 and 48
6. Proportion of participants with minimal clinically important improvement from baseline in ESSPRI: decrease of ≥ 1 point or ≥ 15% at weeks 24 and 48
7. Change from baseline in ESSDAI score at weeks 24 and 48
8. Change from baseline in clinESSDAI score at weeks 24 and 48
9. Change from baseline in ESSPRI score at weeks 24 and 48
10. Proportion of STAR responders (score of ≥ 5) at weeks 24 and 48 when compared to baseline
11. Values, changes from baseline, and percent reduction from baseline in total IgG levels in serum over the 48 week treatment period
12. Values, changes from baseline, and percent reduction from baseline in autoantibodies in serum over the 48-week treatment period: Anti-Ro/SS A; Anti-La/SS B
13. Efgartigimod serum concentrations over the 48-week treatment period
14. Incidence and prevalence of ADA against efgartigimod over the 48-week treatment period

Investigational products

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

Test 1

Sponsors and contacts

Sponsor organisations, regulatory contacts, third parties

Argenx

Sponsor organisation

Argenx

Address

Industriepark-Zwijnaarde 7

City

Gent

Postcode

9052

Country

Belgium

Scientific contact point

Organisation

Argenx

Contact name

Peter Ulrichts PhD

Public contact point

Organisation

Argenx

Contact name

Sabine Coppieters, MD

Third parties 2

Locations

3 EU/EEA countries · 17 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

Summary of results Art. 37(4) CTR

Layperson summary Annex V

Documents 46 files

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

Application history

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