An Open-label Extension Study of ARGX-113-2009 to Evaluate the Long-term Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Adult Participants With Bullous Pemphigoid

Overview

Sponsor-declared trial summary

Key facts

Sponsor

Argenx

Participant type

Patients

Age range

18-64 years, 65+ years

Gender

Male and Female

Therapeutic area

Diseases [C] - Immune System Diseases [C20]

Trial duration

3 May 2022 → 20 Mar 2025

Decision date (initial)

2024-10-25

Transition trial

Yes

Low-intervention

No

Rare-disease indication

Yes

Vulnerable population

Yes

Funding sources

Argenx BV

External identifiers

EU CT number

2024-515832-59-00

EudraCT number

2021-003063-10

ClinicalTrials.gov

NCT05681481

Trial design

CTIS Part I — objectives, methods, condition coding

Main objective

Scope: Others, Efficacy, Safety

To assess the long-term safety and
tolerability of treatment with efgartigimod
PH20 SC in participants with BP

Secondary objectives 4

1. To assess the long-term efficacy and durability of response with efgartigimod PH20 SC treatment in participants with BP
2. To evaluate the impact of efgartigimod PH20 SC treatment in reducing long-term glucocorticoid-associated toxicity in participants with BP
3. To evaluate the impact of efgartigimod PH20 SC treatment on QoL in participants with BP
4. To evaluate the PD and immunogenicity of efgartigimod PH20 SC in participants with BP

Conditions and MedDRA coding

Bullous Pemphigoid

Regulatory references

Plan to share IPD

No

Eligibility criteria

Principal inclusion / exclusion criteria as submitted by sponsor

Inclusion criteria 3

1. Has completed the week 36 visit of ARGX-113-2009
2. Is capable of providing signed informed consent and complying with protocol requirements
3. Agrees to use contraceptive measures consistent with local regulations and the following: a. Male participants: An acceptable method of contraception is a condom. All nonsterilized male participants must use this method from signing of the ICF until the date of the last dose of IMP. b. Women of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. WOCBP must use one of the contraception methods described in the protocol from signing the ICF until the last dose of IMP

Exclusion criteria 3

1. Clinically significant disease, recent major surgery (within 3 months of baseline), 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. Known hypersensitivity to IMP or 1 of its excipients
3. Permanently discontinued IMP in ARGX-113-2009 due to an AE considered related to IMP and for whom the benefit/risk balance is not considered positive

Endpoints

Primary and secondary outcome measures (English text)

Primary endpoints 2

1. • Incidence and severity of treatment-emergent AEs, SAEs, and AESIs
2. • Rate of treatment discontinuation because of safety concerns

Secondary endpoints 12

1. Proportions of participants achieving: − CR while off OCS for ≥8 weeks − CR or PR while off OCS for ≥8 weeks − CR while on minimal OCS therapy for ≥8 weeks. (Minimal OCS therapy is defined as ≤0.10 mg/kg/day of prednisone [or an equivalent dose of another OCS]) − CR while off both OCS and efgartigimod PH20 SC for ≥8 weeks − CR or PR while off both OCS and efgartigimod PH20 SC for ≥8 weeks
2. • Proportions of participants achieving: − CR while off OCS for ≥8 weeks − CR or PR while off OCS for ≥8 weeks − CR while on minimal OCS therapy for ≥8 weeks. (Minimal OCS therapy is defined as ≤0.10 mg/kg/day of prednisone [or an equivalent dose of another OCS]) − CR while off both OCS and efgartigimod PH20 SC for ≥8 weeksb − CR or PR while off both OCS and efgartigimod PH20 SC for ≥8 weeksb
3. Duration of sustained remission
4. Proportion of participants who relapse
5. Time to relapse
6. Incidence and severity of relapse
7. BPDAI activity scores, IGA-BP scores, and itch NRS over time
8. Rate of treatment failure
9. GTI-related scores, including the GTI-AIS, GTI-CWS, and GTI-SL over time
10. EQ-5D-5L, DLQI, and ABQoL scores over time
11. Percent change from baseline over time for anti-BP180 and anti-BP230 antibody levels
12. Incidence and prevalence of ADA

Investigational products

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

Test 1

Placebo 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

Chief Scientific Officer

Public contact point

Organisation

Argenx

Contact name

Vice President Clinical Development

Third parties 19

Locations

10 EU/EEA countries · 35 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 67 files

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

Application history

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