A Study to Assess the Long-term Safety and Efficacy of a Subcutaneous Formulation of Efgartigimod PH20 SC in Adults With Pemphigus (Vulgaris or Foliaceus)

Pemphigus Vulgaris Clinical Trial

— ADDRESS+  

Official title:

An Open-Label, Multicenter, Follow-up Trial of ARGX-113-1904 to Evaluate the Safety, Tolerability, and Efficacy of Efgartigimod PH20 SC in Patients With Pemphigus

Clinical Trial Details — Status: Terminated

Administrative data

• NCT number: NCT04598477
• Other study ID #: ARGX-113-1905
• Status: Terminated
• Phase: Phase 3
• Start date: July 15, 2021
• Est. completion date: March 25, 2024

Study information

• Verified date: April 2024
• Source: argenx
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

This is a prospective, multicenter, open label extension (OLE) trial on the efficacy, safety, patient outcome measures, tolerability, immunogenicity, PK and PD of efgartigimod PH20 SC in adult PV or PF participants, who participated in antecedent trial ARGX-113-1904. This trial provides extension of efgartigimod PH20 SC treatment and retreatment options for participants who have been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and the first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to placebo arm in trial ARGX-113-1904. Trial ARGX-113-1905 evaluates ability to (further) taper prednisone therapy and achieve Clinical Remission (CR) off therapy (CRoff), the ability to achieve CR and CR on minimal therapy (CRmin) for participants who had not yet achieved CRmin, and the ability to treat flare; and assess patient outcome measures and the safety, PD, PK and immunogenicity of efgartigimod PH20 SC over the duration of trial.

Study duration: Up to 60 weeks for participants who receive IMP administration up to 52 weeks and with a follow-up period of 8 weeks after the last IMP administration

Recruitment information / eligibility

• Status: Terminated
• Enrollment: 183
• Est. completion date: March 25, 2024
• Est. primary completion date: March 25, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

Inclusion Criteria:

1. Ability to understand the requirements of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), willingness and ability to comply with the trial protocol procedures (including required trial visits).

2. The participant participated in trial ARGX-113-1904 and completed the study or has the defined criteria for rollover.

3. Contraceptive use by men and women should be consistent with local regulations regarding the methods for contraception for those participating in clinical trials and:

1. Male participants:

Male participants must agree to use an acceptable method of contraception as described in the protocol, from signing the ICF until the last dose of the study drug.

2. Female participants

Women of childbearing potential (WOCBP) must:

• have a negative urine pregnancy test at baseline before the IMP can be administered,

• agree to use a highly effective or acceptable contraception method (as described in the protocol), which should be maintained at minimum until after the last dose of IMP

Exclusion Criteria:

1. Pregnant and lactating women and those intending to become pregnant during the trial.

2. Participants with clinical evidence of other significant serious disease or participants who recently underwent or have planned a major surgery during the period of the trial, or any other condition in the opinion of the investigator, that could confound the results of the trial or put the participant at undue risk.

3. Known hypersensitivity to any of the components of the administered treatments.

Related Conditions & MeSH terms

• Pemphigus
• Pemphigus Foliaceus
• Pemphigus Vulgaris

Intervention

Biological:
• efgartigimod PH20 SC
Subcutaneous injection of efgartigimod using rHuPH20 (PH20) as a permeation enhancer

Drug:
• prednisone
Oral prednisone tablets

Locations

Country	Name	City	State
Australia	Investigator site 92 - AU0610013	Melbourne
Australia	Investigator site 11 - AU0610007	Parkville	Victoria
Australia	Investigator site 15 - AU0610006	Sydney	New South Wales
Bulgaria	Investigator site 17 - BG3590012	Pleven
Bulgaria	Investigator site 18 - BG3590013	Plovdiv
Bulgaria	Investigator site 16 - BG3590009	Sofia
Bulgaria	Investigator site 2 - BG3590010	Sofia
Bulgaria	Investigator site 3 - BG3590011	Sofia
China	Investigator site 101 - CN0860017	Beijing
China	Investigator site 107 - CN0860018	Chengdu
China	Investigator site 118 - CH0860027	Chongqing
China	Investigator site 120 - CH0860023	Fuzhou
China	Investigator site 116 - CH0860053	Guangzhou
China	Investigator site 119 - CH0860022	Guangzhou
China	Investigator site 100 - CN0860021	Guanzhou
China	Investigator site 113 - CN0860024	Nanjing
China	Investigator site 102 - CN0860020	Shanghai
China	Investigator site 99 - CN0860016	Shanghai
China	Investigator site 112 - CN0860019	Wuhan
China	Investigator site 121 - CH0860025	Wuhan
China	Investigator site 117 - CH0860026	Zhengzhou
France	Investigator site 77 - FR0330028	Bobigny
France	Investigator site 60 - FR0330027	La Tronche
France	Investigator site 108 - FR0330029	Rouen
France	Investigator site 51 - FR0330026	Saint-Étienne
Georgia	Investigator site 127 - GE9950030	Tbilisi
Georgia	Investigator site 31 - GE9950015	Tbilisi
Georgia	Investigator site 32 - GE9950013	Tbilisi
Georgia	Investigator site 78 - GE9950014	Tbilisi
Germany	Investigator site 45 - DE0490029	Berlin
Germany	Investigator site 34 - DE0490030	Dresden
Germany	Investigator site 33 - DE0490024	Frankfurt am main
Germany	Investigator site 53 - DE0490023	Freiburg
Germany	Investigator site 35 - DE0490028	Kiel
Germany	Investigator site 20 - DE0490002	Lübeck
Germany	Investigator site 52 - DE0490001	Marburg
Germany	Investigator site 19 - DE0490025	Tübingen
Germany	Investigator site 93 - DE0490027	Ulm
Germany	Investigator site 4 - DE0490026	Würzburg
Greece	Investigator site 21 - GR030004	Athens
Greece	Investigator site 37 - GR030006	Athens
Greece	Investigator site 54 - GR0300001	Athens
Greece	Investigator site 22 - GR030003	Chaïdári
Greece	Investigator site 23 - GR030005	Thessaloníki
Greece	Investigator site 36 - GR0300002	Thessaloníki
Hungary	Investigator site 6 - HU0360003	Debrecen
Hungary	Investigator site 5 - HU0360001	Pécs
Hungary	Investigator site 24 - HU0360002	Szeged
India	Investigator site 65 - IN0910002	Ahmedabad
India	Investigator site 94 - IN0910001	Chandigarh
India	Investigator site 79 - IN0910004	Lucknow
India	Investigator site 80 - IN0910003	Nagpur
Israel	Investigator site 85 - IL9720002	Tel Aviv
Italy	Investigator site 95 - IT0390039	Catania
Italy	Investigator site 38 - IT0390031	Firenze
Italy	Investigator site 81 - IT0390030	Genova
Italy	Investigator site 55 - IT0390038	Perugia
Italy	Investigator site 12 - IT0390006	Roma
Italy	Investigator site 25 - IT-0390005	Roma
Italy	Investigator site 61 - IT0390040	Siena
Japan	Investigator site 82 - JP0810046	Aichi
Japan	Investigator site 68 - JP0810040	Hiroshima
Japan	Investigator site 66 - JP0810042	Kofu
Japan	Investigator site 69 - JP0810050	Kurume
Japan	Investigator site 70 - JP0810041	Okayama
Japan	Investigator site 73 - JP0810047	Okayama
Japan	Investigator site 71 - JP0810049	Osaka
Japan	Investigator site 72 - JP0810045	Sapporo
Japan	Investigator site 114 - JP0810067	Sendai
Japan	Investigator site 67 - JP0810043	Tokyo
Poland	Investigator site 27 - PL0480027	Katowice
Poland	Investigator site 56 - PL0480032	Lódz
Poland	Investigator site 86 - PL0480036	Poznan
Poland	Investigator site 28 - PL0480025	Rzeszów
Poland	Investigator site 26 - PL0480028	Wroclaw
Romania	Investigator site 97 - RO0400013	Bucharest
Romania	Investigator 96 - RO0400014	Cluj-Napoca
Romania	Investigator site 98 - RO0400015	Iasi
Russian Federation	Investigator site 40 - RU0070035	Chelyabinsk
Russian Federation	Investigator site 41 - RU0070033	Ekaterinburg
Russian Federation	Investigator site 48 - RU0070029	Kazan
Russian Federation	Investigator site 49 - RU0070030	Krasnodar
Russian Federation	Investigator site 39 - RU0070032	Rostov-on-Don
Russian Federation	Investigator site 46 - RU0070031	Saint Petersburg
Russian Federation	Investigator site 50 - RU0070034	Saint Petersburg
Russian Federation	Investigator site 47 - RU0070028	Saratov
Serbia	Investigator site 109 - RS3810011	Belgrade
Serbia	Investigator site 110 - RS3810010	Belgrade
Serbia	Investigator site 105 - RS3810012	Niš
Serbia	Investigator site 104 - RS3810009	Novi Sad
Spain	Investigator site 13 - ES0340032	Barcelona
Spain	Investigator site 62 - ES0340026	Barcelona
Spain	Investigator site 122 - ES0340053	Granada
Spain	Investigator site 42 - ES0340025	Madrid
Spain	Investigator site 57 - ES0340027	Madrid
Spain	Investigator site 59 - ES0340034	Madrid
Spain	Investigator site 7 - ES0340029	Madrid
Spain	Investigator site 58 - ES0340028	Sevilla
Turkey	Investigator site 64 - TR0900020	Gaziantep
Turkey	Investigator site 63 - TR0900012	Istanbul
Turkey	Investigator site 74 - TR0900011	Istanbul
Ukraine	Investigator site 75 - UA3800017	Dnipro
Ukraine	Investigator site 29 - UA3800023	Ivano-Frankivs'k
Ukraine	Investigator site 14 - UA3800020	Kyiv
Ukraine	Investigator site 8 - UA3800019	Kyiv
Ukraine	Investigator site 43 - UA3800021	Lviv
Ukraine	Investigator site 9 - UA3800018	Zaporizhzhia
United Kingdom	Investigator site 106 - UK0440021	Birmingham
United Kingdom	Investigator site 83 - UK0440022	Bristol
United Kingdom	Investigator site 123 - UK0440037	Southampton
United States	Investigator site 115 - US0010086	Birmingham	Alabama
United States	Investigator site 1 - US0010087	Boca Raton	Florida
United States	Investigator site 10 - US0010088	Buffalo	New York
United States	Investigator site 30 - US0010094	Cleveland	Ohio
United States	Investigator site 125 - US0010107	Dallas	Texas
United States	Investigator site 87 - US0010084	Dripping Springs	Texas
United States	Investigator site 76 - US0010096	Durham	North Carolina
United States	Investigator site 88 - US0010114	Houston	Texas
United States	Investigator site 90 - US0010117	Miami	Florida
United States	Investigator site 126 - US0010090	Minneapolis	Minnesota
United States	Investigator site 44 - US0010106	Norfolk	Virginia
United States	Investigator site 91 - US0010109	Orlando	Florida
United States	Investigator site 103 - US0010097	Philadelphia	Pennsylvania
United States	Investigator site 84 - US0010089	Philadelphia	Pennsylvania
United States	Investigator site 124 - US0010092	Redwood City	California
United States	Investigator site 111 - US0010098	Saint Louis	Missouri
United States	Investigator site 89 - US0010091	Scottsdale	Arizona

Sponsors (1)

Lead Sponsor	Collaborator
argenx

Countries where clinical trial is conducted

United States,  Australia,  Bulgaria,  China,  France,  Georgia,  Germany,  Greece,  Hungary,  India,  Israel,  Italy,  Japan,  Poland,  Romania,  Russian Federation,  Serbia,  Spain,  Turkey,  Ukraine,  United Kingdom, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)	Incidence of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)	Up to 60 weeks
Primary	Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)	Severity of Treatment-Emergent Adverse Events (TEAE), Adverse Events of Special Interest (AESI), and Serious Adverse Events (SAE)	Up to 60 weeks
Secondary	Proportion of Pemphigus Vulgaris (PV) participants who achieve complete clinical remission (CR) on minimal prednisone therapy	Proportion of participants with Pemphigus Vulgaris who achieve complete clinical remission on minimal prednisone therapy.	Up to 52 weeks treatment period
Secondary	Proportion of Pemphigus Vulgaris (PV) and Pemphigus Foliaceus (PF) participants who achieve complete clinical remission (CR) on minimal prednisone dose therapy	Proportion of participants with Pemphigus Vulgaris and Pemphigus Foliaceus who achieve complete clinical remission on minimal prednisone therapy.	Up to 52 weeks treatment period
Secondary	Time to Disease Control (DC)	Time to Disease Control	Up to 60 weeks
Secondary	Time to complete clinical remission (CR)	Time to complete clinical remission	Up to 60 weeks
Secondary	Time to complete clinical remission (CR) on minimal prednisone therapy	Time to complete clinical remission on minimal prednisone therapy	Up to 60 weeks
Secondary	Time to complete clinical remission (CR) off prednisone therapy	Time to complete clinical remission off prednisone therapy	Up to 60 weeks
Secondary	Time to flare	Time to flare	Up to 60 weeks
Secondary	Rate of treatment failure	Rate of treatment failure	Up to 60 weeks
Secondary	Rate of flare	Rate of flare	Up to 60 weeks
Secondary	Cumulative prednisone dose over the trial	Cumulative prednisone dose over the trial	Up to 52 weeks treatment period
Secondary	Pemphigus Disease Area Index (PDAI) at each visit	Pemphigus Disease Area Index at each visit	Up to 52 weeks treatment period
Secondary	EuroQol 5-Dimension 5-Level (EQ-5D-5L) score	EuroQol 5-Dimension 5-Level score	Up to 52 weeks treatment period
Secondary	Autoimmune Blister Quality of Life (ABQOL) score	Autoimmune Blister Quality of Life score	Up to 52 weeks treatment period
Secondary	Efgartigimod serum concentrations	Efgartigimod serum concentrations	Up to 60 weeks
Secondary	Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels	Total Immunoglobulin G and subtype (IgG1, IgG2, IgG3, IgG4) serum levels	Up to 60 weeks
Secondary	Anti-desmoglein (Dsg) -1 and -3 autoantibodies serum levels	Anti-desmoglein (Dsg) -1 and -3 autoantibodies serum levels	Up to 60 weeks
Secondary	Incidence of antidrug antibodies (ADA) against efgartigimod PH20 SC	Incidence of antidrug antibodies (ADA) against efgartigimod PH20 SC	Up to 60 weeks
Secondary	Prevalence of antidrug antibodies (ADA) against efgartigimod PH20 SC	Prevalence of antidrug antibodies (ADA) against efgartigimod PH20 SC	Up to 60 weeks
Secondary	Composite Glucocorticoid Toxicity Index (C-GTI) comprising the Aggregate Improvement Score (AIS) and the Cumulative Worsening Score (CWS)	Composite Glucocorticoid Toxicity Index comprising the Aggregate Improvement Score and the Cumulative Worsening Score	Up to 52 weeks treatment period
Secondary	Percentage of participants who performed self-administration	Percentage of participants who performed self-administration	Up to 52 weeks
Secondary	Percentage of caregivers who administered the injection to the participant	Percentage of caregivers who administered the injection to the participant	Up to 52 weeks
Secondary	Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC	Number of visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC	Up to 52 weeks
Secondary	Frequency of self- or caregiver-supported administration at home	Frequency of self- or caregiver-supported administration at home	Up to 52 weeks