Clinical trial NCT04598477: 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)
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
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE3
Conditions: Pemphigus Vulgaris
Pemphigus Foliaceus
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2020-10-22
Enrollment: 183
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This was 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 provided extension of efgartigimod PH20 SC treatment and retreatment options for participants who had been randomized to efgartigimod PH20 SC treatment arm in the trial ARGX-113-1904, and first treatment of efgartigimod PH20 SC and retreatment options for participants who had been randomized to the placebo arm in trial ARGX-113-1904. The participants could also receive concomitant prednisone therapy. Investigators could increase or decrease the prednisone dose based on protocol-specified criteria.

Trial ARGX-113-1905 evaluated the 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 CR or CRmin, and the ability to treat flare; it also assessed 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


Eligibility criteria: 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.

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