Clinical trial NCT04225156: A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
Official title: A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia.
Study type: INTERVENTIONAL
Overall status: ACTIVE_NOT_RECRUITING
Phase: PHASE3
Conditions: Primary Immune Thrombocytopenia
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2020-01-13
Enrollment: 101
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 is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.


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), and to comply with the trial protocol procedures (including required trial visits).
2. Patients enrolled in the ARGX-113-1801 trial who completed the 24-weeks trial period.
3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered.
4. Women of childbearing potential should use a highly effective or acceptable method of contraception during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month (as listed in the protocol)

6. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits).

7. Patient has completed a 52-week treatment period.

Exclusion criteria:

1. Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins or live/live-attenuated vaccines).
2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.
4. Use of any other investigational drug or participation in any other investigational trial.

Primary outcomes:
measure: Frequency and severity of Adverse Events
timeFrame: Up to 60 weeks
measure: Frequency and severity of vital signs
timeFrame: Up to 60 weeks
measure: Frequency and severity of laboratory assessments
timeFrame: Up to 60 weeks
Secondary outcomes:
measure: Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L.
timeFrame: Over the 52 weeks of treatment
measure: Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period.
timeFrame: Over the 52 weeks of treatment
measure: Mean change from baseline in platelet count at each visit.
timeFrame: Up to 60 weeks, at each visit
measure: For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L
timeFrame: Up to 60 weeks, at each visit
measure: The percentage of weeks in the trial with platelet counts of ≥30×109/L and at least 20×10E9/L above baseline.
timeFrame: Over the 52 weeks of treatment
measure: In patients with baseline platelet count of <15×10E9/L in the current trial (ARGX-113-1803), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and at least 20×10E9/L above baseline.
timeFrame: Over the 52 weeks of treatment
measure: In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial.
timeFrame: Up to 5 weeks, between visit 19 and 24 of the trial
measure: In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial.
timeFrame: Up to 7 weeks, between visit 17 and 24 of the trial
measure: Rate of receipt of rescue therapy (rescue per patient per month).
timeFrame: Up to 60 weeks, at each visit
measure: Reduction in concurrent ITP therapy.
timeFrame: Up to 60 weeks, at each visit
measure: Incidence and severity of the WHO-classified bleeding events.
timeFrame: Up to 60 weeks, at each visit
measure: Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits.
timeFrame: Up to 52 weeks
measure: Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits.
timeFrame: Up to 52 weeks
measure: Change from baseline in Quality of Life (SF-36) at planned visits.
timeFrame: Up to 52 weeks
measure: Incidence of anti-drug antibodies (ADA) to efgartigimod.
timeFrame: Up to 216 weeks
measure: Pharmacokinetic parameter of efgartigimod: serum concentration observed predose (Ctrough).
timeFrame: Up to 60 weeks
measure: Pharmacodynamics markers: total IgG.
timeFrame: Up to 60 weeks
Other outcomes:
Locations:
city: Washington
country: United States
facility: Investigator Site 0010045
state: District of Columbia
city: Ocala
country: United States
facility: Investigator Site 0010037
state: Florida
city: Iowa City
country: United States
facility: Investigator Site 0010042
state: Iowa
city: Columbus
country: United States
facility: Investigator Site 0010040
state: Ohio
city: Vienna
country: Austria
facility: Investigator Site 0430002
city: Vienna
country: Austria
facility: Investigator Site 0430003
city: Brasschaat
country: Belgium
facility: Investigator Site 0320012
city: Brugge
country: Belgium
facility: Investigator Site 0320011
city: Turnhout
country: Belgium
facility: Investigator Site 0320014
city: Yvoir
country: Belgium
facility: Investigator Site 0320002
city: Pleven
country: Bulgaria
facility: Investigator Site 3590001
city: Sofia
country: Bulgaria
facility: Investigator Site 3590002
city: Brno
country: Czechia
facility: Investigator Site 4200001
city: Olomouc
country: Czechia
facility: Investigator Site 4200008
city: Ostrava
country: Czechia
facility: Investigator Site 4200006
city: Praha
country: Czechia
facility: Investigator Site 4200007
city: Créteil
country: France
facility: Investigator Site 0330009
city: Montpellier
country: France
facility: Investigator Site 0330018
city: Pessac
country: France
facility: Investigator Site 0330008
city: Périgueux
country: France
facility: Investigator Site 0330016
city: Tbilisi
country: Georgia
facility: Investigator site 9950007
city: Tbilisi
country: Georgia
facility: Investigator site 9950008
city: Tbilisi
country: Georgia
facility: Investigator site 9950009
city: Tbilisi
country: Georgia
facility: Investigator site 9950011
city: Tbilisi
country: Georgia
facility: Investigator site 9950012
city: Düsseldorf
country: Germany
facility: Investigator Site 0490010
city: Essen
country: Germany
facility: Investigator Site 0490008
city: Budapest
country: Hungary
facility: Investigator Site 0360004
city: Debrecen
country: Hungary
facility: Investigator Site 0360006
city: Győr
country: Hungary
facility: Investigator Site 0360015
city: Nyíregyháza
country: Hungary
facility: Investigator Site 0360010
city: Szombathely
country: Hungary
facility: Investigator Site 0360014
city: Milano
country: Italy
facility: Investigator Site 0390014
city: Monza
country: Italy
facility: Investigator Site 0390020
city: Novara
country: Italy
facility: Investigator Site 0390015
city: Ravenna
country: Italy
facility: Investigator Site 0390010
city: Reggio Calabria
country: Italy
facility: Investigator Site 0390011
city: Reggio Emilia
country: Italy
facility: Investigator Site 0390018
city: Rimini
country: Italy
facility: Investigator Site 0390019
city: Siena
country: Italy
facility: Investigator Site 0390009
city: Trieste
country: Italy
facility: Investigator Site 0390016
city: Hirakata
country: Japan
facility: Investigator Site 0810015
city: Hiroshima
country: Japan
facility: Investigator Site 0810010
city: Iruma
country: Japan
facility: Investigator Site 0810017
city: Kashiwa
country: Japan
facility: Investigator Site 0810022
city: Maebashi
country: Japan
facility: Investigator Site 0810018
city: Niigata
country: Japan
facility: Investigator Site 0810021
city: Sapporo
country: Japan
facility: Investigator Site 0810014
city: Shibukawa
country: Japan
facility: Investigator Site 0810016
city: Shimotsuke
country: Japan
facility: Investigator Site 0810023
city: Den Haag
country: Netherlands
facility: Investigator Site 0310006
city: Rotterdam
country: Netherlands
facility: Investigator Site 0310005
city: Gdańsk
country: Poland
facility: Investigator Site 0480012
city: Katowice
country: Poland
facility: Investigator Site 0480013
city: Lublin
country: Poland
facility: Investigator Site 0480014
city: Nowy Sącz
country: Poland
facility: Investigator Site 0480026
city: Łódź
country: Poland
facility: Investigator Site 0480011
city: Kaluga
country: Russian Federation
facility: Investigator Site 0070006
city: Moscow
country: Russian Federation
facility: Investigator Site 0070008
city: Petrozavodsk
country: Russian Federation
facility: Investigator Site 0070007
city: Rostov-on-Don
country: Russian Federation
facility: Investigator Site 0070013
city: Syktyvkar
country: Russian Federation
facility: Investigator Site 0070015
city: Tula
country: Russian Federation
facility: Investigator Site 0070012
city: Ufa
country: Russian Federation
facility: Investigator Site 0070010
city: Barcelona
country: Spain
facility: Investigator Site 0340006
city: Barcelona
country: Spain
facility: Investigator Site 0340007
city: Madrid
country: Spain
facility: Investigator Site 0340009
city: Madrid
country: Spain
facility: Investigator Site 0340014
city: Palma De Mallorca
country: Spain
facility: Investigator Site 0340012
city: Pozuelo De Alarcón
country: Spain
facility: Investigator Site 0340015
city: Sevilla
country: Spain
facility: Investigator Site 0340013
city: Valencia
country: Spain
facility: Investigator Site 0340004
city: Valencia
country: Spain
facility: Investigator Site 0340011
city: Ankara
country: Turkey
facility: Investigator Site 0900003
city: Ankara
country: Turkey
facility: Investigator Site 0900006
city: Ankara
country: Turkey
facility: Investigator Site 0900015
city: Edirne
country: Turkey
facility: Investigator Site 0900016
city: Istanbul
country: Turkey
facility: Investigator Site 0900013
city: İzmir
country: Turkey
facility: Investigator Site 0900004
city: Mersin
country: Turkey
facility: Investigator Site 0900010
city: Sakarya
country: Turkey
facility: Investigator Site 0900007
city: Samsun
country: Turkey
facility: Investigator Site 0900009
city: Tekirdağ
country: Turkey
facility: Investigator Site 0900017
city: Trabzon
country: Turkey
facility: Investigator Site 0900019
city: Mykolaiv
country: Ukraine
facility: Investigator Site 3800006
city: London
country: United Kingdom
facility: Investigator Site 0440008
city: Southampton
country: United Kingdom
facility: Investigator Site 0440012
Study officials:
References: