Clinical trial NCT04188379: A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP).
Official title: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Primary Immune Thrombocytopenia
Keywords: immune thrombocytopenia
sustained platelet count response
bleeding events
Lead sponsor: argenx
Sponsor class: INDUSTRY
First posted: 2019-12-05
Enrollment: 131
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP.


Eligibility criteria: Inclusion criteria:

* 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).
* Male or female patient aged ≥18 years.
* Confirmed ITP diagnosis, at least 3 months before randomization and according to the American Society of Hematology Criteria, and no known other etiology for thrombocytopenia.
* Diagnosis supported by a response to a prior ITP therapy (other than thrombopoietin receptor agonists \[TPO-RAs\]), in the opinion of the investigator.
* Mean platelet count of \<30×10E9/L from 2 counts: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization.
* At the start of the trial, the patient is either on concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the patient is not on treatment for ITP but has received at least 2 prior treatments for ITP. Patients receiving permitted concurrent ITP treatment(s) at baseline, must have been stable in dose and frequency for at least 4 weeks prior to randomization.
* Women of childbearing potential must have a negative serum pregnancy test at the screening visit and a negative urine pregnancy test at baseline before trial medication (infusion) can be administered.
* 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.

Exclusion criteria:

* ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
* Use of certain medications before the start of the studies (more details in the protocol)
* Patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before screening. Patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted at any time.
* Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments.
* History of any major thrombotic or embolic event within 12 months prior to randomization.
* History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
* History of a recent or planned major surgery (that involves major organs eg, brain, heart, lung, liver, bladder, or gastrointestinal tract) within 4 weeks of randomization.
* Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV), Hepatitis C virus (HCV), Human immunodeficiency virus (HIV)
* Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITPdespite appropriate treatments which could put the patient at undue risk.
* Patients with known medical history of hypersensitivity to any of the ingredients of the IMP.
* Patients who previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP.
* Pregnant or lactating females. More details in the protocol

Primary outcomes:
measure: Percentage of Participants With Chronic ITP With a Sustained Platelet Count 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: From Week 19 up to Week 24
Secondary outcomes:
measure: Extent of Disease Control Defined as the Number of Cumulative Weeks Over the Planned 24-week Treatment Period With Platelet Counts of ≥50×10^9/L in the Chronic ITP Population
timeFrame: From Week 1 up to Week 24
measure: Percentage of Participants With a Sustained Platelet Count Response for at Least 4 of the 6 Visits Between Week 19 and 24 of the Study
timeFrame: From Week 19 up to Week 24
measure: Incidence and Severity of the WHO-classified Bleeding Events
timeFrame: From Week 1 to Week 24
measure: Percentage of Participants in the Overall Population Achieving Platelet Counts of at Least 50×10^9/L for at Least 6 of the 8 Visits Between Week 17 and Week 24
timeFrame: From Week 17 up to Week 24
Other outcomes:
Locations:
city: Tucson
country: United States
facility: Investigator Site 0010038
state: Arizona
city: Washington
country: United States
facility: Investigator Site 0010045
state: District of Columbia
city: Jacksonville
country: United States
facility: Investigator Site 0010034
state: Florida
city: Ocala
country: United States
facility: Investigator site 0010037
state: Florida
city: Iowa City
country: United States
facility: Investigator Site 0010042
state: Iowa
city: Greenville
country: United States
facility: Investigator Site 0010046
state: North Carolina
city: Cleveland
country: United States
facility: Investigator Site 0010049
state: Ohio
city: Columbus
country: United States
facility: Investigator Site 0010040
state: Ohio
city: Philadelphia
country: United States
facility: Investigator Site 0010041
state: Pennsylvania
city: Linz
country: Austria
facility: Investigator Site 0430004
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: Leuven
country: Belgium
facility: Investigator Site 0320015
city: Turnhout
country: Belgium
facility: Investigator Site 0320014
city: Verviers
country: Belgium
facility: Investigator Site 0320020
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: Clermont-Ferrand
country: France
facility: Investigator Site 0330019
city: Créteil
country: France
facility: Investigator Site 0330009
city: Le Mans
country: France
facility: Investigator Site 0330015
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: Rouen
country: France
facility: Investigator Site 0330017
city: Tbilisi
country: Georgia
facility: Investigator Site 9950006
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: Gießen
country: Germany
facility: Investigator Site 0490012
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: Nyiregyhaza
country: Hungary
facility: Investigator site 0360010
city: Szombathely
country: Hungary
facility: Investigator Site 0360014
city: Campobasso
country: Italy
facility: Investigator Site 0390012
city: Milan
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: Roma
country: Italy
facility: Investigator Site 0390021
city: Siena
country: Italy
facility: Investigator Site 0390009
city: Torino
country: Italy
facility: Investigator Site 0390017
city: Trieste
country: Italy
facility: Investigator Site 0390016
city: Bunkyō-Ku
country: Japan
facility: Investigator Site 0810024
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: Isehara
country: Japan
facility: Investigator site 0810011
city: Kashiwa
country: Japan
facility: Investigator Site 0810022
city: Maebashi
country: Japan
facility: Investigator site 0810018
city: Minato-Ku
country: Japan
facility: Investigator site 0810020
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: Shinjuku-Ku
country: Japan
facility: Investigator Site 0810025
city: Den Haag
country: Netherlands
facility: Investigator site 0310006
city: Rotterdam
country: Netherlands
facility: Investigator Site 0310005
city: Rotterdam
country: Netherlands
facility: Investigator Site 0310007
city: Białystok
country: Poland
facility: Investigator Site 0480030
city: Brzozów
country: Poland
facility: Investigator Site 0480015
city: Bydgoszcz
country: Poland
facility: Investigator Site 0480010
city: Chorzów
country: Poland
facility: Investigator Site 0480013
city: Gdańsk
country: Poland
facility: Investigator Site 0480012
city: Katowice
country: Poland
facility: Investigator Site 0480008
city: Lodz
country: Poland
facility: Investigator Site 0480011
city: Lublin
country: Poland
facility: Investigator Site 0480014
city: Nowy Sącz
country: Poland
facility: Investigator site 0480026
city: Wrocław
country: Poland
facility: Investigator Site 0480016
city: Kaluga
country: Russian Federation
facility: Investigator site 0070006
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: Burgos
country: Spain
facility: Investigator Site 0340030
city: Madrid
country: Spain
facility: Investigator Site 0340009
city: Madrid
country: Spain
facility: Investigator Site 0340014
city: Madrid
country: Spain
facility: Investigator Site 0340015
city: Palma De Mallorca
country: Spain
facility: Investigator site 0340012
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: Adana
country: Turkey
facility: Investigator Site 0900002
city: Adapazarı
country: Turkey
facility: Investigator Site 0900007
city: Ankara
country: Turkey
facility: Investigator Site 0900003
city: Ankara
country: Turkey
facility: Investigator Site 0900006
city: Ankara
country: Turkey
facility: Investigator Site 0900008
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: Izmir
country: Turkey
facility: Investigator Site 0900004
city: Kocaeli
country: Turkey
facility: Investigator Site 0900014
city: Malatya
country: Turkey
facility: Investigator Site 0900018
city: Manisa
country: Turkey
facility: Investigator Site 0900005
city: Mersin
country: Turkey
facility: Investigator Site 0900010
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: Kharkiv
country: Ukraine
facility: Investigator Site 3800022
city: Mykolayiv
country: Ukraine
facility: Investigator site 3800006
city: London
country: United Kingdom
facility: Investigator Site 0440008
city: Rhyl
country: United Kingdom
facility: Investigator Site 0440010
city: Southampton
country: United Kingdom
facility: Investigator Site 0440012
city: Truro
country: United Kingdom
facility: Investigator Site 0440014
Study officials:
References: