Actively Recruiting
A Phase 3 Study to Evaluate the Efficacy and Safety of Efgartigimod IV in Adults With Primary Immune Thrombocytopenia
Led by argenx · Updated on 2026-05-01
69
Participants Needed
94
Research Sites
N/A
Total Duration
On this page
AI-Summary
What this Trial Is About
Researchers are evaluating the effects and safety of efgartigimod given intravenously in adults with primary immune thrombocytopenia (ITP), a condition where the immune system attacks platelets. This phase 3 clinical trial aims to determine how well efgartigimod controls platelet counts compared to a placebo during a 24-week double-blinded treatment period. The study is sponsored by argenx and includes follow-up periods to monitor ongoing effects and safety. Participants will be randomly assigned in a 2:1 ratio to receive either efgartigimod IV or placebo IV during the double-blinded treatment period. After this, all participants will receive efgartigimod IV during a first 52-week open-label treatment phase, followed by an optional second 52-week open-label phase. After completing these treatment periods, participants will have an approximately 8-week follow-up while off the study drug, with total study participation lasting up to 138 weeks. During the study, participants will have regular blood tests to measure platelet counts and assess disease control. Researchers will track the number of weeks participants maintain platelet counts above specified levels and monitor any bleeding events or adverse effects. Safety is closely observed throughout the trial, including antibody responses to the drug. Participants will have scheduled visits and assessments throughout treatment and follow-up to gather comprehensive data on efgartigimod's impact.
CONDITIONS
Brief Title
A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia
Who Can Participate
Eligibility Criteria
You may qualify if you...
- At least 18 years old and able to consent to participate
- Documented baseline mean platelet count below 30 x 10^9/L before randomization
- Diagnosis of primary immune thrombocytopenia (ITP) lasting more than 12 months
- Prior treatment with corticosteroids, intravenous immunoglobulin, anti-D immunoglobulin, thrombopoietin receptor agonist, or rituximab
- Insufficient response to prior ITP treatment as defined in the study protocol
- Prior platelet count response to certain ITP treatments within the past 3 years
You will not qualify if you...
- Known autoimmune diseases other than ITP or medical conditions interfering with ITP assessment
- Secondary immune thrombocytopenia
- Nonimmune thrombocytopenia
- Autoimmune hemolytic anemia
- Critical or severe bleeding associated with ITP
AI-Screening
AI-Powered Screening
Complete this quick 3-step screening to check your eligibility
Your Study Journey
Duration - 2 to 4 weeks
Participants are screened for eligibility to participate in the trial.
Duration - Up to 24 weeks
Participants receive intravenous infusions of either efgartigimod or placebo during the double-blinded treatment period and may continue with open-label treatment periods.
Multiple visits during the double-blinded treatment period
Trial Site Locations
Total: 94 locations
1
Mayo Clinic Hospital Scottsdale
Phoenix, Arizona, United States, 85054
Actively Recruiting
2
University of Southern California Norris Comprehensive Cancer Center
Los Angeles, California, United States, 90033
Actively Recruiting
3
Sharp Memorial Hospital
Oceanside, California, United States, 92056
Actively Recruiting
4
University of Colorado Hospital
Aurora, Colorado, United States, 80045
Actively Recruiting
5
Yale University School of Medicine
New Haven, Connecticut, United States, 06510
Actively Recruiting
6
Georgetown University Hospital
Washington D.C., District of Columbia, United States, 20007
Actively Recruiting
7
The University of Chicago Medicine
Chicago, Illinois, United States, 60637
Actively Recruiting
8
The University of Iowa Hospitals & Clinics
Iowa City, Iowa, United States, 52242
Actively Recruiting
9
Tulane University
New Orleans, Louisiana, United States, 70112
Actively Recruiting
10
Henry Ford Health
Detroit, Michigan, United States, 48202
Actively Recruiting
11
University of Minnesota
Minneapolis, Minnesota, United States, 55422
Actively Recruiting
12
Regional Cancer Care Associates, LLC (RCCA)
Little Silver, New Jersey, United States, 07739
Actively Recruiting
13
Clinical Research Alliance Inc.
Westbury, New York, United States, 11590
Terminated
14
Duke University Medical Center
Durham, North Carolina, United States, 27710
Actively Recruiting
15
Brody School of Medicine at East Carolina University
Greenville, North Carolina, United States, 27834
Actively Recruiting
16
University Hospitals Cleveland Medical Center
Cleveland, Ohio, United States, 44106
Actively Recruiting
17
Ohio State University Hospital East
Columbus, Ohio, United States, 43203
Actively Recruiting
18
INTEGRIS Cancer Institute of Oklahoma
Oklahoma City, Oklahoma, United States, 73109
Actively Recruiting
19
Allgemeines Krankenhaus der Stadt Linz
Linz, Austria, 4020
Actively Recruiting
20
Ordensklinikum Linz GmbH Elisabethinen
Linz, Austria, 4020
Actively Recruiting
21
Klinikum Kreuzschwestern Wels
Wels, Austria, 4600
Actively Recruiting
22
Acibadem City clinic Tokuda hospital
Sofia, Bulgaria, 1407
Actively Recruiting
23
UMHAT Aleksandrovska
Sofia, Bulgaria, 1431
Actively Recruiting
24
University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski
Sofia, Bulgaria, 1431
Actively Recruiting
25
National Specialized Hospital for Active Treatment of Hematology Diseases
Sofia, Bulgaria, 1797
Actively Recruiting
26
UMHAT SofiaMed
Sofia, Bulgaria, 1797
Actively Recruiting
27
University Hospital Prof. Dr. Stoyan Kirkovich AD
Stara Zagora, Bulgaria, 6003
Actively Recruiting
28
Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University
Beijing, China, 100050
Actively Recruiting
29
Peking University Third Hospital
Beijing, China, 100191
Actively Recruiting
30
The First Affiliated Hospital Of Bengbu Medical College
Bengbu, China, 233004
Actively Recruiting
31
The Affiliated Hospital of Chengde Medical University
Chengde, China, 067020
Actively Recruiting
32
The Affiliated Hospital of Inner Mongolia Medical University
Hohhot, China, 010050
Actively Recruiting
33
The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University
Huai'an, China, 223300
Actively Recruiting
34
Huizhou Central People's Hospital
Huizhou, China
Actively Recruiting
35
Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
Tianjin, China, 300020
Actively Recruiting
36
Union Hospital Tongji Medical College of Huazhong University of Science and Technology (HUST)
Wuhan, China
Actively Recruiting
37
Wuxi People's Hospital (Wuxi No.1 People's Hospital)
Wuxi, China, 214023
Actively Recruiting
38
Affiliated Hospital of Jiangsu University
Zhenjiang, China, 212001
Actively Recruiting
39
University Hospital Center Zagreb
Zagreb, Croatia, 10000
Actively Recruiting
40
Fakultni Nemocnice Ostrava
Ostrava, Czechia, 708 52
Actively Recruiting
41
Fakultni nemocnice Kralovske Vinohrady
Prague, Czechia, 100 34
Actively Recruiting
42
CHU d'Amiens-Picardie - Hopital SUD
Amiens, France, 80054
Actively Recruiting
43
Centre Hospitalier Universitaire (CHU) - Hopital Henri Mondor
Créteil, France, 94010
Actively Recruiting
44
Centre Hospitalier Universitaire Dijon Bourgogne Medecine Interne et Immunologie Clinique
Dijon, France, 21079
Actively Recruiting
45
University Hospital Carl Gustav Carus
Dresden, Germany, 01307
Actively Recruiting
46
Universitaetsklinikum Essen (AoR)
Essen, Germany, 45147
Actively Recruiting
47
Semmelweis University-Internal Medicine and Haematology
Budapest, Hungary, 1088
Actively Recruiting
48
Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz
Győr, Hungary, 9024
Actively Recruiting
49
Mater Misericordiae University Hospital
Dublin, Ireland, D07 R2WY
Actively Recruiting
50
St James's Hospital - Cancer Clinical Trials Office
Dublin, Ireland, D08 NHY1
Actively Recruiting
51
Azienda ULSS 7 Pedemontana
Bassano del Grappa, Italy, 36061
Actively Recruiting
52
Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC)
Florence, Italy, 50134
Actively Recruiting
53
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Milan, Italy, 20122
Actively Recruiting
54
Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico
Milan, Italy, 20122
Actively Recruiting
55
ASST Grande Ospedale Metropolitano Niguarda
Milan, Italy, 20162
Actively Recruiting
56
AO Maggiore Della Carita
Novara, Italy, 28100
Actively Recruiting
57
U.O.C. Ematologia Ospedale Santa Maria delle Croci
Ravenna, Italy, 48121
Actively Recruiting
58
Arcispedale Santa Maria Nuova (ASMN) Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS)
Reggio Emilia, Italy, 42123
Actively Recruiting
59
A.O.U. Citta della Salute e della Scienza di Torino
Torino, Italy, 10126
Actively Recruiting
60
AO Ordine Mauriziano di Torino
Torino, Italy, 10128
Actively Recruiting
61
Azienda Sanitaria Universitaria Integrata
Trieste, Italy, 34125
Actively Recruiting
62
ASST Sette Laghi Ospedale di circolo e Fondazione Macchi
Varese, Italy, 21100
Actively Recruiting
63
Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy
Bydgoszcz, Poland, 85-168
Actively Recruiting
64
Pratia Onkologia Katowice
Katowice, Poland, 40-519
Actively Recruiting
65
Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodzi
Lodz, Poland, 93-513
Actively Recruiting
66
NZOZ Neuromed M. i M. Nastaj Sp.P.
Lublin, Poland, 20-064
Actively Recruiting
67
Uniwersytecki Szpital Kliniczny Nr 4 w Lublinie Centrum Innowacyjnych Terapii
Lublin, Poland, 20-090
Actively Recruiting
68
Szpital Specjalistyczny im. Jedrzeja Sniadeckiego w Nowym Saczu
Nowy Sącz, Poland, 33-300
Actively Recruiting
69
Wojewodzki Szpital Zespolony im. Ludwika Rydygiera w Toruniu
Torun, Poland, 87-100
Actively Recruiting
70
Unidade Local de Saude de Braga
Braga, Portugal, 4710
Actively Recruiting
71
Unidade Local de Saude Gaia e Espinho, E.P.E.
Vila Nova de Gaia, Portugal, 4434-502
Actively Recruiting
72
Spitalul Clinic Coltea, Clinica de Hematologie
Bucharest, Romania, 030171
Actively Recruiting
73
Spitalul Clinic Colentina
Bucharest, Romania, 20125
Actively Recruiting
74
Spitalul Clinic Judetean de Urgenta Sibiu (SCJS) (Clinical County Hospital Sibiu)
Sibiu, Romania, 550245
Actively Recruiting
75
Spitalul Clinic Judetean de Urgenta Targu-Mures
Târgu Mureş, Romania, 540136
Actively Recruiting
76
Clinical Center of Serbia
Belgrade, Serbia, 11000
Actively Recruiting
77
University Clinical Centre of Kragujevac
Kragujevac, Serbia, 34000
Actively Recruiting
78
Clinical Center of Nis
Niš, Serbia, 18000
Actively Recruiting
79
University Clinical Hospital Center Zemun
Zemun, Serbia, 11080
Actively Recruiting
80
General Hospital Djordje Joanovic
Zrenjanin, Serbia, 23101
Actively Recruiting
81
Complejo Hospitalario Universitario A Coruna
A Coruña, Spain, 15006
Actively Recruiting
82
Instituto Catalan de Oncologia - Hospital Duran i Reynals
L'Hospitalet de Llobregat, Spain, 08908
Actively Recruiting
83
Hospital Universitario Ramon y Cajal
Madrid, Spain, 28034
Actively Recruiting
84
Hospital General Universitario Morales Meseguer
Murcia, Spain, 30008
Actively Recruiting
85
University Hospital Quironsalud Madrid
Pozuelo de Alarcón, Spain, 28223
Actively Recruiting
86
Hospital Clinico Universitario - University of Valencia
Valencia, Spain, 46010
Actively Recruiting
87
Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust
Birmingham, United Kingdom, B15 2GW
Actively Recruiting
88
Bradford Royal Infirmary - Bradford Teaching Hospitals NHS Foundation
Bradford, United Kingdom, BD9 6RJ
Actively Recruiting
89
University Hospitals Coventry and Warwickshire NHS Trust
Coventry, United Kingdom, CV2 2DX
Actively Recruiting
90
Glasgow Royal Infirmary - North Glasgow University Hospital Division
Glasgow, United Kingdom, G4 0ET
Actively Recruiting
91
University Hospitals of Leicester NHS Trust-Leicester Royal Infirmary
Leicester, United Kingdom, LE1 5WW
Actively Recruiting
92
Barts and the London Pathology & Pharmacy Building - Barts Health NHS Trust
London, United Kingdom, E1 2ES
Actively Recruiting
93
Hammersmith Hospital, Imperial College School Of Medicine - Imperial College Healthcare NHS Trust
London, United Kingdom, W12 0HS
Actively Recruiting
94
Royal Cornwall Hospital (RCH)
Truro, United Kingdom, TR1 3LJ
Actively Recruiting
Research Team
S
Sabine Coppieters, MD
How is the study designed?
Study Type
INTERVENTIONAL
Masking
QUADRUPLE
Allocation
RANDOMIZED
Model
PARALLEL
Primary Purpose
TREATMENT
Number of Arms
2
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