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Recruiting NCT06544499

A Study to Assess the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

Phase III Interventional Primary Immune Thrombocytopenia (ITP)

For patients and families

In plain language

An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.

What is being studied
The protocol lists: Efgartigimod IV, Placebo IV.
Who it may be relevant to
Registry conditions: Primary Immune Thrombocytopenia (ITP). Basic parameters: from 18 years · All.
What needs checking
Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
Where it takes place
United States, Austria, Bulgaria, China, Croatia +12
Next step
Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →

Unsure about the terms? Read our patient guide →

Official title

A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled, Parallel-Arm Study Followed by an Open-Label Arm to Evaluate the Efficacy and Safety of Efgartigimod IV in Adult Participants With Primary Immune Thrombocytopenia

Overview

The main purpose of this study is to look at the effect (efficacy) and safety of efgartigimod IV in participants with primary immune thrombocytopenia (ITP). After an up to 2 weeks screening period, eligible participants will be randomized in a 2:1 ratio to receive either efgartigimod IV or placebo IV, respectively during the double-blinded treatment period (DBTP). At the end of the treatment period (up to 24 weeks), all participants will receive efgartigimod IV during the first 52-week open-label treatment period (OLTP1). At the end of the first OLTP1, participants may begin a second 52-week OLTP2. After the OLTP2, the participants will enter a follow-up period (approximately 8 weeks) while off study drug. The participants will be in the study for up to 138 weeks. More information can be found here: https://clinicaltrials.argenx.com/advancenext

Interventions

  • Biological Efgartigimod IV
    Intravenous infusion of efgartigimod
  • Other Placebo IV
    Intravenous infusion of placebo

Primary outcome measures

  • Extent of disease control, defined as the number of cumulative weeks during the 24-week Double-Blinded Treatment Period with platelet counts of at least 50 × 10^9/L [Time frame: Up to 24 weeks]
Secondary outcome measures (12)
  • Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 study visits between study weeks 19 and 24 of the DBTP [Time frame: Up to 6 weeks]
  • Proportion of participants achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 study visits between study weeks 17 and 24 of the DBTP [Time frame: Up to 8 weeks]
  • Proportion of participants achieving a platelet counts of at least 50 × 10^9/L for at least 8 of the 12 study visits between weeks 13 and 24 of the DBTP [Time frame: Up to 12 weeks]
  • Proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions at any time until study week 12 during the DBTP [Time frame: Up to 12 weeks]
  • Time to response, defined as the time to achieve 2 consecutive platelet counts of at least 50 × 10^9/L at any time during the DBTP [Time frame: up to 24 weeks]
  • Proportion of participants with an IWG response during the DBTP [Time frame: Up to 24 weeks]
  • Proportion of participants with initial response, defined as a platelet count of at least 30 × 10^9/L and a 2-fold increase from baseline in platelet count at study week 5 of the DBTP [Time frame: Up to 20 weeks]
  • Time to achieve a platelet count of at least 30 × 10^9/L and a 2-fold increase from baseline in platelet count during the DBTP [Time frame: Up to 24 weeks]
  • Number of cumulative weeks during the DBTP with platelet counts of at least 30 × 10^9/L and ≥20 × 10^9/L above baseline [Time frame: Up to 24 weeks]
  • Number of cumulative weeks during the DBTP with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline in participants with baseline platelet counts of <15 × 10^9/L [Time frame: Up to 24 weeks]
  • Extent of disease control, defined as the percentage of weeks with platelet counts of at least 50 × 10^9/L [Time frame: Up to 76 weeks]
  • In participants receiving placebo IV in the DBTP, the extent of disease control, defined as the number of cumulative weeks with platelet counts of at least 50 × 10^9/L [Time frame: Up to 76 weeks]

Eligibility criteria

Inclusion criteria

  • Is at least 18 years of age and the local legal age of consent for clinical studies when signing the informed consent form (ICF).
  • Has documented baseline mean platelet count of <30 x 10\^9/L before randomization
  • Has a documented duration of primary immune thrombocytopenia (ITP) of more than 12 months on the date of informed consent form (ICF) signature.
  • Has documented prior ITP treatment with at least 1 of the following treatments: corticosteroids, intravenous immunoglobulin (IVIg), anti-D immunoglobulin, thrombopoietin receptor agonist (TPO-RAs), or rituximab.
  • Has documented insufficient response to a prior ITP treatment (the specific criteria can be found in the protocol).
  • Has documented prior response defined as 1 platelet count of ≥50 × 109/L to at least 1 of the following ITP treatments in the 3 years before the date of ICF signature: prednisone, dexamethasone, other or nonspecified corticosteroids, IVIg, or anti-D immunoglobulin

Exclusion criteria

  • Other than the indication under study, known autoimmune disease or any medical condition that would interfere with an accurate assessment of clinical symptoms of ITP, confound the results of the study or put the participant at undue risk.
  • Secondary ITP
  • Nonimmune thrombocytopenia
  • Autoimmune hemolytic anemia
  • ITP-associated critical or severe bleeding The complete list of criteria can be found in the protocol.

Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.

Healthy volunteers: No

Study design

Allocation
Randomized
Model
Parallel assignment
Masking
Quadruple blind
Primary purpose
Treatment

Study locations

United States · 18 centers
  • Mayo Clinic Hospital Scottsdale — Phoenix
  • University of Southern California Norris Comprehensive Cancer Center — Los Angeles
  • Sharp Memorial Hospital — Oceanside
  • University of Colorado Hospital — Aurora
  • Yale University School of Medicine — New Haven
  • Georgetown University Hospital — Washington D.C.
  • The University of Chicago Medicine — Chicago
  • The University of Iowa Hospitals & Clinics — Iowa City
  • … and 10 more centers
Italy · 12 centers
  • Azienda ULSS 7 Pedemontana — Bassano del Grappa
  • Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC) — Florence
  • Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico — Milan
  • Fondazione IRCCS Ca' Granda - Ospedale Maggiore Policlinico — Milan
  • ASST Grande Ospedale Metropolitano Niguarda — Milan
  • AO Maggiore Della Carita — Novara
  • U.O.C. Ematologia Ospedale Santa Maria delle Croci — Ravenna
  • Arcispedale Santa Maria Nuova (ASMN) Istituto di Ricovero e Cura a Carattere Scientifico ( — Reggio Emilia
  • … and 4 more centers
China · 11 centers
  • Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University — Beijing
  • Peking University Third Hospital — Beijing
  • The First Affiliated Hospital Of Bengbu Medical College — Bengbu
  • The Affiliated Hospital of Chengde Medical University — Chengde
  • The Affiliated Hospital of Inner Mongolia Medical University — Hohhot
  • The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University — Huai'an
  • Huizhou Central People's Hospital — Huizhou
  • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & P — Tianjin
  • … and 3 more centers
United Kingdom · 8 centers

Center list to be confirmed — check the primary protocol.

Poland · 7 centers
  • Szpital Uniwersytecki nr 2 im. Dr Jana Biziela w Bydgoszczy — Bydgoszcz
  • Pratia Onkologia Katowice — Katowice
  • Wojewodzkie Wielospecjalistyczne Centrum Onkologii i Traumatologii im. M. Kopernika w Lodz — Lodz
  • NZOZ Neuromed M. i M. Nastaj Sp.P. — Lublin
  • Uniwersytecki Szpital Kliniczny Nr 4 w Lublinie Centrum Innowacyjnych Terapii — Lublin
  • Szpital Specjalistyczny im. Jedrzeja Sniadeckiego w Nowym Saczu — Nowy Sącz
  • Wojewodzki Szpital Zespolony im. Ludwika Rydygiera w Toruniu — Torun
Bulgaria · 6 centers
  • Acibadem City clinic Tokuda hospital — Sofia
  • UMHAT Aleksandrovska — Sofia
  • University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski — Sofia
  • National Specialized Hospital for Active Treatment of Hematology Diseases — Sofia
  • UMHAT SofiaMed — Sofia
  • University Hospital Prof. Dr. Stoyan Kirkovich AD — Stara Zagora
Spain · 6 centers

Center list to be confirmed — check the primary protocol.

Serbia · 5 centers
  • Clinical Center of Serbia — Belgrade
  • University Clinical Centre of Kragujevac — Kragujevac
  • Clinical Center of Nis — Niš
  • University Clinical Hospital Center Zemun — Zemun
  • General Hospital Djordje Joanovic — Zrenjanin
Romania · 4 centers
  • Spitalul Clinic Coltea, Clinica de Hematologie — Bucharest
  • Spitalul Clinic Colentina — Bucharest
  • Spitalul Clinic Judetean de Urgenta Sibiu (SCJS) (Clinical County Hospital Sibiu) — Sibiu
  • Spitalul Clinic Judetean de Urgenta Targu-Mures — Târgu Mureş
Austria · 3 centers
  • Allgemeines Krankenhaus der Stadt Linz — Linz
  • Ordensklinikum Linz GmbH Elisabethinen — Linz
  • Klinikum Kreuzschwestern Wels — Wels
France · 3 centers
  • CHU d'Amiens-Picardie - Hopital SUD — Amiens
  • Centre Hospitalier Universitaire (CHU) - Hopital Henri Mondor — Créteil
  • Centre Hospitalier Universitaire Dijon Bourgogne Medecine Interne et Immunologie Clinique — Dijon
Czechia · 2 centers
  • Fakultni Nemocnice Ostrava — Ostrava
  • Fakultni nemocnice Kralovske Vinohrady — Prague
Germany · 2 centers
  • University Hospital Carl Gustav Carus — Dresden
  • Universitaetsklinikum Essen (AoR) — Essen
Hungary · 2 centers
  • Semmelweis University-Internal Medicine and Haematology — Budapest
  • Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz — Győr
Ireland · 2 centers
  • Mater Misericordiae University Hospital — Dublin
  • St James's Hospital - Cancer Clinical Trials Office — Dublin
Portugal · 2 centers
  • Unidade Local de Saude de Braga — Braga
  • Unidade Local de Saude Gaia e Espinho, E.P.E. — Vila Nova de Gaia
Croatia · 1 center
  • University Hospital Center Zagreb — Zagreb

Identifiers

NCT: NCT06544499 · ARGX-113-2402 · 2024-515451-38-00

Primary sources (government registries)

View this study on ClinicalTrials.gov ↗