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

April 29, 2026 updated by: argenx

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

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

Study Overview

Status

Recruiting

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Estimated)

69

Phase

  • Phase 3

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Contact

Study Locations

  • Austria
      • Linz, Austria, 4020
      • Linz, Austria, 4020
      • Wels, Austria, 4600
  • Bulgaria
      • Sofia, Bulgaria, 1407
        • Recruiting
        • Acibadem City Clinic Tokuda Hospital
        • Contact:
      • Sofia, Bulgaria, 1431
        • Recruiting
        • UMHAT Aleksandrovska
        • Contact:
      • Sofia, Bulgaria, 1797
        • Recruiting
        • UMHAT SofiaMed
        • Contact:
      • Sofia, Bulgaria, 1431
        • Recruiting
        • University Multi-Profile Hospital for Active Treatment (UMHAT) St. Ivan Rilski
        • Contact:
      • Sofia, Bulgaria, 1797
        • Recruiting
        • National Specialized Hospital for Active Treatment of Hematology Diseases
        • Contact:
      • Stara Zagora, Bulgaria, 6003
        • Recruiting
        • University Hospital Prof. Dr. Stoyan Kirkovich AD
        • Contact:
  • China
      • Beijing, China, 100191
        • Recruiting
        • Peking University Third Hospital
        • Contact:
      • Beijing, China, 100050
        • Recruiting
        • Beijing YouYi Hospital-Beijing Friendship Hospital Capital Medical University
        • Contact:
      • Bengbu, China, 233004
        • Recruiting
        • The First Affiliated Hospital of Bengbu Medical College
        • Contact:
      • Chengde, China, 067020
        • Recruiting
        • The Affiliated Hospital of Chengde Medical University
        • Contact:
      • Hohhot, China, 010050
        • Recruiting
        • The Affiliated Hospital of Inner Mongolia Medical University
        • Contact:
      • Huai'an, China, 223300
        • Recruiting
        • The Affiliated Huaian No.1 People's Hospital of Nanjing Medical University
        • Contact:
      • Huizhou, China
        • Recruiting
        • Huizhou Central People's Hospital
        • Contact:
      • Tianjin, China, 300020
        • Recruiting
        • Institute of Hematology & Blood Diseases Hospital, Chinese Academy of Medical Sciences & Peking Union Medical College
        • Contact:
      • Wuhan, China
        • Recruiting
        • Union Hospital Tongji Medical College of Huazhong University of Science and Technology (HUST)
        • Contact:
      • Wuxi, China, 214023
        • Recruiting
        • Wuxi People's Hospital (Wuxi No.1 People's Hospital)
        • Contact:
      • Zhenjiang, China, 212001
        • Recruiting
        • Affiliated Hospital of Jiangsu University
        • Contact:
  • Croatia
      • Zagreb, Croatia, 10000
        • Recruiting
        • University Hospital Center Zagreb
        • Contact:
  • Czechia
      • Ostrava, Czechia, 708 52
        • Recruiting
        • Fakultni Nemocnice Ostrava
        • Contact:
      • Prague, Czechia, 100 34
        • Recruiting
        • Fakultni nemocnice Kralovske Vinohrady
        • Contact:
  • France
      • Amiens, France, 80054
      • Créteil, France, 94010
        • Recruiting
        • Centre Hospitalier Universitaire (CHU) - Hopital Henri Mondor
        • Contact:
      • Dijon, France, 21079
        • Recruiting
        • Centre Hospitalier Universitaire Dijon Bourgogne Medecine Interne et Immunologie Clinique
        • Contact:
  • Germany
  • Hungary
      • Budapest, Hungary, 1088
      • Győr, Hungary, 9024
        • Recruiting
        • Gyor-Moson-Sopron Varmegyei Petz Aladar Egyetemi Oktato Korhaz
        • Contact:
  • Ireland
      • Dublin, Ireland, D07 R2WY
      • Dublin, Ireland, D08 NHY1
        • Recruiting
        • St James's Hospital - Cancer Clinical Trials Office
        • Contact:
  • Italy
      • Bassano del Grappa, Italy, 36061
      • Florence, Italy, 50134
        • Recruiting
        • Universita Degli Studi Di Firenze - Azienda Ospedaliero-Universitaria Careggi (AOUC)
        • Contact:
      • Milan, Italy, 20162
      • Milan, Italy, 20122
      • Novara, Italy, 28100
        • Recruiting
        • AO Maggiore Della Carita
        • Contact:
      • Ravenna, Italy, 48121
        • Recruiting
        • U.O.C. Ematologia Ospedale Santa Maria delle Croci
        • Contact:
      • Reggio Emilia, Italy, 42123
      • Torino, Italy, 10126
      • Torino, Italy, 10128
        • Recruiting
        • AO Ordine Mauriziano di Torino
        • Contact:
      • Trieste, Italy, 34125
      • Varese, Italy, 21100
  • Poland
      • Bydgoszcz, Poland, 85-168
        • Recruiting
        • Szpital Uniwersytecki nr 2 im. dr Jana Biziela w Bydgoszczy
        • Contact:
      • Katowice, Poland, 40-519
      • Lodz, Poland, 93-513
      • Lublin, Poland, 20-064
      • Lublin, Poland, 20-090
        • Recruiting
        • Uniwersytecki Szpital Kliniczny Nr 4 w Lublinie Centrum Innowacyjnych Terapii
        • Contact:
      • Nowy Sącz, Poland, 33-300
        • Recruiting
        • Szpital Specjalistyczny im. Jedrzeja Sniadeckiego w Nowym Saczu
        • Contact:
        • Contact:
          • Szymon Fornagiel, MD
          • Phone Number: +48501079942
          • Email: szf@wp.pl
      • Torun, Poland, 87-100
        • Recruiting
        • Wojewodzki Szpital Zespolony im. Ludwika Rydygiera w Toruniu
        • Contact:
  • Portugal
      • Braga, Portugal, 4710
        • Recruiting
        • Unidade Local de Saude de Braga
        • Contact:
      • Vila Nova de Gaia, Portugal, 4434-502
  • Romania
  • Serbia
      • Belgrade, Serbia, 11000
      • Kragujevac, Serbia, 34000
        • Recruiting
        • University Clinical Centre of Kragujevac
        • Contact:
      • Niš, Serbia, 18000
        • Recruiting
        • Clinical Center of Nis
        • Contact:
      • Zemun, Serbia, 11080
        • Recruiting
        • University Clinical Hospital Center Zemun
        • Contact:
      • Zrenjanin, Serbia, 23101
  • Spain
      • A Coruña, Spain, 15006
        • Recruiting
        • Complejo Hospitalario Universitario A Coruña
        • Contact:
      • L'Hospitalet de Llobregat, Spain, 08908
      • Madrid, Spain, 28034
        • Recruiting
        • Hospital Universitario Ramon y Cajal
        • Contact:
      • Murcia, Spain, 30008
        • Recruiting
        • Hospital General Universitario Morales Meseguer
        • Contact:
          • Maria Luisa Lozano Almela, MD
          • Phone Number: +34968360900
          • Email: mllozano@um.es
      • Pozuelo de Alarcón, Spain, 28223
        • Recruiting
        • University Hospital Quironsalud Madrid
        • Contact:
      • Valencia, Spain, 46010
        • Recruiting
        • Hospital Clinico Universitario - University of Valencia
        • Contact:
  • United Kingdom
      • Birmingham, United Kingdom, B15 2GW
        • Recruiting
        • Queen Elizabeth Hospital Birmingham - University Hospitals Birmingham NHS Foundation Trust
        • Contact:
      • Bradford, United Kingdom, BD9 6RJ
      • Coventry, United Kingdom, CV2 2DX
        • Recruiting
        • University Hospitals Coventry and Warwickshire NHS Trust
        • Contact:
      • Glasgow, United Kingdom, G4 0ET
        • Recruiting
        • Glasgow Royal Infirmary - North Glasgow University Hospital Division
        • Contact:
      • Leicester, United Kingdom, LE1 5WW
        • Recruiting
        • University Hospitals of Leicester NHS Trust-Leicester Royal Infirmary
        • Contact:
      • London, United Kingdom, W12 0HS
        • Recruiting
        • Hammersmith Hospital, Imperial College School Of Medicine - Imperial College Healthcare NHS Trust
        • Contact:
      • London, United Kingdom, E1 2ES
        • Recruiting
        • Barts and the London Pathology & Pharmacy Building - Barts Health NHS Trust
        • Contact:
      • Truro, United Kingdom, TR1 3LJ
  • United States
    • Arizona
      • Phoenix, Arizona, United States, 85054
    • California
      • Los Angeles, California, United States, 90033
        • Recruiting
        • University of Southern California Norris Comprehensive Cancer Center
        • Contact:
      • Oceanside, California, United States, 92056
    • Colorado
      • Aurora, Colorado, United States, 80045
    • Connecticut
      • New Haven, Connecticut, United States, 06510
        • Recruiting
        • Yale University School of Medicine
        • Contact:
    • District of Columbia
      • Washington D.C., District of Columbia, United States, 20007
        • Recruiting
        • Georgetown University Hospital
        • Contact:
    • Illinois
      • Chicago, Illinois, United States, 60637
    • Iowa
      • Iowa City, Iowa, United States, 52242
        • Recruiting
        • The University of Iowa Hospitals & Clinics
        • Contact:
    • Louisiana
      • New Orleans, Louisiana, United States, 70112
        • Recruiting
        • Tulane University
        • Contact:
    • Michigan
      • Detroit, Michigan, United States, 48202
        • Recruiting
        • Henry Ford Health
        • Contact:
    • Minnesota
      • Minneapolis, Minnesota, United States, 55422
        • Recruiting
        • University of Minnesota
        • Contact:
    • New Jersey
      • Little Silver, New Jersey, United States, 07739
    • New York
      • Westbury, New York, United States, 11590
        • Terminated
        • Clinical Research Alliance Inc.
    • North Carolina
      • Durham, North Carolina, United States, 27710
        • Recruiting
        • Duke University Medical Center
        • Contact:
      • Greenville, North Carolina, United States, 27834
        • Recruiting
        • Brody school of Medicine at East Carolina University
        • Contact:
    • Ohio
      • Cleveland, Ohio, United States, 44106
        • Recruiting
        • University Hospitals Cleveland Medical Center
        • Contact:
      • Columbus, Ohio, United States, 43203
        • Recruiting
        • Ohio State University Hospital East
        • Contact:
    • Oklahoma
      • Oklahoma City, Oklahoma, United States, 73109

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

  • Adult
  • Older Adult

Accepts Healthy Volunteers

No

Description

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.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: Randomized
  • Interventional Model: Parallel Assignment
  • Masking: Quadruple

Arms and Interventions

Participant Group / Arm
Intervention / Treatment
Experimental: Efgartigimod IV
Participants receiving efgartigimod IV during the double-blinded treatment period and the open-label treatment period(s)
Biological: Efgartigimod IV
Intravenous infusion of efgartigimod
Experimental: Placebo IV
Participants receiving placebo IV during the double-blinded treatment period and receiving efgartigimod IV during the open-label treatment period(s)
Biological: Efgartigimod IV
Intravenous infusion of efgartigimod
Other: Placebo IV
Intravenous infusion of placebo

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
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
Up to 24 weeks

Secondary Outcome Measures

Outcome Measure
Measure Description
Time Frame
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
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
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
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
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
up to 24 weeks
Proportion of participants with an IWG response during the DBTP
Time Frame: Up to 24 weeks
International Working Group (IWG)
Up to 24 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
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
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
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
Up to 76 weeks
Overall platelet count response, defined as the proportion of participants achieving a platelet count of at least 50 × 10^9/L on at least 4 occasions during the first 52 weeks of treatment with efgartigimod IV
Time Frame: Up to 52 weeks
Up to 52 weeks
Mean change from baseline in platelet count at each study visit
Time Frame: Up to 76 weeks
Up to 76 weeks
The percentage of weeks with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline
Time Frame: Up to 76 weeks
Up to 76 weeks
In participants with baseline platelet counts <15 × 10^9/L, the percentage of weeks with platelet counts of at least 30 × 10^9/L and at least 20 × 10^9/L above baseline
Time Frame: Up to 76 weeks
Up to 76 weeks
Proportion of participants who achieve a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10^9/L for at least 4 occasions during 6-week intervals
Time Frame: Up to 76 weeks
Up to 76 weeks
In participants receiving placebo IV in the DBTP, proportion of participants who achieve sustained platelet count response, defined as achieving platelet counts of at least 50 × 10^9/L for at least 6 of 8 visits between weeks 17 and 24 of the OLTP1
Time Frame: Up to 8 weeks
Up to 8 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 during the first 24 weeks of treatment with efgartigimod IV
Time Frame: Up to 24 weeks
Up to 24 weeks
Occurrence rate of rescue ITP therapy
Time Frame: Up to 76 weeks
Up to 76 weeks
Proportion of participants with reduction in concurrent ITP therapy during the OLTP1
Time Frame: Up to 52 weeks
Up to 52 weeks
Incidence and severity of bleeding, assessed by the ITP Bleeding Scale (IBLS)
Time Frame: Up to 76 weeks
Up to 76 weeks
Efgartigimod Cmax over time
Time Frame: Up to 136 weeks
Up to 136 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
Up to 20 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
Up to 76 weeks
In participants receiving placebo IV in the DBTP, the proportion of participants achieving 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 OLTP1
Time Frame: Up to 12 weeks
Up to 12 weeks
Incidence of AEs (including AEs of clinical interest) and SAEs
Time Frame: Up to 136 weeks
AE = adverse event; SAE = serious adverse event; An adverse event of clinical interest is a pre-specified medically significant event that has the potential to be causally associated with the study drug.
Up to 136 weeks
Incidence of ADA against efgartigimod in serum over time
Time Frame: Up to 136 weeks
ADA = antidrug antibodies
Up to 136 weeks
Incidence of NAb against efgartigimod in serum over time
Time Frame: Up to 136 weeks
NAb = Neutralizing antibodies
Up to 136 weeks
Percent change from baseline in total IgG levels in serum over time
Time Frame: Up to 136 weeks
IgG = immunoglobulin G
Up to 136 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

argenx

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

Helpful Links

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

October 18, 2024

Primary Completion (Estimated)

June 1, 2028

Study Completion (Estimated)

June 1, 2028

Study Registration Dates

First Submitted

July 31, 2024

First Submitted That Met QC Criteria

August 5, 2024

First Posted (Actual)

August 9, 2024

Study Record Updates

Last Update Posted (Actual)

May 1, 2026

Last Update Submitted That Met QC Criteria

April 29, 2026

Last Verified

April 1, 2026

More Information

Terms related to this study

Additional Relevant MeSH Terms

Other Study ID Numbers

  • ARGX-113-2402
  • 2024-515451-38-00 (Ctis)

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

Yes

Studies a U.S. FDA-regulated device product

No

product manufactured in and exported from the U.S.

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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