A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With ITP
A Randomized, Double-blind, Placebo-controlled, Phase II Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With Primary Immune Thrombocytopenia
2 other identifiers
interventional
38
10 countries
30
Brief Summary
The purpose of the study is to determine safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia.
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for phase_2
Started Mar 2017
30 active sites
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
Study Start
First participant enrolled
March 13, 2017
CompletedFirst Submitted
Initial submission to the registry
March 14, 2017
CompletedFirst Posted
Study publicly available on registry
April 6, 2017
CompletedPrimary Completion
Last participant's last visit for primary outcome
April 9, 2019
CompletedStudy Completion
Last participant's last visit for all outcomes
April 9, 2019
CompletedJuly 25, 2023
July 1, 2023
2.1 years
March 14, 2017
July 24, 2023
Conditions
Outcome Measures
Primary Outcomes (1)
Incidence and severity of serious adverse events (SAEs).
Changes from Baseline in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities and clinical laboratory assessments.
After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
Secondary Outcomes (1)
Frequency and proportion of patients with initial response
Over the study period (up to 13 weeks).
Study Arms (3)
ARGX-113 Dose A + SoC
EXPERIMENTALPatients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
ARGX-113 Dose B +SoC
EXPERIMENTALPatients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
Placebo + SoC
PLACEBO COMPARATORPatients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
Interventions
Eligibility Criteria
You may qualify if:
- Male or female patients aged ≥ 18 to ≤ 85 years.
- Must receive SoC treatment for ITP that has been stable in dose and frequency for at least 4 weeks prior to Screening. SoC may include oral corticosteroids and/or permitted oral immunosuppressants and/or TPO-R agonist.
- Confirmed diagnosis of ITP with blood platelet counts \< 30 × 109/L and who have not experienced major bleeding in the last 4 weeks prior to Screening.
You may not qualify if:
- Use of anticoagulants, or any drug with antiplatelet effect within 3 weeks prior to Screening.
- Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
- Use of Intravenous immunoglobulin G (IVIg) or anti-D immunoglobulin treatment within 4 weeks prior to screening.
- Use of recombinant thrombopoietin at any time.
- Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
- Use of immunosuppressants is not permitted within 4 weeks prior to Screening, with the exception of the following oral immunosuppressants: azathioprine, danazol, mycophenolate mofetil, mycophenolate sodium which must have been stable for at least 4 weeks prior to Screening.
- Use of any other biological therapy or investigational drug than those previously indicated within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
- Received vaccinations within 4 weeks prior to Screening or planned during the study.
- At Screening, have clinically significant laboratory abnormalities
- History of any thrombotic or embolic event within 12 months prior to Screening.
- Known auto-immune disease other than ITP.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
- argenxlead
- Quintiles, Inc.collaborator
Study Sites (30)
Vienna
Vienna, Austria
Wien
Vienna, Austria
Leuven
Leuven, Belgium
Mont-Godinne
Namur, Belgium
Brno
Brno, Czechia
Praha
Prague, Czechia
Bordeaux
Bordeaux, France
Grenoble
Grenoble, France
Paris
Paris, France
Berlin
Berlin, Germany
Hanover
Hanover, Germany
Tubingen
Tübingen, Germany
Budapest
Budapest, Hungary
Debrecen
Debrecen, Hungary
Gyula
Gyula, Hungary
Kaposvar
Kaposvár, Hungary
Nyiregyhaza
Nyíregyháza, Hungary
Pecs
Pécs, Hungary
Lublin
Lublin, Poland
Opole
Opole, Poland
Wroclaw
Wroclaw, Poland
A Coruna
A Coruña, Spain
Barcelona
Barcelona, Spain
Madrid
Madrid, Spain
Valencia
Valencia, Spain
Dnipro
Dnipro, Ukraine
Ivano-Frankivsk
Ivano-Frankivsk, Ukraine
Nikolaev
Mykolayiv, Ukraine
Uzhgorod
Uzhhorod, Ukraine
London
London, United Kingdom
Related Publications (1)
Newland AC, Sanchez-Gonzalez B, Rejto L, Egyed M, Romanyuk N, Godar M, Verschueren K, Gandini D, Ulrichts P, Beauchamp J, Dreier T, Ward ES, Michel M, Liebman HA, de Haard H, Leupin N, Kuter DJ. Phase 2 study of efgartigimod, a novel FcRn antagonist, in adult patients with primary immune thrombocytopenia. Am J Hematol. 2020 Feb;95(2):178-187. doi: 10.1002/ajh.25680. Epub 2019 Dec 10.
PMID: 31821591RESULT
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Study Officials
- STUDY CHAIR
Adrian Newland
Barts Hospital, Cancer Centre in London
Study Design
- Study Type
- interventional
- Phase
- phase 2
- Allocation
- RANDOMIZED
- Masking
- QUADRUPLE
- Who Masked
- PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
- Purpose
- TREATMENT
- Intervention Model
- PARALLEL
- Sponsor Type
- INDUSTRY
- Responsible Party
- SPONSOR
Study Record Dates
First Submitted
March 14, 2017
First Posted
April 6, 2017
Study Start
March 13, 2017
Primary Completion
April 9, 2019
Study Completion
April 9, 2019
Last Updated
July 25, 2023
Record last verified: 2023-07