A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With ITP

July 24, 2023 updated by: argenx

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

The purpose of the study is to determine safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia.

Study Overview

Status

Completed

Intervention / Treatment

Detailed Description

This is a randomized, double-blind, placebo-controlled Phase II study in which approximately 36 patients will be randomized in a 1:1:1 ratio to receive either ARGX-113 Dose A, or ARGX-113 Dose B body weight or placebo in 4 infusions administered 1-week apart in addition to Standard-of-Care (SoC) treatment. Patients aged 18 to 85 years (inclusive) with confirmed primary immune thrombocytopenia (ITP) who have a platelet count ˂ 30 × 109/L and who are receiving oral corticosteroids and/or permitted oral immunosuppressants and/or Thrombopoietin receptor (TPO-R) agonist as SoC which must be maintained on a stable dose and frequency for at least 4 weeks prior to Screening.

The study will include a 2-week Screening, a 3-week Treatment period, and an 21-week follow-up (FU) period. The study is followed by an open label period where patients will be given the option to be treated with ARGX-113 Dose A in cycles of 4 weekly infusions with a minimum of 4 weeks apart. Patients may receive rescue therapy during the study at the discretion the investigator when deemed medically necessary.

Study Type

Interventional

Enrollment (Actual)

38

Phase

  • Phase 2

Contacts and Locations

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

Study Locations

      • Vienna, Austria
        • Vienna
      • Wien, Austria
        • Wien
      • Leuven, Belgium
        • Leuven
      • Namur, Belgium
        • Mont-Godinne
      • Brno, Czechia
        • Brno
      • Praha, Czechia
        • Praha
      • Bordeaux, France
        • Bordeaux
      • Grenoble, France
        • Grenoble
      • Paris, France
        • Paris
      • Berlin, Germany
        • Berlin
      • Hanover, Germany
        • Hanover
      • Tubingen, Germany
        • Tübingen
      • Budapest, Hungary
        • Budapest
      • Debrecen, Hungary
        • Debrecen
      • Gyula, Hungary
        • Gyula
      • Kaposvar, Hungary
        • Kaposvar
      • Nyiregyhaza, Hungary
        • Nyíregyháza
      • Pecs, Hungary
        • Pecs
      • Lublin, Poland
        • Lublin
      • Opole, Poland
        • Opole
      • Wroclaw, Poland
        • Wroclaw
      • A Coruña, Spain
        • A Coruna
      • Barcelona, Spain
        • Barcelona
      • Madrid, Spain
        • Madrid
      • Valencia, Spain
        • Valencia
      • Dnipro, Ukraine
        • Dnipro
      • Ivano-Frankivsk, Ukraine
        • Ivano-Frankivsk
      • Nikolaev, Ukraine
        • Nikolaev
      • Uzhgorod, Ukraine
        • Uzhgorod
      • London, United Kingdom
        • London

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

18 years to 85 years (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Male or female patients aged ≥ 18 to ≤ 85 years.
  2. 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.
  3. 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.

Exclusion Criteria:

  1. Use of anticoagulants, or any drug with antiplatelet effect within 3 weeks prior to Screening.
  2. Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
  3. Use of Intravenous immunoglobulin G (IVIg) or anti-D immunoglobulin treatment within 4 weeks prior to screening.
  4. Use of recombinant thrombopoietin at any time.
  5. Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
  6. 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.
  7. 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.
  8. Received vaccinations within 4 weeks prior to Screening or planned during the study.
  9. At Screening, have clinically significant laboratory abnormalities
  10. History of any thrombotic or embolic event within 12 months prior to Screening.
  11. Known auto-immune disease other than ITP.

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: ARGX-113 Dose A + SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
Experimental: ARGX-113 Dose B +SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
Placebo Comparator: Placebo + SoC
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Measure Description
Time Frame
Incidence and severity of serious adverse events (SAEs).
Time Frame: After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
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 Outcome Measures

Outcome Measure
Measure Description
Time Frame
Frequency and proportion of patients with initial response
Time Frame: Over the study period (up to 13 weeks).
Mean change from Baseline in platelet counts
Over the study period (up to 13 weeks).

Collaborators and Investigators

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

Sponsor

Collaborators

Investigators

  • Study Chair: Adrian Newland, Barts Hospital, Cancer Centre in London

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.

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)

March 13, 2017

Primary Completion (Actual)

April 9, 2019

Study Completion (Actual)

April 9, 2019

Study Registration Dates

First Submitted

March 14, 2017

First Submitted That Met QC Criteria

March 30, 2017

First Posted (Actual)

April 6, 2017

Study Record Updates

Last Update Posted (Actual)

July 25, 2023

Last Update Submitted That Met QC Criteria

July 24, 2023

Last Verified

July 1, 2023

More Information

Terms related to this study

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

UNDECIDED

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

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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