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

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ClinicalTrials.gov Identifier: NCT03102593
Recruitment Status : Recruiting
First Posted : April 6, 2017
Last Update Posted : March 2, 2018
Sponsor:
Collaborator:
Quintiles, Inc.
Information provided by (Responsible Party):
argenx BVBA

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

Condition or disease Intervention/treatment Phase
Primary Immune Thrombocytopenia Drug: ARGX-113 Other: Placebo Phase 2

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  (Clinical Trial)
Estimated Enrollment : 36 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Intervention Model Description: Protocol designed to evaluate one or more interventions for treating a disease, syndrome or condition.
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : March 13, 2017
Estimated Primary Completion Date : August 2018
Estimated Study Completion Date : November 2018


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
Drug: ARGX-113
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
Drug: ARGX-113
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
Other: Placebo
ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly




Primary Outcome Measures :
  1. 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.


Secondary Outcome Measures :
  1. 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



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Ages Eligible for Study:   18 Years to 85 Years   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03102593


Contacts
Contact: Jocelyne DeWulf +32 (0) 9 310 34 91 jdewulf@argenx.com
Contact: Thierry Cousin tcousin@argenx.com

Locations
Austria
Vienna Recruiting
Vienna, Austria
Wien Recruiting
Wien, Austria
Belgium
Leuven Recruiting
Leuven, Belgium
Mont-Godinne Recruiting
Namur, Belgium
Czechia
Brno Recruiting
Brno, Czechia
Praha Recruiting
Praha, Czechia
France
Bordeaux Recruiting
Bordeaux, France
Grenoble Recruiting
Grenoble, France
Paris Recruiting
Paris, France
Germany
Berlin Recruiting
Berlin, Germany
Hanover Recruiting
Hanover, Germany
Tubingen Recruiting
Tubingen, Germany
Hungary
Budapest Recruiting
Budapest, Hungary
Debrecen Recruiting
Debrecen, Hungary
Gyula Recruiting
Gyula, Hungary
Kaposvar Recruiting
Kaposvar, Hungary
Nyiregyhaza Recruiting
Nyiregyhaza, Hungary
Pecs Recruiting
Pecs, Hungary
Poland
Lublin Recruiting
Lublin, Poland
Opole Recruiting
Opole, Poland
Wroclaw Recruiting
Wroclaw, Poland
Spain
A Coruna Recruiting
A Coruña, Spain
Barcelona Recruiting
Barcelona, Spain
Madrid Recruiting
Madrid, Spain
Valencia Recruiting
Valencia, Spain
Ukraine
Dnipro Recruiting
Dnipro, Ukraine
Ivano-Frankivsk Recruiting
Ivano-Frankivsk, Ukraine
Nikolaev Recruiting
Nikolaev, Ukraine
Uzhgorod Recruiting
Uzhgorod, Ukraine
United Kingdom
London Recruiting
London, United Kingdom
Sponsors and Collaborators
argenx BVBA
Quintiles, Inc.
Investigators
Study Chair: Adrian Newland Barts Hospital, Cancer Centre in London

Responsible Party: argenx BVBA
ClinicalTrials.gov Identifier: NCT03102593     History of Changes
Other Study ID Numbers: ARGX-113-1603.
2016-003038-26 ( EudraCT Number )
First Posted: April 6, 2017    Key Record Dates
Last Update Posted: March 2, 2018
Last Verified: July 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:
Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations
Signs and Symptoms