Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT03102593
Recruitment Status : Completed
First Posted : April 6, 2017
Last Update Posted : May 6, 2019
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.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 38 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
Actual Primary Completion Date : April 9, 2019
Actual Study Completion Date : April 9, 2019


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



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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


Locations
Show Show 30 study locations
Sponsors and Collaborators
argenx BVBA
Quintiles, Inc.
Investigators
Layout table for investigator information
Study Chair: Adrian Newland Barts Hospital, Cancer Centre in London
Layout table for additonal information
Responsible Party: argenx BVBA
ClinicalTrials.gov Identifier: NCT03102593    
Other Study ID Numbers: ARGX-113-1603.
2016-003038-26 ( EudraCT Number )
First Posted: April 6, 2017    Key Record Dates
Last Update Posted: May 6, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for 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