Back to resultsA Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With ITP
Primary Purpose
Primary Immune Thrombocytopenia
Status
Completed
Phase
Phase 2
Locations
International
Study Type
Interventional
Intervention
ARGX-113
Placebo
Sponsored by
About this trial
This is an interventional treatment trial for Primary Immune Thrombocytopenia
Eligibility Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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.
Sites / Locations
- Vienna
- Wien
- Leuven
- Mont-Godinne
- Brno
- Praha
- Bordeaux
- Grenoble
- Paris
- Berlin
- Hanover
- Tubingen
- Budapest
- Debrecen
- Gyula
- Kaposvar
- Nyiregyhaza
- Pecs
- Lublin
- Opole
- Wroclaw
- A Coruna
- Barcelona
- Madrid
- Valencia
- Dnipro
- Ivano-Frankivsk
- Nikolaev
- Uzhgorod
- London
Arms of the Study
Arm 1
Arm 2
Arm 3
Arm Type
Experimental
Experimental
Placebo Comparator
Arm Label
ARGX-113 Dose A + SoC
ARGX-113 Dose B +SoC
Placebo + SoC
Arm Description
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
Outcomes
Primary Outcome Measures
Incidence and severity of serious adverse events (SAEs).
Changes from Baseline in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities and clinical laboratory assessments.
Secondary Outcome Measures
Frequency and proportion of patients with initial response
Mean change from Baseline in platelet counts
Full Information
NCT ID
NCT03102593
First Posted
March 14, 2017
Last Updated
July 24, 2023
Sponsor
argenx
Collaborators
Quintiles, Inc.
1. Study Identification
Unique Protocol Identification Number
NCT03102593
Brief Title
A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With ITP
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
Study Type
Interventional
2. Study Status
Record Verification Date
July 2023
Overall Recruitment Status
Completed
Study Start Date
March 13, 2017 (Actual)
Primary Completion Date
April 9, 2019 (Actual)
Study Completion Date
April 9, 2019 (Actual)
3. Sponsor/Collaborators
Responsible Party, by Official Title
Sponsor
Name of the Sponsor
argenx
Collaborators
Quintiles, Inc.
4. Oversight
Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes
5. Study Description
Brief Summary
The purpose of the study is to determine safety, efficacy, tolerability and Pharmacokinetics of ARGX-113 in Patients with Primary Immune Thrombocytopenia.
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.
6. Conditions and Keywords
Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Primary Immune Thrombocytopenia
7. Study Design
Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Protocol designed to evaluate one or more interventions for treating a disease, syndrome or condition.
Masking
ParticipantCare ProviderInvestigatorOutcomes Assessor
Allocation
Randomized
Enrollment
38 (Actual)
8. Arms, Groups, and Interventions
Arm Title
ARGX-113 Dose A + SoC
Arm Type
Experimental
Arm Description
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
Arm Title
ARGX-113 Dose B +SoC
Arm Type
Experimental
Arm Description
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
Arm Title
Placebo + SoC
Arm Type
Placebo Comparator
Arm Description
Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
Intervention Type
Drug
Intervention Name(s)
ARGX-113
Intervention Description
ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
Intervention Type
Other
Intervention Name(s)
Placebo
Intervention Description
ARGX-113 (Dose A or Dose B) or matching placebo will be administered IV weekly
Primary Outcome Measure Information:
Title
Incidence and severity of serious adverse events (SAEs).
Description
Changes from Baseline in vital signs, electrocardiogram parameters (ECGs), physical examination abnormalities and clinical laboratory assessments.
Time Frame
After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
Secondary Outcome Measure Information:
Title
Frequency and proportion of patients with initial response
Description
Mean change from Baseline in platelet counts
Time Frame
Over the study period (up to 13 weeks).
10. Eligibility
Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
85 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria:
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.
Exclusion Criteria:
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.
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Adrian Newland
Organizational Affiliation
Barts Hospital, Cancer Centre in London
Official's Role
Study Chair
Facility Information:
Facility Name
Vienna
City
Vienna
Country
Austria
Facility Name
Wien
City
Wien
Country
Austria
Facility Name
Leuven
City
Leuven
Country
Belgium
Facility Name
Mont-Godinne
City
Namur
Country
Belgium
Facility Name
Brno
City
Brno
Country
Czechia
Facility Name
Praha
City
Praha
Country
Czechia
Facility Name
Bordeaux
City
Bordeaux
Country
France
Facility Name
Grenoble
City
Grenoble
Country
France
Facility Name
Paris
City
Paris
Country
France
Facility Name
Berlin
City
Berlin
Country
Germany
Facility Name
Hanover
City
Hanover
Country
Germany
Facility Name
Tubingen
City
Tubingen
Country
Germany
Facility Name
Budapest
City
Budapest
Country
Hungary
Facility Name
Debrecen
City
Debrecen
Country
Hungary
Facility Name
Gyula
City
Gyula
Country
Hungary
Facility Name
Kaposvar
City
Kaposvar
Country
Hungary
Facility Name
Nyiregyhaza
City
Nyiregyhaza
Country
Hungary
Facility Name
Pecs
City
Pecs
Country
Hungary
Facility Name
Lublin
City
Lublin
Country
Poland
Facility Name
Opole
City
Opole
Country
Poland
Facility Name
Wroclaw
City
Wroclaw
Country
Poland
Facility Name
A Coruna
City
A Coruña
Country
Spain
Facility Name
Barcelona
City
Barcelona
Country
Spain
Facility Name
Madrid
City
Madrid
Country
Spain
Facility Name
Valencia
City
Valencia
Country
Spain
Facility Name
Dnipro
City
Dnipro
Country
Ukraine
Facility Name
Ivano-Frankivsk
City
Ivano-Frankivsk
Country
Ukraine
Facility Name
Nikolaev
City
Nikolaev
Country
Ukraine
Facility Name
Uzhgorod
City
Uzhgorod
Country
Ukraine
Facility Name
London
City
London
Country
United Kingdom
12. IPD Sharing Statement
Plan to Share IPD
Undecided
Citations:
PubMed Identifier
31821591
Citation
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.
Results Reference
result
Learn more about this trial
A Study to Evaluate the Safety, Efficacy, and Pharmacokinetics of ARGX-113 in Patients With ITP
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