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

Phase 2

Completed

• Conditions: Primary Immune Thrombocytopenia
• Interventions: Other: Placebo; Drug: ARGX-113

• Registration Number: NCT03102593
• Lead Sponsor: argenx

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.

Study Timeline

• Study Start: 2017-03-13
• Study First Submitted: 2017-03-14
• Study First Submitted QC: 2017-03-30
• Study First Posted: 2017-04-06
• Primary Completion: 2019-04-09
• Study Completion: 2019-04-09
• Status Verified: 2023-07
• Last Update Submitted: 2023-07-24
• Last Update Posted: 2023-07-25

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 38

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 & Design

• Study Type: INTERVENTIONAL
• Study Design: PARALLEL

Arm && Interventions

Group	Intervention	Description
Placebo + SoC	Placebo	Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
ARGX-113 Dose B +SoC	ARGX-113	Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo
ARGX-113 Dose A + SoC	ARGX-113	Patients will be randomized in a 1:1:1 ratio to ARGX-113 (Dose A or Dose B) or placebo

Primary Outcome Measures

Name	Time	Method
Incidence and severity of serious adverse events (SAEs).	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

Name	Time	Method
Frequency and proportion of patients with initial response	Over the study period (up to 13 weeks).	Mean change from Baseline in platelet counts

Trial Locations

Locations (30)

Gyula; 🇭🇺 Gyula, Hungary

Vienna; 🇦🇹 Vienna, Austria

Paris; 🇫🇷 Paris, France

Berlin; 🇩🇪 Berlin, Germany

Leuven; 🇧🇪 Leuven, Belgium

Wien; 🇦🇹 Wien, Austria

Bordeaux; 🇫🇷 Bordeaux, France

Mont-Godinne; 🇧🇪 Namur, Belgium

Grenoble; 🇫🇷 Grenoble, France

Brno; 🇨🇿 Brno, Czechia

Praha; 🇨🇿 Praha, Czechia

Hanover; 🇩🇪 Hanover, Germany

Tubingen; 🇩🇪 Tubingen, Germany

Budapest; 🇭🇺 Budapest, Hungary

Debrecen; 🇭🇺 Debrecen, Hungary

Kaposvar; 🇭🇺 Kaposvar, Hungary

Nyiregyhaza; 🇭🇺 Nyiregyhaza, Hungary

Pecs; 🇭🇺 Pecs, Hungary

Lublin; 🇵🇱 Lublin, Poland

Opole; 🇵🇱 Opole, Poland

A Coruna; 🇪🇸 A Coruña, Spain

Wroclaw; 🇵🇱 Wroclaw, Poland

Barcelona; 🇪🇸 Barcelona, Spain

Madrid; 🇪🇸 Madrid, Spain

Valencia; 🇪🇸 Valencia, Spain

Dnipro; 🇺🇦 Dnipro, Ukraine

Ivano-Frankivsk; 🇺🇦 Ivano-Frankivsk, Ukraine

Uzhgorod; 🇺🇦 Uzhgorod, Ukraine

Nikolaev; 🇺🇦 Nikolaev, Ukraine

London; 🇬🇧 London, United Kingdom