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

Primary Immune Thrombocytopenia Clinical Trial

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03102593
• Other study ID #: ARGX-113-1603
• Secondary ID: 2016-003038-26
• Status: Completed
• Phase: Phase 2
• Start date: March 13, 2017
• Est. completion date: April 9, 2019

Study information

• Verified date: July 2023
• Source: argenx
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 38
• Est. completion date: April 9, 2019
• Est. primary completion date: April 9, 2019
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 85 Years

Eligibility

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.

Related Conditions & MeSH terms

• Primary Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

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

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

Locations

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

Sponsors (2)

Lead Sponsor	Collaborator
argenx	Quintiles, Inc.

Countries where clinical trial is conducted

Austria,  Belgium,  Czechia,  France,  Germany,  Hungary,  Poland,  Spain,  Ukraine,  United Kingdom, 

References & Publications (1)

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 a — View Citation

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence and severity of serious adverse events (SAEs).	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	Frequency and proportion of patients with initial response	Mean change from Baseline in platelet counts	Over the study period (up to 13 weeks).