Clinical trial NCT03102593: 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
Overall status: COMPLETED
Phase: PHASE2
Conditions: Primary Immune Thrombocytopenia
Lead sponsor: argenx
Sponsor class: INDUSTRY
Collaborators: name: Quintiles, Inc.
First posted: 2017-04-06
Enrollment: 38
Sex: ALL
Minimum age (years): 18
Maximum age (years): 85
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Intervention model description: Protocol designed to evaluate one or more interventions for treating a disease, syndrome or condition.
Primary purpose: TREATMENT

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.

Eligibility 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.

Primary outcomes:
measure: Incidence and severity of serious adverse events (SAEs).
timeFrame: After the first administration of Investigational Medicinal Product day 1 to 30 days of a patient's last visit.
Secondary outcomes:
measure: Frequency and proportion of patients with initial response
timeFrame: Over the study period (up to 13 weeks).
Other outcomes:
Locations:
city: Vienna
country: Austria
facility: Vienna
city: Wien
country: Austria
facility: Wien
city: Leuven
country: Belgium
facility: Leuven
city: Namur
country: Belgium
facility: Mont-Godinne
city: Brno
country: Czechia
facility: Brno
city: Praha
country: Czechia
facility: Praha
city: Bordeaux
country: France
facility: Bordeaux
city: Grenoble
country: France
facility: Grenoble
city: Paris
country: France
facility: Paris
city: Berlin
country: Germany
facility: Berlin
city: Hanover
country: Germany
facility: Hanover
city: Tubingen
country: Germany
facility: Tubingen
city: Budapest
country: Hungary
facility: Budapest
city: Debrecen
country: Hungary
facility: Debrecen
city: Gyula
country: Hungary
facility: Gyula
city: Kaposvar
country: Hungary
facility: Kaposvar
city: Nyiregyhaza
country: Hungary
facility: Nyiregyhaza
city: Pecs
country: Hungary
facility: Pecs
city: Lublin
country: Poland
facility: Lublin
city: Opole
country: Poland
facility: Opole
city: Wroclaw
country: Poland
facility: Wroclaw
city: A Coruña
country: Spain
facility: A Coruna
city: Barcelona
country: Spain
facility: Barcelona
city: Madrid
country: Spain
facility: Madrid
city: Valencia
country: Spain
facility: Valencia
city: Dnipro
country: Ukraine
facility: Dnipro
city: Ivano-Frankivsk
country: Ukraine
facility: Ivano-Frankivsk
city: Nikolaev
country: Ukraine
facility: Nikolaev
city: Uzhgorod
country: Ukraine
facility: Uzhgorod
city: London
country: United Kingdom
facility: London
Study officials:
affiliation: Barts Hospital, Cancer Centre in London
name: Adrian Newland
role: STUDY_CHAIR
References:
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.