A Phase 3 Study to Evaluate the Safety and Efficacy of Efgartigimod PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia

Phase 3Active Not RecruitingNCT04812925

argenx173 enrolled

Overview

A Phase 3 study to evaluate the safety and efficacy of efgartigimod PH20 subcutaneous in adult patients with primary immune thrombocytopenia

Study Type

INTERVENTIONAL

Allocation

Purpose

TREATMENT

Masking

NONE

Enrollment

173

Conditions

Primary Immune Thrombocytopenia

Interventions

efgartigimod PH20 SCBIOLOGICAL

Subcutaneous injection with efgartigimod PH20 SC

Eligibility

Sex: ALLMin age: 18 Years

Medical Language ↔ Plain English

Inclusion Criteria: 1. Ability to understand the requirements of the trial and provide written informed consent (including consent for the use and disclosure of research-related health information), willing and able to comply with the trial protocol procedures (including attending the required trial visits). 2. Participants enrolled in the ARGX-113-2004 trial who completed the 24-week trial period. Note: If a participant has had an SAE during the ARGX-113-2004 trial, their eligibility should be evaluated by the investigator and the sponsor's trial physician. The decision of enrolling the participant will be evaluated case by case. 3a. Agree to use contraceptives consistent with local regulations and the following: • Female participants of childbearing potential must have a negative urine pregnancy test at baseline before receiving IMP. In addition to the above criteria, for participants who want to continue receiving efgartigimod during an additional 52-week treatment period (only applicable in case efgartigimod is not yet commercially available for patients with primary ITP or available through another patient program for patients with primary ITP), the following criteria apply: 4\. Ability to understand the requirements of the additional 52-week treatment period of the trial, to provide written informed consent (including consent for the use and disclosure of research-related health information), and to comply with the trial protocol procedures (including required trial visits). 5\. Participant has completed a 52-week treatment period. Exclusion criteria: 1. Introduction or continuation of nonpermitted medications during the ARGX-113-2004 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, Fc fusion proteins, or live/live-attenuated vaccines) 2. Use of any other investigational drug or participation in any other investigational trial 3. Known hypersensitivity reaction to efgartigimod PH20 SC or any of its excipients 4. Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of efgartigimod PH20 SC

Locations (83)

Outcomes

Primary Outcomes

Incidence, frequency, and severity of adverse events (AEs), AEs of special interest (AESIs), and serious AEs (SAEs)

Time frame: 216 weeks

Vital sign measurement: blood pressure in the overall population

Time frame: 216 weeks

ECG: PR, QT and QRS interval in the overall population

Time frame: 216 weeks

Laboratory safety evaluations: CRP analysis in the overall population

Time frame: 216 weeks

Secondary Outcomes

Extent of disease control defined as the percentage of weeks in the trial with platelet counts of ≥50×10E9/L

Time frame: 52 weeks

Proportion of patients with overall platelet count response defined as achieving a platelet count of ≥50×10E9/L on at least 4 occasions at any time during the 52-week treatment period

Time frame: 52 weeks

Mean change from baseline in platelet count at each visit

Time frame: 52 weeks

For patients rolling over from the ARGX-113-2004 trial with a platelet count of <30×10E9/L: time to response defined as the time to achieve 2 consecutive platelet counts of ≥50×10E9/L

Time frame: 52 weeks

The percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline

Time frame: 52 weeks

In patients with a baseline platelet count of <15×10E9/L in the current trial (ARGX-113-2005), the percentage of weeks in the trial with platelet counts of ≥30×10E9/L and ≥20×10E9/L above baseline

Data from ClinicalTrials.gov

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Investigator Site 0010116

Bentonville, Arkansas, United States

Investigator site 0010045

Washington D.C., District of Columbia, United States

Investigator Site 0010062

Fort Wayne, Indiana, United States

Investigator site US0010042

Iowa City, Iowa, United States

Investigator Site 0010095

Oklahoma City, Oklahoma, United States

Investigator Site 0540001

Buenos Aires, Argentina

Investigator site 540004

Buenos Aires, Argentina

Investigator Site 0610012

Garran, Australia

Investigator Site 0610003

West Perth, Australia

Investigator site 610005

Westmead, Australia

...and 73 more locations

Time frame: 52 weeks

In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of ≥50×10E9/L for at least 4 of the 6 visits between week 19 and week 24

Time frame: 5 weeks (week 19-24)

In patients with the first exposure to efgartigimod PH20 SC, the proportion of patients achieving platelet counts of ≥50×10E9/L for at least 6 of the 8 visits between week 17 and week 24

Time frame: 7 weeks (week 17-24)

Proportion of patients for whom dose and/or frequency of concurrent ITP therapies have been reduced compared to baseline

Time frame: 52 weeks

Rate of receipt of rescue therapy (rescue per patient per month)

Time frame: 52 weeks

Incidence of the World Health Organization (WHO)-classified bleeding events

Time frame: 52 weeks

Severity of the World Health Organization (WHO)-classified bleeding events

Time frame: 52 weeks

Serum efgartigimod concentration observed predose (Ctrough)

Time frame: 52 weeks

Change from baseline in PRO (Functional Assessment of Chronic Illness Therapy Fatigue Scale [FACIT-fatigue]) at planned visits

Time frame: 52 weeks

Change from baseline in PRO (Functional Assessment of Cancer Therapy questionnaire-Th6 [FACT-Th6]) at planned visits

Time frame: 52 weeks

Change from baseline in PRO (QoL (Short Form-36 [SF-36]) at planned visits

Time frame: 52 weeks

Pharmacodynamics markers: total IgG

Time frame: 52 weeks

Number of patients who performed self-administration at home over time

Time frame: 52 weeks

Percentage of patients who performed self-administration at home over time

Time frame: 52 weeks

Number of caregivers who administered the injection to the patient at home over time

Time frame: 52 weeks

Percentage of caregivers who administered the injection to the patient at home over time

Time frame: 52 weeks

Number of training visits needed for the participant or caregiver to be competent to start administering efgartigimod PH20 SC

Time frame: 52 weeks

Number of self- or caregiver-supported administrations at home

Time frame: 52 weeks

Percentage of self- or caregiver-supported administrations at home

Time frame: 52 weeks

Incidence and prevalence of antibodies to efgartigimod

Time frame: 216 weeks

Titers of antibodies to efgartigimod

Time frame: 216 weeks

Presence of neutralizing antibodies (NAb) against efgartigimod

Time frame: 216 weeks