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

  • STATUS
    Recruiting
  • End date
    Jul 1, 2022
  • participants needed
    156
  • sponsor
    argenx
Updated on 16 January 2022

Summary

This is a phase 3, multicenter, randomized, double-blinded, placebo-controlled, parallel-group trial to evaluate the efficacy, safety, and effect on QoL/PRO of efgartigimod PH20 SC treatment in adult patients with primary ITP.

Details
Condition Primary Immune Thrombocytopenia
Treatment efgartigimod PH20 SC, Placebo PH20 SC
Clinical Study IdentifierNCT04687072
Sponsorargenx
Last Modified on16 January 2022

Eligibility

Yes No Not Sure

Inclusion Criteria

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)
Male or female, aged ≥18 years at the time the informed consent form (ICF) is signed. Exceptions are made for The Republic of South Korea and Taiwan where, according to local regulatory requirements, legal age is reached at 19 years and 20 years, respectively
Confirmed diagnosis of primary ITP made at least 3 months before randomization and based on the American Society of Hematology Criteria, and no known etiology for thrombocytopenia
Diagnosis supported by a response to a prior ITP therapy (other than TPO-RAs), in the opinion of the investigator
Mean platelet count of <30×10E9/L from at least 3 documented, qualifying counts within the 3 preceding months where at least 2 of the qualifying counts must be taken during the screening period: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization (visit 1). If the third count is not available from the 3 preceding months, this third platelet count can be obtained during the screening period
A documented history of a platelet count of <30×10E9/L before screening
Permitted concurrent ITP medications include corticosteroids, danazol, vinca alkaloids
oral immunosuppressants, dapsone, fostamatinib, and/or oral TPO-RAs
At the start of the trial, the participant either takes concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the participant does not take treatment for ITP (see note) but has received at least 2 prior treatments for ITP. Participants receiving permitted concurrent ITP treatment(s) at baseline must have been stable in dose and frequency for at least 4 weeks before randomization
Note: Participants not receiving concurrent ITP therapy are also eligible for the trial if
they have not received prior ITP therapy for at least 4 weeks before baseline, and 6 months
in case of prior ITP therapy with an anti-CD20 therapy (eg, rituximab)
As defined in Woman of Childbearing Potential in the protocol, women of
childbearing potential must have a negative serum pregnancy test at screening and
a negative urine pregnancy test at baseline before trial medication can be
administered
Women of childbearing potential
Must be on a stable regimen for at least 1 month of a highly effective or
acceptable method of contraception during the trial and for 90 days after the
last administration of IMP
Non-sterilized male participants who are sexually active with a female partner of
childbearing potential must use an acceptable method of contraception, ie, a condom
from signing the ICF through the last administration of the IMP. Male participants are
also not allowed to donate sperm during this time

Exclusion Criteria

Use of any transfusions within 4 weeks prior to randomization
Use of romiplostim within 4 weeks prior to randomization
Secondary ITP/thrombocytopenia associated with another condition, eg, lymphoma
Undergone splenectomy less than 4 weeks prior to randomization
chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune
thrombocytopenia, thrombocytopenia associated with myeloid dysplasia, or hematopoietic
stem cell transplant
At the screening visit, clinically significant laboratory abnormalities as follows
Use of anticoagulants (eg, vitamin K antagonists, direct oral anticoagulants) within 4
weeks prior to randomization
OR -
Use of Ig (IV, SC, or intramuscular route) or plasmapheresis (PLEX) within 4 weeks
prior to randomization
OR -
Adequately treated basal cell or squamous cell skin cancer
Use of an investigational product within 3 months or 5 half-lives (whichever is
Carcinoma in situ of the cervix
longer) before the first dose of the IMP
Use of any monoclonal antibody or Fc fusion proteins, other than those previously
Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
Carcinoma in situ of the breast or
indicated, within 6 months before the first dose of the IMP (eg, anti-CD20)
o Hemoglobin ≤9 g/dL
o International normalized ratio >1.5 or activated partial thromboplastin time
>1.5×upper limit of normal
o total IgG level <6 g/L
History of malignancy unless deemed cured by adequate treatment with no evidence of
Known hypersensitivity reaction to efgartigimod, rHuPH20, or 1 of its excipients
recurrence for ≥3 years before the first administration of IMP. Participants with the
following cancer can be included at any time
Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160
mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments
History of any major thrombotic or embolic event (eg, myocardial infarction, stroke
deep venous thrombosis, or pulmonary embolism) within 12 months prior to randomization
History of coagulopathy or hereditary thrombocytopenia or a family history of
thrombocytopenia
Evidence of an active clinically significant bleeding of an organ or internal mucosal
bleeding, other than expected in ITP, that warrants emergent treatment or therapeutic
procedure based on the investigator's judgment (eg, intracranial hemorrhage, pulmonary
hemorrhage, bleeding with ongoing need for packed red blood cell transfusion)
Estimated high risk of a clinically significant bleeding of an organ or internal
mucosal bleeding, other than expected in ITP, that warrants emergent treatment or
therapeutic procedure according to the investigator's judgment
Clinical evidence of other significant serious diseases, have had a recent major
surgery, or who have any other condition in the opinion of the investigator, that
could confound the results of the trial or put the participant at undue risk
Positive serum test at screening for an active viral infection with any of the
following conditions
Hepatitis B virus (HBV) that is indicative of an acute or chronic infection
unless associated with a negative HBV DNA test
(<https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf>)
Hepatitis C virus (HCV) based on HCV-antibody assay (unless associated with a
negative HCV RNA test)
Human immunodeficiency virus (HIV) based on test results that are associated with
an acquired immunodeficiency syndrome (AIDS)-defining condition or a CD4 count
≤200 cells/mm3
Previously participated in a clinical trial with efgartigimod and have received at
least 1 administration of the IMP
Pregnant or lactating females and those who intend to become pregnant during the trial
or within 90 days after last dose of the IMP
Clinically significant uncontrolled active or chronic bacterial, viral, or fungal
infection at screening
Any other known autoimmune disease that, in the opinion of the investigator, would
interfere with an accurate assessment of clinical symptoms of ITP or put the
participant at undue risk
Clinical evidence of significant unstable or uncontrolled acute or chronic diseases
other than ITP (eg, cardiovascular, pulmonary, hematologic, gastrointestinal
endocrine, hepatic, renal, neurological, malignancy, infectious diseases, uncontrolled
diabetes) despite appropriate treatments which could put the participant at undue risk
Current or history of (ie, within 12 months of screening) alcohol, drug, or medication
abuse
Received a live/live-attenuated vaccine less than 4 weeks before screening. The
receipt of any inactivated, sub-unit, polysaccharide, or conjugate vaccine at any time
before screening is not considered an exclusion criterion
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