PER-016-21尚未招募3 期
A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and the Safety of Efgartigimod (ARGX-113) PH20 Subcutaneous in Adult Patients With Primary Immune Thrombocytopenia
适应症
试验速览
- 阶段
- 3 期
- 状态
- 尚未招募
- 发起方
- Argenx BV
研究概览
简要总结
暂无简介。
研究设计
- 研究类型
- Interventional
入排标准
- 性别
- All
入选标准
- •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. Male or female, aged =18 years at the time the informed consent form (ICF) is signed
- •3. 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
- •4. Diagnosis supported by a response to a prior ITP therapy (other than TPO-RAs), in
- •the opinion of the investigator
- •5. Mean platelet count of <30×109/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.
- •6. A documented history of a platelet count of <30×109/L before screening
- •7. 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.
- •Permitted concurrent ITP medications include corticosteroids, danazol, vinca
- •alkaloids, oral immunosuppressants, dapsone, fostamatinib, and/or oral TPO-RAs.
- •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).
- •8. Women of childbearing potential:
- •As defined in Woman of Childbearing Potential, 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
- •Must be on a stable regimen for at least 1 month of a highly effective or
- •acceptable method of contraception (see Female Contraception) during the trial
- •and for 90 days after the last administration of IMP
- •9. 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 (see Male Contraception) from signing the ICF through the last administration of the IMP. Male participants are also not allowed to donate sperm during this time.
排除标准
- •1. Secondary ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, thrombocytopenia associated with myeloid dysplasia, or hematopoietic stem cell transplant
- •2. Use of anticoagulants (eg, vitamin K antagonists, direct oral anticoagulants) within
- •4 weeks prior to randomization
- •3. Use of any transfusions within 4 weeks prior to randomization
- •4. Use of Ig (IV, SC, or intramuscular route) or plasmapheresis (PLEX) within 4 weeks
- •prior to randomization
- •5. Use of romiplostim within 4 weeks prior to randomization
- •6. Undergone splenectomy less than 4 weeks prior to randomization
- •7. Use of an investigational product within 3 months or 5 half-lives (whichever is
- •longer) before the first dose of the IMP
- •8. Use of any monoclonal antibody or Fc fusion proteins, other than those previously
- •indicated, within 6 months before the first dose of the IMP (eg, anti-CD20)
- •9. At the screening visit, clinically significant laboratory abnormalities as follows:
- •Hemoglobin =9 g/dL
- •International normalized ratio >1.5 or activated partial thromboplastin
- •time >1.5×upper limit of normal
- •total IgG level <6 g/L
- •History of malignancy unless deemed cured by adequate treatment with no evidence
- •of recurrence for =3 years before the first administration of IMP. Participants with the
- •following cancer can be included at any time:
- •a. Adequately treated basal cell or squamous cell skin cancer
- •b. Carcinoma in situ of the cervix
- •c. Carcinoma in situ of the breast or
- •d. Incidental histological finding of prostate cancer (TNM stage T1a or T1b)
- •11. Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding
- •160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments
- •12. History of any major thrombotic or embolic event (eg, myocardial infarction, stroke,
- •deep venous thrombosis, or pulmonary embolism) within 12 months prior to
- •randomization
- •13. History of coagulopathy or hereditary thrombocytopenia or a family history of
- •thrombocytopenia
- •14. 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
- •15. Positive serum test at screening for an active viral infection with any of the following
- •conditions:
- •a. Hepatitis B virus (HBV) that is indicative of an acute or chronic infection
- •(https://www.cdc.gov/hepatitis/HBV/PDFs/SerologicChartv8.pdf)
- •b. Hepatitis C virus (HCV) based on HCV-antibody assay (unless associated with a
- •negative HCV RNA test)
- •c. 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
- •16. Known hypersensitivity reaction to efgartigimod, rHuPH20, or 1 of its excipients
- •17. Previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP
- •18. Pregnant or lactating females and those who intend to become pregnant during the trial or within 90 days after last dose of the IMP
- •19. Clinically significant uncontrolled active or chronic bacterial, viral, or fungal
- •infection at screening
- •20. Any other known autoimmune disease that, in the opinion of the investigator, would interfere with an accurate as
研究者
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