A Long-term study to assess the efficacy and safety of efgartigimod in adult patients with primary immune thrombocytopenia

JRCT ID: jRCT2080225021

Registered date:20/01/2020

Basic Information

• Recruitment status: completed
• Health condition(s) or Problem(s) studied: Primary immune thrombocytopenia
• Date of first enrollment: 15/02/2021
• Target sample size: 5
• Countries of recruitment: Austria,Belgium,Bulgaria,The Czech Republic,France,Georgia,Germany,Hungary,Italy,The Netherlands,Poland,Russia,Spain,Turkey,Ukraine,The United Kingdom,The United States
• Study type: Interventional
• Intervention(s): Patients will receive 10 mg/kg of ARGX-113 weekly or biweekly (continue as in the ARGX-113-1801 trial), and a change in dosing frequency is permitted as from visit 1 (baseline).

Outcome(s)

• Primary Outcome: Frequency and severity of AEs, vital signs, and laboratory assessments.

Key inclusion & exclusion criteria

• Age minimum: >= 20age old
• Age maximum: Not applicable
• Gender: Both

Include criteria

1. Ability to understand the requirements 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).
2. Patients enrolled in the ARGX-113-1801 trial who completed the 24 week trial period.
3. Women of childbearing potential must have a negative urine pregnancy test at baseline before trial medication (infusion) can be administered. Women are considered of childbearing potential unless they are post-menopausal (defined by continuous amenorrhea) for at least 1 year with a follicle-stimulating hormone (FSH) of >40 IU/L or are surgically sterilized (i.e. women who had a hysterectomy, a bilateral salpingectomy, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle-stimulating hormone can be used to confirm post-menopausal status in amenorrheic patients not on hormonal replacement therapy.
4. Women of childbearing potential should use a highly effective or acceptable method of contraception during the trial and for 90 days after the last administration of the IMP. They must be on a stable regimen, for at least 1 month: combined (estrogen and progestogen containing) hormonal contraception associated with inhibition of ovulation (oral, intravaginal, transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, implantable), intrauterine device (IUD), intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner (provided that the partner is the sole sexual partner of the trial participant and that aspermia was documented post procedure), continuous abstinence from heterosexual sexual contact. Sexual abstinence is only allowable if it is the preferred and usual lifestyle of the patient. Periodic abstinence (calendar, symptothermal, post-ovulation methods) is not acceptable. Male or female condom with or without spermicide. Cap, diaphragm, or sponge with spermicide.
5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use an acceptable method of contraception, ie, a condom. Male patients practicing true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant) can be included. Sterilized male patients who have had a vasectomy with documented aspermia post procedure can be included. In addition, male patients are not allowed to donate sperm during this period from signing of informed consent form, throughout the duration of the trial, and for 90 days after the last administration of IMP.
In addition to the above criteria, for patients 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 becomes available through another patient program for patients with primary ITP)
6. 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).
7. Patient has completed a 52 week treatment period.

Exclude criteria

1. Introduction or continuation of non-permitted medications during the ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal antibodies, or Fc fusion proteins, or live/live-attenuated vaccines).
2. Pregnant or lactating women, and those intending to become pregnant during the trial or within 90 days after the last dosing.
3. Patients with known medical history of hypersensitivity to any of the ingredients of efgartigimod.
4. Use of any other investigational drug or participation in any other investigational trial.

Related Information

• Primary Sponsor: argenx BV (ICCC: ICON JAPAN K.K.)
• Secondary ID(s): 2019-002101-21