A Phase 3, Multicenter, Open-label, Long-term Trial to Evaluate the Safety and Efficacy of Efgartigimod (ARGX-113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia

Phase 3

Withdrawn

• Conditions: Werlhof's disease; Primary Immune Thrombocytopenia; 10003816

• Registration Number: NL-OMON55305
• Lead Sponsor: argenx BV

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Last Update Posted: 15 April 2024

Recruitment & Eligibility

• Status: Withdrawn
• Sex: Not specified
• Target Recruitment: 20

Inclusion 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
o oral
o Intravaginal
o Transdermal
* progestogen-only hormonal contraception associated with inhibition of
ovulation:
o Oral
o Injectable
o 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 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 patient 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 t

Exclusion Criteria

1. Introduction or continuation of non-permitted medications during the
ARGX-113-1801 trial (such as anti-CD20 therapy, romiplostim, monoclonal
antibodies, 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.

Study & Design

• Study Type: Interventional
• Study Design: Not specified

Primary Outcome Measures

Name	Time	Method
<p>Frequency and severity of AEs, vital signs, and laboratory assessments.</p><br>