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A Long-term Study to Assess the Safety and Efficacy of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). (ADVANCE+)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04225156
Recruitment Status : Recruiting
First Posted : January 13, 2020
Last Update Posted : November 3, 2020
Sponsor:
Information provided by (Responsible Party):
argenx

Brief Summary:
This is an open-label long-term multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in adult patients with primary ITP.

Condition or disease Intervention/treatment Phase
Primary Immune Thrombocytopenia Biological: efgartigimod Phase 3

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 156 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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.
Actual Study Start Date : June 2, 2020
Estimated Primary Completion Date : June 2022
Estimated Study Completion Date : October 2022


Arm Intervention/treatment
Experimental: efgartigimod
patients receiving efgartigimod
Biological: efgartigimod
Intravenous infusion of efgartigimod
Other Name: ARGX-113




Primary Outcome Measures :
  1. Frequency and severity of Adverse Events [ Time Frame: Up to 56 weeks ]
  2. Frequency and severity of vital signs [ Time Frame: Up to 56 weeks ]
  3. Frequency and severity of laboratory assessments [ Time Frame: Up to 56 weeks ]

Secondary Outcome Measures :
  1. Extent of disease control defined as the number of cumulative weeks over the planned 52-week treatment period with platelet counts of ≥ 50×10^9/L [ Time Frame: Over the 52 weeks of treatment ]
  2. Percentage of patients with overall platelet count response defined as achieving a platelet count of ≥50×10^9/L on at least 4 occasions at any time during the 52-week treatment period. [ Time Frame: Over the 52 weeks of treatment ]
  3. Mean change from baseline in platelet count at each visit. [ Time Frame: Up to 56 weeks, at each visit ]
  4. For patients rolling-over from the ARGX-113-1801 trial with a platelet count of <30×10^9/L: time to response is defined as the time to achieve 2 consecutive platelet counts of ≥50×10^9/L [ Time Frame: UP to 56 weeks, at each visit ]
  5. The number of cumulative weeks over the planned 52-week treatment period with platelet counts of ≥30×10^9/L and at least 20×10^9/L above baseline. [ Time Frame: Over the 52 weeks of treatment ]
  6. In patients with baseline platelet count of <15×10^9/L in the current trial (ARGX-113-1803), the number of cumulative weeks over the planned 52-week treatment period with platelet counts of ≥30×10^9/L and at least 20×10^9/L above baseline. [ Time Frame: Over the 52 weeks of treatment ]
  7. In patients with first exposure to efgartigimod: proportion of patients who achieve a sustained platelet response defined as achieving platelet counts of at least 50×10^9/L for at least 4 of the 6 visits between week 19 and 24 of the trial. [ Time Frame: Up to 5 weeks, between visit 19 and 24 of the trial ]
  8. In patients with first exposure to efgartigimod: proportion of patients in the overall population achieving platelet counts of at least 50x10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial. [ Time Frame: Up to 7 weeks, between visit 17 and 24 of the trial ]
  9. Rate of receipt of rescue therapy (rescue per patient per month). [ Time Frame: Up to 56 weeks, at each visit ]
  10. Reduction in concurrent ITP therapy. [ Time Frame: Up to 56 weeks, at each visit ]
  11. Incidence and severity of the WHO-classified bleeding events. [ Time Frame: Up to 56 weeks, at each visit ]
  12. Change from baseline in Patient reported Outcomes (FACIT-Fatigue) at planned visits. [ Time Frame: Up to 52 weeks ]
  13. Change from baseline in Patient reported Outcomes (Fact-Th6) at planned visits. [ Time Frame: Up to 52 weeks ]
  14. Change from baseline in Quality of Life (SF-36) at planned visits. [ Time Frame: Up to 52 weeks ]
  15. Incidence of anti-drug antibodies (ADA) to efgartigimod. [ Time Frame: Up to 56 weeks ]
  16. Pharmacokinetic parameters of efgartigimod: maximum observed serum concentration (Cmax). [ Time Frame: Up to 56 weeks ]
  17. Serum levels of pharmacodynamics markers: total IgG, IgG isotypes (IgG1, IgG2, IgG3, IgG4). [ Time Frame: Up to 56 weeks ]


Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

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-weeks 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 postmenopausal (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, both ovaries surgically removed, or have a documented permanent female sterilization procedure including tubal ligation). Follicle stimulating hormone can be used to confirm postmenopausal status in amenorrheic patients not on hormonal replacement therapy.
  4. Women of childbearing potential should use a highly effective method of contraception (i.e. pregnancy rate of less than 1% per year) 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.
  5. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective double contraception, being a condom for male patients and a highly effective form of contraception for the female partner of childbearing (same as for female patients described in inclusion criterion 4). 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.

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04225156


Contacts
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Contact: Antonio Guglietta, MD +1 857-350-4834 Clinicaltrials@argenx.com

Locations
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Sponsors and Collaborators
argenx
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Responsible Party: argenx
ClinicalTrials.gov Identifier: NCT04225156    
Other Study ID Numbers: ARGX-113-1803
2019-002101-21 ( EudraCT Number )
First Posted: January 13, 2020    Key Record Dates
Last Update Posted: November 3, 2020
Last Verified: November 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
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Thrombocytopenia
Immune System Diseases
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations