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Trial record 1 of 1 for:    NCT04188379
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A Study to Assess the Efficacy and Safety of Efgartigimod in Adult Patients With Primary Immune Thrombocytopenia (ITP). (ADVANCE)

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ClinicalTrials.gov Identifier: NCT04188379
Recruitment Status : Completed
First Posted : December 5, 2019
Last Update Posted : March 7, 2024
Sponsor:
Information provided by (Responsible Party):
argenx

Brief Summary:
This is a randomized, double-blind placebo-controlled multicenter phase 3 trial to evaluate the efficacy and safety of ARGX-113 in participants with primary ITP.

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

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 131 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Quadruple (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: A Phase 3, Multicenter, Randomized, Double-Blinded, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Efgartigimod (ARGX 113) 10 mg/kg Intravenous in Adult Patients With Primary Immune Thrombocytopenia
Actual Study Start Date : December 16, 2019
Actual Primary Completion Date : February 3, 2022
Actual Study Completion Date : February 3, 2022


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

Placebo Comparator: Placebo
Patients receiving placebo
Other: Placebo
Intravenous infusion of placebo




Primary Outcome Measures :
  1. Proportion of patients with chronic ITP with a sustained platelet count response defined as achieving platelet counts of at least 50×10E9/L for at least 4 of the 6 visits between week 19 and 24 of the trial. [ Time Frame: Up to five weeks (between visits 19 and 24) ]
    Proportion of subjects with chronic ITP with a sustained platelet count response was defined as achieving platelet counts of at least 50 × 10^9 per litre for at least 4 of the 6 visits between weeks 19 and 24 of the study.


Secondary Outcome Measures :
  1. Extent of disease control defined as the number of cumulative weeks over the planned 24-week treatment period with platelet counts of ≥50×10E9/L in the chronic ITP population [ Time Frame: Up to 24 weeks (treatment period) ]
    Extent of disease control, defined as the cumulative number of weeks over the planned 24-week treatment period with platelet counts of ≥50 × 10^9/L in the chronic ITP population.

  2. Proportion of patients in the overall population (chronic and persistent ITP) with a sustained platelet count response defined as achieving platelet counts of at least 50×10E9/L for at least 4 of the 6 visits between week 19 and 24 of the trial [ Time Frame: Up to five weeks (between visits 19 and 24) ]
    Proportion of subjects in the overall population achieving platelet counts of at least 50 × 10^9/L for at least 6 of the 8 visits between weeks 17 and 24 of the study.

  3. Incidence and severity of the WHO-classified bleeding events [ Time Frame: Up to 24 weeks (From Week 1 to Week 24) ]

    Incidence of the World Health Organization (WHO)-classified bleeding events in the overall population.

    Analysis was performed on Full Analysis Set population that included all randomized subjects. This secondary endpoint used the WHO-classified bleeding scale. Bleeding was the predominant clinical manifestation of ITP and was typically related to platelet count. Accordingly, measuring bleeding was important for monitoring this subject population. The WHO bleeding scale was neither specific to nor validated for ITP, but had been implemented in ITP clinical studies; no specific and validated tools for assessing bleeding in ITP were available.


  4. Proportion of patients in the overall population achieving platelet counts of at least 50 x 10^9/L for at least 6 of the 8 visits between week 17 and 24 of the trial. [ Time Frame: Up to 7 weeks (between visits 17 and 24) ]

    Proportion of subjects in the overall population (chronic and persistent ITP) with a sustained platelet count response, defined as achieving platelet counts of at least 50 × 10^9/L for at least 4 of the 6 visits between weeks 19 and 24 of the study.

    The analysis was performed on Full Analysis set population that included all randomized subjects in the study.




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:

  • 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).
  • Male or female patient aged ≥18 years.
  • Confirmed ITP diagnosis, at least 3 months before randomization and according to the American Society of Hematology Criteria, and no known other etiology for thrombocytopenia.
  • Diagnosis supported by a response to a prior ITP therapy (other than thrombopoietin receptor agonists [TPO-RAs]), in the opinion of the investigator.
  • Mean platelet count of <30×10E9/L from 2 counts: 1 platelet count collected during the screening period and the predose platelet count on the day of randomization.
  • At the start of the trial, the patient is either on concurrent ITP treatment(s) and has received at least 1 prior therapy for ITP in the past, or the patient is not on treatment for ITP but has received at least 2 prior treatments for ITP. Patients receiving permitted concurrent ITP treatment(s) at baseline, must have been stable in dose and frequency for at least 4 weeks prior to randomization.
  • Women of childbearing potential must have a negative serum pregnancy test at the screening visit and a negative urine pregnancy test at baseline before trial medication (infusion) can be administered.
  • 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.

Exclusion criteria:

  • ITP/thrombocytopenia associated with another condition, eg, lymphoma, chronic lymphocytic leukemia, viral infection, hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
  • Use of certain medications before the start of the studies (more details in the protocol)
  • Patients who have a history of malignancy, including malignant thymoma, or myeloproliferative or lymphoproliferative disorders, unless deemed cured by adequate treatment with no evidence of recurrence for ≥3 years before screening. Patients with completely excised non-melanoma skin cancer (such as basal cell carcinoma or squamous cell carcinoma) or cervical carcinoma in situ would be permitted 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 within 12 months prior to randomization.
  • History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
  • History of a recent or planned major surgery (that involves major organs eg, brain, heart, lung, liver, bladder, or gastrointestinal tract) within 4 weeks of randomization.
  • Positive serum test at screening for an active viral infection with any of the following conditions: Hepatitis B virus (HBV), Hepatitis C virus (HCV), Human immunodeficiency virus (HIV)
  • Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITPdespite appropriate treatments which could put the patient at undue risk.
  • Patients with known medical history of hypersensitivity to any of the ingredients of the IMP.
  • Patients who previously participated in a clinical trial with efgartigimod and have received at least 1 administration of the IMP.
  • Pregnant or lactating females. More details in the protocol

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): NCT04188379


Locations
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Sponsors and Collaborators
argenx
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Responsible Party: argenx
ClinicalTrials.gov Identifier: NCT04188379    
Other Study ID Numbers: ARGX-113-1801
2019-002100-41 ( EudraCT Number )
First Posted: December 5, 2019    Key Record Dates
Last Update Posted: March 7, 2024
Last Verified: March 2024
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by argenx:
immune thrombocytopenia
sustained platelet count response
bleeding events
Additional relevant MeSH terms:
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Thrombocytopenia
Purpura, Thrombocytopenic, Idiopathic
Blood Platelet Disorders
Hematologic Diseases
Cytopenia
Purpura, Thrombocytopenic
Purpura
Blood Coagulation Disorders
Thrombotic Microangiopathies
Hemorrhagic Disorders
Autoimmune Diseases
Immune System Diseases
Hemorrhage
Pathologic Processes
Skin Manifestations