Working…
Help guide our efforts to modernize ClinicalTrials.gov.
Send us your comments by March 14, 2020.
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Study to Assess the Efficacy and Safety 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: NCT04188379
Recruitment Status : Recruiting
First Posted : December 5, 2019
Last Update Posted : February 7, 2020
Sponsor:
Information provided by (Responsible Party):
argenx BVBA

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

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

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 156 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 (ITP)
Actual Study Start Date : December 16, 2019
Estimated Primary Completion Date : June 6, 2021
Estimated Study Completion Date : October 31, 2021


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 x 10^9/L for at least 4 of the 6 visits between visits 19 and 24 of the trial. [ Time Frame: Up to five weeks (between visits 19 and 24) ]

Secondary Outcome Measures :
  1. Number of cumulative weeks over the planned 24-week treatment period with platelet counts of at least 50x10^9/L in the chronic ITP population [ Time Frame: Up to 24 weeks (treatment period) ]
  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 x 10^9/L for at least 4 of the 6 visits between visit 19 and 24 of the trial. [ Time Frame: Up to five weeks (between visits 19 and 24) ]
  3. Incidence and severity of the WHO-classified bleeding events [ Time Frame: Up to 31 weeks, at each visit ]
  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 visits 17 and 24 of the trial. [ Time Frame: Up to 7 weeks (between visits 17 and 24) ]


Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
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. Male or female patient aged ≥18 years.
  3. Confirmed ITP diagnosis, at least 3 months before randomization and according to the American Society of Hematology Criteria 2011, and no known other etiology for thrombocytopenia.
  4. Diagnosis supported by a response to a prior ITP therapy (other than thrombopoietin receptor agonists [TPO-RAs]), in the opinion of the investigator.
  5. Mean platelet count of <30×10^9/L (and no single platelet count of >35×10^9/L) from 3 qualifying counts, 2 during the screening period and the pre-dose platelet count at visit 1. The 3 platelet counts must be over the course of 7 to 14 days, with at least 2 days between any 2 counts.
  6. 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.

    Permitted concurrent ITP medications include oral corticosteroids, oral immunosuppressants, dapsone/danazol, and/or eltrombopag.

    Patients not receiving concurrent ITP therapy are also eligible for the trial if they have not received prior ITP therapy for at least 4 weeks prior to baseline, and 6 months in case of prior ITP therapy with an anti-CD20 therapy (e.g. rituximab).

  7. Women of childbearing potential must have a negative serum pregnancy test at the screening visit and a negative urine pregnancy test at baseline prior to infusion. Women of childbearing potential are defined as all female patients 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 sterile (i.e. women who had a hysterectomy, both ovaries surgically removed, or have documented tubal ligation or any other documented permanent female sterilization procedure). Follicle-stimulating hormone can be used to confirm post-menopausal status in amenorrheic patients not on hormonal replacement therapy.
  8. 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, of combined estrogen and progestogen containing hormonal contraception with inhibition of ovulation, progestogen-only hormonal contraception associated with inhibition of ovulation, intrauterine device (IUD), intrauterine hormone-releasing system, bilateral tubal occlusion, vasectomized partner, or agree upon 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.
  9. 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 potential (same as for female patients described in inclusion criterion 8). 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 ICF, throughout the duration of the trial, and for 90 days after the last administration of IMP.

Exclusion Criteria:

  1. ITP/thrombocytopenia associated with another condition, e.g. lymphoma, chronic lymphocytic leukemia, viral infection, autoimmune disorders, thyroid disease, human immunodeficiency virus (HIV), hepatitis, induced or alloimmune thrombocytopenia, or thrombocytopenia associated with myeloid dysplasia.
  2. Use of anticoagulants (e.g. 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 IVIg, subcutaneous or intramuscular route, or plasmapheresis (PLEX), 4 weeks prior to randomization.
  5. Use of anti-CD20 therapy (e.g. rituximab) within 6 months prior to randomization.
  6. Use of romiplostim within 4 weeks prior to randomization.
  7. Use of fostamatinib within 4 weeks prior to randomization.
  8. Undergone splenectomy less than 4 weeks prior to randomization.
  9. Use of any other investigational drug within 3 months or 5 half-lives of the drug (whichever is longer) prior to randomization.
  10. Use of monoclonal antibodies or crystallized fragment (Fc) fusion proteins, other than those previously indicated, within 3 months prior to randomization.
  11. At the screening visit, clinically significant laboratory abnormalities as below: · Hemoglobin ≤9 g/dL. - OR - · International normalized ratio >1.5 or activated partial thromboplastin time >1.5×ULN. - OR - · Total IgG level <6 g/L.
  12. 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.
  13. Uncontrolled hypertension, defined as a repeated elevated blood pressure exceeding 160 mmHg (systolic) and/or 100 mmHg (diastolic) despite appropriate treatments.
  14. History of any thrombotic or embolic event within 12 months prior to randomization.
  15. History of coagulopathy or hereditary thrombocytopenia or a family history of thrombocytopenia.
  16. History of a recent or planned major surgery (that involves major organs e.g. brain, heart, lung, liver, bladder, or gastrointestinal tract) within 4 weeks of randomization.
  17. Patients with known serum-positivity or who test positive for an active viral infection at screening with: Hepatitis B Virus (HBV) (except patients who are anti-HBs Ab positive because of HBV vaccination), Hepatitis C Virus, HIV.
  18. Clinical evidence of significant unstable or uncontrolled acute or chronic diseases other than ITP (e.g. cardiovascular, pulmonary, hematologic, gastrointestinal, endocrine, hepatic, renal, neurological, malignancy, infectious diseases, uncontrolled diabetes) despite appropriate treatments which could put the patient at undue risk.
  19. Patients with known medical history of hypersensitivity to any of the ingredients of the IMP.
  20. Patients who previously participated in a clinical trial with efgartigimod.
  21. Pregnant or lactating females.
  22. Employees of the investigator or trial center, with direct involvement in the proposed trial or other trials under the direction of that investigator or trial center, as well as family of the employees or the investigator.

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


Contacts
Layout table for location contacts
Contact: Antonio Guglietta, MD +1 857-350-4834 clinicaltrials@argenx.com

Locations
Layout table for location information
United States, Florida
Investigator Site 1 Recruiting
Jacksonville, Florida, United States, 32204
Contact: Antonio Guglietta, MD    857-350-4834    clinicaltrials@argenx.com   
Investigator site 2 Recruiting
Ocala, Florida, United States, 34474
Contact: Antonio Guglietta, MD    857-350-4834    clinicaltrials@argenx.com   
United States, Illinois
Investigator Site 3 Recruiting
Quincy, Illinois, United States, 62301
Contact: Antonio Guglietta, MD    857-350-4834    clinicaltrials@argenx.com   
Sponsors and Collaborators
argenx BVBA

Layout table for additonal information
Responsible Party: argenx BVBA
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: February 7, 2020
Last Verified: February 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Additional relevant MeSH terms:
Layout table for MeSH terms
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
Signs and Symptoms