A Study of the safety and effectiveness of ARGX-113 in Patients with Primary Immune Thrombocytopenia

Phase 1

• Conditions: Primary Immune Thrombocytopenia; MedDRA version: 19.0 Level: LLT Classification code 10074678 Term: Primary immune thrombocytopenic purpura System Organ Class: 100000004851; Therapeutic area: Body processes [G] - Immune system processes [G12]

• Registration Number: EUCTR2016-003038-26-HU
• Lead Sponsor: Argenx BVBA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-11-30
• Last Update Posted: 30 June 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: Not specified
• Target Recruitment: 36

Inclusion Criteria

1. Ability to understand the requirements of the study, provide written informed consent (including consent for the use and disclosure of research-related health information), and comply with the study protocol procedures (including required study visits).
2. Male or female patients aged = 18 to = 85 years.
3. Eligible patients must receive standard-of-care treatment for ITP following the ASH guidelines and International Working Group (IWG) that has been stable in dose and frequency for at least 4 weeks prior to Screening. Standard-of-care may include oral corticosteroids and/or permitted oral immunosuppressants and/or TPO-R agonist (see Section 7.9.2).
4. Confirmed diagnosis of ITP according to the American Society of Hematology Criteria 2010 with blood platelet counts < 30 × 109/L and who have not experienced major bleeding in the last 4 weeks prior to Screening.1 Note: The average platelet counts must be < 30 × 109/L with no single reading > 35 × 109/L as measured on 2 separate occasions at least 1 day apart during the Screening.
5. Women of childbearing potential must have a negative serum pregnancy test at Screening and a negative urine pregnancy test at Baseline prior to administration of IMP.
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 follicle-stimulating hormone (FSH) > 40 IU/L or are surgically sterile (i.e., women who had a hysterectomy, both ovaries surgically removed, or have current documented tubal ligation or any other permanent female sterilization procedure). Follicle-stimulating hormone can be used to confirm post-menopausal status in amenorrheic patients not on hormonal replacement therapy if the value is within the post-menopausal range per the central laboratory.
6. Female participants of childbearing potential must agree to use a highly effective method of birth control (i.e., pregnancy rate of less than 1% per year) during the study and for 90 days after the discontinuation of IMP.
Contraceptive methods that result in a low failure rate when used consistently and correctly include methods such as combined hormonal contraception associated with inhibition of ovulation (oral, intravaginal, and transdermal), progestogen-only hormonal contraception associated with inhibition of ovulation (oral, injectable, and implantable), intrauterine devices (IUDs), intrauterine hormone-releasing system, true sexual abstinence (when this is in line with the preferred and usual lifestyle of the participant-periodic abstinence [e.g., calendar, ovulation] and withdrawal methods are not acceptable methods of contraception), bilateral tubal occlusion, or a male partner who has had a vasectomy with documented aspermia post procedure. All female participants using hormonal contraceptive, especially those using mycophenolate, must also use a barrier method (i.e., condom or occlusive cap [diaphragm or cervical/vault caps]) and should have been stable on their hormonal contraceptive treatment for at least 4 weeks prior to Screening.
7. Non-sterilized male patients who are sexually active with a female partner of childbearing potential must use effective double contraception, of which 1 method must be a barrier method and the other another barrier method or highly effective form of cont

Exclusion Criteria

1. Use of anticoagulants, or any drug with antiplatelet effect (e.g., acetylsalicylic acid [aspirin] or other salicylate containing medications, cyclooxygenase inhibitors, adenosine diphosphate (ADP) receptor inhibitors, phosphodiesterase inhibitors, glycoprotein IIB/IIIA inhibitors, adenosine reuptake inhibitors, thromboxane inhibitors, antimalarials, and prostacyclins) during the study and within 3 weeks prior to Screening.
2. Patients who have received any blood support or transfusion within 4 weeks prior to Screening.
3. Use of IVIg or anti-D immunoglobulin treatment within 4 weeks prior to Screening.
4. Use of recombinant thrombopoietin at any time.
5. Use of rituximab within 6 months prior to Screening. Use of any anti-CD20 other than rituximab at any time is not permitted.
6. Use of corticosteroids which has not been stable for at least 4 weeks prior to Screening.
7. Use of immunosuppressants is not permitted within 4 weeks prior to Screening, with the exception of the following oral immunosuppressants: azathioprine [up to 2.5 mg/kg/day], danazol [up to 15 mg/kg/day], mycophenolate mofetil [up to 3 g/day], mycophenolate sodium [up to 2160 mg/day]) which must have been stable for at least 4 weeks prior to Screening.
8. Use of any other biological therapy or investigational drug than those previously indicated within 3 months or 5 half-lives of the drug (whichever is longer) prior to Screening.
9. Received vaccinations within 4 weeks prior to Screening or planned during the study.
10. At Screening, have clinically significant laboratory abnormalities given as below:
a. Aspartate aminotransferase (AST) or ALT > 3 × upper limit of normal (ULN).
b. Total serum bilirubin of > 1.5 x ULN (except for Grade 1 hyperbilirubinemia as defined by the National Cancer Institute [NCI] Common Terminology Criteria for Adverse Events [CTCAE], due solely to a documented medical diagnosis of Gilbert’s syndrome).
c. Serum creatinine of > 1.5 mg/dL or creatinine clearance < 50 mL/min (using Chronic Kidney Disease Epidemiology - Creatinine formula).
d. Hemoglobin = 9 g/L.
e. Thyroid stimulating hormone or thyroglobulin outside of the central laboratory normal range.
f. International normalized ratio (INR) > 1.5 or activated partial thromboplastin time > 1.5 × ULN.
g. Total immunoglobulin G (IgG) level < 6 g/L.
h. Presence of = 1+ proteinuria
11. History of myeloproliferative or lymphoproliferative disorders at any time; or have a history of malignancy at any time unless deemed cured by adequate treatment with no evidence of recurrence for = 5 years prior to Screening. Patients with completely excised nonmelanoma skin cancers or cervical carcinoma in situ would be permitted at any time.
12. History of cerebrovascular accident or myocardial infarction within the last 12 months, before Screening, or current severe/unstable angina, arrhythmia, or at risk of ventricular arrhythmia, symptomatic congestive heart failure (New York Heart Association Class III or IV), or uncontrolled hypertension (Grade 3 to 4 CTCAE). Uncontrolled hypertension is defined as a repeated elevation in the blood pressure exceeding 140 mmHg and over 90 mmHg despite appropr

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified