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

Phase 1

• Conditions: Primary Immune Thrombocytopenia; MedDRA version: 20.1 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-DE
• Lead Sponsor: Argenx BVBA

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2016-12-08
• Last Update Posted: 10 December 2019

Recruitment & Eligibility

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

Inclusion Criteria

Main study
1. Ability to understand the requirements of the study, and comply
withthe 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)
stable in dose and frequency for at least 4 weeks prior to Screening.
4. Confirmed diagnosis of ITP according to the American Society of
Hematology Criteria 2011 with (average) blood platelet counts < 30 ×
10E9/L and who have not experienced major bleeding in the last 4
weeks prior to 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.
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.
7. Non-sterilized male patients who are sexually active with a female
partner of childbearing potential must use effective double contraception
Additional Inclusion Criteria for the Extended Follow-up Period
- Sign the amended ICF of the main study including its extended
followup
period
- Completed Visit 16 of the FU period of the protocol with a platelet
count = 30 × 10E9/L and/or at least doubling of the Baseline platelet
count and absence of bleeding and remained on the same SoC
Eligibility criteria for the open-label treatment period (first treatment
cycle)
1. Please refer to inclusion criteria 5, 6 and 7 from the main study.
2. Provide written informed consent
3. Received at least 3 doses of the IMP and had at least 2 weeks of
follow-up in the main study.
4. Patient is at the same SoC as in the main study. Dose and/or
frequency increase is allowed, changing or stopping the SoC is not
allowed.
5. During up to 21 weeks of FU, the patient is relapsing i.e. platelet count
decreases to below 30 x 10E9/L or the patient's platelets never went up
to 30 x 10E9/L and are still below 30 x 10E9/L, and absence of bleeding.
Eligibility criteria for subsequent open-label retreatment cycles
The patient has the right to receive more than 1 retreatment cycle if:
1. Patient reached a platelet count of at least twice the platelet count
measured on the day of the first IMP administration during the previous
(re)treatment cycle, confirmed on at least 2 separate consecutive
occasions (at least 1 day apart but with maximum 7 days in between the
2 measurements), and measured during the treatment period up to
minimum 4 weeks of follow-up.
2. Patient

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/dL.
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
Note: A patient with isolated laboratory values that meet the thresholds listed above may be considered eligible. The agreed upon medical rationale, discussed between the investigator and sponsor, must be documented in the patient's chart prior to patient enrolment in the study
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, ar

Study & Design

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