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

Phase 1

• Conditions: Primary Immune Thrombocytopenia; MedDRA version: 20.1Level: LLTClassification code 10074678Term: Primary immune thrombocytopenic purpuraSystem Organ Class: 100000004851; Therapeutic area: Body processes [G] - Immune system processes [G12]

• Registration Number: EUCTR2016-003038-26-BE
• 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: 25 November 2019

Recruitment & Eligibility

• Status: ot Recruiting
• Sex: All
• 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 received at least 3 doses of the IMP and had at least 4 weeks
follow-up in the previous treatment cycle.
3. Patient is at the same SoC as in the previous treatment cycle. Dose
and/or frequency increase is allowed, changing or stopping the SoC is
not allowed.
4. 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.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 22
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 14

Exclusion Criteria

1. Use of anticoagulants, or any drug with antiplatelet effect 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.
5. Use of rituximab within 6 months prior to Screening. other anti-CD20s
not permitted
6. Use of corticosteroids not been stable for at least 4 weeks prior to
Screening
7. Use of immunosuppressants not permitted within 4 weeks prior to
Screening except azathioprine, danazol, mycophenolate mofetil,
mycophenolate sodium 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
11. History of myeloproliferative or lymphoproliferative disorders at any
time; or history of malignancy at any time unless deemed cured
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
13. History of any thrombotic or embolic event within 12 months prior to
Screening
14. History of coagulopathy or hereditary thrombocytopenia or a family
history of thrombocytopenia
15. Known history or symptoms of systemic lupus erythematosus,
antiphospholipid antibody syndrome or any other clinically documented
auto-immune disease other than ITP
16. Prior history or symptoms suggestive of untreated Helicobacter
pylori infection
17. History of a recent major surgery
18. Active infection; a recent serious infection within the 8 weeks prior
to Screening.
19. Clinical evidence of significant unstable or uncontrolled acute or
chronic diseases other than ITP, uncontrolled diabetes
20. History of alcoholism or drug/chemical/substance abuse within the
past 2 years prior to Screening per investigator's opinion
21. Body Mass Index (BMI) at Screening = 35 kg/m2
22. Female patient who is pregnant or lactating or have been lactating
within 3 months of Screening
Additional Exclusion Criterion for the Extended Follow-up Period
23. At the moment of start of extended FU period, being enrolled in a
clinical study with another investigational drug or device
Exclusion criteria for the open-label treatment period (first treatment
cycle)
1. At the moment of start of the open-label treatment period, being
enrolled in a clinical study with another investigational drug or device
2. Please refer to the following exclusion criteria from the main study: 1,
4, 19, 21, and 22
3. Use of IVIg or anti-D immunoglobulin treatment as rescue therapy at
any time during the main study or extended FU period
4. Received vaccinations within 4 weeks prior to any (re)treatment cycle
5. At Treatment Evaluation Visit, have clinically significant laboratory
abnormalities
6. Patient has a history of severe and/or serious hypersensitivity
reaction to IMP in ARGX- 113-1603 main study
Exclusion criteria for subsequent open-label retreatment cycles
1. Please refer to the exclusion criteria for the first treatment cycle of
the open-label treatment period
2

Study & Design

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