Clinical trial NCT02859909: This Clinical Study is to Test Efficacy and Safety of BT595 in Chronic Primary Immune Thrombocytopenia (ITP)
Official title: An Open Label, Prospective, Randomized, Multicenter Study Investigating Clinical Efficacy and Safety of the Human Normal Immunoglobulin for Intravenous Administration BT595 in Patients With Chronic Primary Immune Thrombocytopenia (ITP)
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE3
Conditions: Immune Thrombocytopenia
Lead sponsor: Biotest
Sponsor class: INDUSTRY
Collaborators: name: Syneos Health
First posted: 2016-08-09
Enrollment: 34
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The main purpose of this study is to assess the efficacy and safety of BT595 in adult subjects with chronic ITP. The primary objective of this study is to determine the rate of subjects with a response. A response is defined as a platelet count of ≥30×10\^9/L and at least a 2 fold increase of the baseline count, confirmed on at least 2 separate occasions at least 7 days apart, and the absence of bleeding. The secondary objectives of this study, in addition to further efficacy assessments, are to evaluate the safety of BT595.


Eligibility criteria: Main Inclusion Criteria:

* Diagnosis of chronic ITP (\>12 months' duration), including diagnosis of refractory ITP, and as defined by the International Working Group (Rodeghiero et al, 2009), where ITP is described as an autoimmune disorder characterized by isolated thrombocytopenia in the absence of other causes or disorders that may be associated with thrombocytopenia
* Treatment is indicated because of a high risk of bleeding or a need to raise the platelet count
* Mean screening platelet count of \<30×10\^9/L from 3 qualifying platelet counts performed within approximately 7 to 14 days before the start of treatment, with no individual platelet count above 35×10\^9/L. The subject may be rescreened if the mean screening platelet count is ≥30×10\^9/L. (Note: If a subject is rescreened, all screening laboratory tests must be repeated.)

Main Exclusion Criteria:

* Secondary thrombocytopenia or acquired medical conditions known to be associated with secondary thrombocytopenia, such as chronic lymphocytic leukemia; lymphoma; multiple myeloma; thyroid disease; or other forms of thrombocytopenia, such as drug induced thrombocytopenia; cirrhotic liver diseases; antiphospholipid syndrome; environmental thrombocytopenia; and bone marrow diseases
* Severe concomitant diseases that in the judgment of the investigator will interfere with the study, such as autoimmune hemolytic anemia, acute renal failure, and noncontrolled arterial hypertension
* Laboratory findings (e.g., abnormal laboratory values for hemoglobin, transaminase levels \[alanine aminotransferase, aspartate aminotransferase\], total bilirubin, creatinine, blood urea nitrogen, and immunoglobulins G, A, M) that preclude participation
* Positive Coombs test (direct and indirect)
* Planned invasive procedures during the time frame of the study
* Maintenance therapy with intravenous immunoglobulins (IVIgs) or infusion of IVIgs within 3 months before start of the study
* Unresponsive to previous IVIg treatment
* Additional therapy with high dose corticosteroids (equivalent to \>30 mg prednisone/day), thrombopoietin receptor agonists, and/or immunosuppressives and/or other therapies (e.g., infusion of platelets) within 1 month before the start of the study (Note: Subjects on stable doses of ITP active treatment must not have modified the dose in the preceding 2 weeks and must maintain their prestudy dose during the study. Corticosteroids should not be given as a premedication. Rescue therapy with short courses \[i.e., 1 to 4 days\] of high dose steroids and IVIgs are allowed up to 2 weeks before study inclusion.)
* History of thrombotic events (including myocardial infarction, cerebral vascular accident \[including stroke\], pulmonary embolism, and deep vein thrombosis) 6 months before treatment start with BT595 or the presence of significant risk factors for thrombotic events
* Therapy with live attenuated virus vaccines 3 months before start of the study
* Selective, absolute immunoglobulin A (IgA) deficiency or known antibodies to IgA

Primary outcomes:
measure: Rate of subjects with response (R)
timeFrame: 1 month
Secondary outcomes:
measure: The number of subjects with complete response (CR)
timeFrame: 1 month
measure: The percentage of subjects with complete response (CR)
timeFrame: 1 month
measure: The number of subjects with no response (NR)
timeFrame: 1 month
measure: The percentage of subjects with no response (NR)
timeFrame: 1 month
measure: The number of subjects with a loss of response
timeFrame: 1 month
measure: The percentage of subjects with a loss of response
timeFrame: 1 month
measure: Time to Response (R)
timeFrame: 1 month
measure: Duration of response (R),
timeFrame: 1 month
measure: The number of subjects with response to ≥50×10^9/L
timeFrame: 1 month
measure: The percentage of subjects with response to ≥50×10^9/L
timeFrame: 1 month
measure: Subject's maximum platelet count achieved
timeFrame: 1 month
measure: Time to subject's maximum platelet count
timeFrame: 1 month
measure: Time course of platelet count
timeFrame: 1 month
measure: Occurrence of bleeding symptoms
timeFrame: 1 month
Other outcomes:
Locations:
city: Pleven
country: Bulgaria
facility: Investigational site # 3597
city: Plovdiv
country: Bulgaria
facility: Investigational site # 3593
city: Sofia
country: Bulgaria
facility: Investigational site # 3598
city: Sofia
country: Bulgaria
facility: Investigational site # 3591
city: Varna
country: Bulgaria
facility: Investigational site # 3596
city: Praha
country: Czechia
facility: Investigational site # 4202
city: Berlin
country: Germany
facility: Investigational site # 4901
city: München
country: Germany
facility: Investigational Site #4902
city: Budapest
country: Hungary
facility: Investigational site # 3601
city: Debrecen
country: Hungary
facility: Investigational site # 3604
city: Győr
country: Hungary
facility: Investigational site # 3607
city: Miskolc
country: Hungary
facility: Investigational site # 3602
city: Nyiregyhaza
country: Hungary
facility: Investigational site # 3603
city: Pécs
country: Hungary
facility: Investigational site # 3606
city: Belgrade
country: Serbia
facility: Investigational site # 3811
city: Belgrade
country: Serbia
facility: Investigational site # 3813
city: Niš
country: Serbia
facility: Investigational site #3814
city: Novi Sad
country: Serbia
facility: Investigational site # 3812
city: Madrid
country: Spain
facility: Investigational site # 3403
city: Madrid
country: Spain
facility: Investigational site # 3404
city: Malaga
country: Spain
facility: Investigational site #3401
city: Palma de Mallorca
country: Spain
facility: Investigational site # 3402
Study officials:
affiliation: Semmelweis University Medical School, First Department of Medicine, Department of Hematology, 1083 Budapest, Korányi S. u. 2/a, Hungary
name: Judit Demeter, MD, PhD, DSc
role: PRINCIPAL_INVESTIGATOR
References: