- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02859909
This Clinical Study is to Test Efficacy and Safety of BT595 in Chronic Primary Immune Thrombocytopenia (ITP)
August 12, 2019 updated by: Biotest
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)
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.
Study Overview
Study Type
Interventional
Enrollment (Actual)
34
Phase
- Phase 3
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
-
Pleven, Bulgaria, 5800
- Investigational site # 3597
-
Plovdiv, Bulgaria, 4000
- Investigational site # 3593
-
Sofia, Bulgaria, 1431
- Investigational site # 3598
-
Sofia, Bulgaria, 1756
- Investigational site # 3591
-
Varna, Bulgaria, 9010
- Investigational site # 3596
-
-
-
-
-
Praha, Czechia, 10034
- Investigational site # 4202
-
-
-
-
-
Berlin, Germany, 13353
- Investigational site # 4901
-
München, Germany, 81377
- Investigational Site #4902
-
-
-
-
-
Budapest, Hungary, 1083
- Investigational site # 3601
-
Debrecen, Hungary, 4032
- Investigational site # 3604
-
Győr, Hungary, 9023
- Investigational site # 3607
-
Miskolc, Hungary, 3529
- Investigational site # 3602
-
Nyiregyhaza, Hungary, 4400
- Investigational site # 3603
-
Pécs, Hungary, 7621
- Investigational site # 3606
-
-
-
-
-
Belgrade, Serbia, 11000
- Investigational site # 3811
-
Belgrade, Serbia, 11080
- Investigational site # 3813
-
Niš, Serbia, 18000
- Investigational site #3814
-
Novi Sad, Serbia, 21000
- Investigational site # 3812
-
-
-
-
-
Madrid, Spain, 28006
- Investigational site # 3403
-
Madrid, Spain, 28007
- Investigational site # 3404
-
Malaga, Spain
- Investigational Site #3401
-
Palma de Mallorca, Spain, 07012
- Investigational Site # 3402
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years to 75 years (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
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
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: Randomized
- Interventional Model: Parallel Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: 2 day treatment schedule
Patients will receive a dosage of 1 g/kg bw per day of BT595 for 2 consecutive days
|
Other Names:
|
Experimental: 5 day treatment schedule
Patients will receive a dosage of 0.4 g/kg bw per day of BT595 for 5 consecutive days
|
Other Names:
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Rate of subjects with response (R)
Time Frame: 1 month
|
The rate of subjects with response is defined as subjects with 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
|
1 month
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
The number of subjects with complete response (CR)
Time Frame: 1 month
|
The number of subjects with CR, which is defined as a platelet count ≥100×10^9/L, confirmed on at least 2 separate occasions at least 7 days apart, and the absence of bleeding
|
1 month
|
The percentage of subjects with complete response (CR)
Time Frame: 1 month
|
The percentage of subjects with CR, which is defined as a platelet count ≥100×10^9/L, confirmed on at least 2 separate occasions at least 7 days apart, and the absence of bleeding
|
1 month
|
The number of subjects with no response (NR)
Time Frame: 1 month
|
The number of subjects with NR, which is defined as subjects without R, i.e. a platelet count <30×10^9/L or less than a 2 fold increase of baseline platelet count, confirmed on at least 2 separate occasions, approximately 1 day apart, or bleeding
|
1 month
|
The percentage of subjects with no response (NR)
Time Frame: 1 month
|
The percentage of subjects with NR, which is defined as subjects without R, i.e. a platelet count <30×10^9/L or less than a 2 fold increase of baseline platelet count, confirmed on at least 2 separate occasions, approximately 1 day apart, or bleeding
|
1 month
|
The number of subjects with a loss of response
Time Frame: 1 month
|
The number of subjects with a loss of response (only in subjects who previously had CR or R), which is defined as a platelet count <100×10^9/L or bleeding (from CR) or platelet count <30×10^9/L or less than 2 fold increase of the baseline platelet count, or bleeding (from R).
Platelet counts will be confirmed on at least 2 separate occasions approximately 1 day apart
|
1 month
|
The percentage of subjects with a loss of response
Time Frame: 1 month
|
The percentage of subjects with a loss of response (only in subjects who previously had CR or R), which is defined as a platelet count <100×10^9/L or bleeding (from CR) or platelet count <30×10^9/L or less than 2 fold increase of the baseline platelet count, or bleeding (from R).
Platelet counts will be confirmed on at least 2 separate occasions approximately 1 day apart
|
1 month
|
Time to Response (R)
Time Frame: 1 month
|
Time to response (R), which is defined as the time from the start of treatment to the time of achievement of CR or R
|
1 month
|
Duration of response (R),
Time Frame: 1 month
|
Duration of response (R), which is defined as the time from the achievement of CR or R to loss of CR or R
|
1 month
|
The number of subjects with response to ≥50×10^9/L
Time Frame: 1 month
|
The number of subjects with response to ≥50×10^9/L, which is defined as a platelet count increase to at least ≥50×10^9/L within 7 days of the first BT595 infusion
|
1 month
|
The percentage of subjects with response to ≥50×10^9/L
Time Frame: 1 month
|
The percentage of subjects with response to ≥50×10^9/L, which is defined as a platelet count increase to at least ≥50×10^9/L within 7 days of the first BT595 infusion
|
1 month
|
Subject's maximum platelet count achieved
Time Frame: 1 month
|
1 month
|
|
Time to subject's maximum platelet count
Time Frame: 1 month
|
1 month
|
|
Time course of platelet count
Time Frame: 1 month
|
1 month
|
|
Occurrence of bleeding symptoms
Time Frame: 1 month
|
1 month
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Collaborators
Investigators
- Principal Investigator: Judit Demeter, MD, PhD, DSc, Semmelweis University Medical School, First Department of Medicine, Department of Hematology, 1083 Budapest, Korányi S. u. 2/a, Hungary
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
November 1, 2016
Primary Completion (Actual)
December 1, 2018
Study Completion (Actual)
December 1, 2018
Study Registration Dates
First Submitted
July 25, 2016
First Submitted That Met QC Criteria
August 4, 2016
First Posted (Estimate)
August 9, 2016
Study Record Updates
Last Update Posted (Actual)
August 14, 2019
Last Update Submitted That Met QC Criteria
August 12, 2019
Last Verified
April 1, 2018
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Immune System Diseases
- Autoimmune Diseases
- Hematologic Diseases
- Hemorrhage
- Hemorrhagic Disorders
- Blood Coagulation Disorders
- Skin Manifestations
- Blood Platelet Disorders
- Thrombotic Microangiopathies
- Purpura, Thrombocytopenic
- Purpura
- Purpura, Thrombocytopenic, Idiopathic
- Thrombocytopenia
- Physiological Effects of Drugs
- Immunologic Factors
- Immunoglobulins
- Immunoglobulins, Intravenous
Other Study ID Numbers
- 992
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
Undecided
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
Clinical Trials on Immune Thrombocytopenia
-
Institute of Hematology & Blood Diseases Hospital...Henan Cancer Hospital; Beijing Children's Hospital; Tianjin Medical University... and other collaboratorsRecruitingPrimary Immune Thrombocytopenia (ITP)China
-
argenxWithdrawnPrimary Immune Thrombocytopenia (ITP)
-
Novartis PharmaceuticalsRecruitingPrimary Immune Thrombocytopenia (ITP)China, United States, Spain, Singapore, Austria, Germany, Belgium, Italy, Japan, Czechia, Hong Kong, Hungary, Malaysia, Argentina, Bulgaria, Turkey, Vietnam, Australia, Thailand, Mexico, United Kingdom, France, Romania, Norway, India
-
University Children's Hospital BaselNovartis Pharmaceuticals; Stiftung zur Förderung medizinischer und biologischer... and other collaboratorsActive, not recruiting
-
Gruppo Italiano Malattie EMatologiche dell'AdultoCompletedAdult Patients | Immune Primary Thrombocytopenia | Splenectomy | TPO-mimeticsItaly
-
National Heart, Lung, and Blood Institute (NHLBI)Enrolling by invitationImmune Mediated Anemia | Immune Mediated Thrombocytopenia | Chronic GVHDUnited States
-
Changzhou No.2 People's HospitalRui Therapeutics Co., LtdNot yet recruitingThrombocytopenia Alloimmune
-
Nahda UniversityRecruiting
-
National Heart, Lung, and Blood Institute (NHLBI)RecruitingImmune Mediated Anemia | Immune Mediated Thrombocytopenia | Chronic GVHDUnited States
-
National Institute of Blood and Marrow Transplant...RecruitingImmune Thrombocytopenia PurpuraPakistan
Clinical Trials on BT595
-
BiotestSyneos HealthCompletedPrimary Immunodeficiency DiseaseUnited States, Germany, Hungary, Russian Federation, Spain