This Clinical Study is to Test Efficacy and Safety of BT595 in Chronic Primary Immune Thrombocytopenia (ITP)

Immune Thrombocytopenia Clinical Trial

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)

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT02859909
• Other study ID #: 992
• Status: Completed
• Phase: Phase 3
• Start date: November 2016
• Est. completion date: December 2018

Study information

• Verified date: April 2018
• Source: Biotest
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 34
• Est. completion date: December 2018
• Est. primary completion date: December 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 75 Years

Eligibility

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

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Biological:
• BT595

Locations

Country	Name	City	State
Bulgaria	Investigational site # 3597	Pleven
Bulgaria	Investigational site # 3593	Plovdiv
Bulgaria	Investigational site # 3591	Sofia
Bulgaria	Investigational site # 3598	Sofia
Bulgaria	Investigational site # 3596	Varna
Czechia	Investigational site # 4202	Praha
Germany	Investigational site # 4901	Berlin
Germany	Investigational Site #4902	München
Hungary	Investigational site # 3601	Budapest
Hungary	Investigational site # 3604	Debrecen
Hungary	Investigational site # 3607	Gyor
Hungary	Investigational site # 3602	Miskolc
Hungary	Investigational site # 3603	Nyiregyhaza
Hungary	Investigational site # 3606	Pécs
Serbia	Investigational site # 3811	Belgrade
Serbia	Investigational site # 3813	Belgrade
Serbia	Investigational site #3814	Niš
Serbia	Investigational site # 3812	Novi Sad
Spain	Investigational site # 3403	Madrid
Spain	Investigational site # 3404	Madrid
Spain	Investigational site #3401	Malaga
Spain	Investigational site # 3402	Palma de Mallorca

Sponsors (2)

Lead Sponsor	Collaborator
Biotest	Syneos Health

Countries where clinical trial is conducted

Bulgaria,  Czechia,  Germany,  Hungary,  Serbia,  Spain, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Rate of subjects with response (R)	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	The number of subjects with complete response (CR)	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
Secondary	The percentage of subjects with complete response (CR)	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
Secondary	The number of subjects with no response (NR)	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
Secondary	The percentage of subjects with no response (NR)	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
Secondary	The number of subjects with a loss of response	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
Secondary	The percentage of subjects with a loss of response	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
Secondary	Time to Response (R)	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
Secondary	Duration of response (R),	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
Secondary	The number of subjects with response to =50×10^9/L	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
Secondary	The percentage of subjects with response to =50×10^9/L	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
Secondary	Subject's maximum platelet count achieved		1 month
Secondary	Time to subject's maximum platelet count		1 month
Secondary	Time course of platelet count		1 month
Secondary	Occurrence of bleeding symptoms		1 month