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

NCT ID: NCT02859909

Last Updated: 2019-08-14

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE3
• Total Enrollment: 34 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-11-30
• Study Completion Date: 2018-12-31

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.

Conditions

Immune Thrombocytopenia

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

2 day treatment schedule

Patients will receive a dosage of 1 g/kg bw per day of BT595 for 2 consecutive days

Group Type: EXPERIMENTAL

BT595

Intervention Type: BIOLOGICAL

5 day treatment schedule

Patients will receive a dosage of 0.4 g/kg bw per day of BT595 for 5 consecutive days

Group Type: EXPERIMENTAL

BT595

Intervention Type: BIOLOGICAL

Interventions

BT595

Intervention Type: BIOLOGICAL

Other Intervention Names

Human Immunoglobulin

Eligibility Criteria

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.)

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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 75 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Syneos Health

OTHER

Sponsor Role: collaborator

Biotest

INDUSTRY

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Judit Demeter, MD, PhD, DSc

Role: PRINCIPAL_INVESTIGATOR

Semmelweis University Medical School, First Department of Medicine, Department of Hematology, 1083 Budapest, Korányi S. u. 2/a, Hungary

Locations

Investigational site # 3597

Pleven, , Bulgaria

Investigational site # 3593

Plovdiv, , Bulgaria

Investigational site # 3598

Sofia, , Bulgaria

Investigational site # 3591

Sofia, , Bulgaria

Investigational site # 3596

Varna, , Bulgaria

Investigational site # 4202

Prague, , Czechia

Investigational site # 4901

Berlin, , Germany

Investigational Site #4902

München, , Germany

Investigational site # 3601

Budapest, , Hungary

Investigational site # 3604

Debrecen, , Hungary

Investigational site # 3607

Győr, , Hungary

Investigational site # 3602

Miskolc, , Hungary

Investigational site # 3603

Nyíregyháza, , Hungary

Investigational site # 3606

Pécs, , Hungary

Investigational site # 3811

Belgrade, , Serbia

Investigational site # 3813

Belgrade, , Serbia

Investigational site #3814

Niš, , Serbia

Investigational site # 3812

Novi Sad, , Serbia

Investigational site # 3403

Madrid, , Spain

Investigational site # 3404

Madrid, , Spain

Investigational site #3401

Málaga, , Spain

Investigational site # 3402

Palma de Mallorca, , Spain

Countries

Bulgaria; Czechia; Germany; Hungary; Serbia; Spain

References

Demeter J, Hamed A, Laszlo S, Suvajdzic N, Aigner S, Borner B, Staiger C. Efficacy and safety of BT595 (10% human intravenous immunoglobulin) in adult patients with chronic immune thrombocytopenia. Transfus Med. 2023 Apr;33(2):165-173. doi: 10.1111/tme.12943. Epub 2022 Nov 30.

Reference Type: DERIVED

PMID: 36448274 (View on PubMed)

Other Identifiers

992

Identifier Type: -

Identifier Source: org_study_id