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

NCT02859909 | Completed Phase 3 DMC

• 1 other identifier: 992
• Study Type: interventional
• Target: 34
• Locations: 6 countries
• Sites: 22

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

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (14)

• The number of subjects with complete response (CR)

  1 month

• The percentage of subjects with complete response (CR)

  1 month

• The number of subjects with no response (NR)

  1 month

• The percentage of subjects with no response (NR)

  1 month

• The number of subjects with a loss of response

  1 month

Study Arms (2)

2 day treatment schedule

EXPERIMENTAL

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

Biological: BT595

5 day treatment schedule

EXPERIMENTAL

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

Biological: BT595

Interventions

BT595 BIOLOGICAL

Also known as: Human Immunoglobulin

2 day treatment schedule; 5 day treatment schedule

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.)

You may not qualify if:

• 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
• 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

Sponsors & Collaborators

• Biotest: lead
• Syneos Health: collaborator

Study Sites (22)

Investigational site # 3597

Pleven, 5800, Bulgaria

Location

Investigational site # 3593

Plovdiv, 4000, Bulgaria

Location

Investigational site # 3598

Sofia, 1431, Bulgaria

Location

Investigational site # 3591

Sofia, 1756, Bulgaria

Location

Investigational site # 3596

Varna, 9010, Bulgaria

Location

Investigational site # 4202

Prague, 10034, Czechia

Location

Investigational site # 4901

Berlin, 13353, Germany

Location

Investigational Site #4902

München, 81377, Germany

Location

Investigational site # 3601

Budapest, 1083, Hungary

Location

Investigational site # 3604

Debrecen, 4032, Hungary

Location

Investigational site # 3607

Győr, 9023, Hungary

Location

Investigational site # 3602

Miskolc, 3529, Hungary

Location

Investigational site # 3603

Nyíregyháza, 4400, Hungary

Location

Investigational site # 3606

Pécs, 7621, Hungary

Location

Investigational site # 3811

Belgrade, 11000, Serbia

Location

Investigational site # 3813

Belgrade, 11080, Serbia

Location

Investigational site #3814

Niš, 18000, Serbia

Location

Investigational site # 3812

Novi Sad, 21000, Serbia

Location

Investigational site # 3403

Madrid, 28006, Spain

Location

Investigational site # 3404

Madrid, 28007, Spain

Location

Investigational site #3401

Málaga, Spain

Location

Investigational site # 3402

Palma de Mallorca, 07012, Spain

Location

Related Publications (1)

• 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.

  PMID: 36448274 DERIVED

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

Immunoglobulins, Intravenous

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Intervention Hierarchy (Ancestors)

Immunoglobulin G; Immunoglobulin Isotypes; Antibodies; Immunoglobulins; Immunoproteins; Blood Proteins; Proteins; Amino Acids, Peptides, and Proteins; Serum Globulins; Globulins

Study Officials

• Judit Demeter, MD, PhD, DSc

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

  PRINCIPAL INVESTIGATOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: July 25, 2016
• First Posted: August 9, 2016
• Study Start: November 1, 2016
• Primary Completion: December 1, 2018
• Study Completion: December 1, 2018
• Last Updated: August 14, 2019

Record last verified: 2018-04

Locations

BU (5); SE (4); DE (2); HU (6); CZ (1); ES (4)