Immune ThrombocytopeniaPhase 3NCT02859909
This Clinical Study is to Test Efficacy and Safety of BT595 in Chronic Primary Immune Thrombocytopenia (ITP)
A Phase 3 study testing BT595 in people with Immune Thrombocytopenia, run by Biotest at 22 sites in 6 countries.
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. Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Immune Thrombocytopenia
- What is being tested?
- BT595
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 34 participants
- Where
- 22 sitesin 6 countries
- Study dates
- Nov 2016to Dec 2018
- Phase
- Phase 3
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
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
Trial locations and contacts
22 sites in 6 countries.
22 sites in 6 countries
Hungary6 sites
Investigational site # 3601
Budapest, 1083
Investigational site # 3604
Debrecen, 4032
Investigational site # 3607
Győr, 9023
Investigational site # 3602
Miskolc, 3529
Investigational site # 3603
Nyíregyháza, 4400
Investigational site # 3606
Pécs, 7621
Bulgaria5 sites
Investigational site # 3597
Pleven, 5800
Investigational site # 3593
Plovdiv, 4000
Investigational site # 3598
Sofia, 1431
Investigational site # 3591
Sofia, 1756
Investigational site # 3596
Varna, 9010
Serbia4 sites
Investigational site # 3811
Belgrade, 11000
Investigational site # 3813
Belgrade, 11080
Investigational site #3814
Niš, 18000
Investigational site # 3812
Novi Sad, 21000
Spain4 sites
Investigational site # 3403
Madrid, 28006
Investigational site # 3404
Madrid, 28007
Investigational site #3401
Málaga
Investigational site # 3402
Palma de Mallorca, 07012
Germany2 sites
Investigational site # 4901
Berlin, 13353
Investigational Site #4902
München, 81377
About this study
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)
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
- Being tested
- BT595
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BT595Biological
Also called Human Immunoglobulin
| Study group | Role | What they receive |
|---|---|---|
| 2 day treatment schedule | Experimental | Patients will receive a dosage of 1 g/kg bw per day of BT595 for 2 consecutive days Receives: 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 Receives: Biological: BT595 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 1 month
Secondary outcomes (14)
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
Measured: 1 month
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
Measured: 1 month
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
Measured: 1 month
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
Measured: 1 month
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
Measured: 1 month
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
Measured: 1 month
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
Measured: 1 month
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
Measured: 1 month
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
Measured: 1 month
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
Measured: 1 month
Subject's maximum platelet count achieved
Measured: 1 month
Time to subject's maximum platelet count
Measured: 1 month
Time course of platelet count
Measured: 1 month
Occurrence of bleeding symptoms
Measured: 1 month
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 Aug 2016 (estimated)
- Study startNov 2016
- Primary completionDec 2018
- Study completionDec 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Immune ThrombocytopeniaStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Enrolment
- 34 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biotest
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Syneos Health
- Responsible party
- Sponsor
- Investigators
- Judit Demeter, MD, PhD, DSc, Principal investigator, Semmelweis University Medical School, First Department of Medicine, Department of Hematology, 1083 Budapest, Korányi S. u. 2/a, Hungary
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02859909
- Sponsor's protocol ID
- 992
- Status last verified
- Apr 2018
- First submitted
- 25 Jul 2016
- Last updated
- 14 Aug 2019
- Results posted
- No
- Data monitoring committee
- Yes
- Individual data sharing
- Undecided
Publications
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.
PubMed 36448274
Study at a glance
From the registry record, last updated by the sponsor on 14 Aug 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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)
- ClinicalTrials.gov ID
- NCT02859909
- Acronym / sponsor ID
- 992
- Conditions
- Immune Thrombocytopenia
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 34 participants (actual)
- Lead sponsor
- BiotestIndustry (pharma, biotech, devices)
- Collaborators
- Syneos Health
- Study start
- Nov 2016 (actual)
- Primary completion
- Dec 2018 (actual)
- Study completion
- Dec 2018 (actual)
- First posted
- 9 Aug 2016 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- None (open label)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| 2 day treatment schedule | Experimental | Biological: BT595Patients will receive a dosage of 1 g/kg bw per day of BT595 for 2 consecutive days |
| 5 day treatment schedule | Experimental | Biological: BT595Patients will receive a dosage of 0.4 g/kg bw per day of BT595 for 5 consecutive days |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BT595Also: Human Immunoglobulin | Biological | 2 day treatment schedule, 5 day treatment schedule |
Primary outcome
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
14 secondary outcomes
The number of subjects with complete response (CR)
Time frame: 1 month
The percentage of subjects with complete response (CR)
Time frame: 1 month
The number of subjects with no response (NR)
Time frame: 1 month
The percentage of subjects with no response (NR)
Time frame: 1 month
The number of subjects with a loss of response
Time frame: 1 month
The percentage of subjects with a loss of response
Time frame: 1 month
Time to Response (R)
Time frame: 1 month
Duration of response (R),
Time frame: 1 month
The number of subjects with response to ≥50×10^9/L
Time frame: 1 month
The percentage of subjects with response to ≥50×10^9/L
Time frame: 1 month
Subject's maximum platelet count achieved
Time frame: 1 month
Time to subject's maximum platelet count
Time frame: 1 month
Time course of platelet count
Time frame: 1 month
Occurrence of bleeding symptoms
Time frame: 1 month
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 14 Aug 2019.
- Derived from this study: PubMed 36448274Demeter 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.
- Individual participant dataSharing planned: Undecided
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Judit Demeter, MD, PhD, DSc principal investigator
Semmelweis University Medical School, First Department of Medicine, Department of Hematology, 1083 Budapest, Korányi S. u. 2/a, Hungary
22 sites in 6 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing BT595 in people with Immune Thrombocytopenia, run by Biotest at 22 sites in 6 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 14 Aug 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 22 sites in Hungary, Bulgaria, Serbia, Spain, Germany and 1 more country. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Rate of subjects with response (R) (time frame: 1 month). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02859909, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 14 Aug 2019.