Chronic adult idiopathic thrombocytopenic purpura Haematological Disorders Purpura and other haemorrhagic conditions
Conditions
Interventions
SM101 is a recombinant, soluble, non-glycosylated version of the human Fc- receptor Fc-RIIb.
This trial consists of a dose escalation and an extension part. In the dose escalation part, each dose gr
Sponsors
SuppreMol GmbH (Germany)
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Written informed consent prior to any study related procedure 2. Male or female subjects aged 18 to 75 years, with or without splenectomy 3. Diagnosis of chronic idiopathic thrombocytopenic purpura (ITP) based on subject's history, physical examination, blood count and blood film examination according to the British Society for Haematology (BSH) and American Society of Hematology (ASH) guidelines for at least 6 months 4. Subject has previously received at least one ITP therapy 5. Platelet count less than 30,000/µL from at least two measurements 6. Subjects greater than 60 years of age must have had a documented history of chronic ITP with a bone marrow report to confirm the diagnosis
Exclusion criteria
Exclusion criteria: 1. Female subjects who are nursing or pregnant, who may be pregnant, or who contemplate pregnancy during the study period 2. Secondary thrombocytopenia 3. Subject received rituximab or any other B-cell depleting agent within 24 months preceding the first dose of investigational medical product (IMP) 4. All other previously completed ITP treatment must achieve at least 5 times their terminal half-life prior to first administration of IMP 5. Subject receives concomitant ITP medication other than corticosteroids, except rescue medication during the clinical trial 6. Splenectomy within 4 weeks prior to screening 7. History of or current alcohol or drug abuse 8. Any condition which in the judgment of the Investigator would place the subject at undue risk or interfere with the results of the study
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence, severity, causality and seriousness of adverse events (AEs), laboratory results and AEs of special interest including bleeding events. AEs will be graded using the Common Terminology Criteria for Adverse Events (CTCAE), version 3.0. Adverse Events will be recorded at an ongoing basis at every visit performed throughout the study (dose escalation part: screening, week 1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20; extension part: screening, week 1, 2, 3, 4, 5, 8, 12, 16). | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Proportion of subjects with a substantial platelet response defined as: 1.1. Platelet count greater than or equal to 30,000/µL from at least two blood samples, and 1.2. At least two-fold increase of platelet count from baseline values from at least two blood samples 2. The following will be defined as efficacy end-points: 2.1. Mean time to reach substantial platelet response 2.2. Mean duration of platelet count greater than or equal to 30,000/µL 2.3. Mean time to reach first platelet count of greater than or equal to 30,000/µL 2.4. Proportion of subjects requiring rescue medication until end of study 2.5. Proportion of subjects with World Health Organisation (WHO) bleeding events grade 2 (mild blood loss) or higher until end of study 2.6. Immunological markers and IMP PK parameters The platelet count will be determined at every visit performed throughout the study (dose escalation part: screening, week 1, 2, 3, 4, 5, 6, 7, 8, 10, 12, 14, 16, 18, 20; extension part: screening, week 1, 2, 3, 4, 5, 8, 12, 16). On treatment days (escalation part: days 1, 36, 43, 50 and 47; extension part: days 1, 8, 15 and 22) the platelet count will be measured 0.5 hours prior to dosing and 2, 4, 12, 24 and 48 hours after start of IMP administration. | — |
Countries
Belgium, Germany, Poland, Russian Federation, Ukraine
Outcome results
None listed