Immune Thrombocytopenic Purpura
Conditions
Keywords
Idiopathic, ITP
Brief summary
This trial is designed as a multi-centre, single-dose, exploratory dose-finding, open label trial evaluating the safety and efficacy of Sym001 in 4-9 consecutive cohorts. Subjects will receive a single IV dose of Sym001.
Interventions
DRUGSym001
Each cohort will receive one single dose of Sym001 according the assigned dose level.
Sponsors
Swedish Orphan Biovitrum
Symphogen A/S
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
Healthy volunteers
No
Inclusion criteria
* Confirmed presence of thrombocytopenia with platelet count \< 30,000/mm3 at the pre-dose visit. * History of isolated ITP * RhD-positive serology. * Previous treatment and response to first line therapy for ITP
Exclusion criteria
* Known clinical picture suggestive of other causes of thrombocytopenia, especially systematic lupus erythematosus,antiphospholipid syndrome, Evans syndrome, immunodeficiency states, lymphoproliferative disorders, liver disease,ingestion of drugs such as quinidine/quinine, heparin and sulfonamides and hereditary thrombocytopenia confirmed by relevant laboratory findings. * Suspected infection with HIV, Hepatitis C, H. pylori. * Clinical splenomegaly * History of abnormal bone marrow examination. * Ongoing haemorrhage corresponding to a grade 3 or 4 on the WHO bleeding scale. * Underlying haemolytic condition * History of splenectomy. * Subject is pregnant, breast feeding or intends to become pregnant.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Incidence and severity of Adverse Events (AEs), including Serious Adverse Events (SAEs) | 6 weeks post dosing |
Secondary
| Measure | Time frame |
|---|---|
| Measurements of platelet counts | From day 1 through week 6 |
Countries
Belgium, Germany, India, Israel, Poland, Romania, Russia, Serbia, Spain, Ukraine, United Kingdom, United States
Outcome results
None listed