Immune Thrombocytopenic Purpura (ITP) in RhD positive, non-splenectomized adult subjects MedDRA version: 9.1 Level: LLT Classification code 10043561 Term: Thrombocytopenic purpura
Conditions
Interventions
Product Name: sym001
Product Code: sym001
Pharmaceutical Form: Solution for injection
Current Sponsor code: sym001
Concentration unit: mg/ml milligram(s)/millilitre
Concentration type: equal
Concentra
Sponsors
Symphogen A/S
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: Informed consent obtained before any trial-related procedures. 2. Age >18 years. 3. Confirmed presence of thrombocytopenia with platelet count 200 µg/kg a maximum of 1 subject with platelet counts 1 hour interval between each other and both being =65 years) yes F.1.3.1 Number of subjects for this age range
Exclusion criteria
Exclusion criteria: Known clinical picture suggestive of other causes of thrombocytopenia, especially systemic 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. 2. Suspected infection with HIV, hepatitis C, H. pylori unless corresponding laboratory test are negative. 3. Clinical splenomegaly (the spleen should not be palpable at more than 1 finger breadth below the costal margin). 4. History of abnormal bone marrow examination (except ITP-typical megacaryocytosis). 5. At pre-dose visit: an ongoing haemorrhage corresponding to a grade 3 or 4 on the WHO bleeding scale. 6. History of anaphylaxis or hypersensitivity reactions following anti-D treatment. 7. Current immune haemolytic anaemia. 8. Underlying haemolytic condition (e.g. reticulocyte count > 3%). 9. Planned surgery during the 15 days post dosing. 10. Haemoglobin pre-dose value lower than 2.0 g/dL below the lower limit of the laboratory normal range for gender and age. 11. History of splenectomy. 12. Known current malignancy (except basal cell carcinoma). 13. Received other investigational agent within 3 months prior to enrolment. 14. Positive DAT (direct Coombs-test) at screening unless subject has received treatment with IVIg or anti-D products within 3 months prior to screening with prior negative DAT. 15. Known non-responders to most recent anti-D treatment (despite any initial response to treatment). 16. Any other current treatment for ITP except corticosteroids (Prednisone or Dexamethasone) at doses equivalent to =30 mg prednisone/day if the daily dose has been constant for 2 weeks or more before trial drug administration. 17. Therapy with IVIg within 2 weeks prior to enrollment or with anti-D or any other treatment of ITP within 4 weeks prior to enrolment. 18. Therapy with tranexamic acid, alkylating agents or any anti-CD20 antibodies within 8 weeks prior to enrolment 19. Any antithrombotic treatment (except acetyl salicylic acid at doses up to 150 mg daily) within 7 days prior to pre-dosing visit or planned during the trial. 20. PT/INR and aPTT out of normal range at the pre-dose visit. 21. History of venous or arterial thrombosis or know thrombophilia. 22. Diseases other than ITP that may influence the result of the trial as judged by the Investigator. 23. Creatinine 25% or more above normal range value, alanine aminotransferase (ALT) and alkaline phosphatase (ALP) 100% above normal range value, and albumin 25% or more below normal range value (according to the local laboratory) at pre-dose visit. 24. Current or planned treatment with erythropoietin. 25. Subject is pregnant, breast feeding or intends to become pregnant. 26. Previous dosing with Sym001 in this trial.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: To evaluate the safety of Sym001 following a single intravenous dose, at multiple dose levels, in the treatment of ITP in RhD positive, non-splenectomized adult subjects;Secondary Objective: To evaluate the efficacy of Sym001 following a single intravenous dose in the treatment of ITP in RhD positive, non-splenectomized adult subjects. • To evaluate the binding of Sym001 to RBCs following a single intravenous dose of Sym001 in the treatment of ITP in RhD positive, non-splenectomized adult subjects. • To evaluate an appropriate dose of Sym001 for Phase 3. ;Primary end point(s): Incidence and severity of AEs, including SAEs and Adverse Events of Special Interest (AESIs) during the 6-week trial period. | — |
Countries
Belgium, Germany, Spain, United Kingdom
Outcome results
None listed