Moderate Aplastic Anemia (MAA)
Conditions
Interventions
Trade Name: Revolade 75 mg
Product Name: Eltrombopag
Product Code: SB497115
Pharmaceutical Form: Film-coated tablet
INN or Proposed INN: Eltrombopag
CAS Number: 496775-62-3
Current Sponsor code: SB497
Sponsors
Universitätsklinikum Ulm
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Current diagnosis of a Moderate Aplastic Anemia requiring standard treatment with CSA without prior specific therapy. MAA is defined as Aplastic Anemia fulfilling the following criteria: • no evidence for other disease, causing marrow failure • hypocellular bone marrow for age • depression of at least two out of three peripheral blood counts below the normal values in two different blood samples in a time span from at least two weeks: • absolute neutrophil count (ANC) =65 years) yes F.1.3.1 Number of subjects for this age range 90
Exclusion criteria
Exclusion criteria: 1. Age 3 times the upper limit of normal if this elevation is progressive, or persistent during the 4 weeks before study entry or accompanied by increased direct bilirubin, or accompanied by clinical symptoms of liver injury or evidence for hepatic decompensation 7. Infection not adequately responding to appropriate therapy 8. HIV-positivity (patients with Hepatitis B or Hepatitis C are only in combination with hepatic failure - see criteria 7- excluded) 9. Moribund status with a likely death within 3 months. 10. History of malignancy other than localized tumors diagnosed more than one year previously and treated surgically with curative intent (for instance squamous cell or other skin cancers, stage 1, breast cancer in situ, cervical carcinoma in situ...). 11. Prior specific treatment of Aplastic Anemia with immuno-suppression or androgens or interleukin2- receptor-antibodies. The use of these drugs in context with other disorders before diagnosis of aplastic anemia is not an exclusion criteria if these treatments were finished longer than 6 months before study entry. 12. Treatment with other hematological effective drugs (including erythropoetin) within 3 months before study entry as well as treatment with corticosteroids and GCSF within 3 weeks before enrollment. 13. Known hypersensitivity to Eltrombopag or its components 14. Known hypersensitivity to Ciclosporin. 15. Current nursing, pregnancy, or unwillingness to take oral contraceptives or use a barrier method of birth control to refrain from pregnancy as well as a missing or positive pregnancy test within the last 14 days before inclusion for women of childbearing potential during the course of this study. 16. Inability to understand the investigational nature of the study or to give informed consent. 17. Renal failure with creatinine > 2× upper limit of normal. 18. Uncontrolled hypertension. 19. Participation in any study using an investigational drug or treatment with an investigational drug within 30 days preceding the first dose of study medication.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Main Objective: The aim of this trial is to improve treatment of Moderate Aplastic Anemia (MAA) by evaluating the safety and efficiency of Eltrombopag as a new treatment option in patients with MAA requiring therapy. The primary objective of this trial is the evaluation of the superiority of Eltrombopag on top of background treatment with Ciclosporin (CSA) regarding hematologic response (PR +CR) at 6 months in comparison with treatment with CSA alone in untreated MAA patient. ;Secondary Objective: The secondary objective of this trial is to investigate the impact of Eltrombopag added to background therapy with CSA on all outcome measures, safety and quality of life in untreated MAA patients. As well as the evaluation of telomere lengths and telomerase mutations as biomarkers for response to Eltrombopag therapy in MAA and the evaluation of the new Aplastic Anemia and Paroxysmal Nocturnal Hemoglobinuria (PNH) specific quality of life questionnaire QLQ-AA/PNH. ;Primary end point(s): The primary endpoint of the study is the hematologic response rate (CR + PR) at 6 months.;Timepoint(s) of evaluation of this end point: 6 months | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Secondary endpoints are: • Trilineage (CR and PR) and single lineage hematological response rate at 3, 6, 12 and 18 months. • cumulative incidence of response • time to best hematological trilineage and single lineage response • Proportion of patients with need for transfusions and number of units transfused (PRBC and PC) since start of treatment • cumulative incidence of progress to SAA/VSAA or intensive immunosuppressive treatment with ATG • toxicity profile as measured using the CTCAE criteria • relapse rate at 6, 12 and 18 months • cumulative incidence of relapse (from best trilineage hematological response) • overall survival • failure-free survival • telomere lengths and presence of telomerase mutations as biomarkers for response. • quality of life as assessed by quality of life instruments (FACIT-F SCALE and EORTC QLQ-C30, QLQAA/PNH) ;Timepoint(s) of evaluation of this end point: • Trilineage hematological response rate (CR and PR) at 3, 6, 12 and 18 months. • single lineage response at 3, 6, 12 and 18 months • relapse rate at 6, 12 and 18 months | — |
Countries
France, Germany, Switzerland
Contacts
Public ContactBritta Höchsmann
Universitätsklinikum Ulm
Outcome results
None listed