Efficacy and Safety of Eltrombopag in Patients with Acquired Moderate Aplastic Anemia (EMAA) who are treated with Ciclosporin A Prospective Randomized Multicenter Study comparing Thrombopoetin-Receptor agonist Eltrombopag with Placebo in Patients with Acquired Moderate Aplastic Anemia who are treated with Ciclosporin A

Phase 1

• Conditions: Moderate Aplastic Anemia (MAA); Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

• Registration Number: EUCTR2014-000174-19-DE
• Lead Sponsor: niversitätsklinikum Ulm

Brief Summary

Not available

Detailed Description

Not available

Study Timeline

• Results First Posted: 1900-01-01
• Study Start: 2014-09-01
• Last Update Posted: 16 May 2022

Recruitment & Eligibility

• Status: Authorised-recruitment may be ongoing or finished
• Sex: All
• Target Recruitment: 90

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) < 1.2 G/L
• platelet count < 70 G/L
• absolute reticulocyte count < 60 G/L21
• without fulfilling the criteria for SAA (hypocellularity of bone marrow 25 % and depression of two of the three peripheral counts: ANC < 0.5 G/L, platelet count < 20 G/L, reticulocyte count < 20 G/L)

2. In this study need for treatment with CSA is defined as:
a. transfusion-independent MAA and:
• ANC < 1.0 G/L
• or hemoglobin < 8.5 g/dl and reticulocyte count < 60 G/L
• or platelet count < 30 G/L
• or significant clinical symptoms (infections, bleeding, anemia)
b. transfusion-dependent MAA:
• Platelet transfusion dependency is defined as prophylactic transfusion (platelet counts < 10 G/L with no bleeding) or therapeutic transfusion in the 12 weeks prior to study entry.
• Red blood cell transfusion dependency is defined as transfusion of at least 4 units of packed red blood cell
concentrates (PRBC) in the 12 weeks prior to study entry.
3. A signed and dated informed consent is necessary before the conduct of any study-specific procedure.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 90
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 90

Exclusion Criteria

1. Age < 18 years
2. Constitutional Aplastic Anemia (i.e. Fanconi anemia or Dyskeratosis congenita)
3. Clonal myeloid disorders based on cytogenetic findings performed within 12 weeks of study entry. Especially patients with cytogenetic abnormalities which are recurrent in MDS are not eligible for the
study.
4. Bone marrow reticulin fibrosis of grade 3 or greater
5. Severe concurrent diseases precluding the patient's ability to tolerate protocol therapy
6. ALT > 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.

Study & Design

• Study Type: Interventional clinical trial of medicinal product
• Study Design: Not specified