Eltrombopag for Moderate Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 27 February 2023
• Main ID: NCT01328587
• Date of registration: 01/04/2011
• Prospective Registration: Yes
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: Eltrombopag for Moderate Aplastic Anemia
• Scientific title: A Pilot Study of a Thrombopoietin-Receptor Agonist (TPO-R Agonist), Eltrombopag, in Moderate Aplastic Anemia Patients
• Date of first enrolment: April 1, 2011
• Target sample size: 34
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT01328587
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: Cynthia E Dunbar, M.D.
• Affiliation: National Heart, Lung, and Blood Institute (NHLBI)

Key inclusion & exclusion criteria

- INCLUSION CRITERIA:

Current diagnosis of moderate aplastic anemia or unilineage bone marrow failure disorders.

- Moderate aplastic anemia is defined as aplastic anemia (hypocellular bone marrow for
age) with no evidence for other disease processes causing marrow failure, and
depression of at least two out of three blood counts below the normal values:

- ANC less than or equal to 1200/mm(3)

- platelet count less than or equal to 70,000/mm(3)

- anemia with hemoglobin less than or equal to 8.5 g/dL and absolute reticulocyte
count less than or equal to 60,000/mm(3) in transfusion-dependent patients but
not fulfilling the criteria for severe disease defined by depression of two of
the three peripheral counts:

- ANC less than or equal to 500/mm(3)

- platelet count less than or equal to 20,000/mm(3)

- reticulocyte count less than or equal to 60,000/mm(3)

- Unilineage bone marrow failure disorders are defined:

- Hemoglobin less than 8.5 g/dL and reticulocyte count less than 60,000 or red cell
transfusion dependent and hypocellular to normocellular bone marrow for age with
significantly reduced erythroid precursors.

- OR thrombocytopenia less than or equal to 30,000/uL or platelet transfusion
dependent and hypocellular to normocellular bone marrow for age with reduced
megakaryocytes.

- No evidence of viral or drug suppression of the marrow, dysplasia, or underproduction
anemias secondary to B12, folate, iron or other reversible causes.

Platelet transfusion dependent is defined as the need for platelet transfusion due to
platelet counts of < 10,000/microL with no bleeding (prophylactic transfusion) or <
20,000/microL with bleeding (therapeutic transfusion). Red cell transfusion dependent is
defined as transfusion of greater than 4 units of blood in the 8 weeks prior to study
entry.

Age greater than or equal to 2 years old

Weight greater than 12 kg

EXCLUSION CRITERIA:

Known diagnosis of Fanconi anemia

Counts that meet criteria for severe aplastic anemia

Infection not adequately responding to appropriate therapy

HIV positivity

Creatinine > 2.5 mg/dL

Bilirubin > 2.0 mg/dL, including congenital abnormalities in the bilirubin count

SGOT or SGPT >5 times the upper limit of normal

Hypersensitivity to eltrombopag or its components

Female subjects who are nursing or pregnant or are unwilling to take oral contraceptives or
refrain from pregnancy if of childbearing potential

Evidence of an active malignant hematological or clonal disorder, or abnormal cytogenetic
studies of the bone marrow performed within 12 weeks of study entry.

Unable to understand the investigational nature of the study or give informed consent or
does not have a legally authorized representative or surrogate that can provide informed
consent

Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary, infectious,
or metabolic disease of such severity that it would preclude the patient's ability to
tolerate protocol therapy, or that death within 7-10 days is likely.

Treatment with horse or rabbit ATG or Campath within 6 months of study entry.

Treatment with cytokines such as G-CSF or Erythropoietin.

Subjects with known cirrhosis in severity that would preclude tolerability of eltrombopag
as evidenced by albumin less than 35g/L.

Life expectancy of less than 3 months

Patients with an active diagnosis of cancer who have received chemotherapeutic treatment or
other specific antineoplastic drugs or radiation therapy within 6 months of study entry.

Unable to take investigational drug



Age minimum: 2 Years
Age maximum: 100 Years
Gender: All

Health Condition(s) or Problem(s) studied

Moderate Aplastic Anemia

Unilineage Bone Marrow Failure Disorders

Intervention(s)

Drug: Eltrombopag

Primary Outcome(s)

Proportion of Drug Responders [Time Frame: 16-20 weeks from start of drug]

Secondary ID(s)

11-H-0134

110134

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 23/04/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01328587