Extended Dosing With Eltrombopag for Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 24 May 2021
• Main ID: NCT01891994
• Date of registration: 28/06/2013
• Prospective Registration: Yes
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: Extended Dosing With Eltrombopag for Severe Aplastic Anemia
• Scientific title: Extended Dosing With Eltrombopag in Refractory Severe Aplastic Anemia
• Date of first enrolment: June 28, 2013
• Target sample size: 40
• Recruitment status: Active, not recruiting
• URL: https://clinicaltrials.gov/show/NCT01891994
• 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:

- Previous diagnosis of refractory severe aplastic anemia and following at least one
treatment course of immunosuppression with a regimen containing antithymocyte
globulin, alemtuzumab or cyclophosphamide.

- One or more of the following three clinically-significant cytopenias: platelet count
less than or equal to 30,000/micro L or platelet-transfusion-dependence (requiring at
least 4 platelet transfusions in the 8 weeks prior to study entry); neutrophil count
less than 500/micro L; hemoglobin less than 9.0 g/dL or red cell
transfusion-dependence (requiring at least 4 units of PRBCs in the eight weeks prior
to study entry)

- Age greater than or equal to 2 years old

- Weight > 12 kg

EXCLUSION CRITERIA:

- Infection not adequately responding to appropriate therapy

- Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study
entry.

- Creatinine > 2.5 mg/dL

- Direct Bilirubin > 2.0 mg/dL

- 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

- Unable to understand the investigational nature of the study or give 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 ATG, cyclophophamide or alemtuzamab within 6 months of study entry.



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

Health Condition(s) or Problem(s) studied

Severe Aplastic Anemia (SAA)

Intervention(s)

Drug: Eltrombopag

Primary Outcome(s)

Number of Participants With Drug Response as Defined by Clinically-signficant Hematologic Improvements [Time Frame: 24 weeks]

Secondary ID(s)

13-H-0133

130133

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Results

• Results available: Yes
• Date Posted: 04/12/2018
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01891994