Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia

• Register: ClinicalTrials.gov
• Last refreshed on: 8 April 2024
• Main ID: NCT01623167
• Date of registration: 14/06/2012
• Prospective Registration: Yes
• Primary sponsor: National Heart, Lung, and Blood Institute (NHLBI)
• Public title: Eltrombopag With Standard Immunosuppression for Severe Aplastic Anemia
• Scientific title: Eltrombopag Added to Standard Immunosuppression in Treatment-Naive Severe Aplastic Anemia
• Date of first enrolment: July 2, 2012
• Target sample size: 207
• Recruitment status: Recruiting
• URL: https://clinicaltrials.gov/ct2/show/NCT01623167
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1/Phase 2

Countries of recruitment

United States

Contacts

• Name: Olga J Rios, R.N.

• Name: For more information at the NIH Clinical Center contact Office of Patient Recruitment (OPR)

• Name: Neal S Young, M.D.
• Affiliation: National Heart, Lung, and Blood Institute (NHLBI)

Key inclusion & exclusion criteria

-INCLUSION CRITERIA:

1. Severe aplastic anemia characterized by Bone marrow cellularity less than 30 percent
(excluding lymphocytes)

AND

At least two of the following:

- Absolute neutrophil count less than 500/microL

- Platelet count less than 20,000/microL

Absolute reticulocyte count less than 60,000/microL

2. Age greater than or equal to 2 years old

3. Weight greater than 12 kg

EXCLUSION CRITERIA:

1. Known diagnosis of Fanconi anemia

2. Evidence of a clonal disorder on cytogenetics performed within 12 weeks of study
entry. Patients with super severe neutropenia (ANC less than 200 /microL) will not be
excluded initially if cytogenetics are not available or pending. If evidence of a
clonal disorder consistent with myelodysplasia is later identified, the patient will
go off study.

3. Prior immunosuppressive therapy with any ATG, alemtuzumab, or high dose
cyclophosphamide

4. SGOT or SGPT >5 times the upper limit of normal

5. Subjects with known liver cirrhosis in severity that would preclude tolerability of
cyclosporine and eltrombopag as evidenced by albumin < 35g/L

6. Hypersensitivity to eltrombopag or its components

7. Infection not adequately responding to appropriate therapy

8. 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

9. Potential subjects with cancer who are on active chemotherapeutic treatment or who
take drugs with hematological effects will not be eligible

10. Current pregnancy, or unwillingness to take oral contraceptives or use a barrier
method of birth control or practice abstinence to refrain from pregnancy if of
childbearing potential during the course of this study

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



Age minimum: 2 Years
Age maximum: 95 Years
Gender: All

Health Condition(s) or Problem(s) studied

Severe Aplastic Anemia

Intervention(s)

Drug: Cohort 1: hATG, CsA, EPAG Day 14 to Month 6

Drug: Extension Cohort

Drug: Cohort 2: hATG, CsA, EPAG Day 14 to Month 3

Drug: Cohort 3: hATG, CsA (dose reduced), EPAG day 1 to month 6

Primary Outcome(s)

Rate of Complete Hematologic Response [Time Frame: 6 months]

Secondary Outcome(s)

Rate of Response at 3 and 12 Months Then Yearly; Rate of Relapse; Rate of Clonal Evolution to PNH, MDS and AML; Rate of Survival; Rate of Response for Relapse Subjects That Re-start Treatment and Effects of CsA Dose Starting at Month 6 to Month 24. [Time Frame: 3 months to 5 years]

Secondary ID(s)

12-H-0150

120150

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Novartis

Results

• Results available: Yes
• Date Posted: 05/06/2020
• URL: https://clinicaltrials.gov/ct2/show/results/NCT01623167