Eltrombopag and the Bcl-extra-large (xL) Pathway in Idiopathic Thrombocytopenic Purpura (ITP)

• Register: ClinicalTrials.gov
• Last refreshed on: 12 December 2020
• Main ID: NCT00902018
• Date of registration: 08/05/2009
• Prospective Registration: No
• Primary sponsor: Weill Medical College of Cornell University
• Public title: Eltrombopag and the Bcl-extra-large (xL) Pathway in Idiopathic Thrombocytopenic Purpura (ITP)
• Scientific title: The Effect of Eltrombopag on Platelet Survival: the Role of the B-cell L Extra Large (BcL-xL) Pathway
• Date of first enrolment: January 2009
• Target sample size: 20
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT00902018
• Study type: Interventional
• Study design: Allocation: Non-Randomized. Intervention model: Sequential Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

United States

Contacts

• Name: James B. Bussel, MD
• Affiliation: Weill Medical College of Cornell University

Key inclusion & exclusion criteria

Inclusion Criteria:

- Subject has signed and dated a written informed consent

- Male or female adults (=18 years) diagnosed with either primary ITP according to the
American Society for Hematology or British Committee for Standards in Haematology
(ASH/BCSH) guidelines [Blood, 1996; British Journal of Haematology, 2003] for at least
three months prior to study entry or with ITP secondary to Evans syndrome, systemic
lupus erythematosus (SLE), or Common Variable Immunodeficiency (including
hypogammaglobulinemia).

- Subjects must have responded with a platelet count > 30,000/µL to a previous ITP
therapy including thrombopoietic agents.

- Platelet count < 30,000/µL

- Female subjects of childbearing potential are practicing an acceptable method of
contraception or are completely abstinent from intercourse.

Exclusion Criteria:

- Active infection

- Previously treated with thrombopoietic agents IF either no response at a therapeutic
dose (peak platelet count < 50k) OR treatment with the agent within the past 4 weeks

- Currently treated with concomitant ITP medication that has not been stable in dose for
at least 2 weeks - only prednisone, azathioprin, and danazol are allowed.

- Female subjects who are nursing or pregnant

- Thrombosis of any kind within past 6 months or on blood thinners because of
thrombosis.

- Intravenous Immunoglobulin (IVIG), IV anti-D, bolus corticosteroids or vinca alkaloids
within the past week

- Other cytotoxic or immunosuppressive ITP therapy within the past 8 weeks or rituximab
within the past 12 weeks

- Active non-dermatologic malignancy defined as presence of known tumor ie. visible by
radiography or evident on blood or bone marrow testing OR receiving chemotherapy
within past 2 months

- Hemoglobin < 10 gm/dl or white blood cell count < 2,500/ul

- Liver function tests (ALT, Aspartate Aminotransferase (AST), or total bilirubin) >
three times upper limit of normal (ULN)

- Creatinine > two times upper limit of normal (ULN)



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Immune Thrombocytopenia

Intervention(s)

Drug: Eltrombopag

Drug: Romiplostim

Other: healthy controls

Primary Outcome(s)

Number of Patients for Whom Eltrombopag Increases the Platelet Count to > 50,000/uL [Time Frame: platelet counts on days 8 and 15]

Number of Patients Who Received Romiplostim and Increased Their Platelet Counts to > 50,000/uL [Time Frame: platelet counts on days 8 and 15]

Secondary Outcome(s)

How Many Patients Developed SAEs and/or Abnormal Liver Tests to a Level > 2 Times the Upper Limit of Normal [Time Frame: on days 8 and 15]

Secondary ID(s)

0809009980

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

GlaxoSmithKline

Results

• Results available: Yes
• Date Posted: 18/03/2019
• URL: https://clinicaltrials.gov/ct2/show/results/NCT00902018