Pilot Study of be Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP)

This study has been completed.
Information provided by (Responsible Party):
ClinicalTrials.gov Identifier:
First received: June 25, 2008
Last updated: August 18, 2011
Last verified: August 2011

The purpose of this study is to determine whether Fostamatinib Disodium is safe and effective in the treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP).

Condition Intervention Phase
Purpura, Thrombocytopenic, Idiopathic
Drug: Fostamatinib Disodium / R935788
Phase 2

Study Type: Interventional
Study Design: Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: A Phase II, Open-Label, Efficacy and Safety, Ascending Dose, Pilot Study of be Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura

Resource links provided by NLM:

Further study details as provided by AstraZeneca:

Primary Outcome Measures:
  • The primary objective of this pilot study is to assess the preliminary efficacy of Fostamatinib Disodium in the treatment of chronic refractory ITP as measured by platelet response. [ Time Frame: Study ] [ Designated as safety issue: No ]

Secondary Outcome Measures:
  • Assess the safety of Fostamatinib Disodium in the treatment of chronic refractory ITP using standard safety tests. [ Time Frame: study ] [ Designated as safety issue: Yes ]

Enrollment: 18
Study Start Date: January 2007
Study Completion Date: April 2010
Primary Completion Date: April 2010 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: 1 Drug: Fostamatinib Disodium / R935788
R935788 tablets
Other Name: R788

Detailed Description:

Patients with chronic refractory ITP are eligible for a 6- to 12-week therapeutic trial. After 24 months of treatment, patients who continue to demonstrate a sustained response, in the investigator's judgment, will be offered the opportunity to receive ongoing therapy.


Ages Eligible for Study:   18 Years to 75 Years
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No

Inclusion Criteria:

  • Patients must be willing and able to give written informed consent by signing an IRB-approved Informed Consent Form prior to admission to this study.
  • Patients may be male or female, between the ages of 18 75. Men, if sexually active, must agree to use at least one medically acceptable form of birth control. Women of childbearing potential must have a negative urine pregnancy test, and agree to use two independent methods of birth control, if sexually active.
  • Patients must have a diagnosis of chronic refractory ITP for at least 3 months. Chronic refractory ITP is defined as:

    1. Platelet count < 30,000/mm3 consistently for 3 months (except for transient nonsustained responses to various therapeutic regimens). There must be at least three separate platelet counts (below 30,000/mm3) over this period, with at least one extending back to three months or more prior to patient entry into the study.
    2. The following conditions will have been excluded either by history or appropriate laboratory investigation: HIV infection (see below), lymphoproliferative disorders, myelodysplasia, SLE, drug-induced or alloimmune thrombocytopenia, or dysglobulinemias.
    3. The patient must have tried at least two typical regimens for the treatment of ITP (George et al., Blood, 1996; Practice Guidelines, American Society of Hematology). At least 50% of the enrolled patients will not be known to be refractory to IVIg. Patients may or may not have been treated with IVIg in the past.
  • Subjects must test negative for HIV, HBV, and HCV by standard serologic tests within the previous six months.

Exclusion Criteria:

  • Patients who have a history or presence of substantial or clinically significant respiratory, gastrointestinal, renal, hepatic, neurological, cardiovascular, psychiatric, musculoskeletal, genitourinary, dermatological, or other disorders that, in the Investigator's opinion, could affect the conduct of the study or the absorption, metabolism or excretion of the study drug are excluded. Specifically excluded are lymphoma/chronic lymphocytic leukemia, hepatitis, or HIV associated with ITP.
  • Patients who have a history of relevant drug hypersensitivity are excluded.
  • Patients who have a history of substance abuse, drug addiction or alcoholism are excluded.
  • Patients with the following laboratory abnormalities: a leukocyte count < 2,500/mm3, a neutrophil count of < 1,800/mm3, lymphocyte count < 750/mm3, Hgb < 10 g/L, or transaminase levels (ALT, AST) > 1.5xULN are excluded.
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Please refer to this study by its ClinicalTrials.gov identifier: NCT00706342

United States, New York
Research Site
New York, New York, United States, 10065
United States, Ohio
Research Site
Cleveland, Ohio, United States, 44195
Sponsors and Collaborators
Study Director: Jeffrey Skolnik, MD AstraZeneca
  More Information

Responsible Party: AstraZeneca
ClinicalTrials.gov Identifier: NCT00706342     History of Changes
Other Study ID Numbers: D4300C00022, C-935788-007
Study First Received: June 25, 2008
Last Updated: August 18, 2011
Health Authority: United States: Food and Drug Administration

Keywords provided by AstraZeneca:
Adult Refractory Immune Thrombocytopenic Purpura (ITP)

Additional relevant MeSH terms:
Purpura, Thrombocytopenic
Purpura, Thrombocytopenic, Idiopathic
Blood Coagulation Disorders
Hematologic Diseases
Pathologic Processes
Skin Manifestations
Signs and Symptoms
Thrombotic Microangiopathies
Blood Platelet Disorders
Immune System Diseases
Hemorrhagic Disorders
Autoimmune Diseases

ClinicalTrials.gov processed this record on July 28, 2014