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

This study has been completed.
Sponsor:
Information provided by (Responsible Party):
AstraZeneca
ClinicalTrials.gov Identifier:
NCT00706342
First received: June 25, 2008
Last updated: July 23, 2014
Last verified: July 2014

June 25, 2008
July 23, 2014
January 2007
April 2010   (final data collection date for primary outcome measure)
  • Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Week 2 [ Time Frame: 2 weeks ] [ Designated as safety issue: No ]
  • Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Week 6 [ Time Frame: 6 Weeks ] [ Designated as safety issue: No ]
  • Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Week 12 [ Time Frame: 12 Weeks ] [ Designated as safety issue: No ]
  • Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Week 24 [ Time Frame: 24 Weeks ] [ Designated as safety issue: No ]
  • Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Month 12 [ Time Frame: 12 Months ] [ Designated as safety issue: No ]
  • Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Month 24 [ Time Frame: 24 Months ] [ Designated as safety issue: No ]
The primary objective of this pilot study is to assess the preliminary efficacy of R788 in the treatment of chronic refractory ITP as measured by platelet response. [ Time Frame: Study ] [ Designated as safety issue: No ]
Complete list of historical versions of study NCT00706342 on ClinicalTrials.gov Archive Site
Not Provided
The secondary objective of this pilot-study is to assess the safety of R788 in the treatment of chronic refractory ITP using standard safety tests. [ Time Frame: study ] [ Designated as safety issue: Yes ]
Not Provided
Not Provided
 
Pilot Study of be Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP)
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

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).

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.

Interventional
Phase 2
Allocation: Non-Randomized
Endpoint Classification: Safety/Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Purpura, Thrombocytopenic, Idiopathic
Drug: Fostamatinib Disodium / R935788
R935788 tablets
Other Name: R788
Experimental: 1
Intervention: Drug: Fostamatinib Disodium / R935788
Podolanczuk A, Lazarus AH, Crow AR, Grossbard E, Bussel JB. Of mice and men: an open-label pilot study for treatment of immune thrombocytopenic purpura by an inhibitor of Syk. Blood. 2009 Apr 2;113(14):3154-60. Epub 2008 Dec 18.

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Completed
18
April 2010
April 2010   (final data collection date for primary outcome measure)

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.
Both
18 Years to 75 Years
No
Contact information is only displayed when the study is recruiting subjects
United States
 
NCT00706342
D4300C00022, C-935788-007
Yes
AstraZeneca
AstraZeneca
Not Provided
Study Director: Jeffrey Skolnik, MD AstraZeneca
AstraZeneca
July 2014

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP