Clinical trial NCT00706342: Pilot Study of Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP)
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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE2
Conditions: Purpura, Thrombocytopenic, Idiopathic
Keywords: Adult Refractory Immune Thrombocytopenic Purpura (ITP)
Lead sponsor: Rigel Pharmaceuticals
Sponsor class: INDUSTRY
First posted: 2008-06-27
Enrollment: 18
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: 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).

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.

Eligibility criteria: 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.

Primary outcomes:
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
timeFrame: 2 weeks
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 6
timeFrame: 6 Weeks
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 12
timeFrame: 12 Weeks
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 24
timeFrame: 24 Weeks
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 - Month 12
timeFrame: 12 Months
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 - Month 24
timeFrame: 24 Months
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: Research Site
state: New York
city: Cleveland
country: United States
facility: Research Site
state: Ohio
Study officials:
affiliation: AstraZeneca
name: Jeffrey Skolnik, MD
role: STUDY_DIRECTOR
References:
citation: 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. doi: 10.1182/blood-2008-07-166439. Epub 2008 Dec 18.