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

NCT00706342 | Completed Phase 2 Results DMC

• 2 other identifiers: D4300C00022C-935788-007
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 2

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).

Conditions

Purpura, Thrombocytopenic, Idiopathic

Keywords

Adult Refractory Immune Thrombocytopenic Purpura (ITP)

Outcome Measures

Primary Outcomes (6)

• 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

  2 weeks

• 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

  6 Weeks

• 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

  12 Weeks

• 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

  24 Weeks

• 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

  12 Months

• 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

  24 Months

Study Arms (1)

1

EXPERIMENTAL

Drug: Fostamatinib Disodium / R935788

Interventions

Fostamatinib Disodium / R935788 DRUG

R935788 tablets

Also known as: R788

1

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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:
• 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.
• 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.
• 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.

You may not qualify if:

• 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.

Sponsors & Collaborators

• Rigel Pharmaceuticals: lead

Study Sites (2)

Research Site

New York, New York, 10065, United States

Location

Research Site

Cleveland, Ohio, 44195, United States

Location

Related Publications (1)

• 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.

  PMID: 19096013 RESULT

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

fostamatinib

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Results Point of Contact

• Title: Anne-Marie Duliege, MD
• Organization: Rigel

clinicaltrials@rigel.com

650-624-1100

Study Officials

• Jeffrey Skolnik, MD

  AstraZeneca

  STUDY DIRECTOR

Publication Agreements

• PI is Sponsor Employee: No
• Restriction Type: LTE60
• Restrictive Agreement: Yes

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: June 25, 2008
• First Posted: June 27, 2008
• Study Start: January 1, 2007
• Primary Completion: April 1, 2010
• Study Completion: April 1, 2010
• Last Updated: June 3, 2016
• Results First Posted: August 13, 2014

Record last verified: 2016-05

Data Sharing

• IPD Sharing: Will not share

Locations

US (2)