Pilot Study of 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

Status

Completed

Phases

Phase 2

Study type

Interventional

Source

ClinicalTrials.gov

Registry ID

NCT00706342

Enrollment

Registered

2008-06-27

Start date

2007-01-31

Completion date

2010-04-30

Last updated

2016-06-03

For informational purposes only — not medical advice. Sourced from public registries and may not reflect the latest updates. Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Keywords

Adult Refractory Immune Thrombocytopenic Purpura (ITP)

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.

Interventions

DRUGFostamatinib Disodium / R935788

R935788 tablets

Study design

Allocation

Intervention model

SINGLE_GROUP

Primary purpose

TREATMENT

Masking

NONE

Eligibility

Sex/Gender

ALL

Age

18 Years to 75 Years

Healthy volunteers

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.

Design outcomes

Primary

Measure	Time frame
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

Countries

United States

Participant flow

Participants by arm

Arm	Count
R788 PO Tablet containing 75mg BID to 225mg BID R788.	18
Total	18

Withdrawals & dropouts

Period	Reason	FG000
Overall Study	Adverse Event	3
Overall Study	Death	1
Overall Study	Investigator discretion	2
Overall Study	Non-responder Week 12	1
Overall Study	Not responding to treatment	2
Overall Study	Ongoing	6
Overall Study	WIthdrawal of consent	1
Overall Study	Withdrawn due to failure to respond	1
Overall Study	Withdrew from the study on his own.	1

Baseline characteristics

Characteristic	R788 PO
Age, Continuous Median (Range)	65 years
Age, Continuous	61.9 years STANDARD_DEVIATION 15.63
Sex: Female, Male Female	10 Participants
Sex: Female, Male Male	8 Participants

Adverse events

Event type	EG000 affected / at risk
deaths Total, all-cause mortality	— / —
other Total, other adverse events	18 / 18
serious Total, serious adverse events	8 / 18