Purpura, Thrombocytopenic, IdiopathicPhase 2NCT00706342
Pilot Study of Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP)
A Phase 2 study testing Fostamatinib Disodium / R935788 in people with Purpura, Thrombocytopenic, Idiopathic, run by Rigel Pharmaceuticals at 2 sites in United States.
Trial 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). Read more about this study
- Who is it for?
- Ages 18 to 75 years
- Conditions the study is for
- Purpura, Thrombocytopenic, Idiopathic
- What is being tested?
- Fostamatinib Disodium / R935788
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 18 participants
- Where
- 2 sitesUnited States
- Study dates
- Jan 2007to Apr 2010
- Phase
- Phase 2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 to 75 years
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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.
Trial locations and contacts
2 sites in 1 country.
United States2 sites
Research Site
Cleveland, Ohio, 44195
Research Site
New York, New York, 10065
About this study
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
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).
Read the 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.
- Conditions
- Purpura, Thrombocytopenic, Idiopathic
- Being tested
- Fostamatinib Disodium / R935788
- Keywords
- Adult Refractory Immune Thrombocytopenic Purpura (ITP)
- Medical subject headings
- Purpura, Thrombocytopenic, Idiopathic
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Fostamatinib Disodium / R935788Drug
Also called R788
R935788 tablets
| Study group | Role | What they receive |
|---|---|---|
| 1 | Experimental | Not described Receives: Drug: Fostamatinib Disodium / R935788 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 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
Measured: 24 Months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted27 Jun 2008 (estimated)
- Study startJan 2007
- Primary completionApr 2010
- Study completionApr 2010
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Purpura, Thrombocytopenic, IdiopathicStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 18 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rigel Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Jeffrey Skolnik, MD, Study director, AstraZeneca
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT00706342
- Sponsor's protocol ID
- D4300C00022
- Other IDs
- C-935788-007
- Status last verified
- May 2016
- First submitted
- 25 Jun 2008
- Last updated
- 3 Jun 2016
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
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.
PubMed 19096013
Study at a glance
From the registry record, last updated by the sponsor on 3 Jun 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT00706342
- Acronym / sponsor ID
- D4300C00022
- Conditions
- Purpura, Thrombocytopenic, Idiopathic
- Keywords
- Adult Refractory Immune Thrombocytopenic Purpura (ITP)
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 18 participants (actual)
- Lead sponsor
- Rigel PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Jan 2007
- Primary completion
- Apr 2010 (actual)
- Study completion
- Apr 2010 (actual)
- First posted
- 27 Jun 2008 (estimated)
- Results
- Posted 13 Aug 2014
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| 1 | Experimental | Drug: Fostamatinib Disodium / R935788 |
Who may qualify
- Ages
- 18 to 75 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (7 listed)
- 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:
- Subjects must test negative for HIV, HBV, and HCV by standard serologic tests within the previous six months.
Key exclusion criteria (4 listed)
- 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.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Fostamatinib Disodium / R935788Also: R788R935788 tablets | Drug | 1 |
Primary outcome
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
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
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
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
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
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
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 3 Jun 2016.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Results publication: PubMed 19096013Podolanczuk 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.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jeffrey Skolnik, MD study director
AstraZeneca
2 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing Fostamatinib Disodium / R935788 in people with Purpura, Thrombocytopenic, Idiopathic, run by Rigel Pharmaceuticals at 2 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 7 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 3 Jun 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 13 Aug 2014). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the 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). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT00706342, which also lists 1 linked publication.
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 3 Jun 2016.