Immune Thrombocytopenic PurpuraPhase 3FITNCT02076399
A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
A Phase 3 study testing Fostamatinib disodium in people with Immune Thrombocytopenic Purpura, run by Rigel Pharmaceuticals at 52 sites in 8 countries.
Trial summary
The purpose of this study is to determine whether fostamatinib is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP). Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Immune Thrombocytopenic Purpura
- What is being tested?
- Fostamatinib disodium
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 76 participants
- Where
- 52 sitesin 8 countries
- Study dates
- 14 Jul 2014to 21 Apr 2016
- Phase
- Phase 3
- 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 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- Clinical diagnosis of persistent/chronic ITP for at least 3 months.
- Average platelet count < 30,000/µL (and none > 35,000 unless as a result of rescue therapy) from at least 3 qualifying counts
Who cannot join
- Clinical diagnosis of autoimmune hemolytic anemia
- Uncontrolled or poorly controlled hypertension
- History of coagulopathy including prothrombotic conditions
Trial locations and contacts
52 sites in 8 countries.
52 sites in 8 countries
United Kingdom14 sites
Colchester General Hospital
Colchester, Essex, CO4 5JL
Kent & Canterbury Hospital
Kent, Canterbury, CT1 3NG
St. James's Hospital
Leeds, LS9 7TF
Leicester Royal Infirmary
Leicester, LE1 5WW
Royal Liverpool University Hospital
Liverpool, UK, L78XP
Royal London Hospital
London, E1 2ES
Hammersmith Hospital, Catherine Lewis Centre
London, W12 0HS
University College Hospital
London, WC1E 6HX
Manchester Royal Infirmary
Manchester, M139WL
Royal Victoria Infirmary
Newcastle upon Tyne, NE1 4LP
James Paget University Hospital
Norfolk, NR31 6LA
Oxford University Hospital
Oxford, OX3 9BQ
University Hospital of North Staffordshire
Stoke-on-Trent, ST4 6QG
Sandwell General Hospital
West Bromwich, B71 4HJ
United States13 sites
Center for Cancer and Blood Disorders
Bethesda, Maryland, 20817
University of Virginia
Charlottesville, Virginia, 22908
Cleveland Clinic
Cleveland, Ohio, 44195
Pitt County Memorial Hospital
Greenville, North Carolina, 27858
Horizon Oncology Research, Inc
Lafayette, Indiana, 47905
Lakeland Regional Cancer Center
Lakeland, Florida, 33805-1965
University of Southern California
Los Angeles, California, 90089
Signal Point Clinical Research Center LLC
Middletown, Ohio, 45042
Weill Cornell Medical College/New York Presbyterian Hospital
New York, New York, 10065
Bleeding & Clotting Disorders Institute
Peoria, Illinois, 61614
Bill Hefner VA Medical Center
Salisbury, North Carolina, 28144
University of Utah Health Sciences Center
Salt Lake City, Utah, 84132
Arizona Oncology Associates
Tucson, Arizona, 85710
Australia9 sites
Concord Repatriation General Hospital
Concord, New South Wales, 2139
Frankston Hospital
Frankston, Victoria, 3199
St George Hospital
Kogarah, New South Wales, 2217
Launceston General Hospital
Launceston, Tasmania, 7250
Liverpool Hospital
Liverpool, New South Wales, 2170
Dept of Haematology, The Alfred Hospital and Monash Medical Centre
Melbourne, Victoria, 3004
Perth Blood Institute
Nedlands, Western Australia, 6009
Prince of Wales Hospital
Randwick, New South Wales, 2031
Westmead Hospital
Westmead, New South Wales, 2145
Italy5 sites
Ematologia - Padigilione 8, Policinico S. Orsola Malpighi, Azienda Ospedaliero Universitaria di Bologna
Bologna, 40138
Ospedale San Raffaele S.r.l. Dipartimento di Oncoematologia
Milan, 20132
Universitã Federico II di Napoli
Naples, 80131
OspedaleCivile-ClinicaEmatologica/PUGD
Udine, 33100
ULSS 6 Vicenza-Ospedale San Bortolo di Vicenza
Vicenza
Denmark4 sites
Aarhus University Hospital
Aalborg, 9000
Herlev Hospital University of Copenhagen, Department of Hematology L124
Herlev, DK, 2730
Dept. of Haematology, Odense University Hospital
Odense C, DK, DK-5000
Hematological department, Roskilde Hospital
Roskilde, DK, 4000
Canada3 sites
Hamilton Health Sciences Corporation
Hamilton, Ontario, L8N 3Z5
Ottawa Hospital Research Institute
Ottawa, Ontario, K1 H8L6
St. Michael's Hospital
Toronto, Ontario, M5B1W8
Hungary3 sites
Semmelweis University 1st
Budapest, H-1083
University of Debrecen
Debrecen, H-1083
Pecs University
Pécs, H-7624
About this study
Official title: A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura
The purpose of this study is to determine whether fostamatinib is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP).
- Conditions
- Immune Thrombocytopenic Purpura
- Being tested
- Fostamatinib disodium, Placebo
- Keywords
- Persistent Immune Thrombocytopenic Purpura, Chronic Immune Thrombocytopenic Purpura
- 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 disodiumDrug
Also called R935788, R788, Fostamatinib
Fostamatinib (100 mg PO bid or 150 mg PO bid)
PlaceboDrug
Placebo tablet PO bid (morning and evening) over the course of 24 weeks
| Study group | Role | What they receive |
|---|---|---|
| Fostamatinib Disodium | Experimental | Subjects begin with Fostamatinib Disodium tablet 100 mg PO bid and increase to 150 mg big after week 4 based on platelet count and tolerability. Receives: Drug: Fostamatinib disodium |
| Placebo | Other | Placebo tablet PO bid (morning and evening) over the course of 24 weeks Receives: Drug: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Number of Participants With Stable Platelet Response (Count of ≥50,000/µL on at Least 4 of the Last 6 Scheduled Visits Between Weeks 14 and 24)
A stable platelet response by Week 24 defined as a platelet count of at least 50,000/μL on at least 4 of the last 6 scheduled visits between Weeks 14 and 24
Measured: From Week 14 to Week 24
Secondary outcomes (6)
Number of Participants With Platelet Count ≥ 50,000/µL at Week 12
Platelet Count ≥ 50,000/µL at Week 12
Measured: Week 12
Number of Participants With Platelet Count ≥ 50,000/µL at Week 24
Platelet Count ≥ 50,000/µL at Week 24
Measured: Week 24
Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 12.
Number of subjects with baseline platelet count <15,000/μL who showed platelet count increase to ≥30,000/μL and ≥20,000/μL from baseline count at Week 12.
Measured: Baseline to Week 12
Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 24.
Number of subjects with baseline platelet count <15,000/μL who showed platelet count increase to ≥30,000/μL and ≥20,000/μL from baseline count at Week 24.
Measured: Baseline to Week 24
Mean of the ITP Bleeding Score (IBLS)
The ITP Bleeding Scale (IBLS) is an immune thrombocytopenic purpura (ITP)-specific bleeding score used to analyze the correlation of clinical and laboratory platelet variables with bleeding. The IBLS comprises of 11 grades from 0 (none) to 2 (marked bleeding) by history over the previous week or by exam; 2 being worse. These 11 grades include: skin by physical exam, oral by physical exam, skin by history, oral by history, epistaxis, gastrointestinal, urinary, gynecological, pulmonary, intracranial hemorrhage, and subconjunctival hemorrhage. After each grade is scored, the mean value for all 11 grades is calculated (lowest score being 0 and highest score being 2) for each subject visit. LOCF method was used to impute any missing data.
The mean of the IBLS scores across visits during the 24-week treatment period was summarized by treatment group using descriptive statistics. A 2-sided, 2-sample t-test was used to test for a difference in means between treatments for this endpoint.
Measured: Assessed over the 24-week study period
Mean of World Health Organization (WHO) Bleeding Scale
The World Health Organization (WHO) bleeding scale is a standardized grading scale created to measure the severity of bleeding. The scale is a clinical investigator-assessed five-point scale with a score range starting at the lowest 0=No bleeding, 1 = Petechiae, 2=Mild blood loss, 3=Gross blood loss, to the worse 4=Debilitating blood loss. The WHO bleeding scale is scored by history over the previous-week or by exam. After each grade is scored, the mean value is calculated (lowest score being 0 [no bleeding] to the highest score being 4 [debilitating blood loss]) for each visit. LOCF method was used to impute any missing data.
The mean of the WHO bleeding scale across visits during the 24-week treatment period was summarized by treatment group using descriptive statistics. A 2-sided, 2-sample t-test was used to test for a difference in means between treatments for this endpoint.
Measured: Assessed over the 24-week study period
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted3 Mar 2014 (estimated)
- Study start14 Jul 2014
- Primary completion21 Apr 2016
- Study completion21 Apr 2016
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 Immune Thrombocytopenic PurpuraStudies 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 3
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 76 participants (actual)
Sponsor and investigators
- Lead sponsor
- Rigel Pharmaceuticals
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
- Investigators
- Rigel Pharmaceuticals, Inc., Study director, Rigel Pharmaceuticals,Inc.
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02076399
- Sponsor's protocol ID
- C-935788-047
- Other IDs
- 2013-005452-15
- Acronym
- FIT
- Status last verified
- Oct 2018
- First submitted
- 26 Feb 2014
- Last updated
- 12 Feb 2019
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- Individual data sharing
- No
Publications
Cooper N, Altomare I, Thomas MR, Nicolson PLR, Watson SP, Markovtsov V, Todd LK, Masuda E, Bussel JB. Assessment of thrombotic risk during long-term treatment of immune thrombocytopenia with fostamatinib. Ther Adv Hematol. 2021 Apr 30;12:20406207211010875. doi: 10.1177/20406207211010875. eCollection 2021.
PubMed 33995988
Study at a glance
From the registry record, last updated by the sponsor on 12 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3, Multi-Center, Randomized, Double-Blind, Placebo-Controlled, Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT02076399
- Acronym / sponsor ID
- FIT / C-935788-047
- Conditions
- Immune Thrombocytopenic Purpura
- Keywords
- Persistent Immune Thrombocytopenic Purpura, Chronic Immune Thrombocytopenic Purpura
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 76 participants (actual)
- Lead sponsor
- Rigel PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- 14 Jul 2014 (actual)
- Primary completion
- 21 Apr 2016 (actual)
- Study completion
- 21 Apr 2016 (actual)
- First posted
- 3 Mar 2014 (estimated)
- Results
- Posted 11 Jan 2019
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 is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP).
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Fostamatinib Disodium | Experimental | Drug: Fostamatinib disodiumSubjects begin with Fostamatinib Disodium tablet 100 mg PO bid and increase to 150 mg big after week 4 based on platelet count and tolerability. |
| Placebo | Other | Drug: PlaceboPlacebo tablet PO bid (morning and evening) over the course of 24 weeks |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Clinical diagnosis of persistent/chronic ITP for at least 3 months.
- Average platelet count < 30,000/µL (and none > 35,000 unless as a result of rescue therapy) from at least 3 qualifying counts
Key exclusion criteria (3 listed)
- Clinical diagnosis of autoimmune hemolytic anemia
- Uncontrolled or poorly controlled hypertension
- History of coagulopathy including prothrombotic conditions
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 disodiumAlso: R935788, R788, FostamatinibFostamatinib (100 mg PO bid or 150 mg PO bid) | Drug | Fostamatinib Disodium |
| PlaceboPlacebo tablet PO bid (morning and evening) over the course of 24 weeks | Drug | Placebo |
Primary outcome
Number of Participants With Stable Platelet Response (Count of ≥50,000/µL on at Least 4 of the Last 6 Scheduled Visits Between Weeks 14 and 24)
Time frame: From Week 14 to Week 24
A stable platelet response by Week 24 defined as a platelet count of at least 50,000/μL on at least 4 of the last 6 scheduled visits between Weeks 14 and 24
6 secondary outcomes
Number of Participants With Platelet Count ≥ 50,000/µL at Week 12
Time frame: Week 12
Number of Participants With Platelet Count ≥ 50,000/µL at Week 24
Time frame: Week 24
Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 12.
Time frame: Baseline to Week 12
Platelet Count ≥ 30,000/μL and ≥ 20,000/μL Above Baseline in Subjects With Baseline Platelet Count of <15,000/μL at Week 24.
Time frame: Baseline to Week 24
Mean of the ITP Bleeding Score (IBLS)
Time frame: Assessed over the 24-week study period
Mean of World Health Organization (WHO) Bleeding Scale
Time frame: Assessed over the 24-week study period
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 12 Feb 2019.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Derived from this study: PubMed 33995988Cooper N, Altomare I, Thomas MR, Nicolson PLR, Watson SP, Markovtsov V, Todd LK, Masuda E, Bussel JB. Assessment of thrombotic risk during long-term treatment of immune thrombocytopenia with fostamatinib. Ther Adv Hematol. 2021 Apr 30;12:20406207211010875. doi: 10.1177/20406207211010875. eCollection 2021.
- 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.
Rigel Pharmaceuticals, Inc. study director
Rigel Pharmaceuticals,Inc.
52 sites in 8 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 3 study testing Fostamatinib disodium in people with Immune Thrombocytopenic Purpura, run by Rigel Pharmaceuticals at 52 sites in 8 countries.
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 2 inclusion and 3 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 12 Feb 2019. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 52 sites in United Kingdom, United States, Australia, Italy, Denmark and 3 more countries. 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 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.
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 11 Jan 2019). 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?
Number of Participants With Stable Platelet Response (Count of ≥50,000/µL on at Least 4 of the Last 6 Scheduled Visits Between Weeks 14 and 24) (time frame: From Week 14 to Week 24). 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 NCT02076399, 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 12 Feb 2019.