Immune Thrombocytopenic PurpuraPhase 3NCT02077192
Open Label Study of R788 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 55 sites in 15 countries.
Trial summary
The primary objective of this study was to assess the long term safety of fostamatinib in subjects with persistent/chronic 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?
- No, everyone receives an active treatment
- How many people
- 123 participants
- Where
- 55 sitesin 15 countries
- Study dates
- Oct 2014to 2 Jun 2020
- 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
- Completed week 24 evaluation of Study C935788-047 or Study C935788-048 or discontinued early due to lack of response.
- Able and willing to give written informed consent
Who cannot join
- Discontinued participation in Study C935788-047 or Study C935788-048 for any reason other than lack of response
- Poorly controlled hypertension during Study C935788-047 or Study C935788-048
- Significant infection, an acute infection such as influenza, or known inflammatory process
Trial locations and contacts
55 sites in 15 countries.
55 sites in 15 countries
United Kingdom11 sites
Kent & Canterbury Hospital
Canterbury, CT1 3NG
Colchester General Hospital
Colchester, Essex, CO4 5JL
James Paget University Hospital
Great Yarmouth, NR31 6LA
St. James's Hospital
Leeds, LS9 7TF
Leicester Royal Infirmary
Leicester, LE1 5WW
Royal Liverpool University Hospital
Liverpool, L78XP
Imperial College Healthcare NHS Trust
London, W12 0HS
University College Hospital
London, WC1E 6AG
Royal Victoria Infirmary
Newcastle upon Tyne, UK, NE1 4LP
Cancer and Haematology Centre
Oxford, OX3 7LE
University Hospital of North Midlands NHS Trust, Royal Stoke University Hospital
Stoke-on-Trent, ST4 6QG
United States9 sites
Center for Cancer and Blood Disorders
Bethesda, Maryland, 20817
East Carolina University, Brody School of Medicine
Greenville, North Carolina, 27834
Horizon Oncology Research, Inc
Lafayette, Indiana, 47905
Signal Point Clinical Research Center LLC
Middletown, Ohio, 45042
Weill Cornell Medical College/New York Presbyterian Hospital
New York, New York, 10065
Weill Cornell Medicine
New York, New York, 10065
Bleeding & Clotting Disorders Institute
Peoria, Illinois, 61615
W.G. "Bill" Hefner VA Medical Center
Salisbury, North Carolina, 28144
Arizona Oncology Associates
Tucson, Arizona, 85710
Poland8 sites
Lkinika Hematologii I Transplantologii Uniwersyteckie Centrum Kliniczne
Gdansk, 80-952
SPZOZ Szpital Uniwersytecki w Krakowie Pracownia Separacji Krwinek i Bank Komórek Krwiotwórczych Klinika Hematologii
Krakow, 31-501
Wojewódzki Szpital Specjalistyczny im. M. Kopernika w Łodzi
Lodz, 93-510
Specjalistyczny Gabinet Lekarski
Lublin, 20-601
Szpital Wojewodzki w Opolu
Opole, 45-061
Wojewodzki Szpital Specjalistyczny im. J. Korczaka
Słupsk, PO, 76-200
Instytut Hematologii I Transfuzjologii
Warsaw, 02-776
Samodzielny Publiczny Szpital Kliniczny Nr 1 we Wrocrlaw
Wroclaw, Dolnoslaski, 50-367
Australia7 sites
Concord Repatriation General Hospital
Concord, New South Wales, 2139
Launceston General Hospital
Launceston, Tasmania, 7250
Liverpool Hospital
Liverpool, New South Wales, 2170
The Alfred
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
Bulgaria4 sites
UMHAT Dr. Georgi Stranski, EAD, Pleven, Clinic of Hematology
Pleven, 5800
Specialized Hospital for Active Treatment of Hematology Diseases, EAD, Sofia, Department of Chemotherapy, Hemotherapy and Blood Inherited Diseases to Clinic of Clinical Hematology;
Sofia, BG, 1756
UMHAT Aleksandrovska, EAD
Sofia, 1431
MHAT Hristo Botev, AD, Vratsa, First Internal Department
Vratsa, 3000
Spain3 sites
Hospital Universitari Vall d'Hebron
Barcelona, 08035
Hospital Universitario La Paz
Madrid, 28046
Hospital Universitari i Politécnic La Fe de Valencia
Valencia, 46026
Canada2 sites
Hamilton Health Sciences Corporation
Hamilton, Ontario, L8N 3Z5
St. Michael's Hospital
Toronto, Ontario, M5B1W8
Czechia2 sites
Fakultni nemocnice Brno
Brno, 625 00
Fakultni nemocnice Ostrava
Ostrava-Poruba, 708 52
Italy2 sites
Istituto di Ematologia "Lorenzo e Ariosto Seràgnoli"
Bologna, BO, 40138
Azienda Ospedaliero-Universitaria "Santa Maria della Misericordia" - Clinica Ematologica
Udine, 33100
Norway2 sites
Haukeland universitetssykehus, Helse Bergen HF
Bergen, 5021
Sykehuset Østfold Kalnes
Grålum, 1714
Hungary1 site
Pecsi Tudomanyegyetem Klinikai Kozpont, I. sz. Belgyogyaszati Klinika
Pécs, H-7624
About this study
Official title: A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura
The primary objective of this study was to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP
- Conditions
- Immune Thrombocytopenic Purpura
- Being tested
- Fostamatinib Disodium
- 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 Disodium tablet 100 mg or 150 mg by mouth twice a day
| Study group | Role | What they receive |
|---|---|---|
| Fostamatinib Disodium | Experimental | Fostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day Receives: Drug: Fostamatinib Disodium |
What is being measured
The primary outcome is the main question the study is designed to answer.
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1
Percentage of subjects who achieved platelet count of at least 50,000/µL within 12 Weeks of beginning treatment up to 12 months was analyzed among all subjects who received active treatment in one of the prior studies (C788-047 or C788-048), in the current extension study (C788-049), or in both prior and current studies. Treated Population was defined as all enrolled and treated subjects.
Measured: Up to 12 months
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Placebo in 047/048 and Fostamatinib 049): Version 2
A within-subject, between-study comparison of platelet counts for subjects who were previously treated with placebo in one of the prior studies (C788-047 or C788-048) was prospectively defined in the protocol (version 2). Achievement of platelet response by 12 weeks and maintenance of platelet response for 12 weeks was analyzed among subjects who had been randomized to placebo in one of the prior studies (C788-047 or C788-048). Treated Population was defined as all enrolled and treated subjects.
Measured: Up to 12 months
Secondary outcomes (2)
Duration of Platelet Response Based on Platelet Count and Rescue Medication
The duration of platelet response was defined as the time from when the subject first achieved a platelet count of at least 50,000/µL, until the first of 2 visits with platelet counts < 50,000/µL that were at least 4 weeks apart without an intervening visit with a platelet count less than equal to (<=) 50,000/µL unrelated to rescue therapy. Duration of platelet response was analyzed using the Kaplan-Meier method. Treated Population was defined as all enrolled and treated subjects. Here, a number of subjects analyzed included all subjects evaluable for this endpoint.
Measured: Up to 12 months
Percentage of Subjects in Whom a Reduction in the Dose of Concomitant ITP Therapy Can be Achieved While Maintaining an Adequate Platelet Count
The percentage of subjects in whom a reduction in the dose of concomitant ITP therapy could be achieved while maintaining an adequate platelet count, the reduction event was clarified to apply only to reductions in the dose of concomitant ITP therapy occurring within a period of platelet response and the reduction event was not be prompted by an adverse event.
Measured: Up to 12 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted4 Mar 2014 (estimated)
- Study startOct 2014
- Primary completion2 Jun 2020
- Study completion2 Jun 2020
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
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 123 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
- NCT02077192
- Sponsor's protocol ID
- C-935788-049
- Other IDs
- 2013-005454-30
- Status last verified
- Dec 2023
- First submitted
- 27 Feb 2014
- Last updated
- 11 Dec 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- No
- 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 11 Dec 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Phase 3 Open Label Extension Study of Fostamatinib Disodium in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura
- ClinicalTrials.gov ID
- NCT02077192
- Acronym / sponsor ID
- C-935788-049
- Conditions
- Immune Thrombocytopenic Purpura
- Phase
- Phase 3
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 123 participants (actual)
- Lead sponsor
- Rigel PharmaceuticalsIndustry (pharma, biotech, devices)
- Study start
- Oct 2014
- Primary completion
- 2 Jun 2020 (actual)
- Study completion
- 2 Jun 2020 (actual)
- First posted
- 4 Mar 2014 (estimated)
- Results
- Posted 11 Dec 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
The primary objective of this study was to assess the long term safety of fostamatinib in subjects with persistent/chronic ITP
Study design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| Fostamatinib Disodium | Experimental | Drug: Fostamatinib DisodiumFostamatinib Disodium tablet 100 mg or 150 mg by mouth twice a day |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (2 listed)
- Completed week 24 evaluation of Study C935788-047 or Study C935788-048 or discontinued early due to lack of response.
- Able and willing to give written informed consent
Key exclusion criteria (3 listed)
- Discontinued participation in Study C935788-047 or Study C935788-048 for any reason other than lack of response
- Poorly controlled hypertension during Study C935788-047 or Study C935788-048
- Significant infection, an acute infection such as influenza, or known inflammatory process
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 Disodium tablet 100 mg or 150 mg by mouth twice a day | Drug | Fostamatinib Disodium |
Primary outcome
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1
Time frame: Up to 12 months
Percentage of subjects who achieved platelet count of at least 50,000/µL within 12 Weeks of beginning treatment up to 12 months was analyzed among all subjects who received active treatment in one of the prior studies (C788-047 or C788-048), in the current extension study (C788-049), or in both prior and current studies. Treated Population was defined as all enrolled and treated subjects.
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Placebo in 047/048 and Fostamatinib 049): Version 2
Time frame: Up to 12 months
A within-subject, between-study comparison of platelet counts for subjects who were previously treated with placebo in one of the prior studies (C788-047 or C788-048) was prospectively defined in the protocol (version 2). Achievement of platelet response by 12 weeks and maintenance of platelet response for 12 weeks was analyzed among subjects who had been randomized to placebo in one of the prior studies (C788-047 or C788-048). Treated Population was defined as all enrolled and treated subjects.
2 secondary outcomes
Duration of Platelet Response Based on Platelet Count and Rescue Medication
Time frame: Up to 12 months
Percentage of Subjects in Whom a Reduction in the Dose of Concomitant ITP Therapy Can be Achieved While Maintaining an Adequate Platelet Count
Time frame: Up to 12 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Dec 2023.
- 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.
55 sites in 15 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 55 sites in 15 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 11 Dec 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 55 sites in United Kingdom, United States, Poland, Australia, Bulgaria and 10 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 Dec 2023). 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?
Percentage of Subjects Who Achieved Platelet Count of at Least 50,000/µL Within 12 Weeks of Beginning Treatment up to 12 Months (Fostamatinib in 047/048 or 049):Version 1 (time frame: Up to 12 months). The study lists 2 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 NCT02077192, 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 11 Dec 2023.