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A Efficacy and Safety Study of R935788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
Phase 3 trial testing Fostamatinib disodium in Immune Thrombocytopenic Purpura in 76 participants. Completed in 21 April 2016.
| Lead sponsor | Rigel Pharmaceuticals |
|---|---|
| Phase | Phase 3 |
| Status | Completed |
| Study type | INTERVENTIONAL |
| Allocation | randomized |
| Design | parallel |
| Masking | quadruple |
| Primary purpose | treatment |
| Enrollment | 76 |
| Start date | 14 July 2014 |
| Primary completion | 21 April 2016 |
| Estimated completion | 21 April 2016 |
| Sites | 52 locations across Denmark, Italy, Netherlands, United Kingdom, Hungary, Canada, Australia, United States |
Rigel Pharmaceuticals — full company profile →
18 and older, any sex, with Immune Thrombocytopenic Purpura. Patients with the condition only — healthy volunteers not accepted.
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
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
| Group | Value | 95% CI |
|---|---|---|
| Fostamatinib Recipient | 9 | |
| Placebo Recipient | 0 |
Platelet Count ≥ 50,000/µL at Week 12
| Group | Value | 95% CI |
|---|---|---|
| Fostamatinib Recipient | 11 | |
| Placebo Recipient | 0 |
Platelet Count ≥ 50,000/µL at Week 24
| Group | Value | 95% CI |
|---|---|---|
| Fostamatinib Recipient | 8 | |
| Placebo Recipient | 0 |
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.
| Group | Value | 95% CI |
|---|---|---|
| Fostamatinib Recipient | 4 | |
| Placebo Recipient | 0 |
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.
| Group | Value | 95% CI |
|---|---|---|
| Fostamatinib Recipient | 4 | |
| Placebo Recipient | 0 |
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
| Group | Value | 95% CI |
|---|---|---|
| Fostamatinib Recipient | 0.13 | ± 0.12 |
| Placebo Recipient | 0.14 | ± 0.10 |
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 metho
| Group | Value | 95% CI |
|---|---|---|
| Fostamatinib Recipient | 0.61 | ± 0.66 |
| Placebo Recipient | 0.46 | ± 0.56 |
Time frame: 24 Weeks. Reporting threshold: 5%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.
| Reaction | System | Fostamatinib Recipient | Placebo Recipient |
|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | — | — |
| Febrile Neutropenia | Blood and lymphatic system disorders | — | — |
| Immune Thrombocytopenic Purpura | Blood and lymphatic system disorders | — | — |
| Thrombocytopenia | Blood and lymphatic system disorders | — | — |
| Cardiac Failure Congestive | Cardiac disorders | — | — |
| Retinal Tear | Eye disorders | — | — |
| Diarrhoea | Gastrointestinal disorders | — | — |
| Gastrointestinal Haemorrhage | Gastrointestinal disorders | — | — |
| Pneumonia | Infections and infestations | — | — |
| Sepsis | Infections and infestations | — | — |
| Syncope | Nervous system disorders | — | — |
| Menorrhagia | Reproductive system and breast disorders | — | — |
| Vaginal Haemorrhage | Reproductive system and breast disorders | — | — |
| Chronic Obstructive Pulmonary Disease | Respiratory, thoracic and mediastinal disorders | — | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — | — |
| Reaction | System | Fostamatinib Recipient | Placebo Recipient |
|---|---|---|---|
| Diarrhoea | Gastrointestinal disorders | — | — |
| Nausea | Gastrointestinal disorders | — | — |
| Hypertension | Vascular disorders | — | — |
| Alanine aminotransferase increased | Investigations | — | — |
| Dizziness | Nervous system disorders | — | — |
| Epistaxis | Respiratory, thoracic and mediastinal disorders | — | — |
| Aspartate aminotransferase increased | Investigations | — | — |
| Headache | Nervous system disorders | — | — |
| Fatigue | General disorders | — | — |
| Upper respiratory tract infection | Infections and infestations | — | — |
| Dysgeusia | Nervous system disorders | — | — |
| Chest pain | General disorders | — | — |
| Rash | Vascular disorders | — | — |
| Constipation | Gastrointestinal disorders | — | — |
| Abdominal pain | Gastrointestinal disorders | — | — |
| Flatulence | Gastrointestinal disorders | — | — |
| Blood pressure increased | Investigations | — | — |
| Dyspnoea | Respiratory, thoracic and mediastinal disorders | — | — |
| Urinary tract infection | Infections and infestations | — | — |
| Contusion | Injury, poisoning and procedural complications | — | — |
| Vomiting | Gastrointestinal disorders | — | — |
| Rectal haemorrhage | Gastrointestinal disorders | — | — |
| Oropharyngeal pain | Respiratory, thoracic and mediastinal disorders | — | — |
| Pyrexia | General disorders | — | — |
| Musculoskeletal pain | Musculoskeletal and connective tissue disorders | — | — |
| Anaemia | Blood and lymphatic system disorders | — | — |
Most-reported serious reactions: Anaemia, Febrile Neutropenia, Immune Thrombocytopenic Purpura, Thrombocytopenia, Cardiac Failure Congestive, Retinal Tear, Diarrhoea, Gastrointestinal Haemorrhage.
Data from ClinicalTrials.gov NCT02076399 adverse events section.
The purpose of this study is to determine whether fostamatinib is safe and effective in the treatment of persistent/chronic Immune Thrombocytopenic Purpura (ITP).
8 peer-reviewed publications reference this trial (live from Europe PMC):
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