Trial Outcomes & Findings for Open Label Study of R788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP) (NCT NCT02077192)
NCT ID: NCT02077192
Last Updated: 2023-12-11
Results Overview
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.
COMPLETED
PHASE3
123 participants
Up to 12 months
2023-12-11
Participant Flow
A total of 124 subjects were screened from studies C788-047/ C788-048 for this extension study. Out of which, 123 subjects were enrolled and treated with study drug.
Participant milestones
| Measure |
Fostamatinib Disodium
Fostamatinib was self-administered twice daily (bid) by orally, once in the morning and once in the evening for up to 5 years or until commercial availability of fostamatinib for all subjects, whichever occurred first, in 2 dosage strengths: 100 milligram (mg) and 150 mg.
|
|---|---|
|
Overall Study
STARTED
|
123
|
|
Overall Study
COMPLETED
|
29
|
|
Overall Study
NOT COMPLETED
|
94
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
Open Label Study of R788 in the Treatment of Persistent/Chronic Immune Thrombocytopenic Purpura (ITP)
Baseline characteristics by cohort
| Measure |
Overall Study
n=123 Participants
Baseline characteristics are analyzed with treated population data analysis set which was defined as all enrolled and treated subjects.
|
|---|---|
|
Age, Continuous
|
52.0 years
n=39 Participants
|
|
Sex: Female, Male
Female
|
74 Participants
n=39 Participants
|
|
Sex: Female, Male
Male
|
49 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Hispanic or Latino
|
2 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Not Hispanic or Latino
|
121 Participants
n=39 Participants
|
|
Ethnicity (NIH/OMB)
Unknown or Not Reported
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
American Indian or Alaska Native
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Asian
|
4 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Native Hawaiian or Other Pacific Islander
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Black or African American
|
4 Participants
n=39 Participants
|
|
Race (NIH/OMB)
White
|
113 Participants
n=39 Participants
|
|
Race (NIH/OMB)
More than one race
|
0 Participants
n=39 Participants
|
|
Race (NIH/OMB)
Unknown or Not Reported
|
2 Participants
n=39 Participants
|
|
Region of Enrollment
Romania
|
1 Participants
n=39 Participants
|
|
Region of Enrollment
Hungary
|
2 Participants
n=39 Participants
|
|
Region of Enrollment
United States
|
17 Participants
n=39 Participants
|
|
Region of Enrollment
Czechia
|
10 Participants
n=39 Participants
|
|
Region of Enrollment
United Kingdom
|
18 Participants
n=39 Participants
|
|
Region of Enrollment
Spain
|
4 Participants
n=39 Participants
|
|
Region of Enrollment
Canada
|
4 Participants
n=39 Participants
|
|
Region of Enrollment
Austria
|
1 Participants
n=39 Participants
|
|
Region of Enrollment
Netherlands
|
1 Participants
n=39 Participants
|
|
Region of Enrollment
Norway
|
2 Participants
n=39 Participants
|
|
Region of Enrollment
Poland
|
34 Participants
n=39 Participants
|
|
Region of Enrollment
Denmark
|
1 Participants
n=39 Participants
|
|
Region of Enrollment
Italy
|
6 Participants
n=39 Participants
|
|
Region of Enrollment
Bulgaria
|
12 Participants
n=39 Participants
|
|
Region of Enrollment
Australia
|
10 Participants
n=39 Participants
|
PRIMARY outcome
Timeframe: Up to 12 monthsPercentage 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.
Outcome measures
| Measure |
Fostamatinib Disodium
n=123 Participants
Fostamatinib was self-administered twice daily (bid) by orally, once in the morning and once in the evening for up to 5 years or until commercial availability of fostamatinib for all subjects, whichever occurred first, in 2 dosage strengths: 100 milligram (mg) and 150 mg.
|
|---|---|
|
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
|
15.4 Percentage of Subjects
Interval 9.6 to 23.1
|
PRIMARY outcome
Timeframe: Up to 12 monthsA 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.
Outcome measures
| Measure |
Fostamatinib Disodium
n=123 Participants
Fostamatinib was self-administered twice daily (bid) by orally, once in the morning and once in the evening for up to 5 years or until commercial availability of fostamatinib for all subjects, whichever occurred first, in 2 dosage strengths: 100 milligram (mg) and 150 mg.
|
|---|---|
|
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
|
2.3 Percentage of Subjects
Interval 0.1 to 12.0
|
SECONDARY outcome
Timeframe: Up to 12 monthsThe 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.
Outcome measures
| Measure |
Fostamatinib Disodium
n=57 Participants
Fostamatinib was self-administered twice daily (bid) by orally, once in the morning and once in the evening for up to 5 years or until commercial availability of fostamatinib for all subjects, whichever occurred first, in 2 dosage strengths: 100 milligram (mg) and 150 mg.
|
|---|---|
|
Duration of Platelet Response Based on Platelet Count and Rescue Medication
|
127.0 Days
Interval 71.0 to 483.0
|
SECONDARY outcome
Timeframe: Up to 12 monthsPopulation: insufficient of number participants with events to perform the analysis
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.
Outcome measures
Outcome data not reported
Adverse Events
Fostamatinib Disodium
Serious adverse events
| Measure |
Fostamatinib Disodium
n=123 participants at risk
Fostamatinib was self-administered twice daily (bid) by orally, once in the morning and once in the evening for up to 5 years or until commercial availability of fostamatinib for all subjects, whichever occurred first, in 2 dosage strengths: 100 milligram (mg) and 150 mg.
|
|---|---|
|
Vascular disorders
Aortic stenosis
|
0.81%
1/123 • Baseline up to 62 months
|
|
General disorders
Chest pain
|
0.81%
1/123 • Baseline up to 62 months
|
|
General disorders
Mucosal haemorrhage
|
0.81%
1/123 • Baseline up to 62 months
|
|
General disorders
Pyrexia
|
0.81%
1/123 • Baseline up to 62 months
|
|
Reproductive system and breast disorders
Menorrhagia
|
0.81%
1/123 • Baseline up to 62 months
|
|
Injury, poisoning and procedural complications
Muscle rupture
|
0.81%
1/123 • Baseline up to 62 months
|
|
Injury, poisoning and procedural complications
Thoracic vertebral fracture
|
0.81%
1/123 • Baseline up to 62 months
|
|
Investigations
Transaminases increased
|
1.6%
2/123 • Baseline up to 62 months
|
|
Cardiac disorders
Atrial fibrillation
|
0.81%
1/123 • Baseline up to 62 months
|
|
Cardiac disorders
Cardiac failure congestive
|
0.81%
1/123 • Baseline up to 62 months
|
|
Cardiac disorders
Cardio-respiratory arrest
|
0.81%
1/123 • Baseline up to 62 months
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
6.5%
8/123 • Baseline up to 62 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
2.4%
3/123 • Baseline up to 62 months
|
|
Nervous system disorders
Headache
|
0.81%
1/123 • Baseline up to 62 months
|
|
Nervous system disorders
Loss of consciousness
|
0.81%
1/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
1.6%
2/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Ascites
|
0.81%
1/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Diarrhoea
|
0.81%
1/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Gastric antral vascular ectasia
|
0.81%
1/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Hemorrhoids
|
0.81%
1/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Hematochezia
|
0.81%
1/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Melaena
|
0.81%
1/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Mouth haemorrhage
|
0.81%
1/123 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Rectal haemorrhage
|
0.81%
1/123 • Baseline up to 62 months
|
|
Renal and urinary disorders
Nephrolithiasis
|
0.81%
1/123 • Baseline up to 62 months
|
|
Hepatobiliary disorders
Cholecystitis
|
0.81%
1/123 • Baseline up to 62 months
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
0.81%
1/123 • Baseline up to 62 months
|
|
Skin and subcutaneous tissue disorders
Purpura
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Sepsis
|
1.6%
2/123 • Baseline up to 62 months
|
|
Infections and infestations
Cellulitis staphylococcal
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Endocarditis bacterial
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Erythema induratum
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Gastroenteritis
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Mastoiditis
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Oral candidiasis
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Pneumonia
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Tuberculosis
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Upper respiratory tract infection
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Urinary tract infection
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Urosepsis
|
0.81%
1/123 • Baseline up to 62 months
|
|
Infections and infestations
Viral Infection
|
0.81%
1/123 • Baseline up to 62 months
|
|
Musculoskeletal and connective tissue disorders
Chondrocalcinosis pyrophosphate
|
0.81%
1/123 • Baseline up to 62 months
|
Other adverse events
| Measure |
Fostamatinib Disodium
n=123 participants at risk
Fostamatinib was self-administered twice daily (bid) by orally, once in the morning and once in the evening for up to 5 years or until commercial availability of fostamatinib for all subjects, whichever occurred first, in 2 dosage strengths: 100 milligram (mg) and 150 mg.
|
|---|---|
|
Vascular disorders
Hypertension
|
17.9%
22/123 • Number of events 32 • Baseline up to 62 months
|
|
Injury, poisoning and procedural complications
Contusion
|
9.8%
12/123 • Number of events 23 • Baseline up to 62 months
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
8.1%
10/123 • Number of events 19 • Baseline up to 62 months
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
15.4%
19/123 • Number of events 27 • Baseline up to 62 months
|
|
Nervous system disorders
Headache
|
12.2%
15/123 • Number of events 26 • Baseline up to 62 months
|
|
General disorders
Fatigue
|
8.1%
10/123 • Number of events 11 • Baseline up to 62 months
|
|
General disorders
Pyrexia
|
5.7%
7/123 • Number of events 13 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Diarrhoea
|
29.3%
36/123 • Number of events 62 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Vomiting
|
8.9%
11/123 • Number of events 14 • Baseline up to 62 months
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
15.4%
19/123 • Number of events 31 • Baseline up to 62 months
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
6.5%
8/123 • Number of events 8 • Baseline up to 62 months
|
|
Infections and infestations
Nasopharyngitis
|
7.3%
9/123 • Number of events 14 • Baseline up to 62 months
|
|
Infections and infestations
Upper respiratory tract infection
|
11.4%
14/123 • Number of events 20 • Baseline up to 62 months
|
|
Nervous system disorders
Dizziness
|
10.6%
13/123 • Number of events 13 • Baseline up to 62 months
|
|
Gastrointestinal disorders
Nausea
|
8.9%
11/123 • Number of events 12 • Baseline up to 62 months
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
8.1%
10/123 • Number of events 12 • Baseline up to 62 months
|
|
General disorders
Edema peripheral
|
5.7%
7/123 • Number of events 7 • Baseline up to 62 months
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.7%
7/123 • Number of events 7 • Baseline up to 62 months
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.7%
7/123 • Number of events 8 • Baseline up to 62 months
|
Additional Information
Director of Clinical Operations
Rigel Pharmaceuticals, Inc.
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place