Trial Outcomes & Findings for Study to Learn if 200mg Test Drug (Fostamatinib) Helps People With Large B-Cell Lymphoma,a Type of Blood Cancer (NCT NCT01499303)
NCT ID: NCT01499303
Last Updated: 2014-08-22
Results Overview
Patients were assessed using the revised response criteria for malignant lymphoma (Cheson). Patients were assessed for response, with CT and FDG-PET scans at 8 weeks, then every 12 weeks until radiological progression by clinical CT. Complete response (CR) was defined as disappearance of all target and non-target lesions in the liver and spleen and all lymph node masses regressed to normal size. Partial response (PR) was defined as ≥50% reduction in sum of the product of the diameters (SPD) for measured lymph nodes, splenic and liver lesions separately compared to baseline SPD. Objective response rate (CR + PR) analysis, exact binomial test, primary analysis.
COMPLETED
PHASE2
101 participants
Week 8
2014-08-22
Participant Flow
This study was conducted in the US and UK. Patient enrolment was completed on 14 June 2013. However three patients, considered by the Investigator to still be receiving clinical benefit, continue to receive fostamatinib. A total of 102 patients were enrolled and 68 received Fostamatinib doses of 100mg or 200mg and 38 Screen Failures.
Participant milestones
| Measure |
100mg BID
100mg Fostmatinib BID
|
200mg BID
200mg Fostmatinib BID
|
|---|---|---|
|
Overall Study
STARTED
|
21
|
47
|
|
Overall Study
COMPLETED
|
18
|
40
|
|
Overall Study
NOT COMPLETED
|
3
|
7
|
Reasons for withdrawal
| Measure |
100mg BID
100mg Fostmatinib BID
|
200mg BID
200mg Fostmatinib BID
|
|---|---|---|
|
Overall Study
Ongoing
|
2
|
1
|
|
Overall Study
Withdrawal by Subject
|
0
|
1
|
|
Overall Study
Adverse Event
|
1
|
4
|
|
Overall Study
Other
|
0
|
1
|
Baseline Characteristics
Study to Learn if 200mg Test Drug (Fostamatinib) Helps People With Large B-Cell Lymphoma,a Type of Blood Cancer
Baseline characteristics by cohort
| Measure |
100mg BID
n=21 Participants
100mg Fostmatinib BID
|
200mg BID
n=47 Participants
200mg Fostmatinib BID
|
Total
n=68 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
62.9 years
STANDARD_DEVIATION 12.38 • n=5 Participants
|
63.9 years
STANDARD_DEVIATION 12.78 • n=7 Participants
|
63.6 years
STANDARD_DEVIATION 12.57 • n=5 Participants
|
|
Sex: Female, Male
Female
|
9 Participants
n=5 Participants
|
12 Participants
n=7 Participants
|
21 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
12 Participants
n=5 Participants
|
35 Participants
n=7 Participants
|
47 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: Week 8Population: Full analysis set - all randomised patients
Patients were assessed using the revised response criteria for malignant lymphoma (Cheson). Patients were assessed for response, with CT and FDG-PET scans at 8 weeks, then every 12 weeks until radiological progression by clinical CT. Complete response (CR) was defined as disappearance of all target and non-target lesions in the liver and spleen and all lymph node masses regressed to normal size. Partial response (PR) was defined as ≥50% reduction in sum of the product of the diameters (SPD) for measured lymph nodes, splenic and liver lesions separately compared to baseline SPD. Objective response rate (CR + PR) analysis, exact binomial test, primary analysis.
Outcome measures
| Measure |
100mg BID
n=21 Participants
100mg Fostmatinib BID
|
200mg BID
n=47 Participants
200mg Fostmatinib BID
|
|---|---|---|
|
Objective Response Rate
|
2 Patients
Interval 1.17 to 30.38
|
0 Patients
Interval 0.0 to 7.55
|
Adverse Events
100mg BID
200mg BID
Serious adverse events
| Measure |
100mg BID
n=21 participants at risk
100mg Fostmatinib BID
|
200mg BID
n=47 participants at risk
200mg Fostmatinib BID
|
|---|---|---|
|
Infections and infestations
PNEUMONIA
|
0.00%
0/21
|
4.3%
2/47
|
|
Infections and infestations
CELLULITIS
|
0.00%
0/21
|
2.1%
1/47
|
|
Infections and infestations
CLOSTRIDIUM DIFFICILE INFECTION
|
0.00%
0/21
|
2.1%
1/47
|
|
Infections and infestations
NEUTROPENIC SEPSIS
|
4.8%
1/21
|
0.00%
0/47
|
|
Cardiac disorders
CARDIAC ARREST
|
0.00%
0/21
|
2.1%
1/47
|
|
Cardiac disorders
CARDIAC FAILURE CONGESTIVE
|
0.00%
0/21
|
2.1%
1/47
|
|
Cardiac disorders
SINUS TACHYCARDIA
|
0.00%
0/21
|
2.1%
1/47
|
|
Cardiac disorders
SUPRAVENTRICULAR TACHYCARDIA
|
0.00%
0/21
|
2.1%
1/47
|
|
General disorders
PYREXIA
|
4.8%
1/21
|
2.1%
1/47
|
|
General disorders
FATIGUE
|
0.00%
0/21
|
2.1%
1/47
|
|
Blood and lymphatic system disorders
ANAEMIA
|
0.00%
0/21
|
2.1%
1/47
|
|
Blood and lymphatic system disorders
PANCYTOPENIA
|
0.00%
0/21
|
2.1%
1/47
|
|
Metabolism and nutrition disorders
HYPONATRAEMIA
|
0.00%
0/21
|
2.1%
1/47
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
4.8%
1/21
|
0.00%
0/47
|
|
Nervous system disorders
CONVULSION
|
0.00%
0/21
|
2.1%
1/47
|
|
Nervous system disorders
SYNCOPE
|
0.00%
0/21
|
2.1%
1/47
|
|
Respiratory, thoracic and mediastinal disorders
PNEUMONITIS
|
0.00%
0/21
|
2.1%
1/47
|
Other adverse events
| Measure |
100mg BID
n=21 participants at risk
100mg Fostmatinib BID
|
200mg BID
n=47 participants at risk
200mg Fostmatinib BID
|
|---|---|---|
|
Gastrointestinal disorders
DIARRHOEA
|
14.3%
3/21
|
34.0%
16/47
|
|
Gastrointestinal disorders
CONSTIPATION
|
19.0%
4/21
|
25.5%
12/47
|
|
Gastrointestinal disorders
NAUSEA
|
19.0%
4/21
|
25.5%
12/47
|
|
Gastrointestinal disorders
VOMITING
|
0.00%
0/21
|
19.1%
9/47
|
|
General disorders
FATIGUE
|
19.0%
4/21
|
27.7%
13/47
|
|
General disorders
PYREXIA
|
19.0%
4/21
|
21.3%
10/47
|
|
Investigations
ASPARTATE AMINOTRANSFERASE INCREASED
|
19.0%
4/21
|
17.0%
8/47
|
|
Investigations
BLOOD ALKALINE PHOSPHATASE INCREASED
|
23.8%
5/21
|
10.6%
5/47
|
|
Investigations
BLOOD CREATININE INCREASED
|
14.3%
3/21
|
6.4%
3/47
|
|
Investigations
ALANINE AMINOTRANSFERASE INCREASED
|
14.3%
3/21
|
4.3%
2/47
|
|
Investigations
BLOOD BILIRUBIN INCREASED
|
14.3%
3/21
|
0.00%
0/47
|
|
Blood and lymphatic system disorders
ANAEMIA
|
9.5%
2/21
|
25.5%
12/47
|
|
Blood and lymphatic system disorders
THROMBOCYTOPENIA
|
23.8%
5/21
|
14.9%
7/47
|
|
Blood and lymphatic system disorders
NEUTROPENIA
|
23.8%
5/21
|
10.6%
5/47
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
14.3%
3/21
|
8.5%
4/47
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
19.0%
4/21
|
4.3%
2/47
|
|
Vascular disorders
HYPERTENSION
|
4.8%
1/21
|
19.1%
9/47
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
19.0%
4/21
|
10.6%
5/47
|
|
Nervous system disorders
HEADACHE
|
9.5%
2/21
|
10.6%
5/47
|
|
Metabolism and nutrition disorders
HYPONATRAEMIA
|
4.8%
1/21
|
10.6%
5/47
|
|
Metabolism and nutrition disorders
DECREASED APPETITE
|
0.00%
0/21
|
10.6%
5/47
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
4.8%
1/21
|
8.5%
4/47
|
|
Gastrointestinal disorders
ABDOMINAL DISTENSION
|
9.5%
2/21
|
4.3%
2/47
|
|
Gastrointestinal disorders
DYSPEPSIA
|
0.00%
0/21
|
8.5%
4/47
|
|
General disorders
OEDEMA PERIPHERAL
|
9.5%
2/21
|
6.4%
3/47
|
|
General disorders
ASTHENIA
|
0.00%
0/21
|
6.4%
3/47
|
|
Investigations
BLOOD LACTATE DEHYDROGENASE INCREASED
|
4.8%
1/21
|
8.5%
4/47
|
|
Investigations
WHITE BLOOD CELL COUNT DECREASED
|
4.8%
1/21
|
6.4%
3/47
|
|
Investigations
BLOOD UREA INCREASED
|
9.5%
2/21
|
2.1%
1/47
|
|
Investigations
NEUTROPHIL COUNT DECREASED
|
9.5%
2/21
|
0.00%
0/47
|
|
Vascular disorders
HYPOTENSION
|
9.5%
2/21
|
6.4%
3/47
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
9.5%
2/21
|
4.3%
2/47
|
|
Musculoskeletal and connective tissue disorders
MYALGIA
|
4.8%
1/21
|
6.4%
3/47
|
|
Nervous system disorders
SYNCOPE
|
0.00%
0/21
|
6.4%
3/47
|
|
Metabolism and nutrition disorders
HYPOKALAEMIA
|
0.00%
0/21
|
8.5%
4/47
|
|
Skin and subcutaneous tissue disorders
NIGHT SWEATS
|
0.00%
0/21
|
8.5%
4/47
|
|
Infections and infestations
PNEUMONIA
|
0.00%
0/21
|
6.4%
3/47
|
|
Psychiatric disorders
ANXIETY
|
9.5%
2/21
|
8.5%
4/47
|
|
Psychiatric disorders
CONFUSIONAL STATE
|
0.00%
0/21
|
6.4%
3/47
|
|
Renal and urinary disorders
RENAL FAILURE ACUTE
|
0.00%
0/21
|
8.5%
4/47
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place