Trial Outcomes & Findings for Pilot Study of Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP) (NCT NCT00706342)
NCT ID: NCT00706342
Last Updated: 2016-06-03
Results Overview
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
18 participants
Primary outcome timeframe
2 weeks
Results posted on
2016-06-03
Participant Flow
Participant milestones
| Measure |
R788 PO
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Overall Study
STARTED
|
18
|
|
Overall Study
COMPLETED
|
0
|
|
Overall Study
NOT COMPLETED
|
18
|
Reasons for withdrawal
| Measure |
R788 PO
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Not responding to treatment
|
2
|
|
Overall Study
Withdrawn due to failure to respond
|
1
|
|
Overall Study
Death
|
1
|
|
Overall Study
Withdrew from the study on his own.
|
1
|
|
Overall Study
Investigator discretion
|
2
|
|
Overall Study
Non-responder Week 12
|
1
|
|
Overall Study
Ongoing
|
6
|
|
Overall Study
WIthdrawal of consent
|
1
|
Baseline Characteristics
Pilot Study of Fostamatinib Disodium/R935788 for the Treatment of Adult Refractory Immune Thrombocytopenic Purpura (ITP)
Baseline characteristics by cohort
| Measure |
R788 PO
n=18 Participants
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Age, Continuous
|
61.9 years
STANDARD_DEVIATION 15.63 • n=5 Participants
|
|
Age, Continuous
Median (Range)
|
65 years
n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
8 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 2 weeksPopulation: Number of participants analyzed refers to the number of patients available at this timepoint for analysis.
Outcome measures
| Measure |
R788 PO
n=17 Participants
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Week 2
|
8 Participants
|
PRIMARY outcome
Timeframe: 6 WeeksPopulation: Number of participants analyzed refers to the number of patients available at this timepoint for analysis.
Outcome measures
| Measure |
R788 PO
n=15 Participants
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Week 6
|
5 Participants
|
PRIMARY outcome
Timeframe: 12 WeeksPopulation: Number of participants analyzed refers to the number of patients available at this timepoint for analysis.
Outcome measures
| Measure |
R788 PO
n=10 Participants
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Week 12
|
5 Participants
|
PRIMARY outcome
Timeframe: 24 WeeksPopulation: Number of participants analyzed refers to the number of patients available at this timepoint for analysis.
Outcome measures
| Measure |
R788 PO
n=7 Participants
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Week 24
|
4 Participants
|
PRIMARY outcome
Timeframe: 12 MonthsPopulation: Number of participants analyzed refers to the number of patients available at this timepoint for analysis.
Outcome measures
| Measure |
R788 PO
n=10 Participants
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Month 12
|
5 Participants
|
PRIMARY outcome
Timeframe: 24 MonthsPopulation: Number of participants analyzed refers to the number of patients available at this timepoint for analysis.
Outcome measures
| Measure |
R788 PO
n=7 Participants
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Summary of Patients Whose Platelet Count Increased by at Least 20,000/mm3 From Baseline to a Total of 30,000/mm3 or More - Month 24
|
2 Participants
|
Adverse Events
R788 PO
Serious events: 8 serious events
Other events: 18 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
R788 PO
n=18 participants at risk
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Gastrointestinal disorders
Diarrhea
|
11.1%
2/18
|
|
Gastrointestinal disorders
Nausea
|
11.1%
2/18
|
|
Gastrointestinal disorders
Vomiting
|
11.1%
2/18
|
|
Gastrointestinal disorders
Gastrointestinal hemorrhage
|
5.6%
1/18
|
|
Gastrointestinal disorders
Gingival bleeding
|
5.6%
1/18
|
|
Gastrointestinal disorders
Mouth hemorrhage
|
5.6%
1/18
|
|
Gastrointestinal disorders
Rectal hemorrhage
|
5.6%
1/18
|
|
Cardiac disorders
Cardiac failure congestive
|
5.6%
1/18
|
|
Cardiac disorders
Palpitations
|
5.6%
1/18
|
|
Infections and infestations
Pneumonia
|
5.6%
1/18
|
|
Infections and infestations
Urinary tract infection
|
5.6%
1/18
|
|
Nervous system disorders
Anoxic encephalopathy
|
5.6%
1/18
|
|
Nervous system disorders
Dizziness
|
5.6%
1/18
|
|
Metabolism and nutrition disorders
Dehydration
|
5.6%
1/18
|
|
Pregnancy, puerperium and perinatal conditions
Abortion spontaneous
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
5.6%
1/18
|
|
Vascular disorders
Deep vein thrombosis
|
5.6%
1/18
|
Other adverse events
| Measure |
R788 PO
n=18 participants at risk
Tablet containing 75mg BID to 225mg BID R788.
|
|---|---|
|
Investigations
Blood glucose increased
|
72.2%
13/18
|
|
Investigations
Blood pressure increased
|
72.2%
13/18
|
|
Investigations
Aspartate aminotransferase increased
|
61.1%
11/18
|
|
Investigations
Blood glucose decreased
|
61.1%
11/18
|
|
Investigations
Blood alkaline phosphatase increased
|
44.4%
8/18
|
|
Investigations
Alanine aminotransferase increased
|
38.9%
7/18
|
|
Investigations
Blood lactate dehydrogenase increased
|
33.3%
6/18
|
|
Investigations
Blood cholesterol increased
|
27.8%
5/18
|
|
Investigations
Protein total decreased
|
27.8%
5/18
|
|
Investigations
BLOOD BILIRUBIN DECREASED
|
16.7%
3/18
|
|
Investigations
BLOOD BILIRUBIN INCREASED
|
16.7%
3/18
|
|
Investigations
BLOOD CALCIUM DECREASED
|
16.7%
3/18
|
|
Investigations
BLOOD POTASSIUM DECREASED
|
16.7%
3/18
|
|
Investigations
BLOOD UREA INCREASED
|
16.7%
3/18
|
|
Investigations
HEART RATE INCREASED
|
16.7%
3/18
|
|
Investigations
ANION GAP INCREASED
|
11.1%
2/18
|
|
Investigations
BILIRUBIN CONJUGATED
|
11.1%
2/18
|
|
Investigations
BLOOD CALCIUM INCREASED
|
11.1%
2/18
|
|
Investigations
BLOOD CHLORIDE DECREASED
|
11.1%
2/18
|
|
Investigations
BLOOD CREATININE INCREASED
|
11.1%
2/18
|
|
Investigations
BLOOD MAGNESIUM DECREASED
|
11.1%
2/18
|
|
Investigations
BLOOD SODIUM DECREASED
|
11.1%
2/18
|
|
Investigations
GLOBULINS INCREASED
|
11.1%
2/18
|
|
Investigations
WEIGHT INCREASED
|
11.1%
2/18
|
|
Investigations
BILIRUBIN CONJUGATED INCREASED
|
5.6%
1/18
|
|
Investigations
BLOOD ALBUMIN DECREASED
|
5.6%
1/18
|
|
Investigations
BLOOD BILIRUBIN UNCONJUGATED INCREASED
|
5.6%
1/18
|
|
Investigations
BLOOD CREATININE DECREASED
|
5.6%
1/18
|
|
Investigations
BLOOD MAGNESIUM INCREASED
|
5.6%
1/18
|
|
Investigations
BLOOD PHOSPHORUS DECREASED
|
5.6%
1/18
|
|
Investigations
BLOOD PHOSPHORUS INCREASED
|
5.6%
1/18
|
|
Investigations
BLOOD POTASSIUM INCREASED
|
5.6%
1/18
|
|
Investigations
BLOOD SODIUM INCREASED
|
5.6%
1/18
|
|
Investigations
BLOOD UREA DECREASED
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
ECCHYMOSIS
|
72.2%
13/18
|
|
Skin and subcutaneous tissue disorders
PETECHIAE
|
66.7%
12/18
|
|
Skin and subcutaneous tissue disorders
RASH
|
33.3%
6/18
|
|
Skin and subcutaneous tissue disorders
BLOOD BLISTER
|
22.2%
4/18
|
|
Skin and subcutaneous tissue disorders
PRURITUS
|
11.1%
2/18
|
|
Skin and subcutaneous tissue disorders
SKIN HAEMORRHAGE
|
11.1%
2/18
|
|
Skin and subcutaneous tissue disorders
URTICARIA
|
11.1%
2/18
|
|
Skin and subcutaneous tissue disorders
DRY SKIN
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
ERYTHEMA
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
INCREASED TENDENCY TO BRUISE
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
PURPURA
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
RASH PAPULAR
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
RASH PRURITIC
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
SKIN DISCOLOURATION
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
SKIN EXFOLIATION
|
5.6%
1/18
|
|
Skin and subcutaneous tissue disorders
SKIN LESION
|
5.6%
1/18
|
|
Gastrointestinal disorders
DIARRHOEA
|
66.7%
12/18
|
|
Gastrointestinal disorders
NAUSEA
|
38.9%
7/18
|
|
Gastrointestinal disorders
GINGIVAL BLEEDING
|
27.8%
5/18
|
|
Gastrointestinal disorders
STOMATITIS
|
27.8%
5/18
|
|
Gastrointestinal disorders
VOMITING
|
27.8%
5/18
|
|
Gastrointestinal disorders
HAEMORRHOIDS
|
16.7%
3/18
|
|
Gastrointestinal disorders
ORAL PAIN
|
16.7%
3/18
|
|
Gastrointestinal disorders
RECTAL HAEMORRHAGE
|
16.7%
3/18
|
|
Gastrointestinal disorders
ABDOMINAL PAIN
|
11.1%
2/18
|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
11.1%
2/18
|
|
Gastrointestinal disorders
CONSTIPATION
|
11.1%
2/18
|
|
Gastrointestinal disorders
MOUTH HAEMORRHAGE
|
11.1%
2/18
|
|
Gastrointestinal disorders
ABDOMINAL PAIN LOWER
|
5.6%
1/18
|
|
Gastrointestinal disorders
DIVERTICULUM
|
5.6%
1/18
|
|
Gastrointestinal disorders
DYSPEPSIA
|
5.6%
1/18
|
|
Gastrointestinal disorders
FAECAL INCONTINENCE
|
5.6%
1/18
|
|
Gastrointestinal disorders
FAECES DISCOLOURED
|
5.6%
1/18
|
|
Gastrointestinal disorders
FREQUENT BOWEL MOVEMENTS
|
5.6%
1/18
|
|
Gastrointestinal disorders
GASTRITIS
|
5.6%
1/18
|
|
Gastrointestinal disorders
GASTROINTESTINAL HAEMORRHAGE
|
5.6%
1/18
|
|
Gastrointestinal disorders
GASTROOESOPHAGEAL REFLUX DISEASE
|
5.6%
1/18
|
|
Gastrointestinal disorders
HAEMATOCHEZIA
|
5.6%
1/18
|
|
Gastrointestinal disorders
TONGUE BLISTERING
|
5.6%
1/18
|
|
Gastrointestinal disorders
TONGUE DISORDER
|
5.6%
1/18
|
|
Respiratory, thoracic and mediastinal disorders
EPISTAXIS
|
50.0%
9/18
|
|
Respiratory, thoracic and mediastinal disorders
COUGH
|
27.8%
5/18
|
|
Respiratory, thoracic and mediastinal disorders
OROPHARYNGEAL PAIN
|
27.8%
5/18
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
16.7%
3/18
|
|
Respiratory, thoracic and mediastinal disorders
NASAL CONGESTION
|
16.7%
3/18
|
|
Respiratory, thoracic and mediastinal disorders
RHINORRHOEA
|
16.7%
3/18
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA EXERTIONAL
|
5.6%
1/18
|
|
Respiratory, thoracic and mediastinal disorders
POSTNASAL DRIP
|
5.6%
1/18
|
|
Respiratory, thoracic and mediastinal disorders
RESPIRATORY TRACT CONGESTION
|
5.6%
1/18
|
|
Respiratory, thoracic and mediastinal disorders
SNEEZING
|
5.6%
1/18
|
|
Nervous system disorders
HEADACHE
|
44.4%
8/18
|
|
Nervous system disorders
DIZZINESS
|
16.7%
3/18
|
|
Nervous system disorders
SOMNOLENCE
|
16.7%
3/18
|
|
Nervous system disorders
ANOXIC ENCEPHALOPATHY
|
5.6%
1/18
|
|
Nervous system disorders
MIGRAINE
|
5.6%
1/18
|
|
Nervous system disorders
MIGRAINE WITH AURA
|
5.6%
1/18
|
|
General disorders
FATIGUE
|
38.9%
7/18
|
|
General disorders
PYREXIA
|
27.8%
5/18
|
|
General disorders
OEDEMA PERIPHERAL
|
22.2%
4/18
|
|
General disorders
PAIN
|
16.7%
3/18
|
|
General disorders
CHEST PAIN
|
11.1%
2/18
|
|
General disorders
AXILLARY PAIN
|
5.6%
1/18
|
|
General disorders
CATHETER SITE INFLAMMATION
|
5.6%
1/18
|
|
General disorders
FACIAL PAIN
|
5.6%
1/18
|
|
General disorders
INFLAMMATION
|
5.6%
1/18
|
|
General disorders
INFLUENZA LIKE ILLNESS
|
5.6%
1/18
|
|
General disorders
LOCAL SWELLING
|
5.6%
1/18
|
|
General disorders
MEDICAL DEVICE COMPLICATION
|
5.6%
1/18
|
|
General disorders
MUCOSAL HAEMORRHAGE
|
5.6%
1/18
|
|
Infections and infestations
NASOPHARYNGITIS
|
27.8%
5/18
|
|
Infections and infestations
HERPES ZOSTER
|
11.1%
2/18
|
|
Infections and infestations
INFLUENZA
|
11.1%
2/18
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
11.1%
2/18
|
|
Infections and infestations
BREAST ABSCESS
|
5.6%
1/18
|
|
Infections and infestations
BRONCHITIS
|
5.6%
1/18
|
|
Infections and infestations
GASTROENTERITIS
|
5.6%
1/18
|
|
Infections and infestations
LOWER RESPIRATORY TRACT INFECTION
|
5.6%
1/18
|
|
Infections and infestations
OSTEOMYELITIS
|
5.6%
1/18
|
|
Infections and infestations
PNEUMONIA
|
5.6%
1/18
|
|
Infections and infestations
SINUSITIS
|
5.6%
1/18
|
|
Infections and infestations
TOOTH ABSCESS
|
5.6%
1/18
|
|
Infections and infestations
TOOTH INFECTION
|
5.6%
1/18
|
|
Infections and infestations
URINARY TRACT INFECTION
|
5.6%
1/18
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
22.2%
4/18
|
|
Musculoskeletal and connective tissue disorders
MUSCLE SPASMS
|
22.2%
4/18
|
|
Musculoskeletal and connective tissue disorders
MUSCULOSKELETAL STIFFNESS
|
16.7%
3/18
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
16.7%
3/18
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
11.1%
2/18
|
|
Musculoskeletal and connective tissue disorders
ARTHRITIS
|
5.6%
1/18
|
|
Musculoskeletal and connective tissue disorders
MUSCULOSKELETAL PAIN
|
5.6%
1/18
|
|
Musculoskeletal and connective tissue disorders
NECK PAIN
|
5.6%
1/18
|
|
Metabolism and nutrition disorders
GOUT
|
11.1%
2/18
|
|
Metabolism and nutrition disorders
DECREASED APPETITE
|
5.6%
1/18
|
|
Metabolism and nutrition disorders
DEHYDRATION
|
5.6%
1/18
|
|
Metabolism and nutrition disorders
HYPERKALAEMIA
|
5.6%
1/18
|
|
Metabolism and nutrition disorders
HYPERLIPIDAEMIA
|
5.6%
1/18
|
|
Metabolism and nutrition disorders
IRON DEFICIENCY
|
5.6%
1/18
|
|
Metabolism and nutrition disorders
VITAMIN D DEFICIENCY
|
5.6%
1/18
|
|
Vascular disorders
HYPERTENSION
|
16.7%
3/18
|
|
Vascular disorders
HAEMORRHAGE
|
11.1%
2/18
|
|
Vascular disorders
THROMBOSIS
|
11.1%
2/18
|
|
Vascular disorders
DEEP VEIN THROMBOSIS
|
5.6%
1/18
|
|
Vascular disorders
HOT FLUSH
|
5.6%
1/18
|
|
Vascular disorders
VENOUS INSUFFICIENCY
|
5.6%
1/18
|
|
Injury, poisoning and procedural complications
CONTUSION
|
22.2%
4/18
|
|
Injury, poisoning and procedural complications
SCRATCH
|
5.6%
1/18
|
|
Cardiac disorders
PALPITATIONS
|
11.1%
2/18
|
|
Cardiac disorders
ATRIAL FIBRILLATION
|
5.6%
1/18
|
|
Cardiac disorders
CARDIAC FAILURE CONGESTIVE
|
5.6%
1/18
|
|
Cardiac disorders
LEFT VENTRICULAR DYSFUNCTION
|
5.6%
1/18
|
|
Eye disorders
EYE HAEMORRHAGE
|
11.1%
2/18
|
|
Eye disorders
VISION BLURRED
|
11.1%
2/18
|
|
Eye disorders
CATARACT
|
5.6%
1/18
|
|
Eye disorders
DRY EYE
|
5.6%
1/18
|
|
Eye disorders
EYE PAIN
|
5.6%
1/18
|
|
Eye disorders
OCULAR HYPERAEMIA
|
5.6%
1/18
|
|
Ear and labyrinth disorders
EAR PAIN
|
11.1%
2/18
|
|
Ear and labyrinth disorders
DEAFNESS
|
5.6%
1/18
|
|
Ear and labyrinth disorders
EAR HAEMORRHAGE
|
5.6%
1/18
|
|
Ear and labyrinth disorders
HYPOACUSIS
|
5.6%
1/18
|
|
Ear and labyrinth disorders
VERTIGO
|
5.6%
1/18
|
|
Reproductive system and breast disorders
PROSTATITIS
|
5.6%
1/18
|
|
Reproductive system and breast disorders
VAGINAL HAEMORRHAGE
|
5.6%
1/18
|
|
Reproductive system and breast disorders
BREAST PAIN
|
5.6%
1/18
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
BENIGN GASTROINTESTINAL NEOPLASM
|
5.6%
1/18
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
HAEMANGIOMA
|
5.6%
1/18
|
|
Psychiatric disorders
ANXIETY
|
5.6%
1/18
|
|
Psychiatric disorders
DEPRESSION
|
5.6%
1/18
|
|
Renal and urinary disorders
DYSURIA
|
5.6%
1/18
|
|
Renal and urinary disorders
POLLAKIURIA
|
5.6%
1/18
|
|
Renal and urinary disorders
URINE ABNORMALITY
|
5.6%
1/18
|
|
Blood and lymphatic system disorders
LYMPHADENOPATHY
|
5.6%
1/18
|
|
Endocrine disorders
AUTOIMMUNE THYROIDITIS
|
5.6%
1/18
|
|
Immune system disorders
HYPERSENSITIVITY
|
5.6%
1/18
|
|
Pregnancy, puerperium and perinatal conditions
ABORTION SPONTANEOUS
|
5.6%
1/18
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee
- Publication restrictions are in place
Restriction type: LTE60