Trial Outcomes & Findings for Effect of SPD422 on Platelet Lowering and Safety in Japanese Adults With At Risk Essential Thrombocythaemia (NCT NCT01214915)
NCT ID: NCT01214915
Last Updated: 2021-06-09
Results Overview
A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
53 participants
Primary outcome timeframe
12 months
Results posted on
2021-06-09
Participant Flow
Participant milestones
| Measure |
Anagrelide Hydrochloride
Subjects will be started at 1.0 mg per day orally and titrated as necessary.
|
|---|---|
|
Overall Study
STARTED
|
53
|
|
Overall Study
COMPLETED
|
42
|
|
Overall Study
NOT COMPLETED
|
11
|
Reasons for withdrawal
| Measure |
Anagrelide Hydrochloride
Subjects will be started at 1.0 mg per day orally and titrated as necessary.
|
|---|---|
|
Overall Study
Adverse Event
|
8
|
|
Overall Study
Lack of Efficacy
|
3
|
Baseline Characteristics
Effect of SPD422 on Platelet Lowering and Safety in Japanese Adults With At Risk Essential Thrombocythaemia
Baseline characteristics by cohort
| Measure |
Anagrelide Hydrochloride
n=53 Participants
|
|---|---|
|
Age, Continuous
|
61.6 Years
STANDARD_DEVIATION 13.1 • n=5 Participants
|
|
Age, Customized
< 50 years
|
12 Participants
n=5 Participants
|
|
Age, Customized
50 - 59 years
|
5 Participants
n=5 Participants
|
|
Age, Customized
60 - 69 years
|
18 Participants
n=5 Participants
|
|
Age, Customized
70 - 79 years
|
17 Participants
n=5 Participants
|
|
Age, Customized
>= 80 years
|
1 Participants
n=5 Participants
|
|
Sex: Female, Male
Female
|
30 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
23 Participants
n=5 Participants
|
|
Region of Enrollment
JAPAN
|
53 Participants
n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Outcome measures
| Measure |
Anagrelide Hydrochloride
n=53 Participants
Subjects will be started at 1.0 mg per day orally and titrated as necessary.
|
|---|---|
|
Percentage of Subjects Who Responded in Platelet Count
|
67.9 percentage of subjects
Interval 53.7 to 80.1
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
Subjects who achieved at least 50% reduction in platelet count from their baseline level across consecutive visits for at least 4 weeks and following 3 months of treatment.
Outcome measures
| Measure |
Anagrelide Hydrochloride
n=53 Participants
Subjects will be started at 1.0 mg per day orally and titrated as necessary.
|
|---|---|
|
Percentage of Subjects With at Least 50% Reduction in Platelet Count
|
50.9 percentage of subjects
Interval 36.8 to 64.9
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
Normalization was defined as platelet counts ≤400x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Outcome measures
| Measure |
Anagrelide Hydrochloride
n=53 Participants
Subjects will be started at 1.0 mg per day orally and titrated as necessary.
|
|---|---|
|
Percentage of Subjects With Normalization in Platelet Count
|
45.3 percentage of subjects
Interval 31.6 to 59.6
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Outcome measures
| Measure |
Anagrelide Hydrochloride
n=47 Participants
Subjects will be started at 1.0 mg per day orally and titrated as necessary.
|
|---|---|
|
Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was ≥600x10^9/L
|
68.1 percentage of subjects
Interval 52.9 to 80.9
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: Full Analysis Set defined as subjects who had taken at least 1 dose of study medication and had at least 1 post-baseline platelet measurement assessment.
A response was defined as platelet counts to \<600x10\^9/L across consecutive visits for at least 4 weeks following at least 3 months of treatment.
Outcome measures
| Measure |
Anagrelide Hydrochloride
n=6 Participants
Subjects will be started at 1.0 mg per day orally and titrated as necessary.
|
|---|---|
|
Percentage of Subjects Who Responded in Platelet Count Whose Baseline Platelet Count Was <600x10^9/L
|
66.7 percentage of subjects
Interval 22.3 to 95.7
|
Adverse Events
Anagrelide Hydrochloride
Serious events: 15 serious events
Other events: 53 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Anagrelide Hydrochloride
n=53 participants at risk
|
|---|---|
|
Blood and lymphatic system disorders
LEUKOCYTOSIS
|
1.9%
1/53 • Number of events 1
|
|
Blood and lymphatic system disorders
SPLENOMEGALY
|
1.9%
1/53 • Number of events 1
|
|
Cardiac disorders
PALPITATIONS
|
1.9%
1/53 • Number of events 1
|
|
Congenital, familial and genetic disorders
CYTOGENETIC ABNORMALITY
|
3.8%
2/53 • Number of events 2
|
|
Eye disorders
VISUAL IMPAIRMENT
|
1.9%
1/53 • Number of events 1
|
|
Gastrointestinal disorders
COLONIC POLYP
|
1.9%
1/53 • Number of events 1
|
|
Gastrointestinal disorders
GASTRIC ULCER
|
1.9%
1/53 • Number of events 1
|
|
Gastrointestinal disorders
MELAENA
|
1.9%
1/53 • Number of events 1
|
|
General disorders
OEDEMA
|
1.9%
1/53 • Number of events 1
|
|
General disorders
PYREXIA
|
1.9%
1/53 • Number of events 1
|
|
Infections and infestations
PNEUMONIA
|
3.8%
2/53 • Number of events 2
|
|
Infections and infestations
PYELONEPHRITIS
|
1.9%
1/53 • Number of events 1
|
|
Injury, poisoning and procedural complications
LACERATION
|
1.9%
1/53 • Number of events 1
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
ANGIOIMMUNOBLASTIC T-CELL LYMPHOMA
|
1.9%
1/53 • Number of events 1
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
PROSTATE CANCER
|
1.9%
1/53 • Number of events 1
|
|
Nervous system disorders
ALTERED STATE OF CONSCIOUSNESS
|
1.9%
1/53 • Number of events 1
|
|
Nervous system disorders
HEADACHE
|
1.9%
1/53 • Number of events 1
|
|
Renal and urinary disorders
HAEMATURIA
|
1.9%
1/53 • Number of events 1
|
|
Respiratory, thoracic and mediastinal disorders
INTERSTITIAL LUNG DISEASE
|
1.9%
1/53 • Number of events 1
|
|
Vascular disorders
CEREBRAL INFARCTION
|
3.8%
2/53 • Number of events 2
|
|
Vascular disorders
LACUNAR INFARCTION
|
1.9%
1/53 • Number of events 1
|
Other adverse events
| Measure |
Anagrelide Hydrochloride
n=53 participants at risk
|
|---|---|
|
Blood and lymphatic system disorders
ANAEMIA
|
47.2%
25/53 • Number of events 27
|
|
Blood and lymphatic system disorders
EOSINOPHILIA
|
5.7%
3/53 • Number of events 3
|
|
Blood and lymphatic system disorders
IRON DEFICIENCY ANAEMIA
|
7.5%
4/53 • Number of events 4
|
|
Cardiac disorders
PALPITATIONS
|
37.7%
20/53 • Number of events 23
|
|
Ear and labyrinth disorders
VERTIGO
|
7.5%
4/53 • Number of events 6
|
|
Gastrointestinal disorders
ABDOMINAL DISTENSION
|
7.5%
4/53 • Number of events 5
|
|
Gastrointestinal disorders
ABDOMINAL PAIN UPPER
|
9.4%
5/53 • Number of events 6
|
|
Gastrointestinal disorders
CONSTIPATION
|
5.7%
3/53 • Number of events 3
|
|
Gastrointestinal disorders
DENTAL CARIES
|
5.7%
3/53 • Number of events 3
|
|
Gastrointestinal disorders
DIARRHOEA
|
32.1%
17/53 • Number of events 20
|
|
Gastrointestinal disorders
EPIGASTRIC DISCOMFORT
|
5.7%
3/53 • Number of events 4
|
|
Gastrointestinal disorders
GASTRITIS
|
7.5%
4/53 • Number of events 8
|
|
Gastrointestinal disorders
GINGIVAL BLEEDING
|
13.2%
7/53 • Number of events 9
|
|
Gastrointestinal disorders
NAUSEA
|
9.4%
5/53 • Number of events 5
|
|
Gastrointestinal disorders
VOMITING
|
9.4%
5/53 • Number of events 6
|
|
General disorders
CHEST PAIN
|
5.7%
3/53 • Number of events 4
|
|
General disorders
FATIGUE
|
17.0%
9/53 • Number of events 12
|
|
General disorders
MALAISE
|
7.5%
4/53 • Number of events 4
|
|
General disorders
OEDEMA
|
5.7%
3/53 • Number of events 3
|
|
General disorders
OEDEMA PERIPHERAL
|
26.4%
14/53 • Number of events 17
|
|
General disorders
PYREXIA
|
17.0%
9/53 • Number of events 10
|
|
Hepatobiliary disorders
HEPATIC FUNCTION ABNORMAL
|
5.7%
3/53 • Number of events 3
|
|
Infections and infestations
BRONCHITIS
|
5.7%
3/53 • Number of events 3
|
|
Infections and infestations
CYSTITIS
|
5.7%
3/53 • Number of events 4
|
|
Infections and infestations
GASTROENTERITIS
|
5.7%
3/53 • Number of events 3
|
|
Infections and infestations
HERPES ZOSTER
|
5.7%
3/53 • Number of events 3
|
|
Infections and infestations
NASOPHARYNGITIS
|
22.6%
12/53 • Number of events 19
|
|
Infections and infestations
PHARYNGITIS
|
5.7%
3/53 • Number of events 3
|
|
Infections and infestations
UPPER RESPIRATORY TRACT INFECTION
|
9.4%
5/53 • Number of events 7
|
|
Injury, poisoning and procedural complications
CONTUSION
|
11.3%
6/53 • Number of events 7
|
|
Injury, poisoning and procedural complications
FALL
|
7.5%
4/53 • Number of events 4
|
|
Investigations
BLOOD ALKALINE PHOSPHATASE INCREASED
|
11.3%
6/53 • Number of events 6
|
|
Investigations
GAMMA-GLUTAMYLTRANSFERASE INCREASED
|
13.2%
7/53 • Number of events 8
|
|
Metabolism and nutrition disorders
DEHYDRATION
|
7.5%
4/53 • Number of events 4
|
|
Metabolism and nutrition disorders
HYPERURICAEMIA
|
5.7%
3/53 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
ARTHRALGIA
|
7.5%
4/53 • Number of events 4
|
|
Musculoskeletal and connective tissue disorders
BACK PAIN
|
11.3%
6/53 • Number of events 6
|
|
Musculoskeletal and connective tissue disorders
PAIN IN EXTREMITY
|
9.4%
5/53 • Number of events 5
|
|
Nervous system disorders
HEADACHE
|
45.3%
24/53 • Number of events 31
|
|
Nervous system disorders
HYPOAESTHESIA
|
11.3%
6/53 • Number of events 8
|
|
Renal and urinary disorders
POLLAKIURIA
|
5.7%
3/53 • Number of events 3
|
|
Respiratory, thoracic and mediastinal disorders
DYSPNOEA
|
11.3%
6/53 • Number of events 6
|
|
Respiratory, thoracic and mediastinal disorders
EPISTAXIS
|
11.3%
6/53 • Number of events 8
|
|
Skin and subcutaneous tissue disorders
DERMATITIS CONTACT
|
7.5%
4/53 • Number of events 4
|
|
Skin and subcutaneous tissue disorders
HAEMORRHAGE SUBCUTANEOUS
|
5.7%
3/53 • Number of events 3
|
|
Skin and subcutaneous tissue disorders
RASH
|
7.5%
4/53 • Number of events 4
|
|
Vascular disorders
HYPERTENSION
|
9.4%
5/53 • Number of events 5
|
Additional Information
Results disclosure agreements
- Principal investigator is a sponsor employee If a multicenter publication is not submitted within twelve (12) months after conclusion, abandonment or termination of the Study at all sites, or after Sponsor confirms there shall be no multicenter Study publication, the Institution and/or such Principal Investigator may publish the results from the Institution site individually.
- Publication restrictions are in place
Restriction type: OTHER