Trial Outcomes & Findings for P3 Study to Evaluate Efficacy and Safety of AMG 531 in Thrombocytopenic Japanese Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (NCT NCT00603642)
NCT ID: NCT00603642
Last Updated: 2022-11-08
Results Overview
Number of weeks with weekly platelet response. A weekly platelet response is defined as a platelet count of ≥ 50 x 10\^9/L on a weekly scheduled dose day from week 2 to week 13.
Recruitment status
COMPLETED
Study phase
PHASE3
Target enrollment
34 participants
Primary outcome timeframe
12 weeks (Weeks 2 - 13)
Results posted on
2022-11-08
Participant Flow
Participants were enrolled from 20 November 2007 through 11 December 2008
Participant milestones
| Measure |
Placebo
Placebo administered subcutaneously once weekly for 12 weeks
|
Romiplostim
Romiplostim administered subcutaneously once weekly for 12 weeks at a starting dose of 3 µg/kg
|
|---|---|---|
|
Overall Study
STARTED
|
12
|
22
|
|
Overall Study
COMPLETED
|
12
|
22
|
|
Overall Study
NOT COMPLETED
|
0
|
0
|
Reasons for withdrawal
Withdrawal data not reported
Baseline Characteristics
P3 Study to Evaluate Efficacy and Safety of AMG 531 in Thrombocytopenic Japanese Subjects With Immune (Idiopathic) Thrombocytopenic Purpura
Baseline characteristics by cohort
| Measure |
Placebo
n=12 Participants
Placebo administered subcutaneously once weekly for 12 weeks
|
Romiplostim
n=22 Participants
Romiplostim administered subcutaneously once weekly for 12 weeks at a starting dose of 3 µg/kg
|
Total
n=34 Participants
Total of all reporting groups
|
|---|---|---|---|
|
Age, Continuous
|
47.6 Years
STANDARD_DEVIATION 13.4 • n=5 Participants
|
58.5 Years
STANDARD_DEVIATION 12.6 • n=7 Participants
|
54.7 Years
STANDARD_DEVIATION 13.7 • n=5 Participants
|
|
Sex: Female, Male
Female
|
10 Participants
n=5 Participants
|
14 Participants
n=7 Participants
|
24 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
2 Participants
n=5 Participants
|
8 Participants
n=7 Participants
|
10 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Japanese
|
12 Participants
n=5 Participants
|
22 Participants
n=7 Participants
|
34 Participants
n=5 Participants
|
|
Race/Ethnicity, Customized
Other
|
0 Participants
n=5 Participants
|
0 Participants
n=7 Participants
|
0 Participants
n=5 Participants
|
|
Platelet Count
|
15.8 10^9/L
STANDARD_DEVIATION 8.6 • n=5 Participants
|
18.4 10^9/L
STANDARD_DEVIATION 8.3 • n=7 Participants
|
17.5 10^9/L
STANDARD_DEVIATION 8.4 • n=5 Participants
|
PRIMARY outcome
Timeframe: 12 weeks (Weeks 2 - 13)Population: Full Analysis Set, composed of all randomized participants who received at least 1 dose of romiplostim or placebo
Number of weeks with weekly platelet response. A weekly platelet response is defined as a platelet count of ≥ 50 x 10\^9/L on a weekly scheduled dose day from week 2 to week 13.
Outcome measures
| Measure |
Placebo
n=12 Participants
Placebo administered subcutaneously once weekly for 12 weeks
|
Romiplostim
n=22 Participants
Romiplostim administered subcutaneously once weekly for 12 weeks at a starting dose of 3 µg/kg
|
|---|---|---|
|
Weeks With Weekly Platelet Response
|
0.0 Weeks
Interval 0.0 to 0.0
|
11.0 Weeks
Interval 9.0 to 12.0
|
SECONDARY outcome
Timeframe: Baseline, 12 weeks (Weeks 2 - 13)Population: Full Analysis Set, composed of all randomized participants who received at least 1 dose of romiplostim or placebo
An increase in platelet count of at least 20 x 10\^9/L from baseline within the participant during the treatment period. Increase was calculated as the maximum observed platelet count during the treatment period minus the baseline platelet count.
Outcome measures
| Measure |
Placebo
n=12 Participants
Placebo administered subcutaneously once weekly for 12 weeks
|
Romiplostim
n=22 Participants
Romiplostim administered subcutaneously once weekly for 12 weeks at a starting dose of 3 µg/kg
|
|---|---|---|
|
Increased Platelet Count From Baseline of at Least 20 x 10^9/L
|
3 Participants
|
21 Participants
|
SECONDARY outcome
Timeframe: 12 weeks (Weeks 2 - 13)Population: Full Analysis Set, composed of all randomized participants who received at least 1 dose of romiplostim or placebo
Change from baseline in the mean of the last 4 weekly platelet counts from week 2 to week 13.
Outcome measures
| Measure |
Placebo
n=12 Participants
Placebo administered subcutaneously once weekly for 12 weeks
|
Romiplostim
n=22 Participants
Romiplostim administered subcutaneously once weekly for 12 weeks at a starting dose of 3 µg/kg
|
|---|---|---|
|
Change From Baseline in Mean of Last 4 Weekly Platelet Counts
|
2.3 10^9/L
Standard Deviation 6.5
|
109.7 10^9/L
Standard Deviation 88.5
|
SECONDARY outcome
Timeframe: 12 weeks (Weeks 2 - 13)Population: Full Analysis Set, composed of all randomized participants who received at least 1 dose of romiplostim or placebo
Number of weeks with platelet count between 50 x 10\^9/L and 200 x 10\^9/L inclusive during week 2 to week 13.
Outcome measures
| Measure |
Placebo
n=12 Participants
Placebo administered subcutaneously once weekly for 12 weeks
|
Romiplostim
n=22 Participants
Romiplostim administered subcutaneously once weekly for 12 weeks at a starting dose of 3 µg/kg
|
|---|---|---|
|
Weeks With Platelet Count Between 50 and 200
|
0.2 Weeks
Standard Deviation 0.4
|
6.3 Weeks
Standard Deviation 3.2
|
SECONDARY outcome
Timeframe: 12 weeks (Weeks 2 - 13)Population: Full Analysis Set, composed of all randomized participants who received at least 1 dose of romiplostim or placebo
Requirement for rescue medication(s) during treatment by the participant
Outcome measures
| Measure |
Placebo
n=12 Participants
Placebo administered subcutaneously once weekly for 12 weeks
|
Romiplostim
n=22 Participants
Romiplostim administered subcutaneously once weekly for 12 weeks at a starting dose of 3 µg/kg
|
|---|---|---|
|
Rescue Medication(s)
|
2 Participants
|
2 Participants
|
Adverse Events
Placebo
Serious events: 1 serious events
Other events: 11 other events
Deaths: 0 deaths
Romiplostim
Serious events: 2 serious events
Other events: 16 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Placebo
n=12 participants at risk
|
Romiplostim
n=22 participants at risk
|
|---|---|---|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Gastrointestinal disorders
Gastrointestinal haemorrhage
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Nervous system disorders
Cerebral haemorrhage
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Nervous system disorders
Subarachnoid haemorrhage
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
Other adverse events
| Measure |
Placebo
n=12 participants at risk
|
Romiplostim
n=22 participants at risk
|
|---|---|---|
|
Blood and lymphatic system disorders
Iron deficiency anaemia
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
0.00%
0/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
9.1%
2/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Ear and labyrinth disorders
Tinnitus
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Ear and labyrinth disorders
Vertigo
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Ear and labyrinth disorders
Vertigo positional
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Eye disorders
Eyelid function disorder
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Gastrointestinal disorders
Diarrhoea
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
General disorders
Fatigue
|
0.00%
0/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
9.1%
2/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
General disorders
Feeling abnormal
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
General disorders
Malaise
|
16.7%
2/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
General disorders
Oedema peripheral
|
0.00%
0/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
18.2%
4/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Hepatobiliary disorders
Gallbladder polyp
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Infections and infestations
Bronchitis
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Infections and infestations
Hordeolum
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Infections and infestations
Nasopharyngitis
|
16.7%
2/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
40.9%
9/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Infections and infestations
Pharyngitis
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Infections and infestations
Skin infection
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Injury, poisoning and procedural complications
Contusion
|
16.7%
2/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Injury, poisoning and procedural complications
Thermal burn
|
0.00%
0/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
9.1%
2/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Investigations
White blood cell count increased
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
9.1%
2/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Musculoskeletal and connective tissue disorders
Arthritis
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Musculoskeletal and connective tissue disorders
Back pain
|
0.00%
0/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
13.6%
3/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Musculoskeletal and connective tissue disorders
Pain in extremity
|
0.00%
0/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
13.6%
3/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Nervous system disorders
Headache
|
16.7%
2/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
31.8%
7/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Nervous system disorders
Hypoaesthesia
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Nervous system disorders
IIIrd nerve paralysis
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Nervous system disorders
Loss of consciousness
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Psychiatric disorders
Depression
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Renal and urinary disorders
Nephrocalcinosis
|
0.00%
0/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
9.1%
2/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Reproductive system and breast disorders
Polymenorrhagia
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Respiratory, thoracic and mediastinal disorders
Dyspnoea exertional
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Skin and subcutaneous tissue disorders
Dry skin
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
0.00%
0/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Skin and subcutaneous tissue disorders
Eczema
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
|
Skin and subcutaneous tissue disorders
Urticaria
|
8.3%
1/12 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
4.5%
1/22 • 12 weeks of treatment plus follow-up period up to 12 weeks
The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.
|
Additional Information
Study Director
Amgen Inc.
Phone: 866-572-6436
Results disclosure agreements
- Principal investigator is a sponsor employee The Clinical Trial Agreement generally does not restrict an investigator's discussion of trial results after completion. The Agreement permits Amgen a limited period of time to review material discussing trial results (typically up to 45 days and possible extension). Amgen may remove confidential information, but authors have final control and approval of publication content. For multi-center studies, the investigator agrees not to publish any results before the first multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER