Trial Outcomes & Findings for Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim (NCT NCT01143038)
NCT ID: NCT01143038
Last Updated: 2022-09-21
Results Overview
The primary endpoint was the number of months a participant achieved a platelet response during the 12-month treatment period. A platelet response for any 1 month was defined as the median of platelet counts measured in the month ≥ 50 x 10\^9/L. Platelet counts within 4 weeks following a rescue medication use or following splenectomy were considered non-response. Months without any platelet count measurement were considered as months with no platelet response.
Recruitment status
COMPLETED
Study phase
PHASE2
Target enrollment
75 participants
Primary outcome timeframe
12 months
Results posted on
2022-09-21
Participant Flow
Ninety-eight adults with immune thrombocytopenia purpura (ITP) were screened for the study; 23 were considered screen failures. Seventy-five participants were enrolled at 32 study centers in Australia, the European Union, and North America from 30 November 2010 to 21 September 2012.
The study included a 12-month romiplostim treatment period, and a romiplostim dose-tapering period. Participants who maintained a platelet count of ≥ 50 x 10\^9/L in the absence of romiplostim and all medications for ITP (concomitant or rescue) were followed for at least 6 months to confirm the incidence of ITP remission.
Participant milestones
| Measure |
Romiplostim
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Overall Study
STARTED
|
75
|
|
Overall Study
COMPLETED
|
59
|
|
Overall Study
NOT COMPLETED
|
16
|
Reasons for withdrawal
| Measure |
Romiplostim
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Overall Study
Withdrawal by Subject
|
4
|
|
Overall Study
Requirement for alternative therapy
|
5
|
|
Overall Study
Adverse Event
|
3
|
|
Overall Study
Lost to Follow-up
|
2
|
|
Overall Study
Protocol Deviation
|
1
|
|
Overall Study
Death
|
1
|
Baseline Characteristics
Interventional Study in Adults With Immune Thrombocytopenia Purpura (ITP) Receiving Romiplostim
Baseline characteristics by cohort
| Measure |
Romiplostim
n=75 Participants
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Age, Continuous
|
44.5 years
STANDARD_DEVIATION 18.7 • n=5 Participants
|
|
Sex: Female, Male
Female
|
44 Participants
n=5 Participants
|
|
Sex: Female, Male
Male
|
31 Participants
n=5 Participants
|
|
Race
American Indian or Alaska Native
|
0 participants
n=5 Participants
|
|
Race
Asian
|
1 participants
n=5 Participants
|
|
Race
Black (or African American)
|
1 participants
n=5 Participants
|
|
Race
Native Hawaiian or Other Pacific Islander
|
0 participants
n=5 Participants
|
|
Race
White
|
72 participants
n=5 Participants
|
|
Race
Other
|
0 participants
n=5 Participants
|
|
Race
Unknown
|
1 participants
n=5 Participants
|
|
Ethnicity
Hispanic/Latino
|
6 participants
n=5 Participants
|
|
Ethnicity
Not Hispanic/Latino
|
69 participants
n=5 Participants
|
|
Time since ITP diagnosis
|
2.2 months
n=5 Participants
|
|
Platelet Count at Screening
|
19.78 x 10^9/L
STANDARD_DEVIATION 15.80 • n=5 Participants
|
PRIMARY outcome
Timeframe: 12 monthsPopulation: Safety Analysis Set includes all participants who received at least 1 dose of romiplostim.
The primary endpoint was the number of months a participant achieved a platelet response during the 12-month treatment period. A platelet response for any 1 month was defined as the median of platelet counts measured in the month ≥ 50 x 10\^9/L. Platelet counts within 4 weeks following a rescue medication use or following splenectomy were considered non-response. Months without any platelet count measurement were considered as months with no platelet response.
Outcome measures
| Measure |
Romiplostim
n=75 Participants
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Number of Months With Platelet Response During the 12-Month Treatment Period
|
9.2 months
Standard Error 0.4
|
SECONDARY outcome
Timeframe: Up to 24 monthsPopulation: Safety analysis set
ITP remission was defined as maintaining every platelet count ≥ 50 x 10\^9/L for at least 6 months in the absence of romiplostim and any other therapies to treat ITP.
Outcome measures
| Measure |
Romiplostim
n=75 Participants
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Percentage of Participants With ITP Remission
|
32.0 percentage of participants
Interval 21.7 to 43.8
|
SECONDARY outcome
Timeframe: 12 monthsPopulation: Safety analysis set
If treatment with romiplostim was deemed ineffective or intolerable by the investigator, a splenectomy may have been performed.
Outcome measures
| Measure |
Romiplostim
n=75 Participants
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Percentage of Participants With Splenectomy During the 12-month Treatment Period
|
1.3 percentage of participants
Interval 0.0 to 7.2
|
SECONDARY outcome
Timeframe: From first dose date of romiplostim to end of study (up to 24 months).Population: Safety analysis set
An adverse event (AE) is defined as any untoward medical occurrence in a clinical trial participant. The event does not necessarily have a causal relationship with study treatment. A serious adverse event is defined as an adverse event that meets at least one of the following serious criteria: • fatal • life threatening • requires in-patient hospitalization or prolongation of existing hospitalization • results in persistent or significant disability/incapacity • congenital anomaly/birth defect • other significant medical hazard. Whether an adverse event was treatment-related (TRAE) or not was determined by investigator.
Outcome measures
| Measure |
Romiplostim
n=75 Participants
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Number of Participants With Adverse Events
All adverse events
|
63 participants
|
|
Number of Participants With Adverse Events
Serious adverse events
|
17 participants
|
|
Number of Participants With Adverse Events
Leading to discontinuation of romiplostim
|
4 participants
|
|
Number of Participants With Adverse Events
Leading to discontinuation from study
|
3 participants
|
|
Number of Participants With Adverse Events
Fatal adverse events
|
0 participants
|
|
Number of Participants With Adverse Events
Treatment-related adverse event
|
21 participants
|
|
Number of Participants With Adverse Events
Treatment-related serious adverse events
|
3 participants
|
|
Number of Participants With Adverse Events
TRAE leading to discontinuation of romiplostim
|
2 participants
|
|
Number of Participants With Adverse Events
TRAE leading to discontinuation from study
|
2 participants
|
|
Number of Participants With Adverse Events
Treatment-related fatal adverse events
|
0 participants
|
SECONDARY outcome
Timeframe: Baseline and at end of treatment (based on response to treatment, this could occur between 12 months and approximately 18 months)Population: Safety analysis set participants with available results
The number of participants who developed antibody formation (defined as negative at baseline and positive at post-baseline, transient or persistent) to romiplostim, endogenous thrombopoietin (eTPO), and thrombopoietin mimetic peptide (TMP, the peptide component of romiplostim) was summarized.
Outcome measures
| Measure |
Romiplostim
n=69 Participants
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Number of Participants Who Developed Antibodies to Romiplostim
Antibodies to romiplostim
|
2 participants
|
|
Number of Participants Who Developed Antibodies to Romiplostim
Antibodies to TPO
|
1 participants
|
|
Number of Participants Who Developed Antibodies to Romiplostim
Antibodies to TMP
|
2 participants
|
|
Number of Participants Who Developed Antibodies to Romiplostim
Neutralizing antibodies to romiplostim
|
1 participants
|
|
Number of Participants Who Developed Antibodies to Romiplostim
Neutralizing antibodies to TPO
|
0 participants
|
|
Number of Participants Who Developed Antibodies to Romiplostim
Neutralizing antibodies to TMP
|
0 participants
|
Adverse Events
Romiplostim
Serious events: 17 serious events
Other events: 50 other events
Deaths: 0 deaths
Serious adverse events
| Measure |
Romiplostim
n=75 participants at risk
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Blood and lymphatic system disorders
Idiopathic Thrombocytopenic Purpura
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Blood and lymphatic system disorders
Thrombocytopenia
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Cardiac disorders
Atrial Fibrillation
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Endocrine disorders
Hypothyroidism
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Abdominal Pain
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Faecaloma
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Gastritis
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Erysipelas
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Injury, poisoning and procedural complications
Tendon Rupture
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Investigations
Transaminases Increased
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Non-Hodgkin's Lymphoma
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Neoplasms benign, malignant and unspecified (incl cysts and polyps)
Papillary Thyroid Cancer
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Nervous system disorders
Reversible Ischaemic Neurological Deficit
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Psychiatric disorders
Delirium Tremens
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Renal and urinary disorders
Renal Failure Acute
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Pleuritic Pain
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Dapsone Syndrome
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Vascular disorders
Peripheral Vascular Disorder
|
1.3%
1/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
Other adverse events
| Measure |
Romiplostim
n=75 participants at risk
Participants received romiplostim administered weekly by subcutaneous injection during the 12-month treatment period. The starting dose was 1 μg/kg with weekly dose increases in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg to reach a target platelet count of ≥ 50 x 10\^9/L. At the completion of the 12-month treatment period, participants receiving only romiplostim and with a platelet count ≥ 50 x 10\^9/L entered the tapering period, during which the romiplostim dose was decreased by 1 µg/kg every 2 weeks, for up to 19 weeks. Participants who had tapered off treatment with romiplostim and whose platelet count dropped below 50 x 10\^9/L could reinitiate romiplostim for up to 8 weeks.
|
|---|---|
|
Ear and labyrinth disorders
Vertigo
|
5.3%
4/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Eye disorders
Conjunctivitis
|
5.3%
4/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Gastrointestinal disorders
Diarrhoea
|
5.3%
4/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
General disorders
Asthenia
|
6.7%
5/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
General disorders
Fatigue
|
8.0%
6/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Influenza
|
9.3%
7/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Nasopharyngitis
|
13.3%
10/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Rhinitis
|
5.3%
4/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Infections and infestations
Upper Respiratory Tract Infection
|
8.0%
6/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Musculoskeletal and connective tissue disorders
Arthralgia
|
14.7%
11/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Musculoskeletal and connective tissue disorders
Myalgia
|
5.3%
4/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Nervous system disorders
Dizziness
|
5.3%
4/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Nervous system disorders
Headache
|
17.3%
13/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Cough
|
9.3%
7/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Respiratory, thoracic and mediastinal disorders
Epistaxis
|
8.0%
6/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Petechiae
|
9.3%
7/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Pruritus
|
5.3%
4/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Skin and subcutaneous tissue disorders
Rash
|
5.3%
4/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Vascular disorders
Haematoma
|
10.7%
8/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
|
Vascular disorders
Hypertension
|
8.0%
6/75 • From first dose date of romiplostim to end of study (up to 24 months).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
|
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 multicenter studies, the investigator agrees not to publish any results before the first multi-center publication.
- Publication restrictions are in place
Restriction type: OTHER