Clinical Trial Results:
A Phase 2, Interventional, Single Arm Study Describing Platelet Responses and ITP Remission Rates in Adult Subjects with Immune Thrombocytopenia Purpura Receiving Romiplostim
|
Summary
|
|
EudraCT number |
2010-019987-35 |
Trial protocol |
IT DE GB ES FR CZ |
Global end of trial date |
26 Dec 2013
|
|
Results information
|
|
Results version number |
v1(current) |
This version publication date |
20 Jun 2016
|
First version publication date |
30 Jul 2015
|
Other versions |
|
Trial Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
Subject Disposition
Baseline Characteristics
End Points
Adverse Events
More Information
|
|||
|
Trial identification
|
|||
Sponsor protocol code |
20080435
|
||
|
Additional study identifiers
|
|||
ISRCTN number |
- | ||
US NCT number |
NCT01143038 | ||
WHO universal trial number (UTN) |
- | ||
|
Sponsors
|
|||
Sponsor organisation name |
Amgen Inc.
|
||
Sponsor organisation address |
One Amgen Center Drive, Thousand Oaks, United States, 91320
|
||
Public contact |
IHQ Medical Info-Clinical Trials, Amgen (EUROPE) GmbH, MedInfoInternational@amgen.com
|
||
Scientific contact |
IHQ Medical Info-Clinical Trials, Amgen (EUROPE) GmbH, MedInfoInternational@amgen.com
|
||
|
Paediatric regulatory details
|
|||
Is trial part of an agreed paediatric investigation plan (PIP) |
No
|
||
Does article 45 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
Does article 46 of REGULATION (EC) No 1901/2006 apply to this trial? |
No
|
||
|
Results analysis stage
|
|||
Analysis stage |
Final
|
||
Date of interim/final analysis |
26 Dec 2013
|
||
Is this the analysis of the primary completion data? |
No
|
||
Global end of trial reached? |
Yes
|
||
Global end of trial date |
26 Dec 2013
|
||
Was the trial ended prematurely? |
No
|
||
|
General information about the trial
|
|||
Main objective of the trial |
To describe the number of months with a subject platelet response over a 12 month treatment period
|
||
Protection of trial subjects |
This study was conducted in accordance with applicable FDA and International Conference on Harmonisation (ICH) Good Clinical Practice (GCP) regulations/guidelines. A copy of the protocol, the proposed informed consent, and all other subject information and/or recruitment materials were submitted to the IEC or IRB of each study center for approval. The investigator or a designee obtained written informed consent from their subjects or legally acceptable representatives after adequate explanation of the aims, methods, anticipated benefits, and potential hazards of the study and before any protocol-specific screening procedures were conducted or investigational product was administered.
|
||
Background therapy |
- | ||
Evidence for comparator |
- | ||
Actual start date of recruitment |
30 Nov 2010
|
||
Long term follow-up planned |
No
|
||
Independent data monitoring committee (IDMC) involvement? |
No
|
||
|
Population of trial subjects
|
|||
Number of subjects enrolled per country |
|||
Country: Number of subjects enrolled |
Poland: 15
|
||
Country: Number of subjects enrolled |
Spain: 8
|
||
Country: Number of subjects enrolled |
United Kingdom: 3
|
||
Country: Number of subjects enrolled |
Czech Republic: 12
|
||
Country: Number of subjects enrolled |
France: 5
|
||
Country: Number of subjects enrolled |
Germany: 11
|
||
Country: Number of subjects enrolled |
Italy: 11
|
||
Country: Number of subjects enrolled |
Australia: 3
|
||
Country: Number of subjects enrolled |
United States: 7
|
||
Worldwide total number of subjects |
75
|
||
EEA total number of subjects |
65
|
||
Number of subjects enrolled per age group |
|||
In utero |
0
|
||
Preterm newborn - gestational age < 37 wk |
0
|
||
Newborns (0-27 days) |
0
|
||
Infants and toddlers (28 days-23 months) |
0
|
||
Children (2-11 years) |
0
|
||
Adolescents (12-17 years) |
0
|
||
Adults (18-64 years) |
61
|
||
From 65 to 84 years |
12
|
||
85 years and over |
2
|
||
|
|||||||||||||||||||||||
|
Recruitment
|
|||||||||||||||||||||||
Recruitment details |
- | ||||||||||||||||||||||
|
Pre-assignment
|
|||||||||||||||||||||||
Screening details |
Ninety-eight adult subjects with ITP were screened for the study; 23 subjects were considered screen failures. Seventy-five subjects were enrolled and received at least 1 dose of romiplostim. | ||||||||||||||||||||||
|
Period 1
|
|||||||||||||||||||||||
Period 1 title |
Overall Study (overall period)
|
||||||||||||||||||||||
Is this the baseline period? |
Yes | ||||||||||||||||||||||
Allocation method |
Not applicable
|
||||||||||||||||||||||
Blinding used |
Not blinded | ||||||||||||||||||||||
|
Arms
|
|||||||||||||||||||||||
|
Arm title
|
Romiplostim | ||||||||||||||||||||||
Arm description |
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 continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. | ||||||||||||||||||||||
Arm type |
Experimental | ||||||||||||||||||||||
Investigational medicinal product name |
Romiplostim
|
||||||||||||||||||||||
Investigational medicinal product code |
AMG 531
|
||||||||||||||||||||||
Other name |
Nplate
|
||||||||||||||||||||||
Pharmaceutical forms |
Powder for solution for injection
|
||||||||||||||||||||||
Routes of administration |
Subcutaneous use
|
||||||||||||||||||||||
Dosage and administration details |
Romiplostim was administered weekly by subcutaneous injection. The starting dose of romiplostim was 1 μg/kg; weekly dose increases continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. Dose adjustments were allowed during the treatment period to maintain a platelet count between 50 x 10^9/L and 200 x 10^9/L.
|
||||||||||||||||||||||
|
|||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Baseline characteristics reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Romiplostim
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
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 continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||
|
End points reporting groups
|
|||
Reporting group title |
Romiplostim
|
||
Reporting group description |
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 continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. | ||
|
|||||||||
End point title |
Number of Months with Platelet Response During the 12-Month Treatment period [1] | ||||||||
End point description |
The primary endpoint was the number of months a subject 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 and months following splenectomy were considered as months with no platelet response.
Safety Analysis Set includes all subjects who have received at least 1 dose of romiplostim.
|
||||||||
End point type |
Primary
|
||||||||
End point timeframe |
12 months
|
||||||||
| Notes [1] - No statistical analyses have been specified for this primary end point. It is expected there is at least one statistical analysis for each primary end point. Justification: A formal hypothesis was not tested in this study. The primary analysis of the primary and secondary endpoints was descriptive. |
|||||||||
|
|||||||||
| Notes [2] - Safety Analysis Set |
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Percentage of subjects with ITP remisssion | ||||||||
End point description |
ITP remission was defined as all platelet counts ≥ 50 x 10^9/L in the absence of romiplostim and all other therapies dosed with the intent to treat ITP for at least 6 months.
|
||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
12 months
|
||||||||
|
|||||||||
| Notes [3] - Safety analysis set |
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||
End point title |
Percentage of subjects with splenectomy during the 12-month treatment period | ||||||||
End point description |
|||||||||
End point type |
Secondary
|
||||||||
End point timeframe |
12 months
|
||||||||
|
|||||||||
| Notes [4] - Safety analysis set |
|||||||||
| No statistical analyses for this end point | |||||||||
|
|||||||||||||||||||
End point title |
Number of subjects who developed antibodies to romiplostim | ||||||||||||||||||
End point description |
The number of subjects developing 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
|
||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||
End point timeframe |
12 months
|
||||||||||||||||||
|
|||||||||||||||||||
| Notes [5] - Safety analysis set participants with available results |
|||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||
|
|||||||||||||||||||||||||||
End point title |
Number of subjects with adverse events | ||||||||||||||||||||||||||
End point description |
An adverse event is defined as any untoward medical occurrence in a clinical trial subject. 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 (places the subject at immediate risk of death)
• 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.
|
||||||||||||||||||||||||||
End point type |
Secondary
|
||||||||||||||||||||||||||
End point timeframe |
From first dose date of romiplostim to end of study (up to 24 months).
|
||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||
| Notes [6] - Safety analysis set |
|||||||||||||||||||||||||||
| No statistical analyses for this end point | |||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Adverse events information
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Timeframe for reporting adverse events |
From first dose date of romiplostim to end of study (up to 24 months)
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Assessment type |
Systematic | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Dictionary used for adverse event reporting
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary name |
MedDRA | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Dictionary version |
16.1
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
Reporting groups
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group title |
Romiplostim
|
||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
Reporting group description |
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 continued in increments of 1 μg/kg/week to a maximum dose of 10 μg/kg in an attempt to reach a target platelet count of ≥ 50 x 10^9/L. | ||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
| Frequency threshold for reporting non-serious adverse events: 5% | |||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||||
|
|||
Substantial protocol amendments (globally) |
|||
| Were there any global substantial amendments to the protocol? Yes | |||
Date |
Amendment |
||
15 Jan 2013 |
- The study schema was updated to illustrate and clarify the various treatment scenarios.
- Clarification was made to describe the various treatment periods and their definitions.
- Clarification was made in the language throughout the protocol for medications for ITP given either concomitantly or as rescue medications.
- Updates to pregnancy, antibody testing parameters, and contraception requirements were updated per the current Amgen requirements.
- Updates were made to the serious adverse event reporting language in Section 9.2 per the current Amgen safety requirements. |
||
Interruptions (globally) |
|||
| Were there any global interruptions to the trial? No | |||
Limitations and caveats |
|||
| Limitations of the trial such as small numbers of subjects analysed or technical problems leading to unreliable data. | |||
| None reported | |||
