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The purpose of this study is to assess the safety and tolerability of AMG 531 (romiplostim), a novel thrombopoiesis-stimulating peptibody, and its effect on platelet counts in adults with immune thrombocytopenic purpura.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Romiplostim | Experimental | Participants will receive a maximum of 2 administrations of romiplostim by subcutaneous injection, the first on day 1 of the study and the second on day 15 or 22 depending on the participant's platelet count. Romiplostim doses to be tested were 30, 100, 300, and 500 μg. |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Romiplostim | Drug | Administered subcutaneously on day 1 and on day 15 or 22 if the platelet count was ≤ 50 x 10⁹/L and not rising, peak platelet count was ≤ 450 x 10⁹/L and no serious adverse events related to treatment were observed. |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants With Adverse Events | From first dose through 8 weeks after last dose of study drug (11 weeks) | |
| Number of Participants With Positive Anti-Romiplostim Antibodies | The presence or development of antibodies to romiplostim and endogenous thrombopoietin was assessed using a neutralizing bioassay. Antibody analyses were conducted on study days 29 and at the end-of-study visit (day 78). The number of participants with positive antibody binding at any time during the study is reported. | Days 29 and 78 |
| Measure | Description | Time Frame |
|---|---|---|
| Number of Participants Who Achieved a Targeted Therapeutic Platelet Response | Targeted therapeutic platelet response was defined as a (single) platelet count that was double the baseline level and between 50 and 450 × 10⁹ cells/L. Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. | Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants) |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| MD | Amgen | Study Director |
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| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 28411254 | Background | Cines DB, Wasser J, Rodeghiero F, Chong BH, Steurer M, Provan D, Lyons R, Garcia-Chavez J, Carpenter N, Wang X, Eisen M. Safety and efficacy of romiplostim in splenectomized and nonsplenectomized patients with primary immune thrombocytopenia. Haematologica. 2017 Aug;102(8):1342-1351. doi: 10.3324/haematol.2016.161968. Epub 2017 Apr 14. | |
| 17061981 |
| Label | URL |
|---|---|
| AmgenTrials clinical trials website | View source |
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Participants were enrolled sequentially into one of four dose cohorts. When the platelet count of the first participant who received the 500 μg dose increased above 650 × 10⁹ cells/L following the first dose, this cohort was discontinued, and subsequent participants were assigned to the 300 μg cohort.
This was a multicenter study conducted in 6 centers in France, the Netherlands, and the United Kingdom.
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| ID | Title | Description |
|---|---|---|
| FG000 | Romiplostim 30 µg | Participants received 30 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| FG001 | Romiplostim 100 µg | Participants received 100 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| FG002 | Romiplostim 300 µg | Participants received 300 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| FG003 | Romiplostim 500 µg | Participants received 500 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
All enrolled participants
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| ID | Title | Description |
|---|---|---|
| BG000 | Romiplostim 30 µg | Participants received 30 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| BG001 | Romiplostim 100 µg |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Number of Participants With Adverse Events | Participants who received at least 1 dose of romiplostim | Posted | Count of Participants | Participants | From first dose through 8 weeks after last dose of study drug (11 weeks) |
|
From first dose through 8 weeks after last dose of study drug (11 weeks).
Other Adverse Events summarizes the non-serious occurrences of adverse events that exceed the indicated frequency threshold.
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Romiplostim 30 µg | Participants received 30 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Anaemia | Blood and lymphatic system disorders | MedDRA 7.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Thrombocythaemia | Blood and lymphatic system disorders | MedDRA 7.0 | Systematic Assessment |
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| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Study Director | Amgen Inc. | 866-572-6436 | medinfo@amgen.com |
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| ID | Term |
|---|---|
| D011696 | Purpura, Thrombocytopenic |
| D016553 | Purpura, Thrombocytopenic, Idiopathic |
| D013921 | Thrombocytopenia |
| ID | Term |
|---|---|
| D011693 | Purpura |
| D001778 | Blood Coagulation Disorders |
| D006402 | Hematologic Diseases |
| D006425 | Hemic and Lymphatic Diseases |
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| ID | Term |
|---|---|
| C488777 | romiplostim |
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|
| Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L From Baseline | Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. | Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants) |
| Number of Participants With Peak Platelet Counts of ≥ 100 x 10⁹ Cells/L | Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. | After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants) |
| Number of Participants With Peak Platelet Counts of ≥ 450 x 10⁹ Cells/L | Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. | After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants) |
| Change From Baseline to Peak Platelet Level | Platelet count data after the use of rescue medication were not included. | Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants) |
| Time to Peak Platelet Count | Time from the date of study drug administration (day 1, 15 or 22) to the day of peak platelet count after each dose. Platelet count data after the use of rescue medication were not included. | From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78 |
| Duration Within the Targeted Therapeutic Range | Targeted therapeutic platelet level was defined as a platelet count that was double the baseline level and between 50 and 450 × 10⁹ cells/L. Platelet count data after the use of rescue medication were not included. | From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78 |
| Newland A, Caulier MT, Kappers-Klunne M, Schipperus MR, Lefrere F, Zwaginga JJ, Christal J, Chen CF, Nichol JL. An open-label, unit dose-finding study of AMG 531, a novel thrombopoiesis-stimulating peptibody, in patients with immune thrombocytopenic purpura. Br J Haematol. 2006 Nov;135(4):547-53. doi: 10.1111/j.1365-2141.2006.06339.x. |
| FDA-approved Drug Labeling | View source |
Participants received 100 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count.
| BG002 | Romiplostim 300 µg | Participants received 300 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| BG003 | Romiplostim 500 µg | Participants received 500 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| BG004 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race/Ethnicity, Customized | Count of Participants | Participants |
|
| Platelet Count | Mean | Standard Deviation | 10⁹cells/L |
|
| OG002 |
| Romiplostim 300 µg |
Participants received 300 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
| OG003 | Romiplostim 500 µg | Participants received 500 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. |
|
|
| Primary | Number of Participants With Positive Anti-Romiplostim Antibodies | The presence or development of antibodies to romiplostim and endogenous thrombopoietin was assessed using a neutralizing bioassay. Antibody analyses were conducted on study days 29 and at the end-of-study visit (day 78). The number of participants with positive antibody binding at any time during the study is reported. | Participants who received at least 1 dose of romiplostim | Posted | Count of Participants | Participants | Days 29 and 78 |
|
|
|
| Secondary | Number of Participants Who Achieved a Targeted Therapeutic Platelet Response | Targeted therapeutic platelet response was defined as a (single) platelet count that was double the baseline level and between 50 and 450 × 10⁹ cells/L. Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. | Participants who received at least 1 dose of romiplostim. | Posted | Count of Participants | Participants | Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants) |
|
|
|
| Secondary | Number of Participants With an Increase in Platelet Count of ≥ 20 x 10⁹ Cells/L From Baseline | Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. | Participants who received at least 1 dose of romiplostim. | Posted | Count of Participants | Participants | Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants) |
|
|
|
| Secondary | Number of Participants With Peak Platelet Counts of ≥ 100 x 10⁹ Cells/L | Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. | Participants who received at least 1 dose of romiplostim. | Posted | Count of Participants | Participants | After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants) |
|
|
|
| Secondary | Number of Participants With Peak Platelet Counts of ≥ 450 x 10⁹ Cells/L | Platelet count data after the use of rescue medication were not included; participants with no platelet count data were considered non-responders. | Participants who received at least 1 dose of romiplostim. | Posted | Count of Participants | Participants | After first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22), and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 for all participants) |
|
|
|
| Secondary | Change From Baseline to Peak Platelet Level | Platelet count data after the use of rescue medication were not included. | Participants who received at least 1 dose of romiplostim with available data. | Posted | Mean | Standard Deviation | 10⁹ cells/L | Baseline and after first dose (days 3, 5, 8, 10, 12, 15, and days 17 and 19 for participants dosed on day 22) and after second dose (days 17, 19, and 22 for participants dosed on day 15, and days 24, 26, 29, 32, 36, 43, 50, 64, and 78 in all participants) |
|
|
|
| Secondary | Time to Peak Platelet Count | Time from the date of study drug administration (day 1, 15 or 22) to the day of peak platelet count after each dose. Platelet count data after the use of rescue medication were not included. | Participants who received at least 1 dose of romiplostim with available data. | Posted | Median | Full Range | days | From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78 |
|
|
|
| Secondary | Duration Within the Targeted Therapeutic Range | Targeted therapeutic platelet level was defined as a platelet count that was double the baseline level and between 50 and 450 × 10⁹ cells/L. Platelet count data after the use of rescue medication were not included. | Participants who received at least 1 dose of romiplostim with a targeted therapeutic response. | Posted | Median | Full Range | days | From first dose of study drug to day 15 or 22, and from the second dose of study drug (day 15 or 22) to day 78 |
|
|
|
| 0 |
| 4 |
| 4 |
| 4 |
| EG001 | Romiplostim 100 µg | Participants received 100 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. | 0 | 4 | 4 | 4 |
| EG002 | Romiplostim 300 µg | Participants received 300 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. | 3 | 7 | 7 | 7 |
| EG003 | Romiplostim 500 µg | Participants received 500 µg romiplostim by subcutaneous injection on day 1 and a possible second dose on day 15 or 22 depending on the participant's platelet count. | 1 | 1 | 1 | 1 |
| Thrombocytopenia | Blood and lymphatic system disorders | MedDRA 7.0 | Systematic Assessment |
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| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Lower respiratory tract infection | Infections and infestations | MedDRA 7.0 | Systematic Assessment |
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| Blood lactate dehydrogenase increased | Investigations | MedDRA 7.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 7.0 | Systematic Assessment |
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| Palpitations | Cardiac disorders | MedDRA 7.0 | Systematic Assessment |
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| Tinnitus | Ear and labyrinth disorders | MedDRA 7.0 | Systematic Assessment |
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| Vertigo | Ear and labyrinth disorders | MedDRA 7.0 | Systematic Assessment |
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| Conjunctival haemorrhage | Eye disorders | MedDRA 7.0 | Systematic Assessment |
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| Aphthous stomatitis | Gastrointestinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Gingival bleeding | Gastrointestinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Mouth haemorrhage | Gastrointestinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Nausea | Gastrointestinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Oral mucosal petechiae | Gastrointestinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Rectal haemorrhage | Gastrointestinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Asthenia | General disorders | MedDRA 7.0 | Systematic Assessment |
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| Chest pain | General disorders | MedDRA 7.0 | Systematic Assessment |
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| Fatigue | General disorders | MedDRA 7.0 | Systematic Assessment |
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| Injection site haemorrhage | General disorders | MedDRA 7.0 | Systematic Assessment |
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| Injection site pain | General disorders | MedDRA 7.0 | Systematic Assessment |
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| Malaise | General disorders | MedDRA 7.0 | Systematic Assessment |
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| Oedema peripheral | General disorders | MedDRA 7.0 | Systematic Assessment |
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| Ear infection | Infections and infestations | MedDRA 7.0 | Systematic Assessment |
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| Gastrointestinal infection | Infections and infestations | MedDRA 7.0 | Systematic Assessment |
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| Influenza | Infections and infestations | MedDRA 7.0 | Systematic Assessment |
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| Nasopharyngitis | Infections and infestations | MedDRA 7.0 | Systematic Assessment |
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| Contusion | Injury, poisoning and procedural complications | MedDRA 7.0 | Systematic Assessment |
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| Head injury | Injury, poisoning and procedural complications | MedDRA 7.0 | Systematic Assessment |
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| Limb injury | Injury, poisoning and procedural complications | MedDRA 7.0 | Systematic Assessment |
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| Muscle strain | Injury, poisoning and procedural complications | MedDRA 7.0 | Systematic Assessment |
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| Anorexia | Metabolism and nutrition disorders | MedDRA 7.0 | Systematic Assessment |
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| Arthralgia | Musculoskeletal and connective tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Back pain | Musculoskeletal and connective tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Pain in extremity | Musculoskeletal and connective tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Headache | Nervous system disorders | MedDRA 7.0 | Systematic Assessment |
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| Lethargy | Nervous system disorders | MedDRA 7.0 | Systematic Assessment |
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| Dyspnoea | Respiratory, thoracic and mediastinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Epistaxis | Respiratory, thoracic and mediastinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Increased viscosity of bronchial secretion | Respiratory, thoracic and mediastinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Pharyngolaryngeal pain | Respiratory, thoracic and mediastinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Sinus congestion | Respiratory, thoracic and mediastinal disorders | MedDRA 7.0 | Systematic Assessment |
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| Ecchymosis | Skin and subcutaneous tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Hyperhidrosis | Skin and subcutaneous tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Purpura | Skin and subcutaneous tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Rash | Skin and subcutaneous tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Skin bleeding | Skin and subcutaneous tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Skin lesion | Skin and subcutaneous tissue disorders | MedDRA 7.0 | Systematic Assessment |
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| Haematoma | Vascular disorders | MedDRA 7.0 | Systematic Assessment |
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| Petechiae | Vascular disorders | MedDRA 7.0 | Systematic Assessment |
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| Breast mass | Reproductive system and breast disorders | MedDRA 7.0 | Systematic Assessment |
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| Menorrhagia | Reproductive system and breast disorders | MedDRA 7.0 | Systematic Assessment |
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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.
| D057049 |
| Thrombotic Microangiopathies |
| D001791 | Blood Platelet Disorders |
| D000095542 | Cytopenia |
| D007154 | Immune System Diseases |
| D006470 | Hemorrhage |
| D010335 | Pathologic Processes |
| D013568 | Pathological Conditions, Signs and Symptoms |
| D012877 | Skin Manifestations |
| D012816 | Signs and Symptoms |
| D006474 | Hemorrhagic Disorders |
| D001327 | Autoimmune Diseases |
| Anti-thrombopoietin antibodies |
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| After Both Doses |
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| After Second Dose |
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| After Both Doses |
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| After Second Dose |
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| After Both Doses |
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| After Second Dose |
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| After Both Doses |
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| After Second Dose |
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| After Second Dose |
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| After Second Dose |
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