Blood and Lymph Conditions

All Conditions

Symptoms and General Pathology

Neoplasms

Rare Diseases

Thrombocytopenia

Syndrome

Cytopenia

Preleukemia

Myelodysplastic Syndromes

Bone Marrow Diseases

Hematologic Diseases

Precancerous Conditions

Blood Platelet Disorders

Cohort 1 in Part A, participants received romiplostim 300 µg subcutaneously once weekly for 3 weeks. Participants who completed Part A could continue to receive weekly injections of romiplostim for up to 1 year in the extension treatment phase.

Cohort 2 in Part A, participants received romiplostim 700 µg subcutaneously once weekly for up to 3 weeks. Participants who completed Part A could continue to receive weekly injections of romiplostim for up to 1 year in the extension treatment phase.

Cohort 3 in Part A, participants received romiplostim 1000 µg subcutaneously once weekly for up to 3 weeks. Participants who completed Part A could continue to receive weekly injections of romiplostim for up to 1 year in the extension treatment phase.

Cohort 4 in Part A, participants received romiplostim 1500 µg subcutaneously once weekly for up to 3 weeks. Participants who completed Part A could continue to receive weekly injections of romiplostim for up to 1 year in the extension treatment phase.

Part B participants received romiplostim 750 µg subcutaneously (SC) once weekly (QW) for 8 weeks. Participants who complete Part B could continue to receive weekly injections of romiplostim for up to 1 year in the extension treatment phase.

Part B participants received romiplostim 750 µg subcutaneously every two weeks (Q2W) for 8 weeks. Participants who complete Part B could continue to receive injections of romiplostim for up to 1 year in the extension treatment phase.

Part B participants received romiplostim 750 µg intravenously (IV) once every two weeks for 8 weeks. Participants who complete Part B could continue to receive romiplostim for up to 1 year in the extension treatment phase.

Romiplostim

The purpose of this study is to evaluate the safety and tolerability of romiplostim in thrombocytopenic patients with low or Intermediate-1 risk MDS. In addition, the study will evaluate the platelet response to romiplostim.

Determination of Safe Dose of Romiplostim (AMG 531) in Patients With Myelodysplastic Syndromes (MDS)

An Open Label, Sequential Cohort, Dose Escalation Study to Evaluate the Safety and Efficacy of AMG 531 in Thrombocytopenic Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS)

The number of participants experiencing one or more adverse events during the treatment phase or extension phase of Part A.

The number of participants experiencing one or more adverse events during the treatment phase or extension phase of Part B.

Participants with a complete or major response during the treatment phase. A complete platelet response was defined as a platelet count ≥ 100 x 10\^9/L during the treatment phase. A major platelet response was defined as an increase in absolute platelet count of ≥ 30 x 10\^9/L for patients starting with \> 20 x 10\^9/L platelets, or an increase from ≤ 20 x 10\^9/L to \> 20 x 10\^9/L and by at least 100%. Any participant receiving rescue medication was considered a non-responder. Platelet transfusions were considered rescue medication.

The number of participants with a platelet response according to the modified International Working Group (IWG) criteria. Response was defined as an absolute increase of ≥ 30 x 10\^9/L with Baseline platelet count \> 20 x 10\^9/L, or with a Baseline count ≤ 20 x 10\^9/L, increasing to above 20 x 10\^9/L and by at least 100% during the treatment or extension period and maintained for at least 8 consecutive weeks. Platelet transfusion was not considered a rescue medication but platelet counts ≤72 hours after platelet transfusion were excluded from the analysis.

Peak platelet count (10\^9/L) during the treatment period.

Participants achieving first platelet response according to IWG criteria, by study week. Platelet response was defined as an absolute increase of ≥ 30 x 10\^9/L with Baseline platelet count \> 20 x 10\^9/L, or with a Baseline ≤ 20 x 10\^9/L increasing the platelet count to above 20 x 10\^9/L and by at least 100% for 8 consecutive weeks. Platelet counts obtained within 72 hours of platelet transfusion were not evaluable for platelet response.

Duration of platelet response per IWG criteria (absolute increase of ≥ 30 x 10\^9/L with Baseline platelet count \> 20 x 10\^9/L, or with a Baseline ≤ 20 x 10\^9/L increasing the platelet count to above 20 x 10\^9/L and by at least 100% for 8 consecutive weeks).

Maximum observed serum concentration (Cmax) of romiplostim during Week 1

Measured romiplostim concentration at the end of the week 1 dosing interval (Ctrough)

Area under the romiplostim concentration-time curve from time zero to the last time point with quantifiable concentration (AUC0-4) during Week 1

Maximum observed serum concentration (Cmax) of romiplostim during Week 7.

Measured romiplostim concentration at the end of the Week 7 dosing interval (Ctrough)

Area under the romiplostim concentration-time curve from time zero to the last time point with quantifiable concentration (AUC0-4) during Week 7.

Time at which the maximum concentration of romiplostum was observed after subcutaneous administration during Week 1

Time at which the maximum concentration of romiplostum was observed after subcutaneous administration during Week 7

Amgen

The table of Other Adverse Events summarizes the most frequent non-serious occurrences of adverse events.

Cohort 1 in Part A participants received romiplostim 300 µg subcutaneously once weekly for 3 weeks. Participants who completed Part A could continue to receive weekly injections of romiplostim for up to 1 year in the extension treatment phase.

Part A: 300 µg Romiplostim

Part A: 700 µg Romiplostim

Part A: 1000 µg Romiplostim

Part A: 1500 µg Romiplostim

Part B participants received romiplostim 750 µg subcutaneously (SC) once weekly (QW) for 8 weeks. Participants who completed Part B could continue to receive weekly injections of romiplostim for up to 1 year in the extension treatment phase.

Part B: 750 µg Romiplostim SC QW

Part B participants received romiplostim 750 µg subcutaneously every two weeks (Q2W) for 8 weeks. Participants who completed Part B could continue to receive injections of romiplostim for up to 1 year in the extension treatment phase.

Part B: 750 µg Romiplostim SC Q2W

Part B participants received romiplostim 750 µg intravenously (IV) once every two weeks for 8 weeks. Participants who completed Part B could continue to receive romiplostim for up to 1 year in the extension treatment phase.

Part B: 750 µg Romiplostim IV Q2W

Cohort 1 in Part A, participants received romiplostim 300 µg subcutaneously once weekly for up to 3 weeks. Participants who completed Part A could continue to receive weekly injections of romiplostim for up to 1 year in the extension treatment phase.

Total

Age, Continuous

Sex: Female, Male

White or Caucasian

Black or African American

Hispanic or Latino

Asian

Japanese

American Indian or Alaska Native

Aborigine

Other

Race/Ethnicity, Customized

Study Director

Efficacy Analysis Set, composed of all enrolled participants who received romiplostim and completed the treatment phase.

Part A: Number of Participants With a Complete or Major Platelet Response

Part B: Number of Participants With a Complete or Major Platelet Response

Subset of Efficacy Analysis Set, composed of all enrolled participants who received romiplostim and completed the treatment phase, who entered the treatment extension.

Part A: Number of Participants With a Platelet Response Per IWG Criteria

Efficacy Analysis Set, composed of all enrolled participants who received romiplostim and completed the treatment phase

Part B: Number of Participants With a Platelet Response Per IWG

Part B: Peak Platelet Count

Week 1 Day 3

Week 2

Week 3

Week 4

Week 5 or later

Not Observed

Part B: Time to First Platelet Response

Subset of Efficacy Analysis Set, composed of all enrolled participants who received romiplostim and completed the treatment phase, who had a platelet response

Part B: Duration of Platelet Response

Safety Analysis Set, composed of all enrolled participants who received at least one dose of romiplostim

Spots Global Cancer Trial Database for Determination of Safe Dose of Romiplostim (AMG 531) in Patients With Myelodysplastic Syndromes (MDS)

Trial Identification

Conditions

Interventions

Study Description

Keywords

Eligibility

Locations

Contact Details

Useful links and downloads for this trial