AMG 531 Treatment of Thrombocytopenic Subjects With Immune (Idiopathic) Thrombocytopenic Purpura (ITP) Prior to Splenectomy

NCT00102336 | Completed Phase 3

• 1 other identifier: 20030212
• Study Type: interventional
• Target: 62
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by the platelet response. This study will also evaluate changes in Patient Reported Outcomes and Health Resource Utilization due to treatment with AMG 531.

Conditions

Thrombocytopenia; Idiopathic Thrombocytopenic Purpura

Keywords

immune (idiopathic) thrombocytopenic purpura; pre-splenectomy; ITP; Thrombopenia

Outcome Measures

Primary Outcomes (1)

• To evaluate the efficacy of AMG 531 in the treatment of thrombocytopenia in subjects with ITP as measured by durable platelet response during the last 8 weeks of treatment and other platelet response parameters

  Last 8 weeks of treatments

Secondary Outcomes (3)

• To evaluate the overall safety of AMG 531

  Entire duration of the study including the follow up period

• To evaluate possible reductions in the dose of concurrent ITP therapies while receiving AMG 531

  Entire duration of the study

• To evaluate changes in Patient Reported Outcomes (PRO) and Health Resource Utilization (HRU) due to treatment with AMG 531

  Entire duration of the study

Study Arms (2)

AMG 531

EXPERIMENTAL

Active investigational product

Biological: AMG 531

Placebo

PLACEBO COMPARATOR

Drug: Placebo

Interventions

AMG 531 BIOLOGICAL

Weekly subcutaneous dosing based on screening weight and platelet count. Starting dose is at 1mcg/kg up to a maximum dose of 15mcg/kg. AMG 531 is supplied in a 5 mL single use glass vial as a sterile, white, preservative-free, lyophilized powder.

AMG 531

Placebo DRUG

Weekly subcutaneous dosing based on screening weight and platelet count. Starting dose is at 1mcg/kg up to a maximum dose of 15mcg/kg. Placebo is supplied as a lyophilized power in a 5 mL single use glass vial.

Placebo

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Diagnosis of ITP according to American Society of Hematology (ASH) guidelines (Appendix F)
• Have completed as least 1 prior treatment for ITP (e.g., prednisone)
• Subjects greater than 60 years of age must have a documented history of chronic ITP with a bone marrow report to confirm the diagnosis
• The platelet count (calculated from the mean of the 2 counts taken during the screening and pre-treatment periods) must be:
• \*less than 30 x 10\^9/L for those subjects not receiving any ITP therapy, with no count greater than 35 x 10\^9/L \*less than 50 x 10\^9/L for those subjects receiving a constant dose schedule of corticosteroids, azathioprine or danazol with no count greater than 55 x 10\^9/L
• A serum creatinine concentration less than or equal to 2 mg/dl (less than or equal to 176.8 µmol/L)
• Adequate liver function, as evidenced by a serum bilirubin less than or equal to 1.5 times the laboratory normal range
• Hemoglobin greater than 11.0 g/dL
• Written informed consent (see Section 12.1)

You may not qualify if:

• Have had a Splenectomy for any reason
• Any known history of bone marrow stem cell disorder (Any abnormal bone marrow findings other than those typical of ITP must be approved by Amgen before a subject may be enrolled in the study)
• Any active malignancy. If prior history of cancer other than basal cell carcinoma or cervical carcinoma in situ, no treatment or active disease within 5 years before randomization
• Documented diagnosis of arterial thrombosis (i.e., stroke, transient ischemic attack or myocardial infarction) in the past year
• History of venous thrombosis (i.e., deep vein thrombosis, pulmonary embolism) including those subjects who are on ant-coagulation therapy
• Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure \[NYHA greater than class II\], uncontrolled hypertension \[diastolic greater than 100 mmHg\] or cardiac arrhythmia)
• Have 3 or more of the following predisposing factors for thromboembolic events: diabetes; smoker; using oral contraceptives; on estrogen therapy; known positive for anti-phospholipid antibodies; hypertriglyceridemia; hypercholesteremia (greater than 240 mg/dL); treatment for hypertension
• Known positive test for human immunodeficiency virus (HIV) infection or hepatitis C virus
• Currently receiving any treatment for ITP except corticosteroids, azathioprine or danazol administered at a constant dose and schedule
• IV Ig or anti-D Ig within 2 weeks before the screening visit
• Rituximab (for any indication) within 14 weeks before the screening visit or anticipated use during the time of the proposed study
• Received hematopoietic growth factors, including IL-11 (oprelvekin) within 4 weeks before the screening visit
• Past or present participation in any study evaluating PEG-rHuMGDF, recombinant human thrombopoietin (rHuTPO), AMG 531 or related platelet product
• Received any aklylating agents within 8 weeks before the screening visit or anticipated use during the time of the proposed study
• Less than 4 weeks since receipt of any therapeutic drug or device that is not FDA approved for any indication before the screening period - Less than 8 weeks since major surgery

Sponsors & Collaborators

• Amgen: lead

Related Publications (6)

• 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.

  PMID: 28411254 BACKGROUND

• Kuter DJ, Newland A, Chong BH, Rodeghiero F, Romero MT, Pabinger I, Chen Y, Wang K, Mehta B, Eisen M. Romiplostim in adult patients with newly diagnosed or persistent immune thrombocytopenia (ITP) for up to 1 year and in those with chronic ITP for more than 1 year: a subgroup analysis of integrated data from completed romiplostim studies. Br J Haematol. 2019 May;185(3):503-513. doi: 10.1111/bjh.15803. Epub 2019 Feb 21.

  PMID: 30793285 BACKGROUND

• Kuter DJ, Arnold DM, Rodeghiero F, Janssens A, Selleslag D, Bird R, Newland A, Mayer J, Wang K, Olie R. Safety and efficacy of self-administered romiplostim in patients with immune thrombocytopenia: Results of an integrated database of five clinical trials. Am J Hematol. 2020 Jun;95(6):643-651. doi: 10.1002/ajh.25776. Epub 2020 Mar 21.

  PMID: 32129511 BACKGROUND

• Weitz I, Sanz MA, Henry D, Schipperus M, Godeau B, Northridge K, Gleeson M, Danese M, Deuson R. A novel approach to the evaluation of bleeding-related episodes in patients with chronic immune thrombocytopenia. Curr Med Res Opin. 2012 May;28(5):789-96. doi: 10.1185/03007995.2012.684046. Epub 2012 Apr 25.

  PMID: 22494019 DERIVED

• Kuter DJ, Mufti GJ, Bain BJ, Hasserjian RP, Davis W, Rutstein M. Evaluation of bone marrow reticulin formation in chronic immune thrombocytopenia patients treated with romiplostim. Blood. 2009 Oct 29;114(18):3748-56. doi: 10.1182/blood-2009-05-224766. Epub 2009 Aug 11.

  PMID: 19671919 DERIVED

• Kuter DJ, Bussel JB, Lyons RM, Pullarkat V, Gernsheimer TB, Senecal FM, Aledort LM, George JN, Kessler CM, Sanz MA, Liebman HA, Slovick FT, de Wolf JT, Bourgeois E, Guthrie TH Jr, Newland A, Wasser JS, Hamburg SI, Grande C, Lefrere F, Lichtin AE, Tarantino MD, Terebelo HR, Viallard JF, Cuevas FJ, Go RS, Henry DH, Redner RL, Rice L, Schipperus MR, Guo DM, Nichol JL. Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. Lancet. 2008 Feb 2;371(9610):395-403. doi: 10.1016/S0140-6736(08)60203-2.

  PMID: 18242413 DERIVED

Related Links

• AmgenTrials clinical trials website
• Notice regarding posted summaries of trial results
• To access clinical trial results information click on this link
• FDA-approved Drug Labeling

MeSH Terms

Conditions

Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic; Purpura, Thrombocytopenic

Interventions

romiplostim

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• MD

  Amgen

  STUDY DIRECTOR

Study Design

• Study Type: interventional
• Phase: phase 3
• Allocation: RANDOMIZED
• Masking: DOUBLE
• Who Masked: PARTICIPANT, INVESTIGATOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: January 27, 2005
• First Posted: January 28, 2005
• Study Start: April 7, 2005
• Primary Completion: December 1, 2006
• Study Completion: April 1, 2007
• Last Updated: November 8, 2022

Record last verified: 2022-11