NPLATE SUMMARY
Romiplostim, a member of the TPO mimetic class, is an Fc-peptide fusion protein (peptibody) that activates intracellular transcriptional pathways leading to increased platelet production via the TPO receptor (also known as cMpl). The peptibody molecule contains two identical single-chain subunits, each consisting of human immunoglobulin IgG1 Fc domain, covalently linked at the C-terminus to a peptide containing two thrombopoietin receptor-binding domains. Romiplostim has no amino acid sequence homology to endogenous TPO. Romiplostim is produced by recombinant DNA technology in Escherichia coli (E coli).Nplate is supplied as a sterile, preservative-free, lyophilized, solid white powder for subcutaneous injection.
Nplate is indicated for the treatment of thrombocytopenia in patients with chronic immune (idiopathic) thrombocytopenic purpura (ITP) who have had an insufficient response to corticosteroids, immunoglobulins or splenectomy. Nplate should be used only in patients with ITP whose degree of thrombocytopenia and clinical condition increases the risk for bleeding. Nplate should not be used in an attempt to normalize platelet counts.
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NEWS HIGHLIGHTS
Published Studies Related to Nplate (Romiplostim Subcutaneous)
Romiplostim for the treatment of chronic immune (idiopathic) thrombocytopenic purpura. [2009.07]
CONCLUSIONS: The FDA approved romiplostim for use among certain patients with chronic ITP. This approval included a Risk Evaluation and Mitigation Strategy to ensure that the benefits of the drug outweigh its risks.
Improved quality of life for romiplostim-treated patients with chronic immune thrombocytopenic purpura: results from two randomized, placebo-controlled trials. [2009.02]
Health-related quality of life (HRQoL) is a major concern for adults with chronic immune thrombocytopenic purpura (ITP) due to the symptoms associated with the disease and its treatment. This study utilized the ITP-patient assessment questionnaire (ITP-PAQ), a specialized HRQoL questionnaire for ITP, to investigate the humanistic burden of ITP and the impact of romiplostim therapy on HRQoL in two, placebo-controlled, phase 3 clinical trials of splenectomized and non-splenectomized patients...
Efficacy of romiplostim in patients with chronic immune thrombocytopenic purpura: a double-blind randomised controlled trial. [2008.02.02]
BACKGROUND: Chronic immune thrombocytopenic purpura (ITP) is characterised by accelerated platelet destruction and decreased platelet production. Short-term administration of the thrombopoiesis-stimulating protein, romiplostim, has been shown to increase platelet counts in most patients with chronic ITP. We assessed the long-term administration of romiplostim in splenectomised and non-splenectomised patients with ITP... INTERPRETATION: Romiplostim was well tolerated, and increased and maintained platelet counts in splenectomised and non-splenectomised patients with ITP. Many patients were able to reduce or discontinue other ITP medications. Stimulation of platelet production by romiplostim may provide a new therapeutic option for patients with ITP.
Romiplostim: a novel thrombopoiesis-stimulating agent. [2009.05.01]
CONCLUSION: Romiplostim is a novel thrombopoietic-stimulating agent for use in patients with chronic ITP who have not responded to other therapies.
Romiplostim management of immune thrombocytopenic purpura. [2009.05]
CONCLUSIONS: Romiplostim is effective for the management of ITP in adults refractory to other therapies, including splenectomy.
Clinical Trials Related to Nplate (Romiplostim Subcutaneous)
A Prospective, Study Evaluating Changes in Bone Marrow Morphology in Adult Subjects Receiving Romiplostim for the Treatment of Thrombocytopenia Associated With Immune (Idiopathic) Thrombocytopenia Purpura (ITP) [Recruiting]
This is a prospective, phase IV, multi-center, open label, study evaluating the changes in
bone marrow reticulin and collagen in adult subjects receiving romiplostim for the treatment
of thrombocytopenia associated with ITP.
The purpose of this study is to evaluate changes in bone marrow morphology (structure) after
long-term exposure to romiplostim.
Subjects, diagnosed with ITP according to the ASH Guidelines, will be sequentially enrolled
into the following groups:
- Bone marrow biopsy at baseline and Year 1
- Bone marrow biopsy at baseline and Year 2
- Bone marrow biopsy at baseline and Year 3
All subjects will receive romiplostim for 3 years, unless withdrawn from the study early.
All subjects will return for 1 visit post treatment for End of Study (EOS) procedures.
An Open Label Study of Romiplostim in Adult Thrombocytopenic Subjects With ITP [Recruiting]
This protocol will provide open label romiplostim to adult thrombocytopenic subjects.
Romiplostim will be administered by subcutaneous injection once per week. Dose adjustment
will be based on platelet counts, and will be allowed throughout the duration of the study.
Rescue therapies are allowed at any time during the study. Reductions in concurrent ITP
therapies may occur at any time when platelet counts are > 50,000.
Romiplostim Treatment of Thrombocytopenia in Subjects With Low or Intermediate-1 Risk Myelodysplastic Syndrome (MDS) [Recruiting]
This is a Phase 2, multicenter, randomized, double blind, placebo controlled study designed
to assess the efficacy and safety of romiplostim (formerly, AMG 531) treatment in
thrombocytopenic MDS subjects. The study is composed of a 26-week placebo controlled test
treatment period (romiplostim versus Placebo), a 2 to 4 week interim wash-out period, a
24-week placebo controlled extended treatment period, and a 4-week follow-up period. During
the interim wash-out period, a bone marrow biopsy will be performed in the absence of growth
factor to assess changes in the marrow. In the extended treatment period, safety assessments
will continue and subjects will be allowed to receive any standard of care treatments for
MDS.
Subjects will be followed for survival for an additional 60 months following the End of
Study (EOS) visit.
Evaluating the Safety of Long Term Dosing of Romiplostim (Formerly AMG 531) in Thrombocytopenic Subjects With Myelodysplastic Syndromes (MDS) [Recruiting]
This is an open label extension study of romiplostim (formerly AMG 531) for treatment of
thrombocytopenia (platelet count ≤ 50 x 109/L) in low or intermediate-1risk MDS subjects not
receiving MDS disease-modifying treatment(s). The study is designed to assess the long-term
safety of treatment with romiplostim, as measured by incidence of overall adverse events,
the incidence of bleeding events, the utilization of platelet transfusions, and the duration
of platelet response.
AMG 531 in Patients With Advanced Malignancy Receiving Treatment With Carboplatin [Recruiting]
Primary Objectives:
1. To determine the clinical safety and tolerability of AMG 531 administered following
chemotherapy in patients with advanced malignancy
2. To determine an optimal biologic dose (OBD) of AMG 531 administered in patients
receiving chemotherapy known to cause severe thrombocytopenia
3. To evaluate the effects of AMG 531 on the degree and duration of thrombocytopenia and
platelet recovery following chemotherapy
Secondary Objectives:
1. To evaluate limited pharmacokinetics of AMG 531 administered by S. C. route
post-chemotherapy
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