Phase 4
N=169
Study on Bone Marrow Morphology in Adults Receiving Romiplostim for Treatment of Thrombocytopenia Associated With Immune Thrombocytopenia Purpura (ITP)
Thrombocytopenia · Idiopathic Thrombocytopenic Purpura
Bottom Line
View on ClinicalTrials.gov: NCT00907478 ↗Enrolled (actual)
169
Serious AEs
33.1%
Results posted
Jan 2015
Primary outcome: Primary: Percentage of Participants With Collagen Fibrosis — 0.0; 0.0; 3.4 percentage of participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- romiplostim (Biological)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- Amgen
- Primary completion
- Jan 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Percentage of Participants With Collagen Fibrosis |
0.0; 0.0; 3.4 | — |
| SECONDARY Number of Participants With Collagen Fibrosis 12 Weeks After Romiplostim Discontinuation in Participants Who Developed Collagen Fibrosis at Years 1, 2, or 3 |
— | — |
| SECONDARY Percentage of Participants Who Developed an Increased Modified Bauermeister Grade |
0.0; 5.1; 12.1 | — |
| SECONDARY Percentage of Participants With Clinically Relevant Changes in Total Cardiac Output Corrected (QTc) Intervals |
0.0; 0.0; 0.0 | — |
| SECONDARY Number of Participants With Improvement of Reticulin to a Grade of ≤ 2 for Participants Who Developed Grade 3 Reticulin |
3 | — |
| SECONDARY Percentage of Participants With CTCAE Grade ≥ 2 Shift in Anemia or Neutropenia |
6.0; 4.0; 8.7; 8.0; 6.0; 13.0 | — |
| SECONDARY Number of Participants With Adverse Events (AEs) |
46; 45; 67; 39; 39; 59 | — |
| SECONDARY Number of Participants Who Developed Antibodies or Neutralizing Antibodies to Romiplostim or to Endogenous Thrombopoietin |
7; 4; 3; 4; 1; 3 | — |
Summary
The purpose of this study is to evaluate changes in bone marrow morphology (structure) after long-term exposure to romiplostim.
Eligibility Criteria
Inclusion Criteria
- Diagnosis of ITP according to American Society of Hematology (ASH) guidelines
- Subject must have had a bone marrow biopsy within one year prior to planned first dose of romiplostim (with available bone marrow tissue block or unstained histological slides to send to a central laboratory for interpretation) or must consent to a pre-treatment bone marrow biopsy within 3 weeks prior to planned first dose of romiplostim. Central laboratory interpretation is required prior to first dose of romiplostim
- Subject must agree to a scheduled bone marrow biopsy at Year 1, Year 2, or Year 3 following romiplostim treatment and any unscheduled biopsies if clinically indicated
- Subject ≥18 years of age
- Baseline bone marrow reticulin grade of 0, 1, 2, or 3 according to the modified Bauermeister grading scheme as assessed by central laboratory interpretation
- Platelet count < 50 x 10^9/L
- Must have received at least 1 prior ITP therapy (examples of ITP therapy include corticosteroids, intravenous immunoglobulin [IVIG], splenectomy)
- Subject (or legally-acceptable representative) is willing and able to provide written informed consent
Exclusion Criteria
- Baseline bone marrow biopsy positive for collagen fibrosis
- Any known history of or currently active bone marrow stem cell disorder, hematological malignancy, myeloproliferative disorder, myelodysplastic syndrome
- Any current active malignancy
- Any prior exposure to cytostatic chemotherapy or radiotherapy for malignancy
- Subject has undergone pacemaker placement, cardiac ablation of arrhythmia, and/or any current treatment with Vaughan Williams Class IA - IC and Class III agents (Vaughan Williams, 1970)
- Subject has participated in any study evaluating pegylated recombinant human megakaryocyte growth and development factor (PEG-rHuMGDF), recombinant human thrombopoietin (rHuTPO), or thrombopoietin receptor agonists (ie romiplostim or eltrombopag)
- Subject has a known hypersensitivity to any recombinant E coli-derived product
- Subject is currently enrolled in or has not yet completed (at least 4 weeks since ending) other investigational device or drug trial(s) or subject is receiving other investigational agent(s)
- Other investigational procedures are excluded
- Subject of child-bearing potential is evidently pregnant (eg positive pregnancy test) or is breast feeding
- Subject is not using adequate contraceptive precautions
- Subject has any kind of disorder that compromises the ability of the subject to give written informed consent and does not have a legally acceptable representative and/or is unable to comply with study procedures.
Data sourced from ClinicalTrials.gov (NCT00907478). Outcome figures and adverse-event rates are extracted automatically from the registry's posted results and are provided for clinician reference, not as a substitute for the primary publication.