Phase 4
N=162
A Longitudinal 2-year Bone Marrow Study of Eltrombopag in Previously Treated Adults, With Chronic Immune (Idiopathic) Thrombocytopenic Purpura (ITP)
Purpura, Thrombocytopaenic, Idiopathic
Bottom Line
View on ClinicalTrials.gov: NCT01098487 ↗Enrolled (actual)
162
Serious AEs
25.9%
Results posted
Mar 2015
Primary outcome: Primary: Number of Participants With Bone Marrow (BM) Fibers of MF Grade 0, 1, 2 and 3 on the European Consensus (EC) Scale at Baseline — 150; 9; 0; 0 Participants
Study Design & Population
- Study type
- Interventional
- Phase
- Phase 4
- Interventions
- Eltrombopag olamine (Drug)
- Age
- Adult, Older Adult · 18+ yrs
- Sex
- All
- Sponsor
- GlaxoSmithKline
- Primary completion
- May 2014
Outcome Measures
| Outcome | Result | p-value |
|---|---|---|
| PRIMARY Number of Participants With Bone Marrow (BM) Fibers of MF Grade 0, 1, 2 and 3 on the European Consensus (EC) Scale at Baseline |
150; 9; 0; 0 | — |
| PRIMARY Number of Participants With a Positive or Negative Collagen Level at Baseline |
159; 0 | — |
| PRIMARY Number of Participants With Indicated Grade Change From Baseline in the EC Grading Scale at 1 Year |
82; 33; 2; 2; 3; 2 | — |
| PRIMARY Number of Participants With Indicated Change From Baseline in the EC Grading Scale at 2 Years |
79; 9; 0; 0; 2; 1 | — |
| PRIMARY Number of Participants With a Positive or Negative Collagen Level at 1 Year |
120; 5; 0; 0; 2; 0 | — |
| PRIMARY Number of Participants With a Positive or Negative Collagen Level at 2 Year |
90; 1; 0; 0; 2; 0 | — |
| SECONDARY Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Clinical Chemistry Parameters at Any Time Post-Baseline During the Study |
151; 5; 6; 0; 0; 125 | — |
| SECONDARY Number of Participants With the Indicated Maximum Toxicity Grade for the Indicated Hematology Parameters at Any Time Post-Baseline During the Study |
143; 17; 1; 1; 0; 55 | — |
| SECONDARY Number of Participants With Any Adverse Event (AE) or Serious Adverse Event (SAE) Started On-therapy + 1 Day, >1 to 30 Days Post Therapy and >30 Days Post Therapy |
141; 41; 12; 5; 9; 1 | — |
Summary
A open-label, multi-center 2-year safety study to ascertain the baseline levels of bone marrow fibers in previously treated adults with chronic immune (idiopathic) thrombocytopenic purpura (ITP) and to evaluate the long-term effect of eltrombopag on bone marrow fibers. The study will also describe the long-term safety and tolerability of oral eltrombopag treatment in subjects with chronic ITP.
Eligibility Criteria
Inclusion Criteria
- Subjects must have signed and dated a written informed consent and be able to understand and comply with protocol requirements and instructions.
- Adults (≥18 years) diagnosed with chronic ITP according to the American Society for Hematology/British Committee for Standards in Hematology (ASH/BCSH) guidelines [George, 1996; BCSH, 2003; Provan, 2009]. In addition, a peripheral blood smear should support the diagnosis of ITP with no evidence of other disease causative of thrombocytopenia (e.g., pseudo thrombocytopenia, myelofibrosis). The physical examination should not suggest any disease, which may cause thrombocytopenia other than ITP.
- Subjects must be physically eligible for serial bone marrow biopsies and must have a bone marrow biopsy performed during screening, and be willing to remain on the study for at least 2 years with annual bone marrow biopsies.
- Subjects, who previously received eltrombopag or romiplostim, must have completed treatment with these therapies at least 6 months prior to the screening bone marrow biopsy.
- Subjects must have the following clinical chemistry values:
- ALT and AST 450 msec or > 480 msec for subjects with Bundle Branch Block.
- Female subjects who are nursing or pregnant (positive serum or urine β-human chorionic gonadotrophin (β-hCG) pregnancy test) at screening.
- Subjects treated with an investigational drug (other than a thrombopopoetin-receptor (TPO-R) agonist) within 30 days or five half-lives (whichever is longer) preceding the first dose of eltrombopag in the study. (For romiplostim or eltrombopag, see inclusion criterion #4).
- Subjects treated with any TPO-R agonist other than romiplostim or eltrombopag.
- Subjects with recent history of alcohol/drug abuse as determined by the investigator.
Data sourced from ClinicalTrials.gov (NCT01098487). 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.