To provide continuing treatment with eltrombopag for subjects who are currently participating in a GSK sponsored investigational study of eltrombopag (parent study) and to collect long term safety data.
ID
Source
Brief title
Condition
- Haematological disorders NEC
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
Adverse effects.
Secondary outcome
None.
Background summary
Eltrombopag is an orally bioavailable, small molecule thrombopoietin receptor
agonist that is being studied in patients with medical disorders associated
with thrombocytopenia. It has been approved for the treatment of chronic
idiopathic thrombocytopenic purpura (ITP) in adults (after splenectomy or if
refractory for other treatments). ITP is a disease a low number of platelets
(<150 Gi/L). In myelodysplastic syndrome (MDS) and acute myeloid leukemia
(AML), megakaryopoiesis can be impaired in both a quantitative (lack of
megakaryocytes), and qualitative way (increased apoptosis in megakaryocytes
from patients with MDS). Interestingly, increased apoptosis of megakaryocytes
has also been observed in ITP. Based on the pathophysiology of thrombocytopenia
in MDS and AML and based on eltrombopag*s known mechanism of action, it is very
likely that eltrombopag will be effective in patients with MDS and AML.
This study is a follow-up study for patients with ITP, MDS and AML who have
completed one of the core studies with eltrombopag in ITP, MDS en AML. The main
aim is to provide the option to these patients to continue the use of
eltrombopag. In Holland only MDS and AML patients from the ASPIRE study
(TRC114968) will participate in this follow-up study.
Study objective
To provide continuing treatment with eltrombopag for subjects who are currently
participating in a GSK sponsored investigational study of eltrombopag (parent
study) and to collect long term safety data.
Study design
Phase IV uncontrolled open-label study.
All patients will be treated with eltrombopag. The starting dose will be the
final dose used in the core study. Thereafter dose adjustments may be performed.
The study may remain open until commercial supply of eltrombopag is available
to patients with the same disease as you, or another way for you to receive
eltrombopag becomes available or until GSK stops the development of
eltrombopag.
Standard of care therapies will be continued.
Approx. 100 patients.
Intervention
Treatment with eltrombopag.
Study burden and risks
Risk: Adverse effects of study medication.
Burden: Treatment every 1-2 weeks or 1-2 months depending on platelet counts.
Each visit: physical examination, blood draw (10-15 ml).
At study start: ECG and pregnancy test (if relevant).
Huis ter Heideweg 62
Zeist 3705 LZ
NL
Huis ter Heideweg 62
Zeist 3705 LZ
NL
Listed location countries
Age
Inclusion criteria
* The subject is participating in a GSK sponsored investigational study of eltrombopag (parent study) within the past 28 days and is receiving clinical benefit without unacceptable toxicity as determined by the investigator.
* Female participants and female partners of male participants of child-bearing potential: subject must not be sexually active or is practicing an acceptable method of contraception.
Exclusion criteria
* Permanent discontinuation of eltrombopag in the parent study based upon the study treatment discontinuation or study withdrawal criteria from the parent study.
Subjects who permanently discontinued treatment because they completed all study related treatments remain eligible.
* Pregnancy and breast feeding. Inadequate contraception, if relevant.
Design
Recruitment
Medical products/devices used
Followed up by the following (possibly more current) registration
No registrations found.
Other (possibly less up-to-date) registrations in this register
No registrations found.
In other registers
| Register | ID |
|---|---|
| Other | clinicaltrials.gov; registratienummer n.n.b. |
| EudraCT | EUCTR2013-001371-20-NL |
| CCMO | NL48025.029.14 |