Primary:To demonstrate the safety of AMG 531 in severely refractory thrombocytopenic subjects with ITPSecondary:To monitor hematological responses to AMG 531To provide open-label use of AMG 531, and to investigate its utility in severely refractory…
ID
Source
Brief title
Condition
- Platelet disorders
Synonym
Research involving
Sponsors and support
Intervention
Outcome measures
Primary outcome
The incidence of adverse events, including clinically significant changes in
laboratory values and incidence of antibody formation.
Secondary outcome
The incidence of platelet response. A platelet response is defined as a
doubling of baseline platelet count and a platelet count equal or bigger then
50 x 10-9 / L.
Background summary
The 20040209 study has been conducted as an individual patient protocol in the
US since 2004. Currently, we have 38 patients enrolled in the study at 23 sites.
With increasing requests from health care professionals for AMG 531 for the
treatment of severely refractory ITP subjects, the protocol has been amended so
that it is scalable for other regions (Europe and Australia). The protocol has
been designed to provide open label AMG 531 to severely thrombocytopenic
patients with ITP who are refractory to other treatments, and who do not
qualify for ongoing AMG 531 ITP studies. The protocol will therefore increase
our understanding of AMG 531 in ITP subjects with severely refractory
thrombocytopenia.
Study objective
Primary:
To demonstrate the safety of AMG 531 in severely refractory thrombocytopenic
subjects with ITP
Secondary:
To monitor hematological responses to AMG 531
To provide open-label use of AMG 531, and to investigate its utility in
severely refractory thrombocytopenic subjects with ITP who do not qualify for
any ongoing ITP studies
Study design
Study design:
This protocol will provide open label AMG 531 to severely thrombocytopenic
subjects with ITP who are refractory to other treatments, and who do not
qualify for ongoing AMG 531 ITP studies. This protocol will therefore expand
our understanding of AMG 531 in ITP subjects with severely refractory
thrombocytopenia. All subjects will enter this study at a starting dose of 3
micrograms per kilogram. AMG 531 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 (see the dose adjustment table in
Section 6.2). Rescue therapy is allowed at any time during the study.
Reductions in concurrent IT therapies may occur at any time when platelet
counts are greater than 50 x 109/L. An end of study visit must be completed for
all subjects that complete or discontinue the study early.
Intervention
AMG 531 will be administered at a starting dose of 3 microgram per kilogram.
Dose adjustment will be governed by the following rules:
Platelet counts (x10-9/ L) less then 200: Dose may be adjusted (increase or
decreased) weekly at the investigator*s decision
Bigger or equal 200 -450: Maintain current dose, or decrease weekly at the
investigators decision
Bigger than 450: Withhold the next scheduled dose. Dose will be reduced on the
next scheduled dosing day that the l\platelet count is less then 200 x 10-9 /
liter
Study burden and risks
Not applicable.
Minervum 7061
4800DH Breda
NL
Minervum 7061
4800DH Breda
NL
Listed location countries
Age
Inclusion criteria
Inclusion Criteria:
* Subject is * 18 years of age
* Subject has a diagnosis of immune (idiopathic)
thrombocytopenic purpura per the American
Society of Hematology guidelines (see Appendix B)
* Subject had a bone marrow biopsy and aspirate
consistent with ITP conducted within 2 years of
screening
* Subject*s platelet count is * 20 x109/L or the subject
is experiencing bleeding that is uncontrolled with
conventional therapies
* Subject has failed at least 3 conventional therapies for
ITP and, in the opinion of the treating physician, is
unlikely to respond to other available therapies
* If a subject has a history of atrial fibrillation, subject
is currently receiving anti-coagulation medication
* Subject is willing and able to provide written informed consent
Exclusion criteria
Exclusion Criteria:
* Subject has a history of arterial thrombosis
(eg, cerebrovascular accident, transient ischemic
attack, myocardial infarction)
* Subject has a history of venous thrombosis (eg, deep
vein thrombosis, pulmonary embolism)
* Subject has a history of human immunodeficiency
virus, hepatitis B virus, hepatitis C virus, or any
other systemic infectious disease known to cause
severe thrombocytopenia
* Subject has a history of disseminated intravascular
coagulation or underlying hypercoaguable state
* Subject has a history of any of the following
autoimmune disorders: systemic lupus
erythematosis, Evans Syndrome, autoimmune
neutropenia, lupus anticoagulant or
antiphospholipid antibody syndrome, or active
vasculitis
* Subject has a history of microangiopathic hemolytic
anemia (ie, hemolytic uremic syndrome, thrombotic
thrombocytopenic purpura)
* Subject has active lymphoproliferative or
immunoproliferative (monoclonal gammopathy of
undeterimined significance, multiple myeloma)
disorder or leukemia
* Subject has a history of a myeloproliferative
disorder (eg, myelofibrosis, chronic myelogenous
leukemia)
* Subject has myelodysplastic syndrome
* Subject with a history of exposure to mutagenic
chemotherapy has either dysplastic cytological
findings or abnormal cytogenetics on bone marrow
study
* Subject has a history of paroxysmal nocturnal
hemoglobinuria
* Subject has participated in any study evaluating PEGrHuMGDF,
recombinant human thrombopoietin
(rHuTPO), or related platelet product
* Subject has a known hypersensitivity to any
recombinant E coli-derived product
* Subject has received any therapeutic drug or device
that is not approved by the local regulatory health
agency for any indication within 4 weeks of screening
* Subject is of reproductive potential and is not using
adequate contraceptive precautions, in the judgment of
the investigator
* Subject is pregnant or breast feeding
* Investigator has concerns regarding the subject*s
ability to comply with the protocol procedures
Design
Recruitment
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
metc-ldd@lumc.nl
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 |
|---|---|
| EudraCT | EUCTR2007-000638-37-NL |
| CCMO | NL20473.098.07 |
| Other | nog niet bekend |