| Eligibility |
Inclusion Criteria:
Patients eligible for inclusion in this Treatment Plan have to meet all of the following
criteria:
- Written informed consent must be obtained prior to any screening procedures or treatment
assignment. If consent cannot be expressed in writing, it must be formally documented and
witnessed, ideally via an independent trusted witness. An acceptable alternative (e.g., an
assent form, or consent from a parent or guardian) should be signed for legally incompetent
patients (e.g., children).
- In addition to informed consent, patients eligible for inclusion in this Treatment Plan
may not be enrolled into any other eltrombopag clinical trial and must meet all of the
following criteria (only one of the diseases listed under criterion No 1 is enough):
1. The patient must have any of the following diseases:
- A confirmed diagnosis of chronic idiopathic thrombocytopenic purpura (ITP)
according to the American Society of Haematology/ICR International consensus
report on the investigation and management of primary immune thrombocytopenia
reported by Neunert (2011) and Provan (2009), and the patient must be refractory
to OR has relapsed after at least one prior ITP treatment recommended by the
ASH/BCSH guidelines (corticosteroids or intravenous immunoglobulin (IVIg) or
anti-D). Alternatively, a patient who is medically not eligible to receive
corticosteroids must have had at least one alternative ITP treatment as
recommended by the guidelines. Previous therapies for ITP and dates of
administration must be recorded on the application form.
- Severe aplastic anemia (SAA) patients who does not have an access to a comparable
or satisfactory alternative treatment (i.e., comparable or satisfactory treatment
is not available or does not exist).
- HCV patients requiring treatment with an interferon based therapy and who have no
access to alternative treatments.
2. The patient must have adequate medical condition and adequate organ function (e.g.
renal, hepatic, cardiac function, etc.), as assessed by the treating physician.
3. Patients in childbearing age must be practicing an acceptable method of contraception
from two weeks prior to administration of eltrombopag, throughout administration of
eltrombopag, and 1 month after the last dose of eltrombopag.
4. Women of childbearing potential must have a negative serum pregnancy test within 14
days of first dose of program treatment and agree to use effective contraception,
during the program and for one week following the last dose of program treatment.
5. The patient is able to understand and comply with all the requirements and
instructions for receiving MAP
Exclusion Criteria:
Patients eligible for this Treatment Plan must not meet any of the following criteria:
1. Presence of moderate to severe impaired renal function as indicated by any or all of
the following criteria:
- Creatinine clearance < 45 mL/min as calculated using Cockcroft-Gault formula
- Serum creatinine > 1.5 mg/dL
2. Total bilirubin > 1.5 × upper limit of normal (ULN)
3. Aspartate transaminase (AST) > 3.0 × ULN
4. Alanine transaminase (ALT) > 3.0 × ULN
5. Patient with liver cirrhosis.
6. Infection not adequately controlled with appropriate therapy.
7. Patients who are human immune deficiency virus (HIV), hepatitis C virus or hepatitis B
surface antigen (HBsAg) positive. HCV-RNA negative patients are allowed to be
enrolled.
8. Moribund status or concurrent hepatic, renal, cardiac, neurologic, pulmonary,
infectious, or metabolic disease of such severity that it would preclude the patient's
ability to consent, be compliant with study procedures, tolerate protocol therapy, or
patients with less than 3 months of life expectancy.
9. History of hypersensitivity to eltrombopag or to any of the excipients in eltrombopag
10. Patients with any prior history of arterial or venous thrombosis (stroke, transient
ischemic attack, myocardial infarction, deep vein thrombosis or pulmonary embolism),
AND = two of the following risk factors: hormone replacement therapy, systemic
contraception (containing oestrogen), smoking, diabetes, hypercholesterolemia,
medication for hypertension, cancer, hereditary thrombophilic disorders (e.g., Factor
V Leiden, ATIII deficiency, etc), or any other family history of arterial or venous
thrombosis.
11. Patients with concurrent malignant disease.
12. Patients with SLE, antiphospholipid antibody syndrome or chronic hepatitis B
infection,
13. Any serious and/or unstable pre-existing medical, psychiatric disorder or other
conditions that could interfere with subject's safety, obtaining informed consent or
compliance with the treatment guidelines.
14. Administration of an investigational drug within 30 days or 5 half-lives, whichever is
longer, preceding the first dose of program treatment.
15. History of non-compliance with medical regimens or patients who are considered
potentially unreliable and/or not cooperative, unwilling or unable to comply with the
treatment guidelines.
16. Women of child-bearing potential, defined as all women physiologically capable of
becoming pregnant, (or female partners of male patients) unless they are using highly
effective methods of contraception while taking study treatment and for 3 months after
stopping medication. Highly effective methods of contraception methods include:
- Total abstinence (when this is in line with the preferred and usual lifestyle of
the patient. Periodic abstinence (e.g., calendar, ovulation, symptothermal,
post-ovulation methods) and withdrawal are not acceptable methods of
contraception
- Female sterilization (have had surgical bilateral oophorectomy with or without
hysterectomy), total hysterectomy, or tubal ligation at least six weeks before
taking study treatment. In case of oophorectomy alone, only when the reproductive
status of the woman has been confirmed by follow up hormone level assessment
- Male sterilization (at least 6 months prior to screening). For female patients on
the study, the vasectomized male partner should be the sole partner for that
patient
- Use of oral (estrogen and progesterone), injected or implanted hormonal methods
of contraception or placement of an intrauterine device (IUD) or intrauterine
system (IUS), or other forms of hormonal contraception that have comparable
efficacy (failure rate < 1%), for example, hormone vaginal ring or transdermal
hormone contraception..
- In case of use of oral contraception women should have been stable on the same
pill for a minimum of 3 months before taking study treatment.
- Women are considered post-menopausal and not of child bearing potential if they
have had 12 months of natural (spontaneous) amenorrhea with an appropriate
clinical profile (e.g. age appropriate history of vasomotor symptoms) or have had
surgical bilateral oophorectomy (with or without hysterectomy), total
hysterectomy or tubal ligation at least six weeks in advance. In the case of
oophorectomy alone, only when the reproductive status of the woman has been
confirmed by follow up hormone level assessment is she considered not of child
bearing potential.
17. Sexually active males must be willing to use a condom during intercourse while taking
study treatment and for 3 months after stopping study treatment. A condom is required
for all sexually active male participants to prevent them from fathering a child AND
to prevent delivery of study treatment via seminal fluid to their partner. In
addition, male participants must not donate sperm for the time period specified above.
If local regulations deviate from the contraception methods listed above to prevent
pregnancy, local regulations apply and will be described in the informed consent form
(ICF).
No additional exclusions may be applied by the investigator, in order to ensure that the
study population will be representative of all eligible patients.
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