Study about the effect of a drug (Eltrombopag)which is administered in patients with thrombocytopenia primary Immune, a relative decrease of platelets in blood (small blood components that help the clotting process by sticking to the lining of blood vessels).

Phase 1

Conditions: Primary Immune Thrombocytopenia
MedDRA version: 18.0Level: PTClassification code 10043554Term: ThrombocytopeniaSystem Organ Class: 10005329 - Blood and lymphatic system disorders
Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]

Registration Number: EUCTR2015-001327-23-IT

Lead Sponsor: Fondazione G.I.M.EM.A. Gruppo Italiano Malattie Ematologiche dell'Adulto

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: All

Target Recruitment: 52

Inclusion Criteria

1.Diagnosis of primary ITP;
2.Age = 18 years;
3.Patients are in the new diagnosis or persistent phase of disease (i.e. within month 12 from diagnosis);
4.Patients not responsive or in relapse after a full course of steroid therapy (prednisone 1 mg/kg/d for at least 28 days or 3 cycles of dexamethasone 40 mg/day for consecutive 4 days, according to the GIMEMA ITP0207 trial) ± IVIG;
5.Patients have a platelet count < 10 x 109/L documented in a single blood cell count;
OR
Patients have bleeding symptoms and a platelet count > 10 < 30 x 109/L documented in a single blood cell count; OR
Patients have no bleeding symptoms and a platelet count >10 < 30 x 109/L in at least 2 blood cell counts at 3 days interval in the week preceding the enrolment with the last count at the day of enrolment;
OR
Patients have corticosteroids or IVIG dependence (the need for ongoing or repeated doses administration of corticosteroids or IVIG to maintain a platelet count = 30 x 109/L and/or to avoid bleeding;
6.Written informed consent obtained from the subject;
7.Men with a female partner of childbearing potential must have either had a prior vasectomy or agree to use effective contraception from time of enrolment randomization until 6 months after the last dose of study treatment;
8.Female subjects of non-childbearing potential may be enrolled in the study; for this study population, non-childbearing potential is defined as current tubal ligation, hysterectomy, ovariectomy or post-menopause;
OR
Female subjects of childbearing potential may be enrolled in the study, if the subject has practiced adequate contraception for 30 days prior to start of Eltrombopag, has a negative pregnancy test within 14 days of first dose of Eltrombopag, and has agreed to continue adequate contraception during the entire treatment period and for 6 months after completion of the treatment.;

Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 32
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 20

Exclusion Criteria

1.Diagnosis of secondary ITP. As far as patients with immune thrombocytopenia and antiphospholipid antibodies positivity, individuals without a previous thromboembolic event are excluded only if lupus anticoagulant (LAC) is associated with the presence of anticardiolipin (aCL) and aß2-Glycoprotein I (aß2GPI) antibodies (triple positivity according to Pengo et al. Blood 2011) (10);
2.Previous treatment with other anti-ITP second line therapies (i.e. Rituximab, Azathioprine, Cyclosporin-A or other); only patients with a previous full course of steroid (see inclusion criteria for definition) ± IVIG are admitted to the study;
3.Previous treatment with any TPO-R agonists;
4.Patients have life threatening bleeding complications;
5.Patients had deep venous thrombosis (DVT) or arterial thrombosis in the 3 months preceding the enrolment;
6.Patients are HIV, HCV, HBsAg positive;
7.Patients with hepatic impairment (i.e. mild, moderate or severe hepatic impairment (Child-Pugh score > 6);
8.Patients have a well established liver disease that represents a contraindication for the use of Eltrombopag;
9.Patients are unable to respect the 4-hour interval between Eltrombopag and other medications (e.g. antacids), calcium-rich foods (e.g. dairy products and calcium fortified juices), or supplements containing polyvalent cations such as iron, calcium, aluminium, magnesium, selenium, and zinc;
10.Patients are unable to stop medications that are known to cause a drug-drug interaction with Eltrombopag;
11.Subjects meeting any of the following criteria must not be enrolled in an Eltrombopag study:
a)Lactating female.
b)History of another malignancy. Exception: subjects who have been disease-free for 5 years, or subjects with a history of completely resected non-melanoma skin cancer or successfully treated in situ carcinoma are eligible.
c)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 study procedures.
d)Hormone replacement therapy. Subjects must discontinue hormone replacement therapy prior to study enrolment due to the potential for inhibition of Cytochrome P450 (CYP) enzymes that metabolize estrogens and progestins.
e)Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
f)Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to Eltrombopag or excipients that contraindicate their participation.