Eltrombopag in Second Line Adult Primary Immune Thrombosytopenia
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ClinicalTrials.gov Identifier: NCT02402998
Recruitment Status : Unknown
Verified August 2018 by Gruppo Italiano Malattie EMatologiche dell'Adulto. Recruitment status was: Active, not recruiting
A clinical study to evaluate the efficacy of a drug called eltrombopag in adult patients affected of primary immune thrombocytopenia as first treatment to address the disease.
Condition or disease
Primacy Immune Thrombocytopenia
This is a prospective, multicenter phase II trial designed to evaluate the activity of Eltrombopag second line treatment in adult patients with primary Immune Thrombocytopenia (ITP) not responsive or in relapse after a full first line steroids treatment (prednisone or dexamethasone) ± Intravenous Immune Globulin (IVIG).
Eltrombopag as Second Line Therapy in Adult Patients With Primary Immune Thrombocytopenia (ESTIT Study) in an Attempt to Achieve Long-term Remission: a Single Arm Multicenter Phase II Clinical and Biological Study
Actual Study Start Date :
Actual Primary Completion Date :
May 22, 2018
Estimated Study Completion Date :
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Number of subjects who achieve long-term response (6 months sustained response) and able to taper down the dose. [ Time Frame: After one year from study treatment. ]
A patient will achieve this endpoint if at the end of the period of treatment he/she reaches to discontinue Eltrombopag and if, after discontinuation, has absence of bleeding and maintains a platelet count ≥ 30 x 109/L and at least a 2-fold increase from the baseline count in blood cell count performed in the 6 months of the period observation, during which no steroid, Eltrombopag, other anti TPO-R agonists and other anti-ITP medications are administered.
Secondary Outcome Measures :
Number of months of response from eltrombopag discontinuation to the last follow-up. [ Time Frame: After one year from study treatment. ]
Number of months of complete response from eltrombopag discontinuation to the last follow-up. [ Time Frame: After one year from study treatment. ]
Number of bleeding events. [ Time Frame: After one year from study treatment. ]
Other Outcome Measures:
Modification of immunological parameters during treatment and their relationship with clinical outcomes. (Composite outcome) [ Time Frame: At baseline, week 24, 36 and 52 ]
This study will be performed and completed in all patients enrolled into the study (responders and no-responders). The following biological markers will be evaluated from peripheral blood at baseline, week 24, 36 and 52:
Cytokine serum levels;
Cytokine production by T cells ex vivo;
Cytokine production by myeloid dendritic cells.
Analysis of the relationship between baseline TPO serum level and response to therapy. [ Time Frame: After six months from treatment start. ]
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Layout table for eligibility information
Ages Eligible for Study:
18 Years and older (Adult, Older Adult)
Sexes Eligible for Study:
Accepts Healthy Volunteers:
Diagnosis of primary ITP;
Age ≥ 18 years;
Patients are in the new diagnosis or persistent phase of disease (i.e. within month 12 from diagnosis);
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;
Patients have a platelet count < 10 x 109/L documented in a single blood cell count;
Patients have bleeding symptoms and a platelet count > 10 < 30 x 109/L documented in a single blood cell count;
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 enrollment with the last count at the day of enrollment;
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;
Written informed consent obtained from the subject;
Men with a female partner of childbearing potential must have either had a prior vasectomy or agree to use effective contraception from time of enrollment until 6 months after the last dose of study treatment;
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.
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 aβ2-Glycoprotein I (aβ2GPI) antibodies;
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;
Previous treatment with any TPO-R agonists;
Patients have life threatening bleeding complications;
Patients had deep venous thrombosis (DVT) or arterial thrombosis in the 3 months preceding the enrollment;
Patients are HIV, HCV, HBsAg positive;
Patients with hepatic impairment (i.e. mild, moderate or severe hepatic impairment (Child-Pugh score > 6);
Patients have a well established liver disease that represents a contraindication for the use of Eltrombopag;
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;
Patients are unable to stop medications that are known to cause a drug-drug interaction with Eltrombopag;
Subjects meeting any of the following criteria must not be enrolled in an Eltrombopag study:
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.
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.
Hormone replacement therapy. Subjects must discontinue hormone replacement therapy prior to study enrollment due to the potential for inhibition of Cytochrome P450 (CYP) enzymes that metabolize estrogens and progestins.
Administration of an investigational drug within 30 days or 5 half-lives, whichever is longer, preceding the first dose of study treatment.
Have a known immediate or delayed hypersensitivity reaction or idiosyncrasy to drugs chemically related to Eltrombopag or excipients that contraindicate their participation.