- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT02402998
Eltrombopag in Second Line Adult Primary Immune Thrombosytopenia
August 13, 2021 updated by: Gruppo Italiano Malattie EMatologiche dell'Adulto
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
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.
Study Overview
Status
Completed
Conditions
Intervention / Treatment
Detailed Description
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).
Study Type
Interventional
Enrollment (Actual)
55
Phase
- Phase 2
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
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Bologna, Italy, 40138
- Azienda Ospedaliera Di Bologna Policlinico S. Orsola - Malpighi
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Milano, Italy
- Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano
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Milano, Italy
- Ospedale Niguarda " Ca Granda" - SC Ematologia Blocco SUD, Ponti Est, Scala E, 4° piano
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Monza, Italy
- Azienda Ospedaliera "S.Gerardo"
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Napoli, Italy
- Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
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Novara, Italy
- S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
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Orbassano, Italy, 10043
- Azienda Ospedale S. Luigi at University of Torino
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Padova, Italy
- AO di Padova Università degli Studi Padova Dipartimento di Medicina Clinica Medica I - Medicina Interna CLOPD
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Rimini, Italy
- Ospedale "Infermi"
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Roma, Italy
- Università degli Studi "Sapienza" - Dip Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia
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Rome, Italy, 00168
- Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore
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Udine, Italy
- Clinica Ematologica-Centro Trapianti e Terapie cellulari Azienda Ospedaliero-Universitaria, Udine
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Verona, Italy
- Università degli Studi di Verona - A. O. - Istituti Ospitalieri di Verona- Div. di Ematologia - Policlinico G.B. Rossi
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Vicenza, Italy, 36100
- Ospedale San Bortolo
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Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
18 years and older (Adult, Older Adult)
Accepts Healthy Volunteers
No
Genders Eligible for Study
All
Description
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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:
- Lactating female.
- 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.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Treatment
- Allocation: N/A
- Interventional Model: Single Group Assignment
- Masking: None (Open Label)
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
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Experimental: Eltrombopag
Eltrombopag 50 mg/daily.
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Eltrombopag 50 mg/daily.
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What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
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.
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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.
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After one year from study treatment.
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Secondary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Number of months of response from eltrombopag discontinuation to the last follow-up.
Time Frame: After one year from study treatment.
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After one year from study treatment.
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Number of months of complete response from eltrombopag discontinuation to the last follow-up.
Time Frame: After one year from study treatment.
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After one year from study treatment.
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Number of bleeding events.
Time Frame: After one year from study treatment.
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After one year from study treatment.
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Other Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Modification of immunological parameters during treatment and their relationship with clinical outcomes. (Composite outcome)
Time Frame: At baseline, week 24, 36 and 52
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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:
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At baseline, week 24, 36 and 52
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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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After six months from treatment start.
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Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Investigators
- Study Chair: Francesco Zaja, Pr., Clinica Ematologica, DISM, Azienda Ospedaliera Universitaria S. M. Misericordia
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
February 24, 2016
Primary Completion (Actual)
May 22, 2018
Study Completion (Actual)
August 28, 2019
Study Registration Dates
First Submitted
March 22, 2015
First Submitted That Met QC Criteria
March 25, 2015
First Posted (Estimate)
March 31, 2015
Study Record Updates
Last Update Posted (Actual)
August 16, 2021
Last Update Submitted That Met QC Criteria
August 13, 2021
Last Verified
August 1, 2021
More Information
Terms related to this study
Additional Relevant MeSH Terms
- Pathologic Processes
- Immune System Diseases
- Autoimmune Diseases
- Hematologic Diseases
- Hemorrhage
- Hemorrhagic Disorders
- Blood Coagulation Disorders
- Skin Manifestations
- Blood Platelet Disorders
- Thrombotic Microangiopathies
- Purpura, Thrombocytopenic
- Purpura
- Purpura, Thrombocytopenic, Idiopathic
- Thrombocytopenia
Other Study ID Numbers
- ITP0815
- 2015-001327-23 (EudraCT Number)
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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