Trial record 6 of 18 for:    0815

Eltrombopag in Second Line Adult Primary Immune Thrombosytopenia

This study is not yet open for participant recruitment. (see Contacts and Locations)
Verified April 2015 by Gruppo Italiano Malattie EMatologiche dell'Adulto
Sponsor:
Information provided by (Responsible Party):
Gruppo Italiano Malattie EMatologiche dell'Adulto
ClinicalTrials.gov Identifier:
NCT02402998
First received: March 22, 2015
Last updated: April 21, 2015
Last verified: April 2015
  Purpose

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 Intervention Phase
Primacy Immune Thrombocytopenia
Drug: Eltrombopag
Phase 2

Study Type: Interventional
Study Design: Endpoint Classification: Efficacy Study
Intervention Model: Single Group Assignment
Masking: Open Label
Primary Purpose: Treatment
Official Title: 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

Resource links provided by NLM:


Further study details as provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:

Primary Outcome Measures:
  • 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. ] [ Designated as safety issue: No ]
    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. ] [ Designated as safety issue: No ]
  • Number of months of complete response from eltrombopag discontinuation to the last follow-up. [ Time Frame: After one year from study treatment. ] [ Designated as safety issue: No ]
  • Number of bleeding events. [ Time Frame: After one year from study treatment. ] [ Designated as safety issue: Yes ]

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 ] [ Designated as safety issue: No ]

    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:

    1. Cytokine serum levels;
    2. Lymphocyte subpopulations;
    3. Cytokine production by T cells ex vivo;
    4. 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. ] [ Designated as safety issue: No ]

Estimated Enrollment: 52
Study Start Date: June 2015
Estimated Study Completion Date: June 2017
Estimated Primary Completion Date: June 2016 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Experimental: Eltrombopag
Eltrombopag 50 mg/daily.
Drug: Eltrombopag
Eltrombopag 50 mg/daily.

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).

  Eligibility

Ages Eligible for Study:   18 Years and older
Genders Eligible for Study:   Both
Accepts Healthy Volunteers:   No
Criteria

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;
  6. Patients have bleeding symptoms and a platelet count > 10 < 30 x 109/L documented in a single blood cell count;
  7. 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;
  8. 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;
  9. Written informed consent obtained from the subject;
  10. 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;
  11. 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
  12. 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:

  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 aβ2-Glycoprotein I (aβ2GPI) antibodies;
  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 enrollment;
  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:

    • 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.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02402998

Contacts
Contact: Paola Fazi p.fazi@gimema.it
Contact: Enrico Crea e.crea@gimema.it

Locations
Italy
Azienda Ospedaliera Di Bologna Policlinico S. Orsola - Malpighi
Bologna, Italy, 40138
Ospedale Niguarda " Ca Granda" - SC Ematologia Blocco SUD, Ponti Est, Scala E, 4° piano
Milano, Italy
Azienda Ospedaliera "S.Gerardo"
Monza, Italy
Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"
Napoli, Italy
S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro
Novara, Italy
Azienda Ospedale S. Luigi at University of Torino
Orbassano, Italy, 10043
AO di Padova Università degli Studi Padova Dipartimento di Medicina Clinica Medica I - Medicina Interna CLOPD
Padova, Italy
Università degli Studi "Sapienza" - Dip Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia
Roma, Italy
Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore
Rome, Italy, 00168
Clinica Ematologica-Centro Trapianti e Terapie cellulari Azienda Ospedaliero-Universitaria, Udine Not yet recruiting
Udine, Italy
Contact: Francesco Zaja         
Principal Investigator: Francesco Zaja         
Università degli Studi di Verona - A. O. - Istituti Ospitalieri di Verona- Div. di Ematologia - Policlinico G.B. Rossi
Verona, Italy
Ospedale San Bortolo
Vicenza, Italy, 36100
Sponsors and Collaborators
Gruppo Italiano Malattie EMatologiche dell'Adulto
Investigators
Study Chair: Francesco Zaja, Pr. Clinica Ematologica, DISM, Azienda Ospedaliera Universitaria S. M. Misericordia
  More Information

No publications provided

Responsible Party: Gruppo Italiano Malattie EMatologiche dell'Adulto
ClinicalTrials.gov Identifier: NCT02402998     History of Changes
Other Study ID Numbers: ITP0815, 2015-001327-23
Study First Received: March 22, 2015
Last Updated: April 21, 2015
Health Authority: Italy: Ethics Committee

Keywords provided by Gruppo Italiano Malattie EMatologiche dell'Adulto:
Primacy immune thrombocytopenia
Adults
Eltrombopag
Long-term remission

Additional relevant MeSH terms:
Purpura, Thrombocytopenic, Idiopathic
Thrombocytopenia
Autoimmune Diseases
Blood Coagulation Disorders
Blood Platelet Disorders
Hematologic Diseases
Hemorrhage
Hemorrhagic Disorders
Immune System Diseases
Pathologic Processes
Purpura
Purpura, Thrombocytopenic
Signs and Symptoms
Skin Manifestations
Thrombotic Microangiopathies

ClinicalTrials.gov processed this record on April 30, 2015