Eltrombopag in Second Line Adult Primary Immune Thrombosytopenia

NCT ID: NCT02402998

Last Updated: 2021-08-16

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE2
• Total Enrollment: 55 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2016-02-24
• Study Completion Date: 2019-08-28

Brief Summary

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.

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).

Conditions

Primacy Immune Thrombocytopenia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Eltrombopag

Eltrombopag 50 mg/daily.

Group Type: EXPERIMENTAL

Eltrombopag

Intervention Type: DRUG

Eltrombopag 50 mg/daily.

Interventions

Eltrombopag

Eltrombopag 50 mg/daily.

Intervention Type: DRUG

Eligibility 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.

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.

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;

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Gruppo Italiano Malattie EMatologiche dell'Adulto

OTHER

Sponsor Role: lead

Responsible Party

Responsibility Role: SPONSOR

Principal Investigators

Francesco Zaja, Pr.

Role: STUDY_CHAIR

Clinica Ematologica, DISM, Azienda Ospedaliera Universitaria S. M. Misericordia

Locations

Azienda Ospedaliera Di Bologna Policlinico S. Orsola - Malpighi

Bologna, , Italy

Fondazione IRCCS Ca' Granda Ospedale Maggiore Policlinico UOC Oncoematologia- Padiglione Marcora 2° piano

Milan, , Italy

Ospedale Niguarda " Ca Granda" - SC Ematologia Blocco SUD, Ponti Est, Scala E, 4° piano

Milan, , 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

AO di Padova Università degli Studi Padova Dipartimento di Medicina Clinica Medica I - Medicina Interna CLOPD

Padua, , Italy

Ospedale "Infermi"

Rimini, , 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

Clinica Ematologica-Centro Trapianti e Terapie cellulari Azienda Ospedaliero-Universitaria, Udine

Udine, , Italy

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

Countries

Italy

Other Identifiers

2015-001327-23

Identifier Type: EUDRACT_NUMBER

Identifier Source: secondary_id

ITP0815

Identifier Type: -

Identifier Source: org_study_id