Eltrombopag in Second Line Adult Primary Immune Thrombosytopenia

• Register: ClinicalTrials.gov
• Last refreshed on: 24 August 2021
• Main ID: NCT02402998
• Date of registration: 22/03/2015
• Prospective Registration: Yes
• Primary sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto
• Public title: Eltrombopag in Second Line Adult Primary Immune Thrombosytopenia
• Scientific 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
• Date of first enrolment: February 24, 2016
• Target sample size: 55
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT02402998
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Italy

Contacts

• Name: Francesco Zaja, Pr.
• Affiliation: Clinica Ematologica, DISM, Azienda Ospedaliera Universitaria S. M. Misericordia

Key inclusion & exclusion 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 aß2-Glycoprotein I (aß2GPI) 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.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Primacy Immune Thrombocytopenia

Intervention(s)

Drug: Eltrombopag

Primary Outcome(s)

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.]

Secondary Outcome(s)

Number of bleeding events. [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 months of response from eltrombopag discontinuation to the last follow-up. [Time Frame: After one year from study treatment.]

Secondary ID(s)

2015-001327-23

ITP0815

Source(s) of Monetary Support

Please refer to primary and secondary sponsors