Eltrombopag in Second Line Adult Primary Immune Thrombosytopenia

NCT02402998 | Completed Phase 2

• 2 other identifiers: ITP08152015-001327-23
• Study Type: interventional
• Target: 55
• Locations: 1 country
• Sites: 14

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.

Conditions

Primacy Immune Thrombocytopenia

Keywords

Primacy immune thrombocytopenia; Adults; Eltrombopag; Long-term remission

Outcome Measures

Primary Outcomes (1)

• Number of subjects who achieve long-term response (6 months sustained response) and able to taper down the dose.

  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.

  After one year from study treatment.

Secondary Outcomes (3)

• Number of months of response from eltrombopag discontinuation to the last follow-up.

  After one year from study treatment.

• Number of months of complete response from eltrombopag discontinuation to the last follow-up.

  After one year from study treatment.

• Number of bleeding events.

  After one year from study treatment.

Other Outcomes (2)

• Modification of immunological parameters during treatment and their relationship with clinical outcomes. (Composite outcome)

  At baseline, week 24, 36 and 52

• Analysis of the relationship between baseline TPO serum level and response to therapy.

  After six months from treatment start.

Study Arms (1)

Eltrombopag

EXPERIMENTAL

Eltrombopag 50 mg/daily.

Drug: Eltrombopag

Interventions

Eltrombopag DRUG

Eltrombopag 50 mg/daily.

Eltrombopag

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• 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.

You may not qualify if:

• 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 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.

Sponsors & Collaborators

• Gruppo Italiano Malattie EMatologiche dell'Adulto: lead

Study Sites (14)

Azienda Ospedaliera Di Bologna Policlinico S. Orsola - Malpighi

Bologna, 40138, Italy

Location

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

Milan, Italy

Location

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

Milan, Italy

Location

Azienda Ospedaliera "S.Gerardo"

Monza, Italy

Location

Azienda Ospedaliera di Rilievo Nazionale "A. Cardarelli"

Napoli, Italy

Location

S.C.D.U. Ematologia - DIMECS e Dipartimento Oncologico - Università del Piemonte Orientale Amedeo Avogadro

Novara, Italy

Location

Azienda Ospedale S. Luigi at University of Torino

Orbassano, 10043, Italy

Location

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

Padua, Italy

Location

Ospedale "Infermi"

Rimini, Italy

Location

Università degli Studi "Sapienza" - Dip Biotecnologie Cellulari ed Ematologia - Divisione di Ematologia

Roma, Italy

Location

Policlinico A. Gemelli - Universita Cattolica del Sacro Cuore

Rome, 00168, Italy

Location

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

Udine, Italy

Location

Università degli Studi di Verona - A. O. - Istituti Ospitalieri di Verona- Div. di Ematologia - Policlinico G.B. Rossi

Verona, Italy

Location

Ospedale San Bortolo

Vicenza, 36100, Italy

Location

MeSH Terms

Interventions

eltrombopag

Study Officials

• Francesco Zaja, Pr.

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

  STUDY CHAIR

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: March 22, 2015
• First Posted: March 31, 2015
• Study Start: February 24, 2016
• Primary Completion: May 22, 2018
• Study Completion: August 28, 2019
• Last Updated: August 16, 2021

Record last verified: 2021-08

Locations

IT (14)