Eltrombopag in Patients With Delayed Post Transplant Thrombocytopenia.

NCT01791101 | Completed Phase 2

• 2 other identifiers: ITP05112011-004608-39
• Study Type: interventional
• Target: 10
• Locations: 1 country
• Sites: 16

Brief Summary

This is a Phase II multicentre study. Patients will be administered eltrombopag 50 mg/daily. If patients don't achieve response after 2 months of therapy they will stop eltrombopag; if patients will achieve response after 2 months of therapy, they will continue eltrombopag for a maximum period of 24 months; 40 patients are needed. In stage I, 22 patients will be enrolled; if ≤ 4 responses at the first evaluation after 2 months (18%) will be seen, the trial will be stopped; if 5 or more responses will be seen, the accrual will continue. In stage II, 18 more patients will be enrolled. If ≤ 12 (30%) responses will be observed out of 40 patients, it will be concluded that the study drug is not active enough. If ≥ 13 responses will be observed, it will be concluded that eltrombopag is worth of further studies.

Conditions

Thrombocytopenia

Keywords

Thrombocytopenia; Eltrombopag

Outcome Measures

Primary Outcomes (1)

• Number of patients who achieve both a platelet count ≥ 50 x 109/L and have doubled their baseline platelet count.

  Response rate (OR), i.e. the number of patients who achieve both a platelet count ≥ 50 x 109/L and have doubled their baseline platelet count, two months after treatment with eltrombopag. The primary endpoint will be considered for all the treated population according to the intention-to-treat and in the evaluable population, i.e. all patients treated with eltrombopag for at least 3 weeks or who interrupted eltrombopag because of toxic events. A transient increase of platelet count after the administration of iv immunoglobulin given for anti- microbial purpose will not be considered criteria of response.

  Two months after treatment with eltrombopag.

Secondary Outcomes (5)

• Number of adverse events.

  After 4 years from study entry.

• Number of surviving patients.

  At 4 years from study entry.

• Number of bleeding events.

  After 4 years from study entry.

• Characteristics of TPO serum level.

  After 4 years from study entry.

• Patients T-reg activity.

  After 4 years from study entry.

Study Arms (1)

Eltrombopag

EXPERIMENTAL

Eltrombopag 50 mg/daily.

Drug: Eltrombopag

Interventions

Eltrombopag DRUG

Eltrombopag

Eligibility Criteria

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

You may qualify if:

• Patients develop delayed thrombocytopenia, i.e. Platelet count 50 x 109/l 3 months after SCT;
• Patients underwent allogeneic SCT with match related or unrelated donor;
• Patients develop CGVHD-related delayed thrombocytopenia. The definition of cGVHD-related delayed thrombocytopenia is: platelet count 50 x 109/l from month 3 from SCT and presence of any clinical, radiological and/or laboratory finding indicative of cGVHD (all grades); - Patients underwent SCT because of lymphoma (Hodgkin or non-Hodgkin, indolent or aggressive), or multiple myeloma;
• Sexually active males who accept to use a condom during intercourse while taking the drug and for 12 months after stopping treatment as they should not father a child in this period. A condom is required to be used also by vasectomised men (as well as during intercourse with a male partner) in order to prevent delivery of the drug via seminal fluid. Refer also to Appendix C. 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 (please refer to Appendix C);
• 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 prior to start of eltrombopag, and has agreed to continue adequate contraception during the entire treatment period and for 6 months after completion of the treatment.
• Written informed consent obtained from the subject.

You may not qualify if:

• Patients underwent SCT with aplo-identical donor or cord blood;
• Patients underwent SCT for diseases different from lymphoma or multiple myeloma;
• Patients have life threatening bleeding complications;
• Patients have an expected survival \< 1 month;
• Patients have delayed thrombocytopenia related to medical conditions other then cGVHD;
• Patients have progressive non stabilized cGVHD necessitating intensification of immune suppressive treatment in the last 2 weeks;
• Patients need to introduce or increase the dosage of steroids, any other immune suppressive or cytotoxic agent at the time of enrolment into the study or start of eltrombopag; patients already in treatment with a fixed, stabilized dosage of steroids or other immune suppressive agents because of cGVHD may be included into the study;
• Patients received concomitant erythropoietin treatment; Patients have active deep venous thrombosis (DVT);
• Patients have venous occlusive disease (VOD);
• Patients with baseline elevation of hepatic enzymes will be monitored carefully in order to point out possible addictive eltrombopag- related hepatotoxicity;
• Patients have hepatic cirrhosis;
• Patients have transplant related-microangiopathy;
• Patients have active infections (CMV reactivation included);
• Patients have hypersensitive to study drug;
• Patients are unable to stop medications that are known to cause a drug-drug interaction with eltrombopag.

Sponsors & Collaborators

• Gruppo Italiano Malattie EMatologiche dell'Adulto: lead

Study Sites (16)

UO Ematologia con trapianto-Università degli Studi di Bari Aldo Moro

Bari, Italy

Location

Divisione di Ematologia - Ospedali Riuniti

Bergamo, Italy

Location

USD Trapianti di midollo per adulti - Cattedra di Ematologia - Università degli Studi di Brescia

Brescia, Italy

Location

Ospedale Santa Croce Divisione di Ematologia Cuneo

Catania, Italy

Location

Ospedale Santa Croce Divisione di Ematologia Cuneo

Cuneo, Italy

Location

Policlinico di Careggi

Florence, Italy

Location

Divisione Ematologia 2 - Azienda Ospedaliera Universitaria - S.Martino

Genova, Italy

Location

Unità Trapianto di Midollo Ist. Nazionale Tumori

Milan, Italy

Location

La Maddalena Casa di Cura di Alta Specialità Dipartimento Oncologico di III Livello

Palermo, Italy

Location

U.O. Ematologia Clinica - Azienda USL di Pescara

Pescara, Italy

Location

Università Cattolica del Sacro Cuore - Policlinico A. Gemelli

Roma, Italy

Location

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

Roma, Italy

Location

Università degli Studi - Policlinico di Tor Vergata

Roma, Italy

Location

Clinica Ematologica - Policlinico Universitario

Udine, Italy

Location

ULSS N. 6 Ospedale S. Bortolo

Vicenza, Italy

Location

ULSS N.6 Osp. S. Bortolo

Vicenza, Italy

Location

MeSH Terms

Conditions

Thrombocytopenia

Interventions

eltrombopag

Condition Hierarchy (Ancestors)

Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Study Officials

• Francesco Zaja

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

  PRINCIPAL INVESTIGATOR

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: February 12, 2013
• First Posted: February 13, 2013
• Study Start: September 16, 2013
• Primary Completion: June 26, 2020
• Study Completion: June 26, 2020
• Last Updated: June 11, 2021

Record last verified: 2021-06

Data Sharing

• IPD Sharing: Will not share

Locations

IT (16)