Eltrombopag in Patients With Delayed Post Transplant Thrombocytopenia.

• Status: Completed
• Trial end date: 2020-06-26
• Target enrollment: 0
• Participant gender: All

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.

• Phase: Phase 2
• Accepts Healthy Volunteers?: No

Details

• Lead Sponsor: Gruppo Italiano Malattie EMatologiche dell'Adulto

Criteria

Inclusion Criteria:

• 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.

Exclusion Criteria:

• 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 have grade 3-4 hyper bilirubinemia; elevation of hepatic enzymes because of cGVHD should not be considered criteria of exclusion.

• 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.