Clinical trial NCT04518878: Fixed Low-dose Eltrombopag and rhTPO for Immune Thrombocytopenia (FLOWER)
Official title: Combination of Fixed Low-dose Eltrombopag and rhTPO for Treatment of Immune Thrombocytopenia
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE1
Conditions: Immune Thrombocytopenia
Keywords: corticosteroid-resistant or relapsed ITP
eltrombopag
recombinant human thrombopoietin (rhTPO)
Lead sponsor: Peking University People's Hospital
Sponsor class: OTHER
First posted: 2020-08-19
Enrollment: 30
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: This is a prospective, single-arm study to investigate the efficacy and safety of the combination of fixed low-dose eltrombopag plus recombinant human thrombopoietin (rhTPO) as treatment for corticosteroid-resistant or relapsed immune thrombocytopenia (ITP) patients during the COVID-19 pandemic.

Detailed description: Eltrombopag, a small molecule agonist of thrombopoietin receptor (TPO-RA), was recommended as the subsequent treatment for ITP patients, which also already showed robust efficacy.Recombinant human thrombopoietin (rhTPO) is a full-length glycosylated-TPO produced by Chinese hamster ovary cells, which showed its effectiveness in ITP in a variety of studies.

Both eltrombopag and rhTPO demonstrated good safety in ITP patients. Because of their non-immunosuppressive nature, both of them serve as a reasonable choice during the global COVID-19 pandemic.

Since they increase the number of platelets through different mechanisms, and previous studies demonstrated that they might exert synergic effect. The investigators hypothesized that the combination of these two agents could be a promising option for treatment of corticosteroid-resistant or relapsed ITP patients.

Eligibility criteria: Inclusion Criteria:

1. Clinically confirmed corticosteroid-resistant or relapsed immune thrombocytopenic purpura (ITP)
2. Subject has signed and provided written informed consent.
3. Fertile patients must use effective contraception during treatment and observational period
4. Negative pregnancy test

Exclusion Criteria:

1. Have an impaired renal function as indicated by a serum creatinine level \> 2.0 mg/dL
2. Have an inadequate liver function as indicated by a total bilirubin level \> 2.0 mg/dL and/or an aspartate aminotransaminase or alanine aminotransferase level \> 3×upper limit of normal
3. Have a New York Heart Classification III or IV heart disease
4. Have a history of severe psychiatric disorder or are unable to comply with study and follow-up procedures
5. Have active hepatitis B or hepatitis C infection
6. Have a HIV infection
7. Have active infection requiring antibiotic therapy within 7 days prior to study entry
8. Are pregnant or lactating women, or plan to become pregnant or impregnated within 12 months of receiving study drug
9. Previous splenectomy
10. Had previous or concomitant malignant disease
11. Not willing to participate in the study.
12. Expected survival of \< 2 years
13. Intolerant to murine antibodies
14. Immunosuppressive treatment within the last 2 weeks
15. Connective tissue disease
16. Autoimmune hemolytic anemia
17. Patients currently involved in another clinical trial with evaluation of drug treatment

Primary outcomes:
measure: Complete response
timeFrame: 6 weeks
measure: Response
timeFrame: 6 weeks
measure: No response
timeFrame: 6 weeks
measure: Relapses
timeFrame: 6 weeks
Secondary outcomes:
measure: Early response
timeFrame: 7 days
measure: Initial response
timeFrame: 1 month
measure: TOR (time to response)
timeFrame: 6 weeks
measure: DOR (duration of response)
timeFrame: 6 weeks
measure: Treatments associated adverse events
timeFrame: 6 weeks
measure: Reduction in bleeding symptoms
timeFrame: 6 weeks
Other outcomes:
Locations:
city: Beijing
country: China
facility: Peking University Insititute of Hematology, Peking University People's Hospital
state: Beijing
Study officials:
affiliation: Peking University People's Hospital, Peking University Insititute of Hematology
name: Xiaohui Zhang, MD
role: PRINCIPAL_INVESTIGATOR
References: