Clinical trial NCT04516837: Eltrombopag Plus rhTPO Versus Eltrombopag for ITP During the COVID-19 Pandemic (ELABORATE-19)
Official title: The Combination of Eltrombopag and Recombinant Human Thrombopoietin (rhTPO) Versus Eltrombopag Monotherapy as Subsequent Treatment for Immune Thrombocytopenia During the COVID-19 Pandemic
Study type: INTERVENTIONAL
Overall status: UNKNOWN
Phase: PHASE2
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: 2020-08-18
Enrollment: 120
Sex: ALL
Minimum age (years): 18
Maximum age (years): 80
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a prospective, multicenter, randomized, open-label study to investigate the efficacy and safety of eltrombopag plus recombinant human thrombopoietin (rhTPO) versus eltrombopag as treatment for corticosteroid-resistant or relapsed immune thrombocytopenia (ITP) during the COVID-19 pandemic.

Detailed description: During the COVID-19 pandemic, the classical subsequent treatment regimen for ITP of immunosuppressants and/or steroids might increase patients' susceptibility of virus infections. To minimize ITP patients' risk during the COVID-19 global crisis and to improve treatment efficacy, this treatment regimen of eltrombopag plus recombinant human thrombopoietin (rhTPO) should be investigated.

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.

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.

Both eltrombopag and rhTPO demonstrated good safety in ITP patients. 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 ITP treatment.

This study aimed to evaluate the sustained responses and safety of eltrombopag plus rhTPO as treatment for corticosteroid-resistant or relapsed ITP patients during the COVID-19 pandemic.

Eligibility criteria: Inclusion Criteria:

1. Clinically confirmed corticosteroid-resistant or relapsed immune thrombocytopenic purpura (ITP)
2. Platelet count less than 30×10\^9/L on two occasions or Platelets above 30×10\^9/L combined with bleeding manifestation (WHO bleeding scale 2 or above)
3. Subject is ≥ 18 years
4. Subject has signed and provided written informed consent.
5. Fertile patients must use effective contraception during treatment and observational period
6. 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 months
measure: Response
timeFrame: 6 months
measure: No response
timeFrame: 6 months
measure: Relapses
timeFrame: 6 months
Secondary outcomes:
measure: Early response
timeFrame: 7 days
measure: Initial response
timeFrame: 1 month
measure: Durable response
timeFrame: 6 months
measure: TOR (time to response)
timeFrame: 6 months
measure: DOR (duration of response)
timeFrame: 6 months
measure: Treatments associated adverse events
timeFrame: 6 months
measure: Reduction in bleeding symptoms
timeFrame: 6 months
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: