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Fixed Low-dose Eltrombopag and rhTPO for Immune Thrombocytopenia (FLOWER)

Primary Purpose

Immune Thrombocytopenia

Status
Unknown status
Phase
Phase 1
Locations
China
Study Type
Interventional
Intervention
Eltrombopag
rhTPO
Sponsored by
Peking University People's Hospital
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Immune Thrombocytopenia focused on measuring corticosteroid-resistant or relapsed ITP, eltrombopag, recombinant human thrombopoietin (rhTPO)

Eligibility Criteria

18 Years - 80 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

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

Sites / Locations

  • Peking University Insititute of Hematology, Peking University People's HospitalRecruiting

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Fixed Low-dose Eltrombopag and rhTPO

Arm Description

Fixed Low-dose Eltrombopag and rhTPO

Outcomes

Primary Outcome Measures

Complete response
A complete response (CR) was defined as a sustained (≥ 3 months) platelet count ≥ 100×10^9/L.
Response
A response (R) was defined as a sustained (≥ 3 months) platelet count ≥ 30×10^9/L without recurrence of thrombocytopenia.
No response
No response (NR) was defined as platelet count < 30 × 10^9/L or a less than two fold increase in platelet count from baseline or the presence of bleeding. Platelet count must be measured on two occasions more than a day apart.
Relapses
A relapses was defined as platelet count falls below 30×10^9/L or bleeding accrues after achieving R or CR.

Secondary Outcome Measures

Early response
Early response was defined as the attainment of a platelet count ≥ 30 × 10⁹ and at least a doubling of baseline platelet count at 1 week.
Initial response
Initial treatment was defined as the attainment of a platelet count ≥ 30 × 10⁹ and at least a doubling of baseline platelet count at 1 month.
TOR (time to response)
The time to achieve platelet count ≥ 30×10^9/L and at least 2-fold increase of the baseline count and absence of bleeding since start of treatment.
DOR (duration of response)
The duration of achieve platelet count ≥ 30×10^9/L and at least 2-fold increase of the baseline count and absence of bleeding since start of treatment.
Treatments associated adverse events
All patients were assessed for safety every week during the first 8 weeks of treatment, and at 2-week intervals thereafter. Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Reduction in bleeding symptoms
Changes of bleeding after treatment. Bleeding was defined in accordance with the WHO bleeding scale (0, no bleeding; 1, petechiae; 2, mild blood loss; 3, gross blood loss; and 4, debilitating blood loss).

Full Information

First Posted
August 16, 2020
Last Updated
August 31, 2020
Sponsor
Peking University People's Hospital
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1. Study Identification

Unique Protocol Identification Number
NCT04518878
Brief Title
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

2. Study Status

Record Verification Date
August 2020
Overall Recruitment Status
Unknown status
Study Start Date
August 31, 2020 (Actual)
Primary Completion Date
December 2021 (Anticipated)
Study Completion Date
June 2022 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking University People's Hospital

4. Oversight

Studies a U.S. FDA-regulated Drug Product
No
Studies a U.S. FDA-regulated Device Product
No
Data Monitoring Committee
Yes

5. Study Description

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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenia
Keywords
corticosteroid-resistant or relapsed ITP, eltrombopag, recombinant human thrombopoietin (rhTPO)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Fixed Low-dose Eltrombopag and rhTPO
Arm Type
Experimental
Arm Description
Fixed Low-dose Eltrombopag and rhTPO
Intervention Type
Drug
Intervention Name(s)
Eltrombopag
Other Intervention Name(s)
Revolade
Intervention Description
Fixed dose of eltrombopag oral 25mg daily
Intervention Type
Drug
Intervention Name(s)
rhTPO
Other Intervention Name(s)
TPIAO, tebiao
Intervention Description
Rh-TPO 300U/kg subcutaneous injection once daily for 7 consecutive days, followed by a tapering dose in maintenance therapy.
Primary Outcome Measure Information:
Title
Complete response
Description
A complete response (CR) was defined as a sustained (≥ 3 months) platelet count ≥ 100×10^9/L.
Time Frame
6 weeks
Title
Response
Description
A response (R) was defined as a sustained (≥ 3 months) platelet count ≥ 30×10^9/L without recurrence of thrombocytopenia.
Time Frame
6 weeks
Title
No response
Description
No response (NR) was defined as platelet count < 30 × 10^9/L or a less than two fold increase in platelet count from baseline or the presence of bleeding. Platelet count must be measured on two occasions more than a day apart.
Time Frame
6 weeks
Title
Relapses
Description
A relapses was defined as platelet count falls below 30×10^9/L or bleeding accrues after achieving R or CR.
Time Frame
6 weeks
Secondary Outcome Measure Information:
Title
Early response
Description
Early response was defined as the attainment of a platelet count ≥ 30 × 10⁹ and at least a doubling of baseline platelet count at 1 week.
Time Frame
7 days
Title
Initial response
Description
Initial treatment was defined as the attainment of a platelet count ≥ 30 × 10⁹ and at least a doubling of baseline platelet count at 1 month.
Time Frame
1 month
Title
TOR (time to response)
Description
The time to achieve platelet count ≥ 30×10^9/L and at least 2-fold increase of the baseline count and absence of bleeding since start of treatment.
Time Frame
6 weeks
Title
DOR (duration of response)
Description
The duration of achieve platelet count ≥ 30×10^9/L and at least 2-fold increase of the baseline count and absence of bleeding since start of treatment.
Time Frame
6 weeks
Title
Treatments associated adverse events
Description
All patients were assessed for safety every week during the first 8 weeks of treatment, and at 2-week intervals thereafter. Adverse events were scaled according to Common Terminology Criteria for Adverse Events (CTCAE) version 5.0.
Time Frame
6 weeks
Title
Reduction in bleeding symptoms
Description
Changes of bleeding after treatment. Bleeding was defined in accordance with the WHO bleeding scale (0, no bleeding; 1, petechiae; 2, mild blood loss; 3, gross blood loss; and 4, debilitating blood loss).
Time Frame
6 weeks

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
80 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Clinically confirmed corticosteroid-resistant or relapsed immune thrombocytopenic purpura (ITP) Subject has signed and provided written informed consent. Fertile patients must use effective contraception during treatment and observational period Negative pregnancy test Exclusion Criteria: Have an impaired renal function as indicated by a serum creatinine level > 2.0 mg/dL 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 Have a New York Heart Classification III or IV heart disease Have a history of severe psychiatric disorder or are unable to comply with study and follow-up procedures Have active hepatitis B or hepatitis C infection Have a HIV infection Have active infection requiring antibiotic therapy within 7 days prior to study entry Are pregnant or lactating women, or plan to become pregnant or impregnated within 12 months of receiving study drug Previous splenectomy Had previous or concomitant malignant disease Not willing to participate in the study. Expected survival of < 2 years Intolerant to murine antibodies Immunosuppressive treatment within the last 2 weeks Connective tissue disease Autoimmune hemolytic anemia Patients currently involved in another clinical trial with evaluation of drug treatment
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaohui Zhang, MD
Phone
+86-13522338836
Email
zhangxh100@sina.com
First Name & Middle Initial & Last Name or Official Title & Degree
Xuelin Dou, MD
Phone
+86-15510491556
Email
dxldw@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiaohui Zhang, MD
Organizational Affiliation
Peking University People's Hospital, Peking University Insititute of Hematology
Official's Role
Principal Investigator
Facility Information:
Facility Name
Peking University Insititute of Hematology, Peking University People's Hospital
City
Beijing
State/Province
Beijing
ZIP/Postal Code
100010
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiaohui Zhang
Email
zhangxh100@sina.com

12. IPD Sharing Statement

Plan to Share IPD
No

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Fixed Low-dose Eltrombopag and rhTPO for Immune Thrombocytopenia (FLOWER)

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