Back to results

The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9

Primary Purpose

Purpura, Thrombocytopenic, Idiopathic

Status

Not yet recruiting

Phase

Phase 2

Locations

China

Study Type

Interventional

Intervention

all-trans retinoic acid

Eltrombopag

About this trial

This is an interventional treatment trial for Purpura, Thrombocytopenic, Idiopathic focused on measuring Resistant/recurrent ITP, MSC-C5b-9 marker, all-trans retinoic acid, Eltrombopag, prospective clinical study

Eligibility Criteria

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

Inclusion Criteria:

1. Isolated thrombocytopenia (platelet count <30 × 109/L); 2. age > 18 years; 3. normal white blood cells and red blood cells on bone marrow examination; 4. increased number of megakaryocytes (bone marrow examination was performed in all patients except for myelofibrosis or other conditions that can cause thrombocytopenia disease); 5. the spleen was normal in size; 6. Eastern Cooperative Oncology Group status score (ECOG score) ≤ 2; 7. ineffective or relapsed after at least 1 course of full-dose full-course hormone therapy; 8. Failure of prior ITP therapy (eg, hormones, splenectomy, and cyclosporine) and at least 4 weeks from enrollment.

Exclusion Criteria:

1. Secondary ITP such as drug-related thrombocytopenia; 2. thrombocytopenia due to viral infection (HIV, hepatitis B virus, or hepatitis C virus); 3. severe cardiac, renal, hepatic, or respiratory insufficiency; 4. severe immunodeficiency; 5. pregnancy or lactation; 6. myelodysplasia or Myelofibrosis; 7. history of malignancy; 8. ongoing immunosuppressive therapy for other diseases; 9. patients previously treated with eltrombopag were excluded from this study.

Sites / Locations

Peking University Insititute of Hematology, Peking University People's Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Active Comparator

Experimental

Arm Label

Eltrombopag

ATRA and Eltrombopag

Arm Description

The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The highest is 75mg/d, if the dose is higher than 200×109/L, the dose is reduced. When the dose is higher than 400×109/L, the drug is temporarily discontinued, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.

ATRA 10 mg, 2 times a day, orally; The initial dose of eltrombopag was 50 mg/time, once a day. According to the clinical standard of eltrombopag dose adjustment in the research protocol set by the research group, the dose was increased when the platelet count was lower than 5×109/L. The maximum dose is 75 mg/d, the dose is reduced if it is higher than 200×109/L, and the drug is temporarily discontinued when it is higher than 400×109/L, and the drug is repeated according to the platelet count. The treatment course is 12 weeks.

Outcomes

Primary Outcome Measures

Sustained Response Rate (SR) at 18 months

The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 18-month follow-up

Secondary Outcome Measures

Complete response rate (CR)

The complete response (CR) was defined as platelet count more than 100×10^9/L and absence of bleeding.

Response rate (R)

The Response rate (R) was defined as platelet count more than 30×10^9/L and more than 2 times higher than baseline, without bleeding

Inefficiency (NR)

Platelet count <30×10^9/L, or less than 2-fold increase from baseline or associated with bleeding

Recurrence rate (relapse)

After the treatment is effective, the platelet count drops below 30×10^9/L or drops to less than 2 times the basal value, or bleeding symptoms occur

Early response

Platelet count ≥30×10^9/L and at least doubling baseline at 1 wk.

Initial response

Initial response as platelet count more than 30×10^9/L and at least 2-fold increase of the baseline count and absence of bleeding.

Time to response (TTR)

The time from starting treatment to time of achievement of CR or R

Time to relapse (duration of efficacy)

The time from achievement of CR or R to time of relapse

Full Information

NCT ID

NCT05438875

First Posted

June 25, 2022

Last Updated

June 25, 2022

Sponsor

Peking University People's Hospital

Collaborators

Beijing Friendship Hospital, Beijing Tongren Hospital, Beijing Hospital, Navy General Hospital, Beijing

1. Study Identification

Unique Protocol Identification Number

NCT05438875

Brief Title

The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9

Official Title

The Combination of ATRA and Eltrombopag as the Treatment Strategy for Glucocorticoid-Resistant/Relapsed ITP Based on the Stratification of the New Biomarker MSC-C5b-9: A Prospective, Randomized, Open-Label, Multicenter Clinical Trial

Study Type

Interventional

2. Study Status

Record Verification Date

June 2022

Overall Recruitment Status

Not yet recruiting

Study Start Date

June 24, 2022 (Anticipated)

Primary Completion Date

December 31, 2024 (Anticipated)

Study Completion Date

March 31, 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Peking University People's Hospital

Collaborators

Beijing Friendship Hospital, Beijing Tongren Hospital, Beijing Hospital, Navy General Hospital, Beijing

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

5. Study Description

Brief Summary

A Prospective, Randomized, Open-Label, Multicenter Clinical Trial study to compare the efficacy and safety of ATRA plus eltrombopag compared to eltrombopag monotherapy in the treatment of steroid-resistant/relapsed immune thrombocytopenia (ITP).

Detailed Description

The investigators are undertaking a parallel group, multicenter, randomized controlled trial of patients with ITP in China. Patients were tested for MSCs, and they were divided into MSC-C5b-9+ group and MSC-C5b-9- group according to the test results, and the two groups were randomized to ATRA + eltrombopag and eltrombopag monotherapy group. Platelet count, bleeding and other symptoms were evaluated before and after treatment. Adverse events are also recorded throughout the study.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Purpura, Thrombocytopenic, Idiopathic

Keywords

Resistant/recurrent ITP, MSC-C5b-9 marker, all-trans retinoic acid, Eltrombopag, prospective clinical study

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 2

Interventional Study Model

Parallel Assignment

Masking

None (Open Label)

Allocation

Randomized

Enrollment

96 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title

Eltrombopag

Arm Type

Active Comparator

Arm Description

Arm Title

ATRA and Eltrombopag

Arm Type

Experimental

Arm Description

Intervention Type

Drug

Intervention Name(s)

all-trans retinoic acid

Other Intervention Name(s)

retinoic acid

Intervention Description

ATRA 10 mg, 2 times a day, orally.The treatment course is 12 weeks.

Intervention Type

Drug

Intervention Name(s)

Eltrombopag

Other Intervention Name(s)

TPO-RA

Intervention Description

The initial dose of eltrombopag is 50 mg/time, once a day, and the dose is increased when the platelet count is lower than 5×109/L, the maximum is 75 mg/d, and the dose is higher than 200×109/L. When the drug is temporarily discontinued, the drug is re-administered according to the platelet count.The treatment course is 12 weeks.

Primary Outcome Measure Information:

Title

Sustained Response Rate (SR) at 18 months

Description

The maintenance of platelet count ≥ 30 x 10^9/L, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 18-month follow-up

Time Frame

18 months

Secondary Outcome Measure Information:

Title

Complete response rate (CR)

Description

The complete response (CR) was defined as platelet count more than 100×10^9/L and absence of bleeding.

Time Frame

18 months

Title

Response rate (R)

Description

The Response rate (R) was defined as platelet count more than 30×10^9/L and more than 2 times higher than baseline, without bleeding

Time Frame

18 months

Title

Inefficiency (NR)

Description

Platelet count <30×10^9/L, or less than 2-fold increase from baseline or associated with bleeding

Time Frame

18 months

Title

Recurrence rate (relapse)

Description

After the treatment is effective, the platelet count drops below 30×10^9/L or drops to less than 2 times the basal value, or bleeding symptoms occur

Time Frame

18 months

Title

Early response

Description

Platelet count ≥30×10^9/L and at least doubling baseline at 1 wk.

Time Frame

1 week

Title

Initial response

Description

Initial response as platelet count more than 30×10^9/L and at least 2-fold increase of the baseline count and absence of bleeding.

Time Frame

1 month

Title

Time to response (TTR)

Description

The time from starting treatment to time of achievement of CR or R

Time Frame

18 months

Title

Time to relapse (duration of efficacy)

Description

The time from achievement of CR or R to time of relapse

Time Frame

18 months

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: 1. Isolated thrombocytopenia (platelet count <30 × 109/L); 2. age > 18 years; 3. normal white blood cells and red blood cells on bone marrow examination; 4. increased number of megakaryocytes (bone marrow examination was performed in all patients except for myelofibrosis or other conditions that can cause thrombocytopenia disease); 5. the spleen was normal in size; 6. Eastern Cooperative Oncology Group status score (ECOG score) ≤ 2; 7. ineffective or relapsed after at least 1 course of full-dose full-course hormone therapy; 8. Failure of prior ITP therapy (eg, hormones, splenectomy, and cyclosporine) and at least 4 weeks from enrollment. Exclusion Criteria: 1. Secondary ITP such as drug-related thrombocytopenia; 2. thrombocytopenia due to viral infection (HIV, hepatitis B virus, or hepatitis C virus); 3. severe cardiac, renal, hepatic, or respiratory insufficiency; 4. severe immunodeficiency; 5. pregnancy or lactation; 6. myelodysplasia or Myelofibrosis; 7. history of malignancy; 8. ongoing immunosuppressive therapy for other diseases; 9. patients previously treated with eltrombopag were excluded from this study.

Central Contact Person:

First Name & Middle Initial & Last Name or Official Title & Degree

Xiaohui Zhang, MD

Phone

+8613522338836

zhangxh100@sina.com

First Name & Middle Initial & Last Name or Official Title & Degree

Qiusha Huang, MD

Phone

+8613051816058

huangfuqs@163.com

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Xiaohui Zhang, MD

Organizational Affiliation

Study Principal Investigator Peking University People's Hospital

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

Facility Contact:

First Name & Middle Initial & Last Name & Degree

Xiao Hui Zhang, MD

zhangxh100@sina.com

First Name & Middle Initial & Last Name & Degree

Menglin Li, doctor

bjyx819@163.com

12. IPD Sharing Statement

Learn more about this trial

The Combination of ATRA and Eltrombopag as the Treatment of Steroid-resistant/Relapse ITP Based on MSC-C5b-9

We'll reach out to this number within 24 hrs