search
Back to results

Low-dose Baricitinib Plus Danazol for Steroid-resistant/Relapse Immune Thrombocytopenia

Primary Purpose

Immune Thrombocytopenia

Status
Recruiting
Phase
Phase 2
Locations
China
Study Type
Interventional
Intervention
Baricitinib 2 MG [Olumiant]
Danazol
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 Immune Thrombocytopenia, baricitinib

Eligibility Criteria

18 Years - 70 Years (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria: Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia; Patients with chronic low platelet count (<30,000/μL) for 6 months who have failed at least one treatment for chronic low platelet count; Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation; Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with clinically significant bleeding symptoms at the enrollment; Willing and able to provide written informed consent, and agreeable to the schedule of assessment. Exclusion Criteria: Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection or patients with confirmed autoimmune disease); Active or a history of malignancy; Pregnancy or lactation; Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection; A history of symptomatic herpes zoster infection within 12 weeks prior to screening; Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV); Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB; Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled; Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure; A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data; Any of the following specific abnormalities on screening laboratory tests: 1) ALT or AST >2 x ULN, or total bilirubin ≥1.5 x ULN 2) hemoglobin <9 g/dL, or total white blood cell (WBC) count <2,500/µL, or neutropenia (absolute neutrophil count <1,200/µL), or lymphopenia (lymphocyte count <750/µL) 3) eGFR <50 mL/min/1.73 m^2.

Sites / Locations

  • Peking University Insititute of Hematology, Peking University People's HospitalRecruiting
  • Beijing Friendship HospitalRecruiting
  • Beijing HospitalRecruiting
  • Beijing Luhe HospitalRecruiting
  • Beijing Tsinghua Changgeng HospitalRecruiting
  • China-Japan Friendship HospitalRecruiting
  • Chinese PLA General HospitalRecruiting
  • Peking University First HospitalRecruiting
  • Peking University Third HospitalRecruiting
  • The Sixth Medical Center of PLA General HospitalRecruiting

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Low-dose baricitinib plus danazol

Danazol

Arm Description

Oral baricitinib is given at a dose of 2 mg daily for 6 months. Danazol is given at a dose of 200 mg twice a day for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request.

Danazol is given at a dose of 200 mg twice a day for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request.

Outcomes

Primary Outcome Measures

Durable response
The maintenance of a platelet count ≥30,000/μL, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.

Secondary Outcome Measures

Complete response (CR)
A platelet count over 100,000/μL and absence of bleeding.
Response (R)
A platelet count over 30,000/μL and at least 2-fold increase of the baseline count and absence of bleeding.
Time to response
The time from starting treatment to time of achievement of CR or R.
Initial response
Achievement of CR or R at day 28
Bleeding events
Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
Health-related quality of life (HRQoL)
ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.
Adverse events
Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.

Full Information

First Posted
May 2, 2023
Last Updated
June 11, 2023
Sponsor
Peking University People's Hospital
Collaborators
Beijing Luhe Hospital, Chinese PLA General Hospital, Navy General Hospital, Beijing, Beijing Hospital, Beijing Friendship Hospital, Peking University First Hospital, Peking University Third Hospital, China-Japan Friendship Hospital, Beijing Tsinghua Changgeng Hospital
search

1. Study Identification

Unique Protocol Identification Number
NCT05852847
Brief Title
Low-dose Baricitinib Plus Danazol for Steroid-resistant/Relapse Immune Thrombocytopenia
Official Title
Low-dose Baricitinib Plus Danazol Versus Danazol for Patients With Steroid-resistant/Relapse Immune Thrombocytopenia: A Multicenter, Randomized, Open-label Phase 2 Trial
Study Type
Interventional

2. Study Status

Record Verification Date
June 2023
Overall Recruitment Status
Recruiting
Study Start Date
May 16, 2023 (Actual)
Primary Completion Date
November 2024 (Anticipated)
Study Completion Date
May 2025 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Principal Investigator
Name of the Sponsor
Peking University People's Hospital
Collaborators
Beijing Luhe Hospital, Chinese PLA General Hospital, Navy General Hospital, Beijing, Beijing Hospital, Beijing Friendship Hospital, Peking University First Hospital, Peking University Third Hospital, China-Japan Friendship Hospital, Beijing Tsinghua Changgeng 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, multicenter, randomized, controlled phase 2 trial to compare the efficacy and safety profiles in ITP patients receiving baricitinib plus danazol to those receiving danazol alone.
Detailed Description
This is a prospective, multicenter, randomized, controlled design of 216 adult patients with steroid-resistant/relapse ITP in China. Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus danazol or danazol alone. Patients in the combination therapy group receive oral baricitinib at a dose of 2 mg daily and oral danazol at a dose of 200 mg twice a day. Those in the monotherapy group receive oral danazol at 200 mg twice daily. The treatment lasts for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request. The primary endpoint is durable response, defined as the maintenance of platelet count ≥ 30,000/μL, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Immune Thrombocytopenia
Keywords
Immune Thrombocytopenia, baricitinib

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 2
Interventional Study Model
Parallel Assignment
Model Description
Patients are randomly assigned at a 1:1 ratio to receive baricitinib plus danazol or danazol alone. Each group requires 108 patients (considering 20% drop-off) to achieve the superiority comparison.
Masking
None (Open Label)
Allocation
Randomized
Enrollment
216 (Anticipated)

8. Arms, Groups, and Interventions

Arm Title
Low-dose baricitinib plus danazol
Arm Type
Experimental
Arm Description
Oral baricitinib is given at a dose of 2 mg daily for 6 months. Danazol is given at a dose of 200 mg twice a day for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request.
Arm Title
Danazol
Arm Type
Active Comparator
Arm Description
Danazol is given at a dose of 200 mg twice a day for 6 months. Treatment will be discontinued if very severe or life-threatening adverse events developed or at the patients' request.
Intervention Type
Drug
Intervention Name(s)
Baricitinib 2 MG [Olumiant]
Other Intervention Name(s)
Olumiant
Intervention Description
Oral baricitinib was given at a dose of 2 mg daily for 6 months. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patients' request.
Intervention Type
Drug
Intervention Name(s)
Danazol
Intervention Description
Danazol was given at a dose of 200 mg bid for 6 months
Primary Outcome Measure Information:
Title
Durable response
Description
The maintenance of a platelet count ≥30,000/μL, at least 2-fold increase of the baseline count, the absence of bleeding, and no need for rescue medication at the 6-month follow-up.
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Complete response (CR)
Description
A platelet count over 100,000/μL and absence of bleeding.
Time Frame
1 month
Title
Response (R)
Description
A platelet count over 30,000/μL and at least 2-fold increase of the baseline count and absence of bleeding.
Time Frame
1 month
Title
Time to response
Description
The time from starting treatment to time of achievement of CR or R.
Time Frame
6 months
Title
Initial response
Description
Achievement of CR or R at day 28
Time Frame
28 days
Title
Bleeding events
Description
Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
Time Frame
6 months
Title
Health-related quality of life (HRQoL)
Description
ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.
Time Frame
6 months
Title
Adverse events
Description
Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.
Time Frame
6 months

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Maximum Age & Unit of Time
70 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia; Patients with chronic low platelet count (<30,000/μL) for 6 months who have failed at least one treatment for chronic low platelet count; Patients who did not achieve a sustained response to treatment with full-dose corticosteroids for a minimum duration of 4 weeks or who relapsed during steroid-tapering or after its discontinuation; Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with clinically significant bleeding symptoms at the enrollment; Willing and able to provide written informed consent, and agreeable to the schedule of assessment. Exclusion Criteria: Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection or patients with confirmed autoimmune disease); Active or a history of malignancy; Pregnancy or lactation; Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection; A history of symptomatic herpes zoster infection within 12 weeks prior to screening; Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV); Have evidence of active tuberculosis (TB), or have previously had evidence of active TB and did not receive appropriate and documented treatment, or have had household contact with a person with active TB and did not receive appropriate and documented prophylaxis for TB; Have experienced a clinically significant thrombotic event within 24 weeks of screening or are on anticoagulants and in the opinion of the investigator are not well controlled; Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure; A history or presence of cardiovascular, respiratory, hepatic, gastrointestinal, endocrine, neurological, or neuropsychiatric disorders or any other serious and/or unstable illness that, in the opinion of the investigator, could constitute an unacceptable risk when taking investigational product or interfere with the interpretation of data; Any of the following specific abnormalities on screening laboratory tests: 1) ALT or AST >2 x ULN, or total bilirubin ≥1.5 x ULN 2) hemoglobin <9 g/dL, or total white blood cell (WBC) count <2,500/µL, or neutropenia (absolute neutrophil count <1,200/µL), or lymphopenia (lymphocyte count <750/µL) 3) eGFR <50 mL/min/1.73 m^2.
Central Contact Person:
First Name & Middle Initial & Last Name or Official Title & Degree
Xiaohui Zhang, MD
Phone
+8610-8832-4672
Email
Zhangxh@bjmu.edu.cn
First Name & Middle Initial & Last Name or Official Title & Degree
Peng Zhao, MD
Phone
+8618810323668
Email
zpeng702@163.com
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Xiaohui Zhang, MD
Organizational Affiliation
Peking University Institute of Hematology, 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
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Xiao Hui Zhang, MD
Phone
+8610-8832-4672
Email
zhangxh100@sina.com
First Name & Middle Initial & Last Name & Degree
Peng Zhao, doctor
Phone
+8618810323668
Email
zpeng702@163.com
First Name & Middle Initial & Last Name & Degree
Xiao Hui Zhang, MD
Facility Name
Beijing Friendship Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zhao Wang, MD
Phone
+861088324672
Email
wangliru2019@126.com
Facility Name
Beijing Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hui Liu, MD
Phone
+861088324672
Email
fengru2019@126.com
Facility Name
Beijing Luhe Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
He Bing Zhou, MD
Phone
+861088324672
Email
zhouhebing2019@126.com
Facility Name
Beijing Tsinghua Changgeng Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Li Hong Li, MD
Phone
+861088324672
Email
1510301227@bjmu.edu.cn
Facility Name
China-Japan Friendship Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Zhen Ling Li, MD
Phone
+861088324672
Email
Lizhenling1994@163.com
Facility Name
Chinese PLA General Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Dai Hong Liu, MD
Phone
+861088326666
Email
1510301227@bjmu.edu.cn
Facility Name
Peking University First Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yu Jun Dong, MD
Phone
+861088324577
Email
1510301227@bjmu.edu.cn
Facility Name
Peking University Third Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Hong Mei Jing, MD
Phone
+861088324672
Email
1510301227@bjmu.edu.cn
Facility Name
The Sixth Medical Center of PLA General Hospital
City
Beijing
Country
China
Individual Site Status
Recruiting
Facility Contact:
First Name & Middle Initial & Last Name & Degree
Yi Liu, MD
Phone
+861088324577
Email
drliuyi@126.com

12. IPD Sharing Statement

Learn more about this trial

Low-dose Baricitinib Plus Danazol for Steroid-resistant/Relapse Immune Thrombocytopenia

We'll reach out to this number within 24 hrs