Low-dose Baricitinib Plus High-dose Dexamethasone for Patients With Newly Diagnosed Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

Official title:

Low-dose Baricitinib Plus High-dose Dexamethasone as First-line Treatment for Patients With Newly Diagnosed Immune Thrombocytopenia: a Multicenter, Open-label, Randomized, Controlled, Phase 2 Trial

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05932524
• Other study ID #: PKU-BAITP-03
• Status: Recruiting
• Phase: Phase 2
• Start date: July 7, 2023
• Est. completion date: June 2025

Study information

• Verified date: July 2023
• Source: Peking University People's Hospital
• Contact: Xiaohui Zhang
• Phone: +8613522338836
• Email: zhangxh[@]bjmu.edu.cn
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

A randomized, open-label, multicenter, phase 2 trial to compare the efficacy and safety of baricitinib plus high-dose dexamethasone compared to high-dose dexamethasone monotherapy for the first-line treatment of adults with primary immune thrombocytopenia (ITP).

Description:

This is a parallel group, multicenter, randomized, controlled trial of patients with ITP in China. Patients were randomly assigned to receive baricitinib plus high-dose dexamethasone or high-dose dexamethasone monotherapy. 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 and 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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 132
• Est. completion date: June 2025
• Est. primary completion date: June 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

Inclusion Criteria:

1. Confirmed newly-diagnosed, treatment-naive ITP;

2. A platelet count <30,000/µL, or a platelet count <50,000/µL with clinically significant bleeding symptoms (WHO bleeding scale 2 or above) at the enrollment;

3. Willing and able to sign written informed consent.

Exclusion Criteria:

1. Received chemotherapy or anticoagulants or other drugs affecting the platelet counts within 6 months before the screening visit;

2. Have a known diagnosis of other autoimmune diseases, established in the medical history and laboratory findings with positive results for the determination of antinuclear antibodies, anti-cardiolipin antibodies, lupus anticoagulant or direct Coombs test;

3. Active or a history of malignancy;

4. Pregnancy or lactation;

5. Received first-line and second-line ITP-modifying therapy;

6. Previously received corticosteroids or immunosuppressive agents for non-ITP diseases within 6 months before enrollment;

7. A history of clinically significant adverse reactions to previous corticosteroid therapy;

8. Unstable or uncontrolled disease or condition related to or impacting cardiac function (e.g., unstable angina, congestive heart failure, uncontrolled hypertension or cardiac arrhythmia);

9. Current or recent (<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection;

10. A history of symptomatic herpes zoster infection within 12 weeks prior to screening;

11. Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV);

12. 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;

13. 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;

14. Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure;

15. 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;

16. 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.

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

Drug:
• Baricitinib 2 MG
Baricitinib 2 mg q.d., p.o., for 6 consecutive months.
• Dexamethasone
Dexamethasone 40 mg q.d. for 4 consecutive days (the 4-day course of dexamethasone will be repeated in the case of lack of response by day 10)

Locations

Country	Name	City	State
China	Beijing Friendship Hospital	Beijing
China	Beijing Hospital	Beijing
China	Beijing Luhe Hospital	Beijing
China	Beijing Tsinghua Changgeng Hospital	Beijing
China	China-Japan Friendship Hospital	Beijing
China	Chinese PLA General Hospital	Beijing
China	Peking University First Hospital	Beijing
China	Peking University Insititute of Hematology, Peking University People's Hospital	Beijing
China	Peking University Third Hospital	Beijing
China	The Sixth Medical Center of PLA General Hospital	Beijing

Sponsors (10)

Lead Sponsor	Collaborator
Peking University People's Hospital	Beijing Friendship Hospital, Beijing Hospital, Beijing Luhe Hospital, Beijing Tsinghua Changgeng Hospital, China-Japan Friendship Hospital, Chinese PLA General Hospital, Navy General Hospital, Beijing, Peking University First Hospital, Peking University Third Hospital

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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.	6 months
Secondary	Complete response (CR)	A platelet count over 100,000/µL and absence of bleeding.	1 month
Secondary	Response (R)	A platelet count over 30,000/µL and at least 2-fold increase of the baseline count and absence of bleeding.	1 month
Secondary	Time to response	The time from starting treatment to time of achievement of CR or R.	6 months
Secondary	Duration of response	Duration of response at 6-month follow up.	6 months
Secondary	Early response	Achievement of CR or R at day 7	7 days
Secondary	Initial response	Achievement of CR or R at day 28	28 days
Secondary	Bleeding events	Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.	From the start of study treatment (Day 1) to the end of week 26
Secondary	Health-related quality of life (HRQoL)	ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.	From the start of study treatment (Day 1) to the end of week 26
Secondary	Adverse events	Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.	From the start of study treatment (Day 1) to the end of week 26