Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia

NCT ID: NCT05446831

Last Updated: 2022-09-28

Basic Information

• Recruitment Status: UNKNOWN
• Clinical Phase: PHASE2
• Total Enrollment: 35 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2022-07-13
• Study Completion Date: 2023-12-01

Brief Summary

Single-arm, open-label, single-center study to evaluate the efficacy and safety of baricitinib for the treatment of adults with steroid-resistant/relapse immune thrombocytopenia (ITP).

Detailed Description

The investigators are undertaking a prospective trial of 20 adults with ITP in China. Baricitinib is administered as 4 mg po. daily. Safety outcomes and efficacy outcomes are assessed on scheduled study visits (primary endpoint defined as durable response at 6-month follow-up).

Conditions

ITP; Immune Thrombocytopenia

Study Design

• Allocation Method: NA
• Intervention Model: SINGLE_GROUP
• Primary Study Purpose: TREATMENT
• Blinding Strategy: NONE

Study Groups

Baricitinib

Oral baricitinib was given at a dose of 4 mg daily for 6 months. Treatment was discontinued if very severe or life-threatening adverse events developed or at the patients' request.

Group Type: EXPERIMENTAL

Baricitinib

Intervention Type: DRUG

Oral baricitinib was given at a dose of 4 mg daily. The decision to initiate rescue therapy was made after assessment of the extent of bleeding, patient preferences, lifestyle and activity, the complications of specific therapies, comorbidities that predisposed patients to bleeding and the tolerance of side effects. If a platelet count over 300,000/μL was observed for two consecutive tests at least 2 weeks apart, baricitinib treatment was interrupted.

Interventions

Baricitinib

Oral baricitinib was given at a dose of 4 mg daily. The decision to initiate rescue therapy was made after assessment of the extent of bleeding, patient preferences, lifestyle and activity, the complications of specific therapies, comorbidities that predisposed patients to bleeding and the tolerance of side effects. If a platelet count over 300,000/μL was observed for two consecutive tests at least 2 weeks apart, baricitinib treatment was interrupted.

Intervention Type: DRUG

Eligibility Criteria

Inclusion Criteria

1. Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia

2. Patients with chronic low platelet count (<30,000/μL) for 6 months who have failed at least one treatment for chronic low platelet count

3. 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

4. Patients with a platelet count <30,000/μL or a platelet count <50,000/μL with clinically significant bleeding symptoms at the enrollment

5. Over 18 years old

6. Willing and able to provide written informed consent, and agreeable to the schedule of assessment

Exclusion Criteria

1. Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection or patients with confirmed autoimmune disease)

2. Active or a history of malignancy

3. Pregnancy or lactation

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

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

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

7. 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

8. 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

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

10. 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

11. 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

• Minimum Eligible Age: 18 Years
• Maximum Eligible Age: 70 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Peking University People's Hospital

OTHER

Sponsor Role: lead

Responsible Party

Xiao Hui Zhang

Vice president of Peking Univeristy Institute of Hematology

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Xiaohui Zhang, MD

Role: PRINCIPAL_INVESTIGATOR

Peking University People's Hospital

Locations

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

Beijing, Beijing Municipality, China

Site Status: RECRUITING

Countries

China

Central Contacts

Xiaohui Zhang, MD

Role: CONTACT

zhangxh100@sina.com

+8613522338836

Peng Zhao, MD

Role: CONTACT

zpeng702@163.com

+8618810323668

Facility Contacts

Xiaohui Zhang, MD

Role: primary

zhangxh100@sina.com

+8613522338836

Peng Zhao, MD

Role: backup

zpeng702@163.com

+8618810323668

Other Identifiers

PKU-ITP2207

Identifier Type: -

Identifier Source: org_study_id