Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia

Phase 2

Recruiting

• Conditions: Immune Thrombocytopenia; ITP
• Interventions: Drug: Baricitinib

• Registration Number: NCT05446831
• Lead Sponsor: Peking University People's Hospital

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).

Study Timeline

• Study First Submitted: 2022-07-01
• Study First Submitted QC: 2022-07-01
• Study First Posted: 2022-07-07
• Study Start: 2022-07-13
• Status Verified: 2022-09
• Last Update Submitted: 2022-09-27
• Last Update Posted: 2022-09-28
• Primary Completion: 2023-06-01
• Study Completion: 2023-12-01

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 35

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

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Baricitinib	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.

Primary Outcome Measures

Name	Time	Method
Durable response	6 months	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

Name	Time	Method
Complete response (CR)	1 month	Complete response (CR) was defined as a platelet count over 100,000/μL and absence of bleeding.
Bleeding events	From the start of study treatment (Day 1) to the end of week 24	Clinically significant bleeding as assessed using the world health organization (WHO) bleeding scale.
Health-related quality of life (HRQoL)	From the start of study treatment (Day 1) to the end of week 24	ITP-PAQ is used to assess the Health Related Quality of Life (HRQoL) before and after treatment.
Time to response	6 months	The time from starting treatment to time of achievement of CR or R.
Duration of response	6 months	Duration of response at 6-month follow up.
Initial response	28 days	Achievement of CR or R at day 28
Response (R)	1 month	Response (R) as a platelet count over 30,000/μL and at least 2-fold increase of the baseline count and absence of bleeding.
Early response	7 days	Achievement of CR or R at day 7
Adverse events	From the start of study treatment (Day 1) to the end of week 24	Adverse events (AEs) are reported and graded in accordance with the Common Terminology Criteria for Adverse Events (CTCAE), version 5.0.

Trial Locations

Locations (1)

Peking University Insititute of Hematology, Peking University People's Hospital; 🇨🇳 Beijing, Beijing, China