Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia (BAITP)

Efficacy and Safety of Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia: A Single-arm, Open-label Phase II Study

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).

Study Overview

• Status: Recruiting
• Conditions: Immune Thrombocytopenia; ITP
• Intervention / Treatment: Drug: Baricitinib

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 Type: Interventional
• Enrollment (Anticipated): 35
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Peng Zhao, MD; Phone Number: +8618810323668; Email: zpeng702@163.com

Study Locations

• China
  • Beijing
    • Beijing, Beijing, China, 100010
      • Recruiting
      • Peking University Insititute of Hematology, Peking University People's Hospital
      • Contact: Peng Zhao, MD; Phone Number: +8618810323668; Email: zpeng702@163.com
      • Principal Investigator: Xiaohui Zhang, MD

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 14 years to 66 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm

Intervention / Treatment

Experimental: 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.

Drug: 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.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
Time to response	The time from starting treatment to time of achievement of CR or R.	6 months
Complete response (CR)	Complete response (CR) was defined as a platelet count over 100,000/μL and absence of bleeding.	1 month
Response (R)	Response (R) as a platelet count over 30,000/μL and at least 2-fold increase of the baseline count and absence of bleeding.	1 month
Duration of response	Duration of response at 6-month follow up.	6 months
Early response	Achievement of CR or R at day 7	7 days
Initial response	Achievement of CR or R at day 28	28 days
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 24
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 24
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 24

Collaborators and Investigators

• Sponsor: Peking University People's Hospital
• Investigators: Principal Investigator: Xiaohui Zhang, MD, Peking University People's Hospital

Study record dates

Study Major Dates

• Study Start (Actual): July 13, 2022
• Primary Completion (Anticipated): June 1, 2023
• Study Completion (Anticipated): December 1, 2023

Study Registration Dates

• First Submitted: July 1, 2022
• First Submitted That Met QC Criteria: July 1, 2022
• First Posted (Actual): July 7, 2022

Study Record Updates

• Last Update Posted (Actual): September 28, 2022
• Last Update Submitted That Met QC Criteria: September 27, 2022
• Last Verified: September 1, 2022

More Information

Terms related to this study

• Additional Relevant MeSH Terms: Pathologic Processes; Immune System Diseases; Autoimmune Diseases; Hematologic Diseases; Hemorrhage; Hemorrhagic Disorders; Blood Coagulation Disorders; Skin Manifestations; Blood Platelet Disorders; Thrombotic Microangiopathies; Purpura, Thrombocytopenic; Purpura; Purpura, Thrombocytopenic, Idiopathic; Thrombocytopenia
• Other Study ID Numbers: PKU-ITP2207

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No