Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia

Immune Thrombocytopenia Clinical Trial

— BAITP  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT05446831
• Other study ID #: PKU-ITP2207
• Status: Recruiting
• Phase: Phase 2
• Start date: July 13, 2022
• Est. completion date: December 1, 2023

Study information

• Verified date: September 2022
• Source: Peking University People's Hospital
• Contact: Xiaohui Zhang, MD
• Phone: +8613522338836
• Email: zhangxh100[@]sina.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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).

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).

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 35
• Est. completion date: December 1, 2023
• Est. primary completion date: June 1, 2023
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years to 70 Years

Eligibility

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

Related Conditions & MeSH terms

• Immune Thrombocytopenia
• ITP
• Purpura, Thrombocytopenic, Idiopathic
• Thrombocytopenia

Intervention

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.

Locations

Country	Name	City	State
China	Peking University Insititute of Hematology, Peking University People's Hospital	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Peking University People's 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)	Complete response (CR) was defined as a platelet count over 100,000/µL and absence of bleeding.	1 month
Secondary	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
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 24
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 24
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 24