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

• ClinicalTrials.gov Identifier: NCT05446831
• Recruitment Status: Recruiting
• First Posted: July 7, 2022
• Last Update Posted: September 28, 2022
• Sponsor: Peking University People's Hospital
• Information provided by (Responsible Party): Xiao Hui Zhang, Peking University People's Hospital

Study Description

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).

Condition or disease	Intervention/treatment	Phase
ITP; Immune Thrombocytopenia	Drug: Baricitinib	Phase 2

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 Design

• Study Type: Interventional (Clinical Trial)
• Estimated Enrollment: 35 participants
• Allocation: N/A
• Intervention Model: Single Group Assignment
• Masking: None (Open Label)
• Primary Purpose: Treatment
• Official Title: Efficacy and Safety of Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia: A Single-arm, Open-label Phase II Study
• Actual Study Start Date: July 13, 2022
• Estimated Primary Completion Date: June 1, 2023
• Estimated Study Completion Date: December 1, 2023

Arms and Interventions

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.

Outcome Measures

Primary Outcome Measures :

1. Durable response [ Time Frame: 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 :

1. Complete response (CR) [ Time Frame: 1 month ]
   Complete response (CR) was defined as a platelet count over 100,000/μL and absence of bleeding.
2. Response (R) [ Time Frame: 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.
3. Time to response [ Time Frame: 6 months ]
   The time from starting treatment to time of achievement of CR or R.
4. Duration of response [ Time Frame: 6 months ]
   Duration of response at 6-month follow up.
5. Early response [ Time Frame: 7 days ]
   Achievement of CR or R at day 7
6. Initial response [ Time Frame: 28 days ]
   Achievement of CR or R at day 28
7. Bleeding events [ Time Frame: 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.
8. Health-related quality of life (HRQoL) [ Time Frame: 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.
9. Adverse events [ Time Frame: 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.

Eligibility Criteria

• Ages Eligible for Study: 18 Years to 70 Years (Adult, Older Adult)
• Sexes Eligible for Study: All
• Accepts Healthy Volunteers: No

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

Contacts and Locations

Contacts

Contact: Xiaohui Zhang, MD; +8613522338836; zhangxh100@sina.com

Contact: Peng Zhao, MD; +8618810323668; zpeng702@163.com

Locations

China, Beijing

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

Beijing, Beijing, China, 100010

Contact: Xiaohui Zhang, MD +8613522338836 zhangxh100@sina.com

Contact: Peng Zhao, MD +8618810323668 zpeng702@163.com

Principal Investigator: Xiaohui Zhang, MD

Sponsors and Collaborators

Peking University People's Hospital

Investigators

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

More Information

• Responsible Party: Xiao Hui Zhang, Vice president of Peking Univeristy Institute of Hematology, Peking University People's Hospital
• ClinicalTrials.gov Identifier: NCT05446831
• Other Study ID Numbers: PKU-ITP2207
• First Posted: July 7, 2022
• Last Update Posted: September 28, 2022
• Last Verified: September 2022

• Studies a U.S. FDA-regulated Drug Product: No
• Studies a U.S. FDA-regulated Device Product: No

Additional relevant MeSH terms:

Thrombocytopenia; Purpura, Thrombocytopenic, Idiopathic; Pathologic Processes; Blood Platelet Disorders; Hematologic Diseases; Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Thrombotic Microangiopathies; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Skin Manifestations