Baricitinib for Steroid-resistant/Relapse Immune Thrombocytopenia

NCT05446831 | Unknown Phase 2 DMC

• 1 other identifier: PKU-ITP2207
• Study Type: interventional
• Target: 35
• Locations: 1 country
• Sites: 1

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).

Conditions

ITP; Immune Thrombocytopenia

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (9)

• Complete response (CR)

  1 month

• Response (R)

  1 month

• Time to response

  6 months

• Duration of response

  6 months

• Early response

  7 days

Study Arms (1)

Baricitinib

EXPERIMENTAL

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

Interventions

Baricitinib 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.

Baricitinib

Eligibility Criteria

• Age: 18 Years - 70 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Primary immune thrombocytopenia (ITP) confirmed by excluding other supervened causes of thrombocytopenia
• Patients with chronic low platelet count (\<30,000/μL) for 6 months who have failed at least one treatment for chronic low platelet count
• 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
• Patients with a platelet count \<30,000/μL or a platelet count \<50,000/μL with clinically significant bleeding symptoms at the enrollment
• Over 18 years old
• Willing and able to provide written informed consent, and agreeable to the schedule of assessment

You may not qualify if:

• Secondary immune thrombocytopenia (e.g. patients with HIV, HCV, Helicobacter pylori infection or patients with confirmed autoimmune disease)
• Active or a history of malignancy
• Pregnancy or lactation
• Current or recent (\<4 weeks prior to screening) clinically serious viral, bacterial, fungal, or parasitic infection
• A history of symptomatic herpes zoster infection within 12 weeks prior to screening
• Active or chronic viral infection from hepatitis B virus (HBV), hepatitis C virus (HCV), or human immunodeficiency virus (HIV)
• 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
• 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
• Myocardial infarction (MI), unstable ischemic heart disease, stroke, or New York Heart Association Stage IV heart failure
• 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
• Any of the following specific abnormalities on screening laboratory tests:
• \) 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

Sponsors & Collaborators

• Peking University People's Hospital: lead

Study Sites (1)

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

Beijing, Beijing Municipality, 100010, China

RECRUITING

MeSH Terms

Conditions

Purpura, Thrombocytopenic, Idiopathic

Interventions

baricitinib

Condition Hierarchy (Ancestors)

Purpura, Thrombocytopenic; Purpura; Blood Coagulation Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Thrombotic Microangiopathies; Thrombocytopenia; Blood Platelet Disorders; Cytopenia; Hemorrhagic Disorders; Autoimmune Diseases; Immune System Diseases; Hemorrhage; Pathologic Processes; Pathological Conditions, Signs and Symptoms; Skin Manifestations; Signs and Symptoms

Study Officials

• Xiaohui Zhang, MD

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xiaohui Zhang, MD

CONTACT

+8613522338836; zhangxh100@sina.com

Peng Zhao, MD

CONTACT

+8618810323668; zpeng702@163.com

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: OTHER
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Vice president of Peking Univeristy Institute of Hematology

Study Record Dates

• First Submitted: July 1, 2022
• First Posted: July 7, 2022
• Study Start: July 13, 2022
• Primary Completion: June 1, 2023
• Study Completion: December 1, 2023
• Last Updated: September 28, 2022

Record last verified: 2022-09

Locations

CN (1)