A Study to Evaluate the Efficacy and Safety of Orelabrutinib in Adult Patients With Immune Thrombocytopenia

A Randomized, Multi-center, Adaptive Phase IIa/IIb Study to Evaluate the Efficacy and Safety of Orelabrutinib in Adult Patients With Persistent or Chronic Primary Immune Thrombocytopenia

The study is designed to be a randomized, open, multi-center, phase IIa/IIb seamless adaptive trial.

Phase IIa: The study consists of a screening period, a core treatment period, an open label extension period, and a safety follow-up period Phase IIb: At present, a preliminary exploratory study (i.e., phase IIa study) will be conducted first. The design of the phase IIb study (including the selection of populations) will be clarified after a relatively clear understanding of the therapeutic effect, value, risks and benefits of the BTK inhibitor for ITP is obtained.

Study Overview

• Status: Recruiting
• Conditions: Primary Immune Thrombocytopenia
• Intervention / Treatment: Drug: Orelabrutinib( lower dose); Drug: Orelabrutinib( higher dose)

• Study Type: Interventional
• Enrollment (Anticipated): 30
• Phase: Phase 2

Contacts and Locations

• Study Contact: Name: Ming Hou, PhD; Phone Number: 18560087007; Email: houming@medmail.com.cn

Study Locations

• China
  • Hainan
    • HaiKou, Hainan, China, 570100
      • Recruiting
      • Hainan People's Hospital
      • Contact: Li'e Lin
  • Henan
    • ZhengZhou, Henan, China, 450000
      • Recruiting
      • Henan Tumor Hospital
    • ZhengZhou, Henan, China, 450000
      • Recruiting
      • The First Affiliated Hospital of Zhengzhou University
      • Contact: Fang Wang
  • Hubei
    • WuHan, Hubei, China, 430000
      • Recruiting
      • Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
      • Contact: Heng Mei, PhD
    • YiChang, Hubei, China, 443000
      • Recruiting
      • Yichang Central People's Hospital
  • Jiangsu
    • WuXi, Jiangsu, China, 214000
      • Recruiting
      • Wuxi People's Hospital
    • XuZhou, Jiangsu, China, 221000
      • Recruiting
      • Affiliated Hospital of Xuzhou Medical University
  • Jiangxi
    • NanChang, Jiangxi, China, 330000
      • Recruiting
      • First Hospital of Nanchang University
      • Contact: Ruibin Huang
  • Shandong
    • JiNan, Shandong, China, 250012
      • Recruiting
      • Qilu Hospital of Shandong University
      • Contact: Ming Hou

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years to 80 years (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

Inclusion Criteria:

1. Subjects have had a detailed understanding of the nature, significance, possible benefits, possible inconveniences, and potential risks of the trial, understood the study procedures, and voluntarily signed a written ICF before the study.
2. Males or females aged from 18 to 80 years (including the marginal values).
3. With a body weight of ≥ 35 kg at screening.
4. Diagnostic criteria:the diagnosis of persistent (3-12 months) or chronic (≥ 12 months) ITP is met
5. Patients who have failed at least 1 prior first-line standard therapy for ITP, or who have failed to tolerate a standard therapy.
6. Women of childbearing potential must take a complementary barrier method of contraception in combination with a highly effective method of contraception at screening, throughout the trial, and within 90 days after the last dose of the investigational drug.
7. The mean of two platelet counts is less than 30 × 109/L and no platelet count is greater than 35 × 109/L during the screening visit and/or before the first dose.

Exclusion Criteria:

1. Severe hemorrhage occurred within 4 weeks prior to screening.
2. Subjects suffer from severe ITP at screening
3. Subjects have other diseases which mention in protocol
4. Subjects develop intracranial hemorrhage within 6 months prior to screening.
5. Active and uncontrollable infection
6. Subjects have a history of coagulopathy other than ITP
7. Subjects with a history of malignancies.
8. History of major organ transplantation or hematopoietic stem cell/bone marrow transplantation.
9. Subjects with a known history of hypersensitivity to the investigational drug as described in the Protocol, or any ingredients.
10. Subjects with a Medication history and surgical history which mention in protocol
11. Subjects do not meet the criterion of the laboratory test in protocol

NOTE: Other protocol defined Inclusion/Exclusion criteria may apply.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: None (Open Label)

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Lower Dose; Orelabrutinib is a white, round, uncoated tablet	Drug: Orelabrutinib( lower dose); Orelabrutinib is a white, round, uncoated tablet, will be taken lower dose QD by patients with persistent or chronic primary immune thrombocytopenia
Experimental: Higher Dose; Orelabrutinib is a white, round, uncoated tablet	Drug: Orelabrutinib( higher dose); Orelabrutinib is a white, round, uncoated tablet, will be taken higher dose QD by patients with persistent or chronic primary immune thrombocytopenia

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Time Frame
Proportion of subjects with the platelet count of ≥ 50 × 109/L after 12 weeks of treatment	12 weeks

Secondary Outcome Measures

Outcome Measure	Time Frame
Proportion of subjects who achieve a complete response (CR) over treatment time. CR is defined as a post-treatment platelet count of ≥100 × 109/L	25 weeks
Occurrence of treatment emergent adverse events (TEAE) and treatment-related adverse events (TRAE) were evaluated according to severity	25 weeks

Other Outcome Measures

Outcome Measure	Measure Description	Time Frame
Cmax	To obtain pharmacokinetic (PK) data of Orelabrutinib include the peak plasma concentration (Cmax)	25 weeks

Collaborators and Investigators

• Sponsor: Beijing InnoCare Pharma Tech Co., Ltd.

Study record dates

Study Major Dates

• Study Start (Actual): February 21, 2022
• Primary Completion (Anticipated): December 30, 2023
• Study Completion (Anticipated): December 30, 2024

Study Registration Dates

• First Submitted: December 20, 2021
• First Submitted That Met QC Criteria: February 7, 2022
• First Posted (Actual): February 9, 2022

Study Record Updates

• Last Update Posted (Actual): January 12, 2023
• Last Update Submitted That Met QC Criteria: January 10, 2023
• Last Verified: January 1, 2023

More Information

Terms related to this study

• Keywords: Persistent or chronic
• 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: ICP-CL-00116

Drug and device information, study documents

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