Clinical trial NCT06900920: A Study of TQB3473 Tablets Compared to Placebo in the Treatment of Adult Primary Immune Thrombocytopenia
Official title: A Randomized, Double-blind, Placebo-controlled, Multicenter Phase III Clinical Trial to Evaluate the Efficacy and Safety of TQB3473 Tablets Compared to Placebo in the Treatment of Adult Primary Immune Thrombocytopenia (ITP)
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: PHASE3
Conditions: Primary Immune Thrombocytopenia
Lead sponsor: Chia Tai Tianqing Pharmaceutical Group Co., Ltd.
Sponsor class: INDUSTRY
First posted: 2025-03-28
Enrollment: 199
Sex: ALL
Minimum age (years): 18
Maximum age (years): 75
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: This is a randomized, double-blind, placebo-controlled Phase III clinical study designed to demonstrate that TQB3473 tablets significantly improve the sustained response rate compared to placebo in adult patients with chronic ITP who have previously received standard corticosteroid therapy and have failed or relapsed after at least one standard ITP treatment.

The study consists of a treatment period and a safety follow-up period.


Eligibility criteria: Inclusion Criteria:

* Subjects voluntarily join this study, sign the informed consent form (ICF), and demonstrate good compliance;
* Male or female subjects aged 18 to 75 years (calculated based on the date of signing the informed consent form);
* Eastern Cooperative Oncology Group (ECOG) score of 0-1;
* Diagnosis of primary ITP prior to randomization, with a duration of ≥12 months;
* An average of at least three independent platelet counts (spaced more than 3 days apart) within the 3 months prior to randomization \<30×10\^9/L, with no count \>35×10\^9/L; and no severe bleeding within 4 weeks prior to screening;
* Previous treatment with glucocorticoids and at least one standard ITP therapy that was ineffective or resulted in relapse.
* Female subjects of childbearing potential must agree to use effective contraception during the study and for 6 months after the end of study treatment, with a negative serum or urine pregnancy test within 7 days prior to study enrollment; male subjects must agree to use effective contraception during the study and for 6 months after the end of study treatment, as detailed in Section 5.5.

Exclusion Criteria:

* Evidence of secondary causes of ITP (e.g., untreated Helicobacter pylori infection, leukemia, lymphoma, history of autoimmune diseases such as systemic lupus erythematosus, Hashimoto's thyroiditis, etc.) or drug-induced causes (e.g., anticonvulsants, antibiotics, heparin, etc.), or immune-mediated cytopenia involving two or more cell lines such as Evans syndrome or immune-related cytopenia;
* Subjects with a history or current diagnosis of myelofibrosis, myelodysplastic syndrome, aplastic anemia, lymphoproliferative disorders, or other malignant hematologic diseases;
* History of or concurrent other malignancies within the past 3 years. Exceptions include: other malignancies treated with a single surgical procedure and achieving continuous 5-year disease-free survival (DFS); cured cervical carcinoma in situ, non-melanoma skin cancer, and superficial bladder tumors \[Ta (non-invasive tumor), Tis (carcinoma in situ), and T1 (tumor invading the basement membrane)\];
* Conditions affecting venous blood sampling or multiple factors affecting oral medication (e.g., inability to swallow, chronic diarrhea, intestinal obstruction, etc.);
* Adverse reactions from previous treatments have not resolved to ≤CTCAE grade 1, except for non-clinically significant and asymptomatic laboratory abnormalities judged by the investigator to pose no safety risks;
* Previous bone marrow/hematopoietic stem cell transplantation or major solid organ transplantation;
* Vaccination within 8 weeks prior to randomization or planned vaccination during the study period;
* Major surgical treatment or significant traumatic injury within 4 weeks prior to randomization, or anticipated need for major elective surgery during the study treatment period (major surgery is defined as level 3 or higher according to the National Surgical Classification Catalog 2022);
* History of intracranial hemorrhage or other severe bleeding in vital organs (≥CTCAE grade 3), or symptomatic gastrointestinal bleeding (e.g., hematemesis, melena, etc., excluding asymptomatic "occult blood test positive" and hemorrhoids) within 6 months prior to screening;
* Arterial/venous thrombotic events within 12 months prior to randomization, such as cerebrovascular accidents (including transient ischemic attacks), deep vein thrombosis, or pulmonary embolism;
* ITP-related symptoms and treatments:

  1. Receipt of immunoglobulins or platelet transfusions within 2 weeks prior to randomization;
  2. Receipt of treatments aimed at increasing platelet counts (including but not limited to glucocorticoids, Thyroid peroxidase (TPO), Thrombopoietin receptor agonists (TPO-RAs), cyclosporine, herbal medicines, etc.) within 4 weeks prior to randomization, except for those meeting inclusion criterion (8);
  3. Use of rituximab or other anti-cluster of differentiation 20 (CD20) drugs within 14 weeks prior to randomization;
  4. Splenectomy within 12 weeks prior to randomization.
* Need for long-term/continuous use of medications affecting platelet function \[including but not limited to aspirin, clopidogrel, ticagrelor, non-steroidal anti-inflammatory drugs (NSAIDs), etc.\] or anticoagulant therapy;
* Known allergy to the active ingredient or excipients of the investigational drug;
* Previous treatment with Syk inhibitors;
* Participation in another clinical trial and use of investigational drugs within 4 weeks prior to randomization;
* Any condition judged by the investigator to pose a significant risk to the subject's safety or to affect the subject's ability to complete the study.

Primary outcomes:
measure: Durable response rate: platelet counts ≥50×10^9/L
timeFrame: During the 24-week randomized double-blind treatment period (Weeks 14-24)
Secondary outcomes:
measure: The effectiveness rate within the first 12 weeks of treatment
timeFrame: Baseline to 12 weeks
measure: The response rate within 24 weeks of treatment
timeFrame: 24 weeks
measure: Time to response: The time from the initiation of treatment to the first platelet count ≥50×10^9/L
timeFrame: The time from the initiation of treatment to the first platelet count ≥50×10^9/L (excluding rescue treatment)
measure: The incidence and severity of bleeding events (Weeks 1-12)
timeFrame: During Weeks 1-12 of the double-blind treatment period
measure: The incidence and severity of bleeding events (Weeks 1-24)
timeFrame: During Weeks 1-24 of the double-blind treatment period
measure: The proportion of subjects who receive rescue treatment
timeFrame: During the 24-week double-blind treatment period
measure: The change of concomitant therapy
timeFrame: During the 24-week double-blind treatment period
measure: The incidence and severity of adverse events
timeFrame: From the signing of the informed consent until 28 days after the last dose of study drug or the initiation of new ITP treatment, whichever occurs first
Other outcomes:
Locations:
city: Hefei
country: China
facility: The first affiliated hospital of ustc anhui provincial hospital
state: Anhui
city: Wuhu
country: China
facility: The First Affiliated Hospital of Wannan Medical College
state: Anhui
city: Beijing
country: China
facility: Beijing Chaoyang Hospital, Capital Medical University
state: Beijing
city: Beijing
country: China
facility: Peking Union Medical College Hospital
state: Beijing
city: Chongqing
country: China
facility: The Second Affiliated Hospital of Chongqing Medical University
state: Chongqing
city: Lanzhou
country: China
facility: The First Hospital of Lanzhou University
state: Gansu
city: Guangzhou
country: China
facility: Nanfang Hospital, Southern Medical University
state: Guangdong
city: Shenzhen
country: China
facility: Shenzhen Nanshan People's Hospital
state: Guangdong
city: Zhanjiang
country: China
facility: Central People's Hospital of Zhanjiang
state: Guangdong
city: Nanning
country: China
facility: The Second Affiliated Hospital of Guangxi Medical University
state: Guangxi
city: Guiyang
country: China
facility: The Affiliated Hospital of Guizhou Medical University
state: Guizhou
city: Haikou
country: China
facility: The First Affiliated Hospital of Hainan Medical University
state: Hainan
city: Baoding
country: China
facility: Affiliated Hospital of Hebei University / School of Clinical Medicine
state: Hebei
city: Chengde
country: China
facility: Affiliated Hospital of ChengDe Medical University
state: Hebei
city: Shijiazhuang
country: China
facility: The First Hospital of Hebei Medical University
state: Hebei
city: Tangshan
country: China
facility: North China University of science and technology
state: Hebei
city: Harbin
country: China
facility: The First Affiliated Hospital of Harbin Medical University
state: Heilongjiang
city: Anyang
country: China
facility: Anyang People's Hospital
state: Henan
city: Nanyang
country: China
facility: Nanyang Central Hospital
state: Henan
city: Xuchang
country: China
facility: Xuchang Central Hospital
state: Henan
city: Zhengzhou
country: China
facility: The First Affiliated Hospital of Zhengzhou University
state: Henan
city: Zhengzhou
country: China
facility: Henan Cancer Hospital
state: Henan
city: Zhengzhou
country: China
facility: The First People's Hospital of Ping Ding Shan
state: Henan
city: Wuhan
country: China
facility: Union Hospital, Tongji Medical College, Huazhong University of Science and Technology
state: Hubei
city: Wuhan
country: China
facility: Zhongnan Hospital of Wuhan University
state: Hubei
city: Changsha
country: China
facility: The Third Hospital of Changsha
state: Hunan
city: Nanjing
country: China
facility: Nanjing Drum Tower Hospital
state: Jiangsu
city: Nanjing
country: China
facility: Jiangsu Province Hospital
state: Jiangsu
city: Nantong
country: China
facility: Affiliated Hospital of Nantong University
state: Jiangsu
city: Yangzhou
country: China
facility: Northern Jiangsu People's Hospital Affiliated to Yangzhou University
state: Jiangsu
city: Ganzhou
country: China
facility: Ganzhou People's Hospital
state: Jiangxi
city: Nanchang
country: China
facility: The First Affiliated Hospital of NanChang University
state: Jiangxi
city: Changchun
country: China
facility: The first hospital of Jilin University
state: Jilin
city: Shenyang
country: China
facility: Shenjing Hospital Of China Medical University
state: Liaoning
city: Xining
country: China
facility: Qinghai University Affiliated Hospital
state: Qinghai
city: Xi'an
country: China
facility: Xijing Hospital
state: Shaanxi
city: Xi'an
country: China
facility: Xi'an No.3 Hospital
state: Shaanxi
city: Xi'an
country: China
facility: The First Affiliated Hospital of Xi'an Jiaotong University
state: Shaanxi
city: Jinan
country: China
facility: Qilu Hospital of Shandong University
state: Shandong
city: Jinan
country: China
facility: Shandong First Medical University Affiliated Central Hospital
state: Shandong
city: Linyi
country: China
facility: Linyi Central Hospital
state: Shandong
city: Yantai
country: China
facility: Yantai Yuhuangding Hospital (Affiliated Yantai Yuhuangding Hospital of Qingdao University)
state: Shandong
city: Shanghai
country: China
facility: Shanghai Sixth People's Hospital
state: Shanghai
city: Shanghai
country: China
facility: Affiliated Zhongshan Hospital of Fudan University，Qingpu Branch
state: Shanghai
city: Changzhi
country: China
facility: Heping Hospital affiliated to Changzhi Medical College
state: Shanxi
city: Taiyuan
country: China
facility: Shanxi Bethune Hospital
state: Shanxi
city: Tianjin
country: China
facility: Tianjin Medical University General Hospital
state: Tianjin
city: Tianjin
country: China
facility: Tianjin People's Hospital
state: Tianjin
city: Ürümqi
country: China
facility: People's Hospital of Xinjiang Uygur Autonomous Region
state: Xinjiang
city: Ürümqi
country: China
facility: The First Affiliated Hospital of Xinjiang Medical University
state: Xinjiang
city: Kunming
country: China
facility: Kunming Medical University Second Affiliated Hospital
state: Yunnan
city: Hangzhou
country: China
facility: The First Affiliated Hospital of Zhejiang Chinese Medical University
state: Zhejiang
city: Ningbo
country: China
facility: The First Affiliated Hospital of Ningbo University
state: Zhejiang
Study officials:
References: