Telitacicept for the Treatment of Connective Tissue Disease-associated Thrombocytopenia

NCT05998759 | Recruiting Phase 2 DMC

• 2 other identifiers: BJ-2021-235RCTAIIRCTD001
• Study Type: interventional
• Target: 296
• Locations: 1 country
• Sites: 23

Brief Summary

The goal of this clinical trial is to evaluate the efficacy and safety of Telitacicept for the treatment of connective tissue disease-associated thrombocytopenia.

Conditions

Connective Tissue Diseases; Thrombocytopenia

Keywords

Connective Tissue Diseases; Thrombocytopenia; biological agents; B cell; targeted therapy

Outcome Measures

Primary Outcomes (1)

• Overall response (CR + PR) rate

  Response is deemed as complete (CR) if the platelet (PLT) count is ≥ 100×10\^9/L, partial (PR) if the platelet count ranges from 50×10\^9/L to 100×10\^9/L and at least doubled from baseline. No active bleeding is allowed in participants classified as CR or PR.

  at week 24

Secondary Outcomes (8)

• Overall response (CR + PR) rate

  at week 12

• Rescue treatment rate

  at week 24

• Time to rescue treatment

  at week 24

• Relapse rate

  at week 24

• Time to relapse

  at week 24

Other Outcomes (13)

• life quality 1

  at week 24

• life quality 2

  at week 24

• Absolute change rate from baseline in serum immunoglobulin G (IgG)

  at week 24

Study Arms (2)

Telitacicept plus standard therapy

EXPERIMENTAL

Telitacicept (160mg ih qw for 24 weeks) combined with standard therapy. Standard therapy refers to the following treatment (monotherapy or in combination): glucocorticoid, hydroxychloroquine, and other immunosuppressants (i.e. cyclophosphamide, cyclosporine, mycophenolate mofetil, azathioprine, tacrolimus, methotrexate and leflunomide, et al.).

Biological: Telitacicept

Placebo plus standard therapy

PLACEBO COMPARATOR

Placebo combined with standard therapy. Standard therapy refers to the following treatment (monotherapy or in combination): glucocorticoid, hydroxychloroquine, and other immunosuppressants (i.e. cyclophosphamide, cyclosporine, mycophenolate mofetil, azathioprine, tacrolimus, methotrexate and leflunomide, et al.).

Drug: Placebo

Interventions

Telitacicept BIOLOGICAL

subcutaneous telitacicept 160 mg weekly for 24 weeks.

Also known as: recombinant human B lymphocyte stimulating factor receptor-Fc fusion protein, Recombinant Human B Lymphocyte(RC18), Tai'ai®

Telitacicept plus standard therapy

Placebo DRUG

subcutaneous placebo weekly for 24 weeks.

Also known as: Placebo control, control

Placebo plus standard therapy

Eligibility Criteria

• Age: 18 Years - 75 Years
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects who have been diagnosed with connective tissue disease (CTD)-associated thrombocytopenia. And CTD includes primary Sjögren syndrome (according to the 2002 American College of Rheumatology (ACR)/ European League against Rheumatism (EULAR) classification criteria), systemic lupus erythematosus (SLE, according to the 1997 or the 2009 ACR classification criteria), and undifferentiated connective tissue disease (according to the 1999 international classification criteria)
• Refractory thrombocytopenia defined as:
• Either: Failure to maintain sustained remission after treatment by glucocorticoid and at least one immunosuppressant (i.e. cyclophosphamide, cyclosporine, mycophenolate mofetil, azathioprine, tacrolimus, methotrexate, leflunomide and hydroxychloroquine, et al.) Or: Relapse during oral glucocorticoid tapering or after withdrawal
• ×10\^9/L\>PLT
• anti-nuclear antibody (ANA) positive (≥1:80, any karyotype) detected in the laboratory of each research center
• Standard therapy should be maintained stable for at least 14 days prior to the first dose of the experimental drug or placebo. Standard therapy refers to the following treatment (monotherapy or in combination): glucocorticoid, hydroxychloroquine, and other immunosuppressants (i.e. cyclophosphamide, cyclosporine, mycophenolate mofetil, azathioprine, tacrolimus, methotrexate and leflunomide, et al.)
• Signed informed consent form, willing or able to participate in all required study evaluations and procedures

You may not qualify if:

• Vital organ lethal bleeding (including but not limited to central nervous system bleeding, digestive tract bleeding) at screening, or intracranial bleeding 6 months prior to screening
• Antiphospholipid syndrome, thrombotic thrombocytopenia purpura, hemolytic uremic syndrome, or thrombocytopenia secondary to other causes (such as sepsis, Epstein-Barr virus infection, cytomegalovirus infection, Corona Virus Disease-19 (COVID-19) infection, drugs, etc.)
• Hematopoietic system disorders, such as myelodysplastic syndrome, paroxysmal sleep hemoglobinuria, aplastic anemia, leukemia, lymphoma, myelofibrosis and so on
• Severe cardiovascular system disease, including: unstable or uncontrollable disease or condition affecting the function of the heart (such as angina pectoris, congestive heart failure, uncontrolled hypertension or arrhythmia)
• Arteriovenous thromboembolism events
• Receiving antiplatelet or anticoagulant therapy at screening
• Clinically significant electrocardiogram changes
• corrected Q-T interval (QTc)\>450ms for male, QTc\>470ms for female
• Severe pulmonary disease, including: unstable or uncontrollable disease or condition affecting respiratory function \[e.g., diffuse alveolar hemorrhage, severe pulmonary hypertension, severe pulmonary interstitial disease (peripheral blood oxygen saturation \<92% at rest without oxygen, or forced vital capacity (FVC)\<50%, or carbon monoxide diffusing capacity (DLCO)\<50%)\]
• Severe kidney disease, including: severe lupus nephritis (urinary protein \> 6 g/24 hours or endogenous creatinine clearance \< 30 ml /min) 8 weeks prior to randomization, active nephritis requiring current protocol disallowed drugs, severe renal insufficiency requiring hemodialysis or prednisone ≥100mg/ day (or equivalent) for ≥14 days
• SLE or non-SLE related central nervous system disease (including epilepsy, psychosis, organic encephalopathy syndrome, cerebrovascular accident, encephalitis, central nervous system vasculitis) 8 weeks prior to randomization
• Active hepatitis, a history of severe liver disease. Subjects positive for hepatitis B surface antigen (HBsAg) or antibodies to hepatitis C virus are excluded. As for subjects with antibodies to hepatitis B core antigen (HBcAb), further hepatitis B virus (HBV)-DNA should be tested. If HBV-DNA is negative, subjects could be enrolled; otherwise, subjects should be excluded
• Abnormal laboratory results (including but not limited to: alanine aminotransferase (ALT) or aspertate aminotransferase (AST)≥3×ULN (upper limit of normal), white blood cell count \<1.5×10\^9/L)
• Subjects with known active infections (e.g., shingles, COVID-19, HIV, active tuberculosis, etc.), and active or recurrent gastrointestinal ulcers
• Pregnant or lactating women, and subjects with a during plan during the trial

Sponsors & Collaborators

• Beijing Hospital: lead
• The First Affiliated Hospital of Anhui Medical University: collaborator
• The First Affiliated Hospital of China University of Science and Technology (Anhui Provincial): collaborator
• Peking University People's Hospital: collaborator
• Peking University Third Hospital: collaborator
• First Affiliated Hospital, Sun Yat-Sen University: collaborator
• Guangdong Provincial People's Hospital: collaborator
• The First Affiliated Hospital of Zhengzhou University: collaborator
• Union Hospital, Tongji Medical College, Huazhong University of Science and Technology: collaborator
• Second Xiangya Hospital of Central South University: collaborator
• Xiangya Hospital of Central South University: collaborator
• The Affiliated Hospital of Inner Mongolia Medical University: collaborator
• First Hospital of China Medical University: collaborator
• Shandong Provincial Hospital: collaborator
• Changhai Hospital: collaborator
• RenJi Hospital: collaborator
• Shanxi Bethune Hospital: collaborator
• West China Hospital: collaborator
• Institute of Hematology & Blood Diseases Hospital, China: collaborator
• Tianjin First Central Hospital: collaborator
• Tianjin Medical University General Hospital: collaborator
• People's Hospital of Xinjiang Uygur Autonomous Region: collaborator
• The First People's Hospital of Yunnan: collaborator

Study Sites (23)

The First Affiliated Hospital of Anhui Medical College

Hefei, Anhui, 230022, China

RECRUITING

The First Affiliated Hospital of China University of Science and Technology (Anhui Provincial)

Hefei, Anhui, China

RECRUITING

Beijing Hospital

Beijing, Beijing Municipality, China

RECRUITING

Peking University People's Hospital

Beijing, Beijing Municipality, China

RECRUITING

Peking University Third Hospital

Beijing, Beijing Municipality, China

RECRUITING

First Affiliated Hospital, Sun Yat-Sen University

Guangzhou, Guangdong, China

RECRUITING

Guangdong Provincial People's Hospital

Guangzhou, Guangdong, China

RECRUITING

The First Affiliated Hospital of Zhengzhou University

Zhengzhou, Henan, China

RECRUITING

Wuhan Union Hospital, China

Wuhan, Hubei, China

RECRUITING

Second Xiangya Hospital of Central South University

Changsha, Hunan, China

RECRUITING

Xiangya Hospital of Central South University

Changsha, Hunan, China

RECRUITING

The Affiliated Hospital of Inner Mongolia Medical University

Hohhot, Inner Mongolia, China

RECRUITING

First Hospital of China Medical University

Shenyang, Liaoning, China

RECRUITING

Shandong Provincial Hospital

Jinan, Shandong, China

RECRUITING

Changhai Hospital

Shanghai, Shanghai Municipality, China

RECRUITING

RenJi Hospital

Shanghai, Shanghai Municipality, China

RECRUITING

Shanxi Bethune Hospital

Taiyuan, Shanxi, China

RECRUITING

West China Hospital

Chengdu, Sichuan, China

RECRUITING

Institute of Hematology & Blood Diseases Hospital

Tianjin, Tianjin Municipality, China

RECRUITING

Tianjin First Central Hospital

Tianjin, Tianjin Municipality, China

RECRUITING

Tianjin Medical University General Hospital

Tianjin, Tianjin Municipality, China

RECRUITING

People's Hospital of Xinjiang Uygur Autonomous Region

Ürümqi, Xinjiang Uygur Autonomous Region, China

RECRUITING

The First People's Hospital of Yunnan

Kunming, Yunnan, China

RECRUITING

MeSH Terms

Conditions

Connective Tissue Diseases; Thrombocytopenia

Interventions

telitacicept

Condition Hierarchy (Ancestors)

Skin and Connective Tissue Diseases; Blood Platelet Disorders; Hematologic Diseases; Hemic and Lymphatic Diseases; Cytopenia

Study Officials

• Xuan Zhang, MD.

  Beijing Hospital

  STUDY CHAIR

• Yongjing Cheng, MD.

  Beijing Hospital

  STUDY CHAIR

• Shengqian Xu, MD.

  The First Affiliated Hospital of Anhui Medical Hospital

  PRINCIPAL INVESTIGATOR

• Zhu Chen, MD.

  The First Affiliated Hospital of China University of Science and Technology (Anhui Provincial)

  PRINCIPAL INVESTIGATOR

• Jing He, MD.

  Peking University People's Hospital

  PRINCIPAL INVESTIGATOR

• Rong Mu, MD.

  Peking University Third Hospital

  PRINCIPAL INVESTIGATOR

• Niansheng Yang, MD.

  First Affiliated Hospital, Sun Yat-Sen University

  PRINCIPAL INVESTIGATOR

• Yang Li, MD.

  Guangdong Provincial People's Hospital

  PRINCIPAL INVESTIGATOR

• Guanmin Gao, MD.

  The First Affiliated Hospital of Zhengzhou University

  PRINCIPAL INVESTIGATOR

• Anbin Huang, MD.

  Union Hospital, Tongji Medical College, Huazhong University of Science and Technology

  PRINCIPAL INVESTIGATOR

• Fen Li, MD.

  Second Xiangya Hospital of Central South University

  PRINCIPAL INVESTIGATOR

• Hui Luo, MD.

  Xiangya Hospital of Central South University

  PRINCIPAL INVESTIGATOR

• Hongbin Li, MD.

  The Affiliated Hospital of Inner Mongolia Medical University

  PRINCIPAL INVESTIGATOR

• Pinting Yang, MD.

  First Hospital of China Medical University

  PRINCIPAL INVESTIGATOR

• Hongsheng Sun, MD.

  Shandong Provincial Hospital

  PRINCIPAL INVESTIGATOR

• Dongbao Zhao, MD.

  Changhai Hospital

  PRINCIPAL INVESTIGATOR

• Sheng Chen, MD.

  RenJi Hospital

  PRINCIPAL INVESTIGATOR

• Liyun Zhang, MD.

  Shanxi Bethune Hospital

  PRINCIPAL INVESTIGATOR

• Qibing Xie, MD.

  West China Hospital

  PRINCIPAL INVESTIGATOR

• Lei Zhang, MD.

  Institute of Hematology & Blood Diseases Hospital, China

  PRINCIPAL INVESTIGATOR

• Wufang Qi, MD.

  Tianjin First Central Hospital

  PRINCIPAL INVESTIGATOR

• Wei Wei, MD.

  Tianjin Medical University General Hospital

  PRINCIPAL INVESTIGATOR

• Lijun Wu, MD.

  People's Hospital of Xinjiang Uygur Autonomous Region

  PRINCIPAL INVESTIGATOR

• Qin Li, MD.

  The First People's Hospital of Yunnan

  PRINCIPAL INVESTIGATOR

Central Study Contacts

Xuan Zhang, MD.

CONTACT

+86-01085136736; zxpumch2003@sina.com

Yongjing Cheng, MD.

CONTACT

+86-01085136736; chengyongjing3427@njhmoh.cn

Study Design

• Study Type: interventional
• Phase: phase 2
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER GOV
• Responsible Party: PRINCIPAL INVESTIGATOR
• PI Title: Professor

Study Record Dates

• First Submitted: August 3, 2023
• First Posted: August 21, 2023
• Study Start: December 2, 2023
• Primary Completion: December 1, 2025
• Study Completion: December 1, 2025
• Last Updated: May 6, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

CN (23)