問卷
Log In
繁中 EN
TPIDB
TPIDB Outstanding PI
TPIDB > Search Result > Clinical Trials List

Clinical Trials List

Protocol NumberR3918-PNH-2021
NCT Number(ClinicalTrials.gov Identfier)NCT05133531
Active

2022-07-01 - 2027-07-31

Phase III

Not yet recruiting9

ICD-10D59.5

Paroxysmal nocturnal hemoglobinuria [Marchiafava-Micheli]

ICD-10D59.6

Hemoglobinuria due to hemolysis from other external causes

ICD-10D59.8

Other acquired hemolytic anemias

ICD-9283.2

Hemoglobinuria due to hemolysis from external causes

A Randomized, Open-Label, C5 Inhibitor-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients With Paroxysmal Nocturnal Hemoglobinuria Who Are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor Therapy

  • Sponsor

  • Trial scale

    Multi-Regional Multi-Center

  • Update

    2026/02/01

Investigators and Locations

Principal Investigator 黃威翰 Division of Hematology & Oncology
Hualien Tzu Chi Hospital

Co-Principal Investigator

  • 黃聖娟 Division of Hematology & Oncology

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator 林炫聿 Division of Hematology & Oncology
CHANGHUA CHRISTIAN HOSPITAL

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator 陳宇欽 Division of Hematology & Oncology
Tri-Service General Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Su-Peng Yeh Division of Hematology & Oncology
China Medical University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Hui-Hua Hsiao Division of Hematology & Oncology
Kaohsiung Medical Univeristy Chung-Ho Memorial Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator HSIN-AN HOU Division of General Internal Medicine
National Taiwan University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Hung Chang Division of Hematology & Oncology
Linkou Chang Gung Medical Foundation

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator HSIN-CHEN LIN Division of Hematology & Oncology
Taichung Veterans General Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Principal Investigator Tsai-Yun Chen Division of Hematology & Oncology
National Taiwan University Hospital

Co-Principal Investigator

The Actual Total Number of Participants Enrolled

0 Not yet recruiting

Condition/Disease

Paroxysmal Nocturnal Hemoglobinuria

Objectives

The primary objective of Arm A was to describe the effect of concomitant pozelimab + cemdisiran therapy compared with ravulizumab therapy on hemolysis, assessed by lactate dehydrogenase (LDH), during 26 weeks of treatment in patients with active paroxysmal nocturnal hemoglobinuria (PNH) who had not received complement inhibitor therapy or had not recently received complement inhibitor therapy. The primary objective of Arm B was to evaluate the effect of concomitant pozelimab + cemdisiran therapy versus eculizumab therapy on hemolysis, as assessed by sustained suppression of LDH and avoidance of red blood cell (RBC) transfusions, during 26 weeks of treatment in patients with active PNH who had not received complement inhibitor therapy or had not recently received complement inhibitor therapy. Secondary objectives for Group A: • Describe the effect of concomitant treatment with pozelimab + cemdisiran compared with treatment with ravulizumab on: - Hemolysis measurement - Transfusion parameters - Hemoglobin concentration - Fatigue as assessed by the Clinical Outcome Assessment (COA) - Health-related quality of life (HRQoL) assessed by COA - Safety and tolerability - Complement activation • Evaluate the total pozelimab concentration in serum versus the total ravulizumab concentration, and the cemdisiran concentration in plasma versus the total complement factor 5 (C5) protein concentration • Evaluate the immunogenicity of pozelimab and cemdisiran Secondary objectives for Group B: • To evaluate the effect of concomitant treatment with pozelimab + cemdisiran compared with eculizumab on: - Hemolysis measurement - Transfusion parameters - Hemoglobin concentration - Fatigue as assessed by COA

Test Drug

Pozelimab
Cemdisiran
Ravulizumab
Eculizumab

Active Ingredient

Pozelimab
Cemdisiran
Ravulizumab
Eculizumab

Dosage Form

IV or SC
SC
IV
IV

Dosage

200 mg/mL
200 mg/mL
100 mg/mL
10 mg/ml

Endpoints

• Percent change in LDH from baseline to week 26
• Transfusion freedom (defined as no need for RBC transfusions based on post-baseline hemoglobin values ​​per the protocol algorithm) from Day 1 to Week 26

Inclution Criteria

Key Inclusion Criteria:

Diagnosis of PNH confirmed by high-sensitivity flow cytometry testing with PNH granulocytes or monocytes as described in the protocol
Active disease, as defined by the presence of 1 or more PNH-related signs or symptoms as described in the protocol
LDH level ≥2 × ULN at the screening visit
Willing and able to comply with clinic/remote visits and study-related procedures, including completion of the full series of meningococcal vaccinations required per protocol

Exclusion Criteria

Key Exclusion Criteria:

Prior treatment with eculizumab within 3 months prior to screening, ravulizumab within 6 months prior to screening, or other complement inhibitors within 5 half-lives of the respective agent prior to screening
Receipt of an organ transplant, history of bone marrow transplantation or other hematologic transplant
Body weight <40 kilograms at screening visit
Planned use of any complement inhibitor therapy other than study drugs during the treatment period
Not meeting meningococcal vaccination requirements and, at a minimum documentation of quadrivalent meningococcal vaccination within 5 years prior to the screening visit and serotype B vaccine within 3 years prior to the screening visit as described in the protocol.
Any contraindication for receiving Neisseria meningitidis vaccinations (serotypes ACWY and B).
Unable to take antibiotics for meningococcal prophylaxis (if required by local ravulizumab [Cohort A] or eculizumab [Cohort B] prescribing information, where available, or national guidelines/local practice, or if necessary when administration of the first dose of the vaccination is less than 2 weeks prior to study treatment initiation)
Any active, ongoing infection or a recent infection requiring ongoing systemic treatment with antibiotics, antivirals, or antifungals within 2 weeks of screening or during the screening period
Documented history of active, uncontrolled, ongoing systemic autoimmune diseases

The Estimated Number of Participants

  • Taiwan

    45 participants

  • Global

    190 participants

聯絡我們

台灣臨床試驗主持人資料庫 TPIDB

All Contents Copyright 2020 台灣臨床試驗主持人資料庫TPIDB