Clinical Trials List
Protocol NumberTP0006
NCT Number(ClinicalTrials.gov Identfier)NCT04224688
2020-10-20 - 2022-04-28
Phase III
Not yet recruiting2
Recruiting3
ICD-10D47.3
Essential (hemorrhagic) thrombocythemia
ICD-10D69.3
Immune thrombocytopenic purpura
ICD-10D69.41
Evans syndrome
ICD-10D69.42
Congenital and hereditary thrombocytopenia purpura
ICD-10D69.49
Other primary thrombocytopenia
ICD-9287.3
Primary thrombocytopenia
A Phase 3 Multicenter, Double-Blind, Randomized, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of Rozanolixizumab in Adult Study Participants With Persistent or Chronic Primary Immune Thrombocytopenia (ITP)
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Trial Applicant
PAREXEL INTERNATIONAL CO., LTD.
-
Sponsor
UCB Biopharma SRL
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Trial scale
Multi-Regional Multi-Center
-
Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- Sin-Syue Li Division of General Internal Medicine
- Ya-Ping Chen Division of General Internal Medicine
- Ya-Ting Hsu Division of General Internal Medicine
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Chieh-Lung Cheng Division of General Internal Medicine
- 田豐銘 無
- Chien-Chin Lin Division of General Internal Medicine
- HSIN-AN HOU 無
- MING YAO 無
- Huai-Hsuan Huang Division of General Internal Medicine
- - - 無
- - - 無
- 林明恩 無
- CHENG-HONG TSAI 無
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
The Actual Total Number of Participants Enrolled
0 Not yet recruiting
Co-Principal Investigator
- Tsu-Yi Chao Division of Hematology & Oncology
- 莊博雅 Division of Hematology & Oncology
- Wei-Hong Cheng Division of Hematology & Oncology
- HUI-WEN LIU Division of Hematology & Oncology
- Yao-Yu Hsieh Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Primary Immune Thrombocytopenia(ITP)
Objectives
Main objective of the trial:
Demonstrate the clinical efficacy of rozanolixizumab in maintenance treatment in study participants with primary immune thrombocytopenia (ITP)
Secondary objectives of the trial:
Assess the safety and tolerability of rozanolixizumab
Test Drug
Rozanolixizumab
Active Ingredient
Rozanolixizumab
Dosage Form
injection
Dosage
140
Endpoints
Primary end point:
Durable Clinically Meaningful Platelet Response of ≥50x10^9/L during the last 12 weeks
Secondary end point(s):
1. Cumulative number of visits with Clinically Meaningful Platelet Response of ≥50x10^9/L
2. Response defined as platelet count ≥30x10^9/L and at least a 2-fold increase of the Baseline count confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit
3. Complete Response defined as platelet count ≥100x10^9/L confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit
4. Time to Clinically Meaningful Platelet Response of ≥50x10^9/L: time from starting treatment to achievement of first response of ≥50x10^9/L
5. Clinically Meaningful Platelet Response of ≥50x10^9/L by Day 8
6. Time to first rescue therapy
7. Response defined as change from Baseline at or above the defined threshold for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
8. Occurrence of treatment-emergent adverse events (TEAEs)
9. Occurrence of TEAEs leading to withdrawal of investigational medicinal product (IMP)
Durable Clinically Meaningful Platelet Response of ≥50x10^9/L during the last 12 weeks
Secondary end point(s):
1. Cumulative number of visits with Clinically Meaningful Platelet Response of ≥50x10^9/L
2. Response defined as platelet count ≥30x10^9/L and at least a 2-fold increase of the Baseline count confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit
3. Complete Response defined as platelet count ≥100x10^9/L confirmed on at least 2 separate occasions at two adjacent nominal visits at least 7 days apart, and absence of bleeding by visit
4. Time to Clinically Meaningful Platelet Response of ≥50x10^9/L: time from starting treatment to achievement of first response of ≥50x10^9/L
5. Clinically Meaningful Platelet Response of ≥50x10^9/L by Day 8
6. Time to first rescue therapy
7. Response defined as change from Baseline at or above the defined threshold for ITP Patient Assessment Questionnaire (ITP-PAQ) Symptoms Score
8. Occurrence of treatment-emergent adverse events (TEAEs)
9. Occurrence of TEAEs leading to withdrawal of investigational medicinal product (IMP)
Inclution Criteria
Inclusion criteria (list the most important) :
-Study participant must be ≥18 years of age at the time of the Screening Visit
-Study participant has a diagnosis of persistent (>3 months duration) or chronic (>12 months duration) primary immune thrombocytopenia (ITP) at the Screening Visit
-Study participant has documented intolerance or insufficient response to one or more appropriate courses of standard of care ITP medication prior to Screening
-Study participants must have prior history of a response to a previous ITP therapy.
-If taking allowed immunosuppressive drugs, study participant must be on stable doses during defined time periods prior to Baseline (Day 1)
-Study participant has a documented history of low platelet count (<30x10^9/L) prior to Screening
-Study participant has a platelet count measurement at Screening and at Baseline (Day 1) with an average of the two <30x10^9/L and no single count may be >35x10^9/L (using local laboratories)
-Study participant has a current or history of a peripheral blood smear consistent with ITP
-Study participants may be male or female:
a. A male participant must agree to use contraception during the Treatment Period and for at least 3 months after the final dose of study treatment and refrain from donating sperm during this period
b. A female participant is eligible to participate if she is not pregnant as confirmed by a negative serum pregnancy test or not planning to get pregnant during the participation in the study, not breastfeeding, and at least one of the following conditions applies:
Not a woman of childbearing potential (WOCBP)
OR
A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 3 months after the dose of study treatment
-Study participant must be ≥18 years of age at the time of the Screening Visit
-Study participant has a diagnosis of persistent (>3 months duration) or chronic (>12 months duration) primary immune thrombocytopenia (ITP) at the Screening Visit
-Study participant has documented intolerance or insufficient response to one or more appropriate courses of standard of care ITP medication prior to Screening
-Study participants must have prior history of a response to a previous ITP therapy.
-If taking allowed immunosuppressive drugs, study participant must be on stable doses during defined time periods prior to Baseline (Day 1)
-Study participant has a documented history of low platelet count (<30x10^9/L) prior to Screening
-Study participant has a platelet count measurement at Screening and at Baseline (Day 1) with an average of the two <30x10^9/L and no single count may be >35x10^9/L (using local laboratories)
-Study participant has a current or history of a peripheral blood smear consistent with ITP
-Study participants may be male or female:
a. A male participant must agree to use contraception during the Treatment Period and for at least 3 months after the final dose of study treatment and refrain from donating sperm during this period
b. A female participant is eligible to participate if she is not pregnant as confirmed by a negative serum pregnancy test or not planning to get pregnant during the participation in the study, not breastfeeding, and at least one of the following conditions applies:
Not a woman of childbearing potential (WOCBP)
OR
A WOCBP who agrees to follow the contraceptive guidance during the Treatment Period and for at least 3 months after the dose of study treatment
Exclusion Criteria
Exclusion criteria (list the most important) :
-Participant has a history of arterial or venous thromboembolism (eg, stroke, transient ischemic attach, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires anticoagulant treatment
-Study participant has clinically significant bleeding that warrants immediate platelet adjustment (eg, menorrhagia with significant in hemoglobin)
-Study participant has a known hypersensitivity to any components of the investigational medicinal product (IMP) or comparative drugs (and/or an investigational device) as stated in this protocol
-Study participant has evidence of a secondary cause of immune thrombocytopenia from the past medical history (eg, bacterial or viral infection, past medical history of leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease) or to drug treatments (eg, heparin, quinine, antimicrobials, anticonvulsants) or participant has a multiple immune cytopenia, eg, Evan’s syndrome
-Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the dose of IMP -Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
-Study participant has a history of a major organ transplant or hematopoietic stem cell/marrow transplant
-Study participant has experienced intracranial bleed in the last 6 months prior to the Screening Visit
-Study participant has a history of coagulopathy disorders other than ITP
-Study participant has a Karnofsky Performance Status rating <60% at the Screening Visit
-Study participant with current or medical history of immunoglobulin A (IgA) deficiency, or a measurement of IgA <50 mg/dL at the Screening Visit
-Study participant has undergone a splenectomy in the 2 years prior to the Baseline Visit
-Participant has a history of arterial or venous thromboembolism (eg, stroke, transient ischemic attach, myocardial infarction, deep vein thrombosis or pulmonary embolism) within the 6 months prior to randomization or requires anticoagulant treatment
-Study participant has clinically significant bleeding that warrants immediate platelet adjustment (eg, menorrhagia with significant in hemoglobin)
-Study participant has a known hypersensitivity to any components of the investigational medicinal product (IMP) or comparative drugs (and/or an investigational device) as stated in this protocol
-Study participant has evidence of a secondary cause of immune thrombocytopenia from the past medical history (eg, bacterial or viral infection, past medical history of leukemia, lymphoma, common variable immunodeficiency, systemic lupus erythematosus, autoimmune thyroid disease) or to drug treatments (eg, heparin, quinine, antimicrobials, anticonvulsants) or participant has a multiple immune cytopenia, eg, Evan’s syndrome
-Study participant has a clinically relevant active infection (eg, sepsis, pneumonia, or abscess) in the opinion of the investigator, or had a serious infection (resulting in hospitalization or requiring parenteral antibiotic treatment) within 6 weeks prior to the dose of IMP -Study participant with a known tuberculosis (TB) infection, at high risk of acquiring TB infection, or latent tuberculosis infection (LTBI), or current/history of nontuberculous mycobacterial infection (NTMBI)
-Study participant has a history of a major organ transplant or hematopoietic stem cell/marrow transplant
-Study participant has experienced intracranial bleed in the last 6 months prior to the Screening Visit
-Study participant has a history of coagulopathy disorders other than ITP
-Study participant has a Karnofsky Performance Status rating <60% at the Screening Visit
-Study participant with current or medical history of immunoglobulin A (IgA) deficiency, or a measurement of IgA <50 mg/dL at the Screening Visit
-Study participant has undergone a splenectomy in the 2 years prior to the Baseline Visit
The Estimated Number of Participants
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Taiwan
8 participants
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Global
105 participants
