Clinical Trials List
Protocol NumberR3918-PNH-1868
NCT Number(ClinicalTrials.gov Identfier)NCT04162470
2019-10-01 - 2022-12-12
Phase III
Recruiting4
ICD-10R82.3
Hemoglobinuria
ICD-9791.2
Hemoglobinuria
An Open-label Extension Study to Evaluate the Long-term Safety, Tolerability, and Efficacy of REGN3918 in Patients With Paroxysmal Nocturnal Hemoglobinuria
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Trial Applicant
Syneos Health
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Sponsor
Regeneron Pharmaceuticals, Inc.
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Trial scale
Multi-Regional Multi-Center
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Update
2025/08/20
Investigators and Locations
Co-Principal Investigator
- Ya-Ting Hsu Division of Hematology & Oncology
- Sin-Syue Li Division of Hematology & Oncology
- Ya-Ping Chen Division of Hematology & Oncology
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- Tung-Liang Lin 無
- 高小雯 無
The Actual Total Number of Participants Enrolled
0 Recruiting
The Actual Total Number of Participants Enrolled
0 Recruiting
Co-Principal Investigator
- 田豐銘 無
- - - 無
- Jih-Luh Tang 無
- Wen-Chien Chou 無
The Actual Total Number of Participants Enrolled
0 Recruiting
Condition/Disease
Paroxysmal Nocturnal Hemoglobinuria
Objectives
The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH).
The secondary objectives of the study are:
To evaluate the long-term effect of REGN3918 on intravascular hemolysis
To assess the concentrations of total REGN3918 in serum
To evaluate the occurrence of the immunogenicity of REGN3918
Test Drug
R3918
Active Ingredient
REGN3918
Dosage Form
injection
Dosage
265
Endpoints
Primary Outcome Measures :
Incidence and severity of treatment-emergent adverse events (TEAEs) [ Time Frame: Up to week 104 ]
TEAEs include adverse events (AEs), serious adverse events (SAEs), AEs of special interest (AESIs), laboratory data, vital signs, and electrocardiograms (ECGs)
Proportion of patients achieving lactate dehydrogenase (LDH) less than or equal to 1.5ULN [ Time Frame: Up to week 26 ]
Secondary Outcome Measures :
Proportion of patients with breakthrough hemolysis [ Time Frame: Up to week 26, 78, 104 ]
Rate/number of units of transfusion [ Time Frame: Up to week 26, 78, 104 ]
Proportions of patients who are transfusion-free (with red blood cell [RBCs]) [ Time Frame: Up to week 26, 78, 104 ]
Proportions of patients achieving adequate control of their intravascular hemolysis [ Time Frame: Up to week 78, 104 ]
Proportions of patients achieving normalization of their intravascular hemolysis [ Time Frame: Up to week 26, 78, 104 ]
Changes in LDH from baseline of the Open-label extension (OLE) study [ Time Frame: Up to week 26, 78, 104 ]
Percent changes in LDH from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
Changes in RBC hemoglobin levels from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
Changes in free hemoglobin levels from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
Concentrations of REGN3918 in serum [ Time Frame: Up to week 104 ]
Incidence of treatment-emergent anti-drug antibodies (ADA) to REGN3918 [ Time Frame: Up to week 104 ]
Incidence and severity of treatment-emergent adverse events (TEAEs) [ Time Frame: Up to week 104 ]
TEAEs include adverse events (AEs), serious adverse events (SAEs), AEs of special interest (AESIs), laboratory data, vital signs, and electrocardiograms (ECGs)
Proportion of patients achieving lactate dehydrogenase (LDH) less than or equal to 1.5ULN [ Time Frame: Up to week 26 ]
Secondary Outcome Measures :
Proportion of patients with breakthrough hemolysis [ Time Frame: Up to week 26, 78, 104 ]
Rate/number of units of transfusion [ Time Frame: Up to week 26, 78, 104 ]
Proportions of patients who are transfusion-free (with red blood cell [RBCs]) [ Time Frame: Up to week 26, 78, 104 ]
Proportions of patients achieving adequate control of their intravascular hemolysis [ Time Frame: Up to week 78, 104 ]
Proportions of patients achieving normalization of their intravascular hemolysis [ Time Frame: Up to week 26, 78, 104 ]
Changes in LDH from baseline of the Open-label extension (OLE) study [ Time Frame: Up to week 26, 78, 104 ]
Percent changes in LDH from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
Changes in RBC hemoglobin levels from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
Changes in free hemoglobin levels from baseline of the OLE study [ Time Frame: Up to week 26, 78, 104 ]
Concentrations of REGN3918 in serum [ Time Frame: Up to week 104 ]
Incidence of treatment-emergent anti-drug antibodies (ADA) to REGN3918 [ Time Frame: Up to week 104 ]
Inclution Criteria
1.Patients with PNH who have completed, without discontinuation, study treatment in 1of the parent studies in which they participated (either R3918-PNH-1852 or R3918-PNH-1853)
2.Willing and able to comply with clinic visits and study-related procedures
3.Provide informed consentsigned by study patient
2.Willing and able to comply with clinic visits and study-related procedures
3.Provide informed consentsigned by study patient
Exclusion Criteria
1.Significant protocol deviation(s) in the parent study based on the investigator’s judgment and to the extent that these would (if continued) impact the study objectives and/or safety of the patient (for example, repetitive non-compliance with dosing by the patient).
2.Any new condition or worsening of an existing condition which,in the opinion of the investigator,would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study.
3.Pregnant or breastfeeding women
4.Women of childbearing potential* who are unwilling to practice highly effective contraception prior to the initialdose/start of the first treatment, during the study, and for at least 21weeks after the last dose. Highly effective contraceptive measures include:
a.stable use of combined (estrogen and progestogen-containing) hormonal contraception (oral, intravaginal,transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) associated with inhibition of ovulation initiated 2or more menstrual cycles prior to screening
b.intrauterine device; intrauterine hormone-releasing system
c.bilateral tubal ligation
d.vasectomized partner
e.and/or sexual abstinence
2.Any new condition or worsening of an existing condition which,in the opinion of the investigator,would make the patient unsuitable for enrollment or could interfere with the patient participating in or completing the study.
3.Pregnant or breastfeeding women
4.Women of childbearing potential* who are unwilling to practice highly effective contraception prior to the initialdose/start of the first treatment, during the study, and for at least 21weeks after the last dose. Highly effective contraceptive measures include:
a.stable use of combined (estrogen and progestogen-containing) hormonal contraception (oral, intravaginal,transdermal) or progestogen-only hormonal contraception (oral, injectable, implantable) associated with inhibition of ovulation initiated 2or more menstrual cycles prior to screening
b.intrauterine device; intrauterine hormone-releasing system
c.bilateral tubal ligation
d.vasectomized partner
e.and/or sexual abstinence
The Estimated Number of Participants
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Taiwan
12 participants
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Global
147 participants
