Paroxysmal nocturnal hemoglobinuria MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Sponsors
Regeneron Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients with PNH who have completed, without discontinuation, study treatment in one of the parent studies in which they participated 2. Willing and able to comply with clinic visits and study related procedures 3. Provide informed consent signed by study patient Are the trial subjects under 18? no Number of subjects for this age range: F.1.2 Adults (18-64 years) yes F.1.2.1 Number of subjects for this age range 111 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 36
Exclusion criteria
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.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Secondary Objective: 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. ;Timepoint(s) of evaluation of this end point: 2 years for the primary safety endpoint 26 weeks for the primary efficacy endpoint;Main Objective: The primary objective of the study is to evaluate the long-term safety, tolerability, and effect on intravascular hemolysis (ie, proportion of patients achieving lactate dehydrogenase (LDH) = 1.5× upper limit of normal (ULN) over 26 weeks) of REGN3918 in patients with paroxysmal nocturnal hemoglobinuria (PNH). ;Primary end point(s): The primary safety endpoint is incidence and severity of treatment-emergent adverse events (TEAEs) and other safety variables during the 2-year open-label treatment period of the study in patients treated with REGN3918. The primary efficacy endpoint is the proportion of patients achieving LDH =1.5×ULN over week 26, defined as LDH =1.5×ULN at every scheduled time point up to week 26 (inclusive) | — |
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): The secondary endpoints include: •The proportion of patients with breakthrough hemolysis over week 26 •The rate and number of units of transfusion over week 26 ;Timepoint(s) of evaluation of this end point: 26 weeks | — |
Countries
Canada, Germany, Hong Kong, Hungary, Italy, Korea, Democratic People's Republic of, Malaysia, Netherlands, Poland, Romania, Singapore, South Africa, Taiwan, United Kingdom, United States
Contacts
Public ContactClinical Trial Information
Regeneron Pharmaceuticals, Inc.
Outcome results
None listed