Paroxysmal Nocturnal Hemoglobinuria MedDRA version: 21.1 Level: LLT Classification code 10055629 Term: Paroxysmal nocturnal hemoglobinuria System Organ Class: 100000004857
Conditions
Interventions
Product Name: Pozelimab
Product Code: REGN3918
Pharmaceutical Form: Solution for injection/infusion
INN or Proposed INN: Pozelimab
Current Sponsor code: REGN3918
Concentration unit: mg/ml milligram(s)
Sponsors
Regeneron Pharmaceuticals, Inc.
Eligibility
Sex/Gender
All
Inclusion criteria
Inclusion criteria: 1. Patients with PNH who are receiving treatment with pozelimab monotherapy in the R3918- PNH-1868 study 2. Provide informed consent signed by study patient 3. Willing and able to comply with clinic/remote visits and study-related procedures 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 20 F.1.3 Elderly (>=65 years) yes F.1.3.1 Number of subjects for this age range 4
Exclusion criteria
Exclusion criteria: 1. Documented, positive polymerase chain reaction (PCR) or equivalent test based on regional recommendations for COVID-19 or suspected SARS-CoV-2 infection as defined in the protocol 2. Participants with documented history of liver cirrhosis or participants with liver disease with evidence of currently impaired liver function; or participants with alanine aminotransferase (ALT) or aspartate aminotransferase (AST) as described in the protocol 3. Significant protocol deviation(s) in the parent study based on the investigator’s judgment as described in the protocol 4. Any new condition or worsening of an existing condition which, in the opinion of the investigator, would make the participant unsuitable for enrollment or would jeopardize the safety of the participant 5. Known hypersensitivity to cemdisiran or any component of cemdisiran formulation NOTE: Other protocol-defined Exclusion Criteria apply
Design outcomes
Secondary
| Measure | Time frame |
|---|---|
| Secondary end point(s): Open-Label Treatment Period (OLTP) 1. Percent change of LDH from pre-treatment to end-of-treatment period 2. Proportion of participants with adequate control of their LDH 3. Proportion of participants with normalization of their LDH 4. Area under the curve (AUC) of LDH over time 5. Proportion of participants with breakthrough hemolysis 6. Proportion of participants with hemoglobin stabilization 7. Change in hemoglobin levels 8. Proportion of participants who are transfusion-free 9. Rate of RBCs transfused 10. Number of units of RBCs transfused 11. Change in CH50 12. Change in fatigue as measured by Functional Assessment of Chronic Illness Therapy-Fatigue (FACIT-Fatigue) scale 13. Mean change in global health status/quality of life scale (GHS/QoL) on the European Organization for Research and Treatment of Cancer: Quality-of-Life Questionnaire core 30 items (EORTC QLQ-C30) 14. Mean change in physical function (PF) scores on the EORTC QLQ-C30 15. Concentrations of total pozelimab in serum 16. Concentrations of cemdisiran in plasma 17. Change from baseline in concentration of total C5 18. Incidence of pozelimab anti-drug antibody (ADA) responses over time 19. Incidence of cemdisiran anti-drug antibody (ADA) responses over time 20. Incidence and severity of TEAEs for participants who received treatment intensification Optional Open-Label Extension Period (OLEP) 21. Change of LDH 22. Percent change of LDH 23. Proportion of participants with adequate control of their LDH 24. Proportion of participants with normalization of LDH 25. AUC of LDH over time 26. Proportion of participants with breakthrough hemolysis 27. Proportion of participants with hemoglobin stabilization 28. Change in hemoglobin levels 29. Proportion of participants who are transfusion-free 30. Rate of RBCs transfused 31. Number of units of RBCs transfused 32. Change in CH50 33. Percent change in CH50 34. Change in fatigue as measured by FACIT-Fatig | — |
Primary
| Measure | Time frame |
|---|---|
| Main Objective: Evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP);Secondary Objective: • To evaluate the effect of the combination treatment on the following parameters of intravascular hemolysis: lactate dehydrogenase (LDH) control, breakthrough hemolysis, and inhibition of total complement hemolysis activity (CH50) • To evaluate the effect of the combination treatment on hemoglobin levels • To evaluate the effect of the combination treatment on red blood cell (RBC) transfusion requirements • To evaluate the effect of the combination treatment on clinical outcome assessments (COAs) measuring fatigue and health related quality of life • To assess the concentrations of total pozelimab in serum and total complement component (C) 5 and cemdisiran in plasma • To assess immunogenicity to pozelimab and cemdisiran • To evaluate the long-term safety and efficacy of pozelimab and cemdisiran in an optional open-label extension period (OLEP) • To assess safety after treatment intensification with pozelimab and cemdisiran;Primary end point(s): Incidence and severity of treatment-emergent adverse events (TEAEs) (OLTP);Timepoint(s) of evaluation of this end point: Through week 28 (OLTP) | — |
Countries
Hong Kong, Hungary, Korea, Republic of, Malaysia, Taiwan, United Kingdom
Contacts
Public ContactClinical Trial Information
Regeneron Pharmaceuticals, Inc.
Outcome results
None listed