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    Summary
    EudraCT Number:2020-005005-17
    Sponsor's Protocol Code Number:R3918-PNH-2092
    National Competent Authority:Hungary - National Institute of Pharmacy
    Clinical Trial Type:EEA CTA
    Trial Status:Completed
    Date on which this record was first entered in the EudraCT database:2021-03-05
    Trial results
    Index
    A. PROTOCOL INFORMATION
    B. SPONSOR INFORMATION
    C. APPLICANT IDENTIFICATION
    D. IMP IDENTIFICATION
    D.8 INFORMATION ON PLACEBO
    E. GENERAL INFORMATION ON THE TRIAL
    F. POPULATION OF TRIAL SUBJECTS
    G. INVESTIGATOR NETWORKS TO BE INVOLVED IN THE TRIAL
    N. REVIEW BY THE COMPETENT AUTHORITY OR ETHICS COMMITTEE IN THE COUNTRY CONCERNED
    P. END OF TRIAL
    Expand All   Collapse All
    A. Protocol Information
    A.1Member State ConcernedHungary - National Institute of Pharmacy
    A.2EudraCT number2020-005005-17
    A.3Full title of the trial
    A Randomized, Open-label, Two-arm Study to Evaluate the Safety, Efficacy, and Pharmacodynamic Effects of Pozelimab and Cemdisiran Combination Treatment in Patients with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
    A.3.1Title of the trial for lay people, in easily understood, i.e. non-technical, language
    Pozelimab and Cemdisiran Combination Treatment in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab Monotherapy
    A.4.1Sponsor's protocol code numberR3918-PNH-2092
    A.7Trial is part of a Paediatric Investigation Plan No
    A.8EMA Decision number of Paediatric Investigation Plan
    B. Sponsor Information
    B.Sponsor: 1
    B.1.1Name of SponsorRegeneron Pharmaceuticals, Inc.
    B.1.3.4CountryUnited States
    B.3.1 and B.3.2Status of the sponsorCommercial
    B.4 Source(s) of Monetary or Material Support for the clinical trial:
    B.4.1Name of organisation providing supportRegeneron Pharmaceuticals, Inc.
    B.4.2CountryUnited States
    B.5 Contact point designated by the sponsor for further information on the trial
    B.5.1Name of organisationRegeneron Pharmaceuticals, Inc.
    B.5.2Functional name of contact pointClinical Trial Information
    B.5.3 Address:
    B.5.3.1Street Address777 Old Saw Mill River Road
    B.5.3.2Town/ cityTarrytown
    B.5.3.3Post code10591
    B.5.3.4CountryUnited States
    B.5.6E-mailclinicaltrials@regeneron.com
    D. IMP Identification
    D.IMP: 1
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product namePozelimab
    D.3.2Product code REGN3918
    D.3.4Pharmaceutical form Solution for injection/infusion
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPIntravenous use
    Subcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNPozelimab
    D.3.9.2Current sponsor codeREGN3918
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin No
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) Yes
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product Yes
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.IMP: 2
    D.1.2 and D.1.3IMP RoleTest
    D.2 Status of the IMP to be used in the clinical trial
    D.2.1IMP to be used in the trial has a marketing authorisation No
    D.2.5The IMP has been designated in this indication as an orphan drug in the Community No
    D.2.5.1Orphan drug designation number
    D.3 Description of the IMP
    D.3.1Product nameCemdisiran
    D.3.2Product code ALN-CC5
    D.3.4Pharmaceutical form Solution for injection
    D.3.4.1Specific paediatric formulation No
    D.3.7Routes of administration for this IMPSubcutaneous use
    D.3.8 to D.3.10 IMP Identification Details (Active Substances)
    D.3.8INN - Proposed INNCemdisiran
    D.3.9.2Current sponsor codeALN-CC5
    D.3.9.3Other descriptive nameCEMDISIRAN
    D.3.9.4EV Substance CodeSUB194793
    D.3.10 Strength
    D.3.10.1Concentration unit mg/ml milligram(s)/millilitre
    D.3.10.2Concentration typeequal
    D.3.10.3Concentration number200
    D.3.11 The IMP contains an:
    D.3.11.1Active substance of chemical origin Yes
    D.3.11.2Active substance of biological/ biotechnological origin (other than Advanced Therapy IMP (ATIMP) No
    The IMP is a:
    D.3.11.3Advanced Therapy IMP (ATIMP) No
    D.3.11.3.1Somatic cell therapy medicinal product No
    D.3.11.3.2Gene therapy medical product No
    D.3.11.3.3Tissue Engineered Product No
    D.3.11.3.4Combination ATIMP (i.e. one involving a medical device) No
    D.3.11.3.5Committee on Advanced therapies (CAT) has issued a classification for this product No
    D.3.11.4Combination product that includes a device, but does not involve an Advanced Therapy No
    D.3.11.5Radiopharmaceutical medicinal product No
    D.3.11.6Immunological medicinal product (such as vaccine, allergen, immune serum) No
    D.3.11.7Plasma derived medicinal product No
    D.3.11.8Extractive medicinal product No
    D.3.11.9Recombinant medicinal product No
    D.3.11.10Medicinal product containing genetically modified organisms No
    D.3.11.11Herbal medicinal product No
    D.3.11.12Homeopathic medicinal product No
    D.3.11.13Another type of medicinal product No
    D.8 Information on Placebo
    E. General Information on the Trial
    E.1 Medical condition or disease under investigation
    E.1.1Medical condition(s) being investigated
    Paroxysmal Nocturnal Hemoglobinuria
    E.1.1.1Medical condition in easily understood language
    Paroxysmal Nocturnal Hemoglobinuria (PNH)
    E.1.1.2Therapeutic area Diseases [C] - Blood and lymphatic diseases [C15]
    MedDRA Classification
    E.1.2 Medical condition or disease under investigation
    E.1.2Version 21.1
    E.1.2Level LLT
    E.1.2Classification code 10055629
    E.1.2Term Paroxysmal nocturnal hemoglobinuria
    E.1.2System Organ Class 100000004857
    E.1.3Condition being studied is a rare disease Yes
    E.2 Objective of the trial
    E.2.1Main objective of the trial
    Evaluate the safety and tolerability of 2 dosing regimens of pozelimab and cemdisiran combination therapy during the open-label treatment period (OLTP)
    E.2.2Secondary objectives of the trial
    • 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
    E.2.3Trial contains a sub-study No
    E.3Principal 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
    E.4Principal 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
    E.5 End points
    E.5.1Primary end point(s)
    Incidence and severity of treatment-emergent adverse events (TEAEs) (OLTP)
    E.5.1.1Timepoint(s) of evaluation of this end point
    Through week 28 (OLTP)
    E.5.2Secondary 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-Fatigue scale
    35. Change in GHS/QoL on the EORTC QLQ-C30
    36. Change in PF scores on the EORTC QLQ-C30
    37. Incidence and severity of TEAEs
    38. Concentrations of total pozelimab in serum
    39. Concentrations of total C5
    40. Concentrations of cemdisiran in plasma
    41. Incidence of pozelimab anti-drug antibody (ADA) responses over time
    42. Incidence of cemdisiran anti-drug antibody (ADA) responses over time
    E.5.2.1Timepoint(s) of evaluation of this end point
    OLTP
    1: End of treatment period, approximately 28 weeks
    2-4: Day 1 through Week 28, Week 4 through Week 28
    5, 6, 17: Baseline through Week 28
    7-14: Baseline to Week 28
    15, 16, 18, 19: Up to Week 28
    20: Through Week 28

    OLEP
    21, 22, 28, 30, 31: Day 1 to Week 24 and Day 1 to Week 52
    32-33: Day 1 to Week 16, Day 1 to Week 24 Day 1 to Week 52
    34-36: Day 1 to Week 52
    23, 24, 26, 27, 29: Day 1 through Week 24 and Day 1 through Week 52
    25: Day 1 through Week 52
    37-42: Up to Week 52
    E.6 and E.7 Scope of the trial
    E.6Scope of the trial
    E.6.1Diagnosis No
    E.6.2Prophylaxis No
    E.6.3Therapy Yes
    E.6.4Safety Yes
    E.6.5Efficacy Yes
    E.6.6Pharmacokinetic Yes
    E.6.7Pharmacodynamic Yes
    E.6.8Bioequivalence No
    E.6.9Dose response No
    E.6.10Pharmacogenetic No
    E.6.11Pharmacogenomic Yes
    E.6.12Pharmacoeconomic No
    E.6.13Others No
    E.7Trial type and phase
    E.7.1Human pharmacology (Phase I) No
    E.7.1.1First administration to humans No
    E.7.1.2Bioequivalence study No
    E.7.1.3Other No
    E.7.1.3.1Other trial type description
    E.7.2Therapeutic exploratory (Phase II) Yes
    E.7.3Therapeutic confirmatory (Phase III) No
    E.7.4Therapeutic use (Phase IV) No
    E.8 Design of the trial
    E.8.1Controlled No
    E.8.1.1Randomised Yes
    E.8.1.2Open Yes
    E.8.1.3Single blind No
    E.8.1.4Double blind No
    E.8.1.5Parallel group Yes
    E.8.1.6Cross over No
    E.8.1.7Other No
    E.8.2 Comparator of controlled trial
    E.8.2.1Other medicinal product(s) No
    E.8.2.2Placebo No
    E.8.2.3Other No
    E.8.2.4Number of treatment arms in the trial2
    E.8.3 The trial involves single site in the Member State concerned Yes
    E.8.4 The trial involves multiple sites in the Member State concerned No
    E.8.4.1Number of sites anticipated in Member State concerned1
    E.8.5The trial involves multiple Member States No
    E.8.6 Trial involving sites outside the EEA
    E.8.6.1Trial being conducted both within and outside the EEA Yes
    E.8.6.2Trial being conducted completely outside of the EEA No
    E.8.6.3If E.8.6.1 or E.8.6.2 are Yes, specify the regions in which trial sites are planned
    Hong Kong
    Korea, Republic of
    Malaysia
    Taiwan
    Hungary
    United Kingdom
    E.8.7Trial has a data monitoring committee No
    E.8.8 Definition of the end of the trial and justification where it is not the last visit of the last subject undergoing the trial
    The end of the main study is defined as the date of the last visit of the last patient in the OLTP, including patients who discontinued treatment during the OLTP and are being followed in the post-treatment safety follow-up period.

    The end of the optional OLEP is defined as the date of the last visit of the last patient in the optional OLEP, including patients who discontinued treatment during the OLEP and are being followed in the post-treatment safety follow-up period.
    E.8.9 Initial estimate of the duration of the trial
    E.8.9.1In the Member State concerned years2
    E.8.9.1In the Member State concerned months10
    E.8.9.1In the Member State concerned days5
    E.8.9.2In all countries concerned by the trial years3
    E.8.9.2In all countries concerned by the trial months0
    E.8.9.2In all countries concerned by the trial days0
    F. Population of Trial Subjects
    F.1 Age Range
    F.1.1Trial has subjects under 18 No
    F.1.1.1In Utero No
    F.1.1.2Preterm newborn infants (up to gestational age < 37 weeks) No
    F.1.1.3Newborns (0-27 days) No
    F.1.1.4Infants and toddlers (28 days-23 months) No
    F.1.1.5Children (2-11years) No
    F.1.1.6Adolescents (12-17 years) No
    F.1.2Adults (18-64 years) Yes
    F.1.2.1Number of subjects for this age range: 20
    F.1.3Elderly (>=65 years) Yes
    F.1.3.1Number of subjects for this age range: 4
    F.2 Gender
    F.2.1Female Yes
    F.2.2Male Yes
    F.3 Group of trial subjects
    F.3.1Healthy volunteers No
    F.3.2Patients Yes
    F.3.3Specific vulnerable populations Yes
    F.3.3.1Women of childbearing potential not using contraception No
    F.3.3.2Women of child-bearing potential using contraception Yes
    F.3.3.3Pregnant women No
    F.3.3.4Nursing women No
    F.3.3.5Emergency situation No
    F.3.3.6Subjects incapable of giving consent personally No
    F.3.3.7Others No
    F.4 Planned number of subjects to be included
    F.4.1In the member state1
    F.4.2 For a multinational trial
    F.4.2.1In the EEA 1
    F.4.2.2In the whole clinical trial 24
    F.5 Plans for treatment or care after the subject has ended the participation in the trial (if it is different from the expected normal treatment of that condition)
    None
    G. Investigator Networks to be involved in the Trial
    N. Review by the Competent Authority or Ethics Committee in the country concerned
    N.Competent Authority Decision Authorised
    N.Date of Competent Authority Decision2021-05-07
    N.Ethics Committee Opinion of the trial applicationFavourable
    N.Ethics Committee Opinion: Reason(s) for unfavourable opinion
    N.Date of Ethics Committee Opinion2021-04-23
    P. End of Trial
    P.End of Trial StatusCompleted
    P.Date of the global end of the trial2023-10-18
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