ALN-CC5 ( DrugBank: - )


4 diseases
IDDisease name (Link within this page)Number of trials
11Myasthenia gravis5
62Paroxysmal nocturnal hemoglobinuria11
66IgA nephropathy3
109Atypical hemolytic uremic syndrome7

11. Myasthenia gravis


Clinical trials : 332 Drugs : 234 - (DrugBank : 81) / Drug target genes : 45 - Drug target pathways : 127
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2020-003272-41-DK
(EUCTR)
15/06/202202/11/2021A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia GravisEfficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis - NIMBLE Generalized Myasthenia Gravis
MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: Cemdisiran
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
235Phase 3Serbia;United States;Czechia;Taiwan;Spain;Turkey;United Kingdom;Italy;India;France;Canada;Belgium;Brazil;Poland;Denmark;Australia;Germany;China;Japan;Korea, Republic of
2EUCTR2020-003272-41-DE
(EUCTR)
04/03/202204/01/2022A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia GravisEfficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis - NIMBLE Generalized Myasthenia Gravis
MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: Cemdisiran
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
235Phase 3United States;Czechia;Taiwan;Spain;Ukraine;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Germany;Korea, Republic of
3EUCTR2020-003272-41-ES
(EUCTR)
03/02/202228/09/2021A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia GravisEfficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis Generalized Myasthenia Gravis
MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: Cemdisiran
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
210Phase 3United States;Czechia;Taiwan;Spain;Ukraine;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Germany;Korea, Republic of
4EUCTR2020-003272-41-IT
(EUCTR)
13/12/202119/10/2021A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia GravisEfficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis - Study of Pozelimab and Cemdisiran combination therapy in patients with Myasthenia Gravis Generalized Myasthenia Gravis
MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Cemdisiran
Product Code: [ALN-CC5]
INN or Proposed INN: Cemdisiran
Product Name: Pozelimab
Product Code: [REGN3918]
INN or Proposed INN: Pozelimab
Trade Name: Bexsero
Product Name: Bexsero
Product Code: [NA]
INN or Proposed INN: Outer membrane vesicles (OMV) from Neisseria meningitidis group B strain NZ98/254measured as amount of total protein containing the PorA P1.4
Other descriptive name: RECOMBINANT NEISSERIA MENINGITIDIS GROUP B FHBP FUSION PROTEIN PRODUCED IN E.COLI CELLS BY RECOMBINANT DNA TECHNOLOGY ADSORBED ON ALUMINIUM HYDROXIDE
INN or Proposed INN: RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NHBA FUSION PROTEIN
Other descriptive name: RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NHBA FUSION PROTEIN
INN or Proposed INN: RECOMBINANT NEISSERIA MENINGITIDIS GROUP B NADA PROTEIN
Other descriptive name: RECOMBINANT NEISSERIA
REGENERON PHARMACEUTICALS, INC.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
210Phase 3United States;Czechia;Taiwan;Spain;Ukraine;Russian Federation;United Kingdom;Italy;France;Canada;Belgium;Poland;Australia;Denmark;Germany;Korea, Republic of
5EUCTR2020-003272-41-PL
(EUCTR)
01/12/202121/10/2021A study to examine the efficacy and safety of pozelimab and cemdisiran combination therapy in patients with symptomatic Generalized Myasthenia GravisEfficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Patients with Symptomatic Generalized Myasthenia Gravis Generalized Myasthenia Gravis
MedDRA version: 21.1;Level: PT;Classification code 10028417;Term: Myasthenia gravis;System Organ Class: 10029205 - Nervous system disorders;Therapeutic area: Diseases [C] - Nervous System Diseases [C10]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: Cemdisiran
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
210Phase 3United States;Czechia;Taiwan;Spain;Ukraine;Russian Federation;United Kingdom;Italy;France;Canada;Poland;Belgium;Australia;Denmark;Germany;Korea, Republic of

62. Paroxysmal nocturnal hemoglobinuria


Clinical trials : 292 Drugs : 151 - (DrugBank : 49) / Drug target genes : 22 - Drug target pathways : 108
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2021-004931-10-GR
(EUCTR)
04/01/202322/11/2022A study to examine the long-term safety, tolerability, and effectiveness of Pozelimab and Cemdisiran combination therapy in adult patients with Paroxysmal Nocturnal HemoglobinuriaAn Open-Label Extension Study to Evaluate the Long-Term Safety, Tolerability, and Efficacy of Pozelimab and Cemdisiran Combination Therapy in Patients with Paroxysmal Nocturnal Hemoglobinuria - ACCESS-EXTENSION Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: Cemdisiran
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
288Phase 3United States;Philippines;Taiwan;Greece;Thailand;Spain;Colombia;Italy;India;France;Jordan;Malaysia;Peru;South Africa;China;Korea, Democratic People's Republic of;Turkey;United Kingdom;Hungary;Mexico;Canada;Brazil;Poland;Singapore;Romania;Germany;Japan
2EUCTR2020-002761-33-FR
(EUCTR)
06/12/202221/03/2022Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal HemoglobinuriaA RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: CEMDISIRAN
Other descriptive name: CEMDISIRAN
Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz)
INN or Proposed INN: Ravulizumab
Other descriptive name: Ravulizumab
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Trade Name: Soliris ™ (eculizumab)
Product Name: Eculizumab
INN or Proposed INN: Eculizumab
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
140Phase 3Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Netherlands;Germany;Japan;Korea, Republic of
3EUCTR2020-002761-33-DE
(EUCTR)
20/06/202203/03/2022Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal HemoglobinuriaA RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: CEMDISIRAN
Other descriptive name: CEMDISIRAN
Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz)
INN or Proposed INN: Ravulizumab
Other descriptive name: Ravulizumab
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Trade Name: Soliris ™ (eculizumab)
Product Name: Eculizumab
INN or Proposed INN: Eculizumab
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
140Phase 3Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Brazil;Poland;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of
4EUCTR2020-004486-40-GR
(EUCTR)
17/06/202208/04/2022Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor TherapyA RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: CEMDISIRAN
Other descriptive name: CEMDISIRAN
Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz)
INN or Proposed INN: Ravulizumab
Other descriptive name: Ravulizumab
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
124Phase 3Portugal;United States;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Canada;Jordan;Poland;Brazil;Malaysia;Singapore;Romania;Peru;South Africa;Netherlands;China;Japan;Korea, Republic of
5EUCTR2020-002761-33-GR
(EUCTR)
17/06/202212/04/2022Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal HemoglobinuriaA RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED, NON-INFERIORITY STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB - ACCESS-2 Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: CEMDISIRAN
Other descriptive name: CEMDISIRAN
Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz)
INN or Proposed INN: Ravulizumab
Other descriptive name: Ravulizumab
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Trade Name: Soliris ™ (eculizumab)
Product Name: Eculizumab
INN or Proposed INN: Eculizumab
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
134Phase 3Portugal;United States;Philippines;Taiwan;Greece;Spain;Thailand;Turkey;Colombia;United Kingdom;Italy;France;Hungary;Mexico;Brazil;Poland;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of
6EUCTR2020-002761-33-ES
(EUCTR)
09/05/202211/03/2022Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal HemoglobinuriaA RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: CEMDISIRAN
Other descriptive name: CEMDISIRAN
Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz)
INN or Proposed INN: Ravulizumab
Other descriptive name: Ravulizumab
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Trade Name: Soliris ™ (eculizumab)
Product Name: Eculizumab
INN or Proposed INN: Eculizumab
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
140Phase 3Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Netherlands;Germany;Japan;Korea, Republic of
7EUCTR2020-004486-40-ES
(EUCTR)
09/05/202211/03/2022Ravulizumab-Controlled Study to Evaluate the Efficacy and Safety of Pozelimab and Cemdisiran Combination Therapy in Adult Patients with Paroxysmal Nocturnal Hemoglobinuria who are Complement Inhibitor Treatment-Naive or Have Not Recently Received Complement Inhibitor TherapyA RANDOMIZED, OPEN-LABEL, RAVULIZUMAB-CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE COMPLEMENT INHIBITOR TREATMENT-NAIVE OR HAVE NOT RECENTLY RECEIVED COMPLEMENT INHIBITOR THERAPY Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: CEMDISIRAN
Other descriptive name: CEMDISIRAN
Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz)
INN or Proposed INN: Ravulizumab
Other descriptive name: Ravulizumab
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
124Phase 3Portugal;United States;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Canada;Jordan;Poland;Brazil;Malaysia;Singapore;Romania;Peru;South Africa;Netherlands;China;Japan;Korea, Republic of
8EUCTR2020-002761-33-PL
(EUCTR)
14/04/202222/03/2022Efficacy and Safety of the Combination of Pozelimab and Cemdisiran versus continued Eculizumab or Ravulizumab Treatment in Adult Patients with Paroxysmal Nocturnal HemoglobinuriaA RANDOMIZED, OPEN-LABEL ECULIZUMAB AND RAVULIZUMAB CONTROLLED STUDY TO EVALUATE THE EFFICACY AND SAFETY OF POZELIMAB AND CEMDISIRAN COMBINATION THERAPY IN PATIENTS WITH PAROXYSMAL NOCTURNAL HEMOGLOBINURIA WHO ARE CURRENTLY TREATED WITH ECULIZUMAB OR RAVULIZUMAB Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: CEMDISIRAN
Other descriptive name: CEMDISIRAN
Trade Name: ULTOMIRIS ™ (ravulizumab-cwvz)
INN or Proposed INN: Ravulizumab
Other descriptive name: Ravulizumab
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Trade Name: Soliris ™ (eculizumab)
Product Name: Eculizumab
INN or Proposed INN: Eculizumab
Regeneron Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
140Phase 3Portugal;United States;Philippines;Taiwan;Greece;Spain;Turkey;Colombia;United Kingdom;Italy;France;Mexico;Poland;Brazil;Singapore;Romania;Germany;Netherlands;Japan;Korea, Republic of
9EUCTR2020-005005-17-HU
(EUCTR)
07/05/202105/03/2021Pozelimab and Cemdisiran Combination Treatment in Adult Participants with Paroxysmal Nocturnal Hemoglobinuria Who Have Received Pozelimab MonotherapyA 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 Paroxysmal Nocturnal Hemoglobinuria
MedDRA version: 21.1;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: Pozelimab
Product Code: REGN3918
INN or Proposed INN: Pozelimab
Product Name: Cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: Cemdisiran
Other descriptive name: CEMDISIRAN
Regeneron Pharmaceuticals, Inc.NULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
24Phase 2Taiwan;Hong Kong;Hungary;Malaysia;United Kingdom;Korea, Republic of
10EUCTR2016-002943-40-ES
(EUCTR)
21/11/201630/09/2016A Clinical Study with Investigational Drug, ALN-CC5, in patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to EculizumabA Phase 2, Open-label, Single Dose, Study of Subcutaneously Administered ALN-CC5 in Patients with Paroxysmal Nocturnal Hemoglobinuria who are Inadequate Responders to Eculizumab Paroxysmal nocturnal hemoglobinuria (PNH)
MedDRA version: 19.0;Level: LLT;Classification code 10055629;Term: Paroxysmal nocturnal hemoglobinuria;System Organ Class: 100000004857;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Code: ALN-CC5
INN or Proposed INN: ALN-62643
Alnylam Pharmaceuticals IncNULLNot RecruitingFemale: yes
Male: yes
15Phase 2Taiwan;Finland;Spain;Turkey;Austria;Russian Federation;Israel;United Kingdom;France;Czech Republic;Canada;Belgium;Australia;Denmark;Bulgaria;Norway;Netherlands;Germany;Korea, Republic of;Sweden
11NCT02352493
(ClinicalTrials.gov)
January 201523/1/2015A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNHA Phase 1/2 Single-ascending and Multiple-ascending Dose, Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics Study of Subcutaneously Administered ALN-CC5 in Healthy Adult Volunteers and Patients With Paroxysmal Nocturnal HemoglobinuriaParoxysmal Nocturnal Hemoglobinuria (PNH)Drug: ALN-CC5;Drug: Sterile Normal Saline (0.9% NaCl)Alnylam PharmaceuticalsNULLCompleted18 Years45 YearsAll62Phase 1/Phase 2Spain;United Kingdom

66. IgA nephropathy


Clinical trials : 275 Drugs : 258 - (DrugBank : 82) / Drug target genes : 36 - Drug target pathways : 140
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1EUCTR2018-002716-27-GB
(EUCTR)
11/06/201907/01/2019A Phase 2 Study of Cemdisiran in Adult Patients with IgA NephropathyA Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Adult Patients with IgA Nephropathy - A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy Immunoglobulin A nephropathy (IgAN);Therapeutic area: Diseases [C] - Immune System Diseases [C20]Product Name: cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: cemdisiran
Other descriptive name: CEMDISIRAN
Alnylam Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 2United States;France;Philippines;Taiwan;Canada;Spain;Malaysia;Singapore;United Kingdom;Sweden
2EUCTR2018-002716-27-SE
(EUCTR)
28/05/201906/02/2019A Phase 2 Study of Cemdisiran in Adult Patients with IgA NephropathyA Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Adult Patients with IgA Nephropathy - A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy Immunoglobulin A nephropathy (IgAN);Therapeutic area: Diseases [C] - Immune System Diseases [C20]Product Name: cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: cemdisiran
Other descriptive name: CEMDISIRAN
Alnylam Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finishedFemale: yes
Male: yes
30Phase 2France;United States;Philippines;Taiwan;Canada;Spain;Malaysia;Singapore;United Kingdom;Sweden
3EUCTR2018-002716-27-ES
(EUCTR)
08/04/201912/04/2019A Phase 2 Study of Cemdisiran in Adult Patients with IgA NephropathyA Phase 2, Randomized, Double-blind, Placebo-controlled Study of Cemdisiran in Adult Patients with IgA Nephropathy - A Phase 2 Study of Cemdisiran in Adult Patients with IgA Nephropathy Immunoglobulin A nephropathy (IgAN);Therapeutic area: Diseases [C] - Immune System Diseases [C20]Product Name: cemdisiran
Product Code: ALN-CC5
INN or Proposed INN: cemdisiran
Other descriptive name: CEMDISIRAN
Alnylam Pharmaceuticals IncNULLAuthorised-recruitment may be ongoing or finished Female: yes
Male: yes
30 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noUnited States;Philippines;Taiwan;Spain;United Kingdom;France;Canada;Malaysia;Singapore;Australia;Germany;Sweden;Korea, Republic of

109. Atypical hemolytic uremic syndrome


Clinical trials : 114 Drugs : 36 - (DrugBank : 7) / Drug target genes : 3 - Drug target pathways : 11
No.TrialIDDate_
enrollment
Date_
registration
Public_titleScientific_titleConditionInterventionPrimary_
sponsor
Secondary_
sponsor
Recruitment_
Status
Inclusion_
agemin
Inclusion_
agemax
Inclusion_
gender
Target_
size
PhaseCountries
1NCT03999840
(ClinicalTrials.gov)
January 202125/6/2019Eculizumab to Cemdisiran Switch in aHUSA Phase II, Randomized, Double-blind, Placebo-controlled Trial to Evaluate the Safety and Efficacy of Cemdisiran (ALN-CC5) Following Withdrawal of Chronic Eculizumab Therapy in Patients With Atypical HUS at High Risk of RecurrenceAtypical Hemolytic Uremic SyndromeDrug: cemdisiran;Drug: PlacebosMario Negri Institute for Pharmacological ResearchAlnylam PharmaceuticalsWithdrawn12 YearsN/AAll0Phase 2Italy
2EUCTR2018-004382-13-IT
(EUCTR)
28/11/201925/05/2021Safety and efficacy of Cemdisiran in athypical hemolytic uremic syndromeA PHASE II, RANDOMIZED, DOUBLE-BLIND, PLACEBO-CONTROLLED TRIAL TO EVALUATE THE SAFETY AND EFFICACY OF CEMDISIRAN (ALN-CC5) FOLLOWING WITHDRAWAL OF CHRONIC ECULIZUMAB THERAPY IN PATIENTS WITH ATYPICAL HUS AT HIGH RISK OF RECURRENCE - Eculizumab to Cemdisiran switch in aHUS Atypical Hemolytic Uremic Syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ALN-CC5
Product Code: [ALN-62643]
INN or Proposed INN: Cemdisiran
IRCCS- ISTITUTO DI RICERCHE FARMACOLOGICHE MARIO NEGRINULLNot RecruitingFemale: yes
Male: yes
12Phase 2Italy
3EUCTR2017-001082-24-SE
(EUCTR)
20/11/201727/09/2017An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injectionA Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome Atypical Hemolytic Uremic Syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ALN-CC5
Product Code: ALN-CC5
INN or Proposed INN: cemdisiran
Alnylam Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
24 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noSerbia;Estonia;Lithuania;India;Canada;Macedonia, the former Yugoslav Republic of;Romania;Albania;Georgia;Latvia;Moldova, Republic of;Sweden;Bosnia and Herzegovina
4NCT03303313
(ClinicalTrials.gov)
September 19, 201725/9/2017A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic SyndromeA Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients With Atypical Hemolytic Uremic SyndromeAtypical Hemolytic Uremic SyndromeDrug: CemdisiranAlnylam PharmaceuticalsNULLWithdrawn18 YearsN/AAll0Phase 2Bosnia and Herzegovina;Canada;Estonia;Georgia;Latvia;Lithuania;Macedonia, The Former Yugoslav Republic of;Moldova, Republic of;Serbia;Sweden
5EUCTR2017-001082-24-EE
(EUCTR)
15/09/201716/08/2017An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injectionA Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome Atypical Hemolytic Uremic Syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ALN-CC5
Product Code: ALN-CC5
INN or Proposed INN: cemdisiran
Alnylam Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
24 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noSerbia;Estonia;Lithuania;India;Canada;Macedonia, the former Yugoslav Republic of;Romania;Albania;Georgia;Latvia;Moldova, Republic of;Sweden;Bosnia and Herzegovina
6EUCTR2017-001082-24-LT
(EUCTR)
23/08/201727/06/2017An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injectionA Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome Atypical Hemolytic Uremic Syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ALN-CC5
Product Code: ALN-CC5
INN or Proposed INN: cemdisiran
Alnylam Pharmaceuticals, Inc.NULLNot RecruitingFemale: yes
Male: yes
24Phase 2Serbia;Estonia;Lithuania;India;Canada;Macedonia, the former Yugoslav Republic of;Romania;Albania;Georgia;Latvia;Moldova, Republic of;Sweden;Bosnia and Herzegovina
7EUCTR2017-001082-24-LV
(EUCTR)
18/08/201720/06/2017An international study at different study sites providing a drug called cemdisiran (ALN-CC5) to patients with Atypical Hemolytic Uremic Syndrome by a subcutaneous injectionA Phase 2, Open Label, Multicenter Study of ALN-CC5 Administered Subcutaneously in Adult Patients with Atypical Hemolytic Uremic Syndrome Atypical Hemolytic Uremic Syndrome
MedDRA version: 20.0;Level: LLT;Classification code 10019515;Term: Hemolytic uremic syndrome;System Organ Class: 100000004851 ;Therapeutic area: Diseases [C] - Blood and lymphatic diseases [C15]
Product Name: ALN-CC5
Product Code: ALN-CC5
INN or Proposed INN: cemdisiran
Alnylam Pharmaceuticals, Inc.NULLNot Recruiting Female: yes
Male: yes
24 Human pharmacology (Phase 1): no Therapeutic exploratory (Phase 2): yes Therapeutic confirmatory - (Phase 3): no Therapeutic use (Phase 4): noSerbia;Estonia;Macedonia, the former Yugoslav Republic of;Canada;Romania;Lithuania;Georgia;Albania;Latvia;Moldova, Republic of;Sweden;Bosnia and Herzegovina