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Results for “RNAi Therapeutic”

52 trials

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Showing 20 of 52 results

Large-scale testing (Phase 3)Study completedNCT03417245
What this trial is testing

Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients Without Inhibitors

Who this might be right for
Hemophilia AHemophilia B
Genzyme, a Sanofi Company 120
Large-scale testing (Phase 3)Active Not RecruitingNCT04153149
What this trial is testing

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Who this might be right for
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Alnylam Pharmaceuticals 655
Testing effectiveness (Phase 2)Looking for participantsNCT07575308
What this trial is testing

HMBeacon: A Phase 2 Study to Evaluate ALN-6400 in Adult and Adolescent Female Patients With VWD and HMB

Who this might be right for
Von Willebrand Disease (VWD)Heavy Menstrual Bleeding (HMB)
Alnylam Pharmaceuticals 24
Early research (Phase 1)Ended earlyNCT02826018
What this trial is testing

ALN-HBV in Healthy Adult Volunteers and Non-cirrhotic Patients With Chronic Hepatitis B Virus (HBV) Infection

Who this might be right for
Hepatitis BChronic Hepatitis BHepatitis B, Chronic+2 more
Alnylam Pharmaceuticals 24
Testing effectiveness (Phase 2)WithdrawnNCT03303313
What this trial is testing

An Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome

Who this might be right for
Atypical Hemolytic Uremic Syndrome
Alnylam Pharmaceuticals
Testing effectiveness (Phase 2)Study completedNCT01981837
What this trial is testing

Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis

Who this might be right for
TTR-mediated Amyloidosis
Alnylam Pharmaceuticals 26
Early research (Phase 1)Study completedNCT02452372
What this trial is testing

Givosiran (ALN-AS1) in Patients With Acute Intermittent Porphyria (AIP)

Who this might be right for
Acute Intermittent Porphyria
Alnylam Pharmaceuticals 40
Testing effectiveness (Phase 2)Study completedNCT02949830
What this trial is testing

Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)

Who this might be right for
Acute Intermittent Porphyria
Alnylam Pharmaceuticals 16
Testing effectiveness (Phase 2)Study completedNCT01617967
What this trial is testing

Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis

Who this might be right for
TTR-mediated Amyloidosis
Alnylam Pharmaceuticals 29
Large-scale testing (Phase 3)Study completedNCT02510261
What this trial is testing

The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Participants Who Have Already Been Treated With ALN-TTR02 (Patisiran)

Who this might be right for
Amyloidosis
Alnylam Pharmaceuticals 211
Large-scale testing (Phase 3)Looking for participantsNCT07052903
What this trial is testing

TRITON-CM: A Study to Evaluate Nucresiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Who this might be right for
Transthyretin Amyloidosis With Cardiomyopathy
Alnylam Pharmaceuticals 1,750
Early research (Phase 1)Ended earlyNCT04565717
What this trial is testing

ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)

Who this might be right for
Nonalcoholic SteatohepatitisNASH
Alnylam Pharmaceuticals 6
Not applicableStudy completedNCT04201418
What this trial is testing

Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation

Who this might be right for
Hereditary Transthyretin-mediated (ATTRv) AmyloidosisPolyneuropathy
Alnylam Pharmaceuticals 67
Testing effectiveness (Phase 2)Study completedNCT02352493
What this trial is testing

An Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH

Who this might be right for
Paroxysmal Nocturnal Hemoglobinuria (PNH)
Alnylam Pharmaceuticals 62
Early research (Phase 1)Looking for participantsNCT07295717
What this trial is testing

Evaluate ALN-4285 in Adult Healthy Volunteers

Who this might be right for
Healthy Volunteers
Alnylam Pharmaceuticals 76
Testing effectiveness (Phase 2)Study completedNCT02706886
What this trial is testing

Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1

Who this might be right for
Primary Hyperoxaluria Type 1 (PH1)
Alnylam Pharmaceuticals 52
Early research (Phase 1)Looking for participantsNCT07624071
What this trial is testing

Evaluate ALN-6222 in Participants With Obesity

Who this might be right for
Obesity
Alnylam Pharmaceuticals 88
Early research (Phase 1)Study completedNCT04555486
What this trial is testing

Study to Evaluate Safety, Tolerability, PK and PD of DCR-PHXC in PH Type 3 Patients

Who this might be right for
Primary Hyperoxaluria Type 3
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company 6
Testing effectiveness (Phase 2)Study completedNCT03847909
What this trial is testing

Evaluate DCR-PHXC in Children and Adults With Primary Hyperoxaluria Type 1 and Primary Hyperoxaluria Type 2

Who this might be right for
Primary Hyperoxaluria Type 1 (PH1)Primary Hyperoxaluria Type 2 (PH2)Kidney Diseases+2 more
Novo Nordisk A/S 35
Large-scale testing (Phase 3)Study completedNCT03338816
What this trial is testing

ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)

Who this might be right for
Acute Hepatic PorphyriaAcute Intermittent PorphyriaPorphyria, Acute Intermittent+4 more
Alnylam Pharmaceuticals 94
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