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Results for “RNAi Therapeutic”

49 trials

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Showing 20 of 49 results

Early research (Phase 1)Study completedNCT01814839
What this trial is testing

A Phase 1, Single- and Multi-Dose, Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Subcutaneously Administered ALN-TTRSC (Revusiran) in Healthy Volunteers

Who this might be right for
TTR-mediated Amyloidosis
Alnylam Pharmaceuticals 85
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
Testing effectiveness (Phase 2)Study completedNCT02554773
What this trial is testing

An Open-label Extension Study of an Investigational Drug, Fitusiran, in Patients With Moderate or Severe Hemophilia A or B

Who this might be right for
Hemophilia AHemophilia B
Genzyme, a Sanofi Company 34
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
Large-scale testing (Phase 3)Study completedNCT04152200
What this trial is testing

Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

Who this might be right for
Primary Hyperoxaluria Type 1Primary Hyperoxaluria
Alnylam Pharmaceuticals 21
Large-scale testing (Phase 3)Enrolling By InvitationNCT06679946
What this trial is testing

Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Who this might be right for
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Alnylam Pharmaceuticals 700
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
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
Early research (Phase 1)Study completedNCT02797847
What this trial is testing

A Safety and Tolerability Study of an Investigational Drug, ALN-TTRSC02, in Healthy Subjects

Who this might be right for
Transthyretin-mediated Amyloidosis (ATTR Amyloidosis)
Alnylam Pharmaceuticals 80
Not applicableApproved For MarketingNCT02939820
What this trial is testing

Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)

Who this might be right for
TTR-mediated AmyloidosisAmyloidosis, HereditaryAmyloid Neuropathies, Familial+3 more
Alnylam Pharmaceuticals
Large-scale testing (Phase 3)Study completedNCT03417102
What this trial is testing

Fitusiran (ALN-AT3SC) in Severe Hemophilia A and B Patients With Inhibitors

Who this might be right for
Hemophilia AHemophilia B
Genzyme, a Sanofi Company 60
Large-scale testing (Phase 3)Looking for participantsNCT07223203
What this trial is testing

TRITON-PN: A Study to Evaluate the Efficacy and Safety of Nucresiran in Patients With Hereditary Transthyretin Amyloidosis With Polyneuropathy

Who this might be right for
Hereditary Transthyretin-Mediated Amyloidosis With PolyneuropathyhATTR-PN
Alnylam Pharmaceuticals 125
Large-scale testing (Phase 3)Study completedNCT01960348
What this trial is testing

APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis

Who this might be right for
TTR-mediated AmyloidosisAmyloidosis, HereditaryAmyloid Neuropathies, Familial+3 more
Alnylam Pharmaceuticals 225
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
Early research (Phase 1)Ended earlyNCT02503683
What this trial is testing

An Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease

Who this might be right for
Antitrypsin Deficiency Liver Disease
Alnylam Pharmaceuticals 26
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
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)Study completedNCT02292186
What this trial is testing

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

Who this might be right for
TTR-mediated Amyloidosis
Alnylam Pharmaceuticals 25
Testing effectiveness (Phase 2)WithdrawnNCT03999840
What this trial is testing

Eculizumab to Cemdisiran Switch in aHUS

Who this might be right for
Atypical Hemolytic Uremic Syndrome
Mario Negri Institute for Pharmacological Research
Large-scale testing (Phase 3)Active Not RecruitingNCT03997383
What this trial is testing

APOLLO-B: A Study to Evaluate Patisiran in Participants With Transthyretin Amyloidosis With Cardiomyopathy (ATTR Amyloidosis With Cardiomyopathy)

Who this might be right for
Transthyretin Amyloidosis (ATTR) With Cardiomyopathy
Alnylam Pharmaceuticals 360
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