Clinical trials: sponsored by Alnylam Pharmaceuticals
Amyloidosis
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)
The purpose of this study is to evaluate the safety and efficacy of long-term dosing with ALN-TTR02 (patisiran) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT02510261
- Testing
- Patisiran
- Where
- 56 sites in United States, Argentina and 19 more
- Volunteers
- 211 people
- Sponsor
- Alnylam Pharmaceuticals
Chronic Inflammatory Demyelinating Polyneuropathy, Guillain-Barre Syndrome
RWE-based Treatment Patterns and Outcomes in CIDP
To describe the demographics, clinical characteristics, treatment patterns and clinical outcomes of chronic inflammatory demyelinating polyneuropathy (CIDP), Guillain-Barre Syndrome (GBS), and heredofamilial amyloidosis (hATTR)…
- Conditions
- Chronic Inflammatory Demyelinating Polyneuropathy, Guillain-Barre Syndrome, Hereditary Amyloidosis
- Trial name
- NCT05940922
- Testing
- No intervention. Retrospective observation only.
- Where
- United Kingdom
- Volunteers
- 170 people
- Sponsor
- RwHealth
TTR-mediated Amyloidosis
Phase 2 Study to Evaluate ALN-TTRSC (Revusiran) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
The purpose of this study is to determine the pharmacokinetics, pharmacodynamics and exploratory clinical activity of ALN-TTRSC (revusiran) in Patients with Transthyretin (TTR) Cardiac Amyloidosis.
- Trial name
- NCT01981837
- Testing
- ALN-TTRSC (revusiran) for subcutaneous administration
- Where
- 4 sites in United States, United Kingdom
- Volunteers
- 26 people
- Sponsor
- Alnylam Pharmaceuticals
Hypertension
A Study to Evaluate ALN-AGT01 in Patients With Hypertension
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetic (PK), and pharmacodynamic (PD) effects of subcutaneous (SC) ALN-AGT01 (zilebesiran) in participants with hypertension.
- Trial name
- NCT03934307
- Testing
- ALN-AGT01, Irbesartan
- Where
- 3 sites in United Kingdom
- Volunteers
- 124 people
- Sponsor
- Alnylam Pharmaceuticals
Hereditary Transthyretin-mediated (ATTRv) Amyloidosis, Polyneuropathy
A Multicenter Observational Study to Evaluate the Effectiveness of Patisiran in Patients With Polyneuropathy of ATTRv Amyloidosis With a V122I or T60A Mutation
To evaluate the effectiveness of patisiran in patients with ATTRv amyloidosis with polyneuropathy who have a V122I or T60A mutation.
- Trial name
- NCT04201418
- Testing
- Patisiran
- Where
- 27 sites in United States
- Volunteers
- 67 people
- Sponsor
- Alnylam Pharmaceuticals
Chronic Hepatitis B
Study of VIR-2218 in Healthy Subjects and Patients With Chronic Hepatitis B
This is a phase 1/2 study in which healthy adult subjects and subjects with chronic hepatitis B virus (HBV) infection will receive VIR-2218 or placebo and will be assessed for safety, tolerability, pharmacokinetics, and antiviral…
- Trial name
- NCT03672188
- Testing
- VIR-2218
- Where
- 14 sites in Australia, Hong Kong and 3 more
- Volunteers
- 82 people, healthy volunteers welcome
- Sponsor
- Vir Biotechnology, Inc.
Healthy
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Pozelimab in Combination With Cemdisiran in Healthy Adult Volunteers
The primary objective of the study is to evaluate the safety and tolerability of ascending doses of subcutaneous (SC) pozelimab and SC cemdisiran when administered on the same day or sequentially 28 days apart.
- Trial name
- NCT04601844
- Testing
- Pozelimab, Cemdisiran
- Where
- Belgium
- Volunteers
- 19 people, healthy volunteers welcome
- Sponsor
- Regeneron Pharmaceuticals
Acute Hepatic Porphyria
EXPLORE: A Natural History Study of Acute Hepatic Porphyria (AHP)
The purpose of this study is to characterize the natural history and clinical management of Acute Hepatic Porphyria (AHP) patients with recurring attacks.
- Trial name
- NCT02240784
- Where
- 30 sites in United States, Australia and 18 more
- Volunteers
- 136 people
- Sponsor
- Alnylam Pharmaceuticals
ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
A Study of ALN-AAT02 in Healthy Participants and Participants With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
The purpose of this study is to evaluate the safety and tolerability of single or multiple doses of ALN-AAT02. The study will be conducted in 2 sequential phases in which Part A will be a single-ascending dose (SAD) phase in…
- Trial name
- NCT03767829
- Testing
- ALN-AAT02
- Where
- United Kingdom
- Volunteers
- 32 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Atypical Hemolytic Uremic Syndrome
Eculizumab to Cemdisiran Switch in aHUS
Atypical Hemolytic Uremic Syndrome (aHUS) is a rare, lifethreatening, chronic disease of complement-mediated thrombotic microangiopathy (TMA) characterized by acute onset of renal impairment, thrombocytopenia, and…
- Trial name
- DANCE (NCT03999840)
- Testing
- cemdisiran
- Where
- Italy
- Sponsor
- Mario Negri Institute for Pharmacological Research
TTR-mediated Amyloidosis
A Extension Study to Evaluate Revusiran (ALN-TTRSC) in Patients With Transthyretin (TTR) Cardiac Amyloidosis
The purpose of this study was to evaluate the safety and clinical activity of long-term dosing with revusiran (ALN-TTRSC). Dosing has been discontinued; patients are being followed-up for safety.
- Trial name
- NCT02292186
- Testing
- Revusiran (ALN-TTRSC)
- Where
- 5 sites in United States, Canada and 1 more
- Volunteers
- 25 people
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis, Leukocyte Chemotactic Factor 2 Amyloidosis
A Study of the Natural History of Leukocyte Chemotactic Factor 2 Amyloidosis (ALECT2) Disease
The purpose of this study is to characterize the natural history of leukocyte chemotactic factor 2 amyloidotic (ALECT2) disease. In this observational study participants with ALECT2 disease will be enrolled.
- Trial name
- NCT03774784
- Where
- 31 sites in United States, Egypt and 4 more
- Volunteers
- 26 people
- Sponsor
- Alnylam Pharmaceuticals
Paroxysmal Nocturnal Hemoglobinuria (PNH)
A Phase 1/2 Study of an Investigational Drug, ALN-CC5, in Healthy Adult Volunteers and Patients With PNH
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics and pharmacodynamics of ALN-CC5 in healthy adult volunteers and subjects with PNH
- Trial name
- NCT02352493
- Testing
- ALN-CC5, Sterile Normal Saline (0.9% NaCl)
- Where
- 4 sites in Spain, United Kingdom
- Volunteers
- 62 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria Type 1 (PH1)
Study of Lumasiran in Healthy Adults and Patients With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of single-ascending doses (SAD) and multiple-ascending doses (MAD) of lumasiran in healthy adult volunteers and subjects…
- Trial name
- NCT02706886
- Testing
- Lumasiran
- Where
- 9 sites in France, Germany and 3 more
- Volunteers
- 52 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP)
The Study of an Investigational Drug, Revusiran (ALN-TTRSC), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Whose Disease Has Continued to Worsen Following Liver Transplant
The purpose of this study was to evaluate the safety and effectiveness of revusiran (ALN-TTRSC) in adults with transthyretin-mediated amyloidosis (ATTR), whose disease has continued to worsen after liver transplantation.
- Conditions
- Transthyretin (TTR)-Mediated Amyloidosis, Familial Amyloidotic Polyneuropathy (FAP), ATTR Amyloidosis, Familial Amyloid Neuropathies
- Trial name
- NCT02595983
- Testing
- Revusiran
- Where
- 6 sites in France, Germany and 4 more
- Volunteers
- 12 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP)
A Study to Investigate the Interaction Between Givosiran and a 5-probe Drug Cocktail in Patients With Acute Intermittent Porphyria (AIP)
The purpose of this study is to evaluate the effect of givosiran on the pharmacokinetics of the 5-probe cocktail of midazolam, caffeine, losartan, omeprazole, and dextromethorphan, and their metabolites, in asymptomatic patients…
- Conditions
- Acute Intermittent Porphyria (AIP), Acute Hepatic Porphyria (AHP), Porphyria, Acute Intermittent, Acute Porphyria
- Trial name
- NCT03505853
- Testing
- Givosiran, 5-probe cocktail
- Where
- Sweden
- Volunteers
- 10 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Hepatic Porphyria, Hepatic Porphyrias
INSIGHT-AHP: A Study to Characterize the Prevalence of Acute Hepatic Porphyria (AHP) in Patients With Clinical Presentation and History Consistent With AHP
This study will use specific diagnostic tests on a group of patients who are experiencing symptoms typical of acute hepatic porphyria (AHP) to determine how many have the condition, and to potentially help improve the diagnostic…
- Conditions
- Acute Hepatic Porphyria, Hepatic Porphyrias, Acute Intermittent Porphyria (AIP), Hereditary Coproporphyria (HCP)
- Trial name
- NCT03547297
- Where
- 12 sites in United States
- Volunteers
- 212 people
- Sponsor
- Alnylam Pharmaceuticals
Antitrypsin Deficiency Liver Disease
A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
The purpose of this study is to evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency…
- Trial name
- NCT02503683
- Testing
- ALN-AAT, Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Atypical Hemolytic Uremic Syndrome
A Study of an Investigational Drug, Cemdisiran (ALN-CC5), in Patients With Atypical Hemolytic Uremic Syndrome
The purpose of this study is to evaluate the safety, tolerability and pharmacokinetics of Cemdisiran in patients with aHUS.
- Trial name
- NCT03303313
- Testing
- Cemdisiran
- Where
- 12 sites in Bosnia and Herzegovina, Canada and 8 more
- Sponsor
- Alnylam Pharmaceuticals
Hepatitis B, Chronic Hepatitis B
A Study of ALN-HBV in Healthy Adult Volunteers and Non-cirrhotic Patients With Chronic Hepatitis B Virus (HBV) Infection
The purpose of this study is to determine the safety, tolerability and pharmacokinetics of ALN-HBV in healthy adult volunteers and patients with chronic hepatitis B virus (HBV) infection.
- Conditions
- Hepatitis B, Chronic Hepatitis B, Hepatitis B, Chronic, Hepatitis B Infection
- Trial name
- NCT02826018
- Testing
- ALN-HBV, Sterile Normal Saline (0.9% NaCl)
- Where
- 9 sites in Australia, Hong Kong and 4 more
- Volunteers
- 24 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals