Clinical trials: sponsored by Alnylam Pharmaceuticals
TTR-mediated Amyloidosis, Amyloidosis, Hereditary
Expanded Access Protocol of Patisiran for Patients With Hereditary ATTR Amyloidosis (hATTR)
The purpose of this study is to provide expanded access of patisiran to patients with hereditary transthyretin-mediated amyloidosis (hATTR).
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies
- Trial name
- NCT02939820
- Testing
- patisiran (ALN-TTR02)
- Sponsor
- Alnylam Pharmaceuticals
Acute Hepatic Porphyria
Expanded Access Protocol of Givosiran for Patients With Acute Hepatic Porphyria
The purpose of this study is to provide expanded access of givosiran to patients with Acute Hepatic Porphyria (AHP).
- Trial name
- NCT04056481
- Testing
- Givosiran
- Sponsor
- Alnylam Pharmaceuticals
Nonalcoholic Steatohepatitis, NASH
A Study of ALN-HSD in Healthy Adult Subjects and Adult Patients With Nonalcoholic Steatohepatitis (NASH)
The purpose of this study is to evaluate the safety and tolerability of single ascending doses of ALN-HSD in healthy participants (Part A) and multiple doses of ALN-HSD in patients with NASH (Parts B and C).
- Trial name
- NCT04565717
- Testing
- ALN-HSD
- Where
- 13 sites in United States, Belgium and 3 more
- Volunteers
- 6 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
PH1, Primary Hyperoxaluria
An Extension Study of an Investigational Drug, Lumasiran (ALN-GO1), in Participants With Primary Hyperoxaluria Type 1
The purpose of this study is to evaluate the long-term safety and tolerability of lumasiran in participants with Primary Hyperoxaluria Type 1.
- Conditions
- PH1, Primary Hyperoxaluria, RNAi Therapeutic, siRNA
- Trial name
- NCT03350451
- Testing
- Lumasiran
- Where
- 9 sites in France, Germany and 3 more
- Volunteers
- 20 people
- Sponsor
- Alnylam Pharmaceuticals
TTR-mediated Amyloidosis, Amyloidosis, Hereditary
APOLLO: The Study of an Investigational Drug, Patisiran (ALN-TTR02), for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis
The purpose of this study is to evaluate the safety and efficacy of patisiran (ALN-TTR02) in patients with transthyretin (TTR) mediated amyloidosis.
- Conditions
- TTR-mediated Amyloidosis, Amyloidosis, Hereditary, Amyloid Neuropathies, Familial, Familial Amyloid Polyneuropathies
- Trial name
- NCT01960348
- Testing
- patisiran (ALN-TTR02), Sterile Normal Saline (0.9% NaCl)
- Where
- 52 sites in United States, Argentina and 19 more
- Volunteers
- 225 people
- Sponsor
- Alnylam Pharmaceuticals
Amyloidosis, Familial, Transthyretin Amyloidosis
Patisiran in Patients With Hereditary Transthyretin-mediated Amyloidosis (hATTR Amyloidosis) Disease Progression Post-Liver Transplant
The purpose of this study is to evaluate the efficacy, safety and pharmacokinetics of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver…
- Trial name
- NCT03862807
- Testing
- Patisiran
- Where
- 9 sites in France, Germany and 5 more
- Volunteers
- 24 people
- Sponsor
- Alnylam Pharmaceuticals
Acute Hepatic Porphyria, Acute Intermittent Porphyria
ENVISION: A Study to Evaluate the Efficacy and Safety of Givosiran (ALN-AS1) in Patients With Acute Hepatic Porphyrias (AHP)
The purpose of this study is to evaluate the effect of subcutaneous givosiran (ALN-AS1), compared to placebo, on the rate of porphyria attacks in patients with Acute Hepatic Porphyrias (AHP).
- Conditions
- Acute Hepatic Porphyria, Acute Intermittent Porphyria, Porphyria, Acute Intermittent, Acute Porphyria
- Trial name
- NCT03338816
- Testing
- Givosiran
- Where
- 36 sites in United States, Australia and 16 more
- Volunteers
- 94 people
- Sponsor
- Alnylam Pharmaceuticals
TTR-mediated Amyloidosis
Safety and Tolerability of Patisiran (ALN-TTR02) in Transthyretin (TTR) Amyloidosis
This was a multiple dose, dose escalation study designed to determine the safety, tolerability, pharmacokinetics and pharmacodynamics of patisiran (ALN-TTR02) in participants with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT01617967
- Testing
- Patisiran
- Where
- 10 sites in United States, Brazil and 5 more
- Volunteers
- 29 people
- Sponsor
- Alnylam Pharmaceuticals
TTR-mediated Amyloidosis
The Study of ALN-TTR02 (Patisiran) for the Treatment of Transthyretin (TTR)-Mediated Amyloidosis in Patients Who Have Already Been Treated With ALN-TTR02 (Patisiran)
The purpose of this study is to evaluate the safety and tolerability of long-term dosing with ALN-TTR02 (patisiran) in patients with transthyretin (TTR) mediated amyloidosis (ATTR).
- Trial name
- NCT01961921
- Testing
- ALN-TTR02 (patisiran) administered by intravenous (IV) infusion
- Where
- 9 sites in United States, Brazil and 5 more
- Volunteers
- 27 people
- Sponsor
- Alnylam Pharmaceuticals
Gout
A Study to Evaluate ALN-XDH in Healthy Subjects and Patients With Gout
The purpose of this study is to: Part A: Evaluate the safety and tolerability of single ascending doses of ALN-XDH in healthy adult participants Part B: Evaluate the safety, tolerability and efficacy of ALN-XDH as monotherapy in…
- Trial name
- NCT05256810
- Testing
- ALN-XDH
- Where
- United Kingdom
- Volunteers
- 44 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals
Acute Intermittent Porphyria
A Study to Evaluate Long-term Safety and Clinical Activity of Givosiran (ALN-AS1) in Patient With Acute Intermittent Porphyria (AIP)
The purpose of this study is to determine the long-term safety, tolerability and pharmacokinetics of givosiran (ALN-AS1) in AIP patients who completed study ALN-AS1-001 (NCT02452372).
- Trial name
- NCT02949830
- Testing
- Givosiran
- Where
- 5 sites in United States, Sweden and 1 more
- Volunteers
- 16 people
- Sponsor
- Alnylam Pharmaceuticals
Transthyretin-mediated Amyloidosis With Cardiomyopathy, ATTR Amyloidosis With Cardiomyopathy
Expanded Access Protocol to Provide Patisiran to Patients With Transthyretin-mediated Amyloidosis With Cardiomyopathy
The objective of the study is to assess the long-term safety of patisiran in patients with ATTR amyloidosis with cardiomyopathy as assessed by a review of adverse events (AEs).
- Trial name
- NCT05505838
- Testing
- Patisiran
- Where
- 20 sites in United States
- Sponsor
- Alnylam Pharmaceuticals
Primary Hyperoxaluria
Expanded Access Protocol to Provide Lumasiran to Patients With Primary Hyperoxaluria Type 1
The purpose of this study is to provide expanded access to lumasiran for adults and pediatric patients with Primary Hyperoxaluria Type 1 (PH1),
- Trial name
- NCT04125472
- Testing
- Lumasiran
- Sponsor
- 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
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
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
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