Evaluating the efficacy and safety of patisiran in participants with hereditary transthyretin-mediated amyloidosis (hATTR amyloidosis) with disease progression after liver transplant.

Location(s):

Italy

For more info:

ClinicalTrials.gov – NCT03862807

Providing expanded access of patisiran to adults with hATTR amyloidosis with polyneuropathy who meet Karnofsky performance status and Polyneuropathy Disability (PND) score requirements.

Location(s):

USA

For more info:

ClinicalTrials.gov – NCT02939820

Evaluating the long-term safety of patisiran in adults with hATTR amyloidosis with symptomatic polyneuropathy who participated in either the Phase 2 OLE study or the APOLLO study.

Location(s):

USA, Argentina, Australia, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Italy, Japan, South Korea, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, Turkey, UK

For more info:

ClinicalTrials.gov – NCT02510261

Evaluated the safety and efficacy of patisiran in adults with hereditary ATTR (hATTR) amyloidosis with symptomatic polyneuropathy (previously known as familial amyloidotic polyneuropathy [FAP]).

Location(s):

USA, Argentina, Australia, Brazil, Bulgaria, Canada, Cyprus, France, Germany, Italy, Japan, South Korea, Malaysia, Mexico, Netherlands, Portugal, Spain, Sweden, Taiwan, Turkey, UK

For more info:

ClinicalTrials.gov – NCT01960348

Evaluated the safety and efficacy of patisiran in adults with hATTR amyloidosis with symptomatic polyneuropathy who participated in the Phase 2 trial.

Location(s):

USA, Brazil, France, Germany, Portugal, Spain, Sweden

For more info:

ClinicalTrials.gov – NCT01961921

Evaluated the safety and efficacy of patisiran in adults with hATTR amyloidosis with symptomatic polyneuropathy.

Location(s):

USA, Brazil, France, Germany, Portugal, Spain, Sweden

For more info:

ClinicalTrials.gov – NCT01617967

Evaluating the effect of givosiran (ALN-AS1) on the rate of porphyria attacks in patients with acute hepatic porphyria (AHP).

Location(s):

USA, Australia, Belgium, Bulgaria, Canada, Denmark, Finland, France, Germany, Italy, Japan, South Korea, Mexico, Netherlands, Poland, Spain, Sweden, Switzerland, Taiwan, UK

For more info:

ClinicalTrials.gov – NCT03338816

A prospective observational study of adults with acute hepatic porphyria (AHP), including acute intermittent porphyria (AIP), variegate porphyria (VP), and hereditary coproporphyria (HCP), with recurrent attacks.

Location(s):

Australia, USA, Bulgaria, Czech Republic, Finland, France, Germany, Italy, Israel, Mexico, Netherlands, Norway, Poland, South Africa, South Korea, Spain, Switzerland, Taiwan, UK

For more info:

ClinicalTrials.gov – NCT02240784

Evaluating the long-term safety and tolerability of givosiran in adults with acute intermittent porphyria (AIP), who participated in the Phase 2 trial.

Location(s):

USA, Sweden, UK

For more info:

ClinicalTrials.gov – NCT02949830

Evaluating the safety and tolerability of givosiran (ALN-AS1) in adults with acute intermittent porphyria (AIP).

Location(s):

USA, Sweden, UK

For more info:

ClinicalTrials.gov – NCT02452372

Evaluated the safety and efficacy of inclisiran in adults with elevated low density lipoprotein cholesterol (LDL-C).

Location(s):

UK

For more info:

ClinicalTrials.gov – NCT02314442

Evaluating the safety and efficacy of lumasiran in healthy volunteers and in patients with primary hyperoxaluria type 1 (PH1).

Location(s):

USA, France, UK, Germany, Israel, Netherlands, Jordan

Interested in learning more?

Visit PH1trial.com for more about the PH1 trial.

For more info:

ClinicalTrials.gov – NCT02706886

Evaluating the long-term safety and tolerability of lumasiran in patients with primary hyperoxaluria type 1.

Location(s):

France, Germany, Netherlands, UK, Israel

For more info:

ClinicalTrials.gov – NCT03350451

Evaluated the safety and efficacy of cemdisiran in healthy volunteers and adults with paroxysmal nocturnal hemoglobinuria (PNH).

Location(s):

Spain, UK

For more info:

ClinicalTrials.gov – NCT02352493

Evaluating the effect of cemdisiran on proteinuria in adults with immunoglobulin A nephropathy (IgAN), who excrete >1 gram (gm) of protein per day despite standard of care, which includes treatment with angiotensin-converting enzyme (ACE) inhibitors and angiotensin receptor blockers (ARBs).

Location(s):

USA

For more info:

ClinicalTrials.gov – NCT03841448

Evaluating the efficacy and safety of vutrisiran (ALN-TTRSC02) in patients with hereditary transthyretin amyloidosis (hATTR amyloidosis).

Location(s):

USA

INTERESTED IN LEARNING MORE?

Visit HELIOSAstudy.com for more about the trial.

For more info:

ClinicalTrials.gov – NCT03759379

Evaluating the safety and tolerability of single or multiple doses of ALN-AAT02 in participants with ZZ type alpha-1 antitrypsin deficiency (PiZZ) and biopsy-proven alpha-1 antitrypsin (AAT) deficiency-associated liver disease.

Location(s):

UK

For more info:

ClinicalTrials.gov – NCT03767829