Clinical Trials on Pyruvate Dehydrogenase E1 Alpha Deficiency in Ireland

Total 13 results

Vertex Pharmaceuticals Incorporated

Completed

Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype

Alpha1-Antitrypsin Deficiency

United States, Germany, Canada, Ireland, Sweden, United Kingdom
CSL Behring

Completed

Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.

Emphysema | Alpha 1-proteinase Inhibitor Deficiency

Australia, Canada, Czech Republic, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden
Arrowhead Pharmaceuticals

Withdrawn

Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels

Alpha-1 Antitrypsin Deficiency

Ireland, Canada, Italy, Sweden
Applied Genetic Technologies Corp
National Heart, Lung, and Blood Institute (NHLBI)

Completed

Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency

Alpha-1 Antitrypsin Deficiency

United States, Ireland
Kamada, Ltd.
Syneos Health

Recruiting

Evaluate Efficacy and Safety of "Kamada-AAT for Inhalation" in Patients With AATD (InnovAATe)

Alpha 1-Antitrypsin Deficiency

United Kingdom, Sweden, Ireland, Netherlands, Belgium, Finland
Vertex Pharmaceuticals Incorporated

Active, not recruiting

A Study to Evaluate Efficacy and Safety of VX-864 in Participants With the PiZZ Genotype

Alpha-1 Antitrypsin Deficiency

United States, United Kingdom, Germany, Ireland
CSL Behring

Completed

Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency

Emphysema | Alpha1-proteinase Inhibitor Deficiency

Australia, Russian Federation, United States, Denmark, Estonia, Finland, Germany, Ireland, Poland, Romania, Sweden, Canada, Czech Republic
Dicerna Pharmaceuticals, Inc., a Novo Nordisk company

Active, not recruiting

A Study of Belcesiran in Patients With AATLD (ESTRELLA)

Alpha 1-Antitrypsin Deficiency

United Kingdom, United States, Ireland, Belgium, New Zealand, Spain, Netherlands, Portugal, Canada, Australia, France, Austria, Germany, Sweden
Agios Pharmaceuticals, Inc.

Completed

A Study Evaluating the Efficacy and Safety of AG-348 in Regularly Transfused Adult Participants With Pyruvate Kinase Deficiency (PKD)

Anemia, Hemolytic | Pyruvate Kinase Deficiency

United States, United Kingdom, Netherlands, Canada, Denmark, France, Italy, Ireland, Thailand
Vertex Pharmaceuticals Incorporated

Terminated

Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype

Alpha 1-Antitrypsin Deficiency

United States, Germany, Canada, Ireland
Agios Pharmaceuticals, Inc.

Active, not recruiting

Extension Study of AG-348 in Adult Participants With Pyruvate Kinase Deficiency Previously Enrolled in AG-348-006 or AG348-C-007

Pyruvate Kinase Deficiency

United States, Spain, Japan, Canada, United Kingdom, Korea, Republic of, Ireland, Thailand, Italy, Turkey, Denmark, Brazil, France, Germany, Netherlands, Switzerland
Kamada, Ltd.

Completed

International Study Evaluating the Safety and Efficacy of Inhaled, Human, Alpha-1 Antitrypsin (AAT) in Alpha-1 Antitrypsin Deficient Patients With Emphysema

Emphysema

United Kingdom, Canada, Denmark, Germany, Ireland, Netherlands, Sweden
Agios Pharmaceuticals, Inc.

Recruiting

Pyruvate Kinase Deficiency Global Longitudinal Registry (PEAK Registry)

Pyruvate Kinase Deficiency

United States, Germany, Spain, Italy, United Kingdom, Czechia, France, Canada, Korea, Republic of, Netherlands, Turkey, Portugal, Denmark, Ireland, Switzerland, Thailand

Clinical Trials on Pyruvate Dehydrogenase E1 Alpha Deficiency in Ireland

Sponsors and Collaborators

Medical Conditions

Drug Interventions

CROs by country

CROs in Tunisia

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Rare Diseases

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Dietary Supplements

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