Clinical trials: Alpha1 Antitrypsin Deficiency
Alpha1-Antitrypsin Deficiency
Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency
This study is a multicenter, open-label trial to evaluate the safety and pharmacokinetics of weekly intravenous infusions of 60 mg/kg of Alpha-1 MP (alpha1-proteinase inhibitor (human), modified process) for 8 weeks.
- Trial name
- NCT02870309
- Testing
- Alpha-1 MP
- Where
- 2 sites in Japan
- Volunteers
- 4 people
- Sponsor
- Grifols Therapeutics LLC
Alpha1-antitrypsin Deficiency
Study of the Effect of Aerosolized, Recombinant Alpha 1-Antitrypsin on Epithelial Lining Fluid Analytes in Subjects With Alpha 1-Antitrypsin Deficiency
The study was a Phase 1B/2A, uncontrolled, open-label, single-center study in individuals with congenital AAT (alpha 1-antitrypsin) deficiency.
- Trial name
- NCT00157092
- Testing
- Aerosolized, Recombinant Alpha 1-Antitrypsin
- Where
- United States
- Volunteers
- 15 people
- Sponsor
- Baxalta now part of Shire
Alpha 1-Antitrypsin Deficiency, Genetic Disease
Alpha-1 Antitrypsin Disease Cohort: Longitudinal Biomarker Study of Disease
Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. The purpose of this study is to examine the density of the lung as measured by chest computed tomography (CT) and determine if…
- Trial name
- A1BC (NCT05297812)
- Where
- 9 sites in United States
- Volunteers
- 286 people
- Sponsor
- Columbia University
Alpha-1 Antitrypsin Deficiency, Emphysema
Alpha-1 Carrier Genomics Study
The goal of this study is to better understand why some Alpha-1 genotype MZ (PiMZ) individuals develop chronic obstructive pulmonary disease (COPD) while others do not.
- Conditions
- Alpha-1 Antitrypsin Deficiency, Emphysema, COPD, Smoking
- Trial name
- NCT02810327
- Testing
- Genetic Sequencing
- Where
- United States
- Volunteers
- 117 people
- Sponsor
- Medical University of South Carolina
Alpha-1 Antitrypsin Deficiency
Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose.
- Trial name
- NCT02900183
- Testing
- ARC-AAT Injection
- Where
- 4 sites in Canada, Ireland and 2 more
- Sponsor
- Arrowhead Pharmaceuticals
Alpha-1 Anti-trypsin Deficiency, AATD
A Phase 1/2a, Single- and Multiple-dose Escalation Study of KRRO-110
The purpose of this first-in human (FIH) study is to assess the safety, tolerability, and pharmacokinetics (PK) of single and multiple doses of KRRO-110 in both healthy adult participants and in clinically stable patients with…
- Trial name
- REWRITE (NCT06677307)
- Testing
- KRRO-110
- Where
- 5 sites in Australia, New Zealand
- Volunteers
- 42 people, healthy volunteers welcome
- Sponsor
- Korro Bio, Inc.
Alpha-1 Antitrypsin Deficiency
Respreeza® Self-administration and Learning Program (AmAREtTI Study)
According to the Respreeza® Summary of Product Characteristics, the initial infusions must be administered under the supervision of a health professional experienced in the treatment of alpha-1 antitrypsin deficiency, although…
- Trial name
- AmAREtTI (NCT04262284)
- Where
- 11 sites in France
- Volunteers
- 15 people
- Sponsor
- CSL Behring
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
Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease
A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency
The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM647a, an investigational Prime Editing therapy, in adults and adolescents with Alpha-1 Antitrypsin Deficiency…
- Conditions
- Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease, Alpha-1 Antitrypsin Deficiency (AATD), AATD
- Trial name
- PM647a (NCT07887347)
- Testing
- PM647a
- Volunteers
- 54 people
- Sponsor
- Prime Medicine, Inc.
Alpha₁-Antitrypsin Deficiency
A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency
Grifols Therapeutics Inc. conducted a multi-center, randomized, double-blind, crossover study to evaluate the safety, immunogenicity, and pharmacokinetics (PK) of Liquid Alpha₁-PI compared to the currently licensed product…
- Trial name
- NCT02282527
- Testing
- Liquid Alpha₁-PI, Prolastin-C
- Where
- 6 sites in United States
- Volunteers
- 32 people
- Sponsor
- Grifols Therapeutics LLC
Alpha1-Antitrypsin Deficiency
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of AAT, called Z-AAT.
- Trial name
- ALPHATUDE (NCT06512454)
- Testing
- No Intervention
- Where
- 8 sites in United States, Austria and 4 more
- Volunteers
- 500 people
- Sponsor
- Takeda
Pulmonary Emphysema in Alpha-1 PI Deficiency
Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156 weeks (i.e., 3 years) using computed…
- Trial name
- SPARTA (NCT01983241)
- Testing
- Alpha-1 MP, 0.9% Sodium Chloride for Injection, USP
- Where
- 52 sites in United States, Argentina and 15 more
- Volunteers
- 345 people
- Sponsor
- Grifols Therapeutics LLC
Alpha-1 Antitrypsin Deficiency
Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"
To evaluate different doses of "Kamada-AAT for Inhalation" on the levels of alpha 1-proteinase inhibitor and other analytes in epithelial lining fluid (ELF) and serum and to assess the safety of the treatment in subjects with AAT…
- Trial name
- NCT02001688
- Testing
- Kamada-AAT for Inhalation, 80mg, Kamada-AAT for Inhalation, 160mg
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Kamada, Ltd.
Alpha 1 Antitrypsin Deficiency
Alpha-1 Foundation DNA and Tissue Bank
This is a long-term follow-up banking protocol that collects medical information and tissue (blood, liver and lung) samples for future research; use of data and samples for research purposes is overseen by the Tissue Bank…
- Trial name
- NCT00884455
- Where
- United States
- Volunteers
- 5,000 people, healthy volunteers welcome
- Sponsor
- University of Florida
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency
The purpose of this study is to conduct a pilot study to evaluate the safety and efficacy of weekly administration of Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy in subjects with A1PI deficiency and emphysema/ chronic…
- Trial name
- NCT02722304
- Testing
- ARALAST NP 60 mg/kg, ARALAST NP 120 mg/kg, GLASSIA 60 mg/kg
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 7 people
- Sponsor
- Baxalta now part of Shire
Retrospective Cohort of Patients Diagnosed With HCC on Non-Cirrhotic Liver
Prevalence of Alpha-1 Antitrypsin Deficiency in Non-Cirrhotic Liver Cancer
Patients with Alpha-1 Antitrypsin Deficiency (AATD), a rare genetic disease, have a 20- to 50-fold higher risk of developing primary liver cancer (PLC), such as hepatocellular carcinoma (HCC), in both cirrhotic and non-cirrhotic…
- Trial name
- CHAMAD (NCT07145385)
- Testing
- multiplex digital PCR
- Where
- France
- Volunteers
- 71 people
- Sponsor
- University Hospital, Bordeaux
Alpha 1-Antitrypsin Deficiency
Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
The purpose of this study is to evaluate the the safety and efficacy of the investigational product, fazirsiran (TAK-999, ARO-AAT), administered subcutaneously to patients with alpha-1 antitrypsin deficiency associated liver…
- Trial name
- NCT03946449
- Testing
- ARO-AAT
- Where
- 4 sites in Austria, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- Arrowhead Pharmaceuticals
Alpha 1-Antitrypsin Deficiency
The Safety and Tolerability of Alpha-1 Modified Process (MP) In Subjects With Alpha-1-antitrypsin (AAT) Deficiency
The purpose of this clinical study is to assess the safety and tolerability of Alpha-1 MP in adult Alpha1-antitrypsin deficient patients.
- Trial name
- STAMP (NCT00301366)
- Testing
- alpha-1 proteinase inhibitor (human)
- Where
- 10 sites in United States, United Kingdom
- Volunteers
- 38 people
- Sponsor
- Grifols Therapeutics LLC
Alpha 1-Antitrypsin Deficiency, Emphysema
Management of Patients With Alpha-1 Antitrypsin Deficiency Associated Emphysema
The aim of this study is to describe the natural history of patients with alpha-1 antitrypsin associated emphysema and to figure out associated prognostic factors.
- Trial name
- NCT00700934
- Where
- France
- Volunteers
- 300 people
- Sponsor
- Assistance Publique - Hôpitaux de Paris
Alpha-1 Antitrypsin Deficiency
Alpha-1 Antitrypsin Deficiency Adult Liver Study
The investigators hypothesize that there is liver injury (inflammation, fibrosis, cirrhosis) in adults with Alpha-1 Antitrypsin Deficiency (AATD), which is asymptomatic, under-recognized, and undiagnosed.
- Trial name
- NCT02014415
- Testing
- Liver Biopsy (Biopsy Group Only)
- Where
- 3 sites in United States
- Volunteers
- 120 people
- Sponsor
- St. Louis University