Clinical trials: Alpha 1-Antitrypsin
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
Type 1 Diabetes Mellitus
Aralast NP in Islet Transplant
Islet transplantation is a relatively new procedure used in people with difficult to control Type 1 diabetes. Insulin producing cells (islets) are isolated from a pancreas donated by the next of kin of a person who is brain dead.
- Trial name
- NCT02520076
- Testing
- Alpha-1 Antitrypsin
- Where
- Canada
- Volunteers
- 5 people
- Sponsor
- University of Alberta
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
Chronic Obstructive Pulmonary Disease, Alpha-1-antitrypsin-deficiency
Effects of Exercise Training in Chronic Obstructive Pulmonary Disease Versus Alpha-1-Antitrypsin-deficiency-patients
Chronic Obstructive Pulmonary Disease (COPD)-patients (caused by smoking-level) and Alpha-1-Antitrypsin-deficiency patients showed different developments during rehabilitation in regard to improvement of 6-minute-walking…
- Trial name
- NCT01380626
- Testing
- exercise training
- Where
- Germany
- Volunteers
- 20 people
- Sponsor
- Schön Klinik Berchtesgadener Land
Alpha 1-Antitrypsin Deficiency
Alpha-1 Research Registry
The Alpha-1 Research Registry is a confidential database made up of individuals diagnosed with Alpha-1 Antitrypsin Deficiency (Alpha-1) and individuals identified as Alpha-1 carriers.
- Trial name
- NCT04157049
- Where
- United States
- Volunteers
- 5,000 people
- Sponsor
- Alpha-1 Foundation
Alpha 1-antitrypsin Deficiency (AATD)
Natural Course and Molecular Basis of Alpha 1- Antitrypsin Deficiency-associated Liver Disease.
To define the course of AATD-associated liver disease. To use the obtained samples for biomedical research which includes: 1. Search for serum-based disease biomarkers and the associated molecular pathways. 2.
- Trial name
- NCT07639996
- Testing
- Multi-omic spatial analysis
- Volunteers
- 45 people
- Sponsor
- Assiut University
Type 1 Diabetes Mellitus
Study of the Safety and Efficacy of Intravenous Alpha-1 Antitrypsin in Type 1 Diabetes Mellitus
Alpha-1 Antitrypsin (AAT), trade name (Glassia ®), is being explored in this phase I/II trial as a potential disease modifying agent in Type 1 Diabetes Mellitus (T1DM) based on its anti-inflammatory properties.
- Trial name
- NCT01304537
- Testing
- Alpha-1 Antitrypsin 40mg (AAT, Glassia®), Alpha-1 Antitrypsin 60mg (AAT, Glassia®), Alpha-1 Antitrypsin 80mg (AAT, Glassia®)
- Where
- 2 sites in Israel
- Volunteers
- 24 people
- Sponsor
- Kamada, Ltd.
Bronchiectasis Adult
Inhaled A1AT in Adult Stable Bronchiectasis
Aim: to investigate the influence of alpha1-antitrypsin (A1-AT) nebulization on levels of A1-AT in BAL and plasma in patients with stable bronchiectasis. Method: single-blind placebo-controlled randomised clinical trial.
- Trial name
- NCT03383939
- Testing
- Group A, Group B
- Volunteers
- 20 people
- Sponsor
- Hospital Universitari Vall d'Hebron Research Institute
Alpha 1-Antitrypsin Deficiency
Study Comparing Weekly Intravenous Administration of OctaAlpha1 With a Marketed Preparation Glassia® in Subjects With Alpha-1-antitrypsin Deficiency
This randomized trial is being conducted to show non-inferiority of OctaAlpha1 compared to Glassia® in terms of the serum trough levels at steady state.
- Trial name
- NCT03385395
- Testing
- OctaAlpha1, Glassia
- Sponsor
- Octapharma
Alpha1-Antitrypsin Deficiency
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
- Trial name
- NCT04474197
- Testing
- VX-864
- Where
- 30 sites in United States, Canada and 4 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Alpha-1 Antitrypsin Deficiency, AATD
Phase 1 Study to Assess the Safety, PK and PD of INBRX-101 in Adults With Alpha-1 Antitrypsin Deficiency
This is an open-label, 2-part, dose-escalating, Phase 1 study of INBRX-101 (rhAAT-Fc). Part 1 will consist of single ascending dose (SAD) administration of INBRX-101 and Part 2 will consist of multiple ascending dose (MAD)…
- Trial name
- rhAAT-Fc (NCT03815396)
- Testing
- INBRX-101/rhAAT-Fc
- Where
- 10 sites in United States, New Zealand and 1 more
- Volunteers
- 31 people
- Sponsor
- Inhibrx Biosciences, Inc
Alpha1-Antitrypsin Deficiency
An Extension Study to Learn About the Long-Term Safety of Fazirsiran and if Fazirsiran Can Help People With Alpha-1 Antitrypsin Liver Disease
The main aim of this study is to learn if fazirsiran is safe during long-term use in people with liver disease caused by the abnormal Z-alpha-1 antitrypsin (Z-AAT) protein.
- Trial name
- NCT05899673
- Testing
- Fazirsiran Injection
- Where
- 10 sites in United States, Austria and 3 more
- Volunteers
- 31 people
- Sponsor
- Takeda
Alpha-1 Antitrypsin Deficiency
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in AAT-deficient adults at three dosage levels [6.0 ×…
- Trial name
- NCT01054339
- Testing
- rAAV1-CB-hAAT
- Where
- 4 sites in United States, Ireland
- Volunteers
- 9 people
- Sponsor
- Beacon Therapeutics
Alpha 1-Antitrypsin Deficiency
Alpha-1 Foundation Research Registry
The Alpha-1 Foundation Research Registry is a confidential database made up of individuals diagnosed with severe alpha-1 antitrypsin deficiency (Alpha-1) or the carrier state.
- Trial name
- NCT00499941
- Where
- United States
- Volunteers
- 6,655 people, healthy volunteers welcome
- Sponsor
- Medical University of South Carolina
Emphysema, Alpha 1-proteinase Inhibitor Deficiency
Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
This study is a continuation of the placebo-controlled study CE1226_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® intravenous (i.v).
- Trial name
- NCT00670007
- Testing
- Alpha1- proteinase inhibitor [human]
- Where
- 21 sites in Australia, Canada and 9 more
- Volunteers
- 140 people
- Sponsor
- CSL Behring