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
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 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease
Prevalence of Alpha-1 Antitrypsin Deficiency in Chronic Obstructive Pulmonary Disease (COPD)
Alpha-1 antitrypsin deficiency (AATD) is considered a rare genetic cause of chronic obstructive pulmonary disease (COPD) and liver disease.
- Trial name
- PFT (NCT01419158)
- Where
- 19 sites in United States, Puerto Rico
- Volunteers
- 3,457 people
- Sponsor
- Alpha-1 Foundation
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
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
Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults
The purpose of this clinical study (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1…
- Trial name
- ChAMP (NCT00295061)
- Testing
- Alpha-1 MP, alpha-1 proteinase inhibitor (human)
- Where
- 8 sites in United States
- Volunteers
- 24 people
- Sponsor
- Grifols Therapeutics LLC
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
Sarcoidosis
Genomic Research in Sarcoidosis
This project is designed to address the following hypothesis: Distinct patterns in lung microbiome are characteristic of sarcoidosis phenotypes and reflected in changes in systemic inflammatory responses as measured by peripheral…
- Trial name
- GRADS Sarc (NCT01831739)
- Where
- 9 sites in United States
- Volunteers
- 368 people
- Sponsor
- University of Pittsburgh
Alpha 1-Antitrypsin Deficiency
Gene Therapy for Alpha 1- Antitrypsin Deficiency
This is a study of gene therapy to treat alpha 1-antitrypsin (AAT) deficiency. This study aims to treat AAT deficiency with a single administration of AAV8hAAT(AVL), a gene therapy that codes for an oxidation resistant form of…
- Trial name
- NCT06996756
- Testing
- AAV8hAAT(AVL)
- Where
- United States
- Volunteers
- 16 people
- Sponsor
- Weill Medical College of Cornell University
Alpha-1-antitrypsin Deficiency
Czech AATD Registry
Alpha-1-antitrypsin deficiency is the most common congenital disease of the respiratory system, leading to early pulmonary emphysema or bronchiectasis. Pulmonary involvement significantly accelerates active cigarette smoking.
- Trial name
- NCT05178277
- Where
- Czechia
- Volunteers
- 300 people
- Sponsor
- Thomayer University Hospital
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
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, Chronic Obstructive Pulmonary Disease (COPD)
Phase 2 Study of Ex-vivo Perfusion and Ventilation of Lungs to Assess Transplant Suitability
The purpose of this research study is to perfect the technique of EVLP and learn about the safety of transplanting lungs that have been ventilated (attached to a breathing machine or ventilator to deliver oxygen) and perfused…
- Conditions
- Emphysema, Chronic Obstructive Pulmonary Disease (COPD), Cystic Fibrosis, Pulmonary Fibrosis
- Trial name
- NCT01241942
- Testing
- Steen Solution™, Conventional Lung Transplant
- Where
- United States
- Volunteers
- 11 people
- Sponsor
- University of North Carolina, Chapel Hill
Alpha 1-Antitrypsin Deficiency
Evaluation of the Efficacy and Safety of VX-814 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
- Trial name
- NCT04167345
- Testing
- VX-814
- Where
- 28 sites in United States, Canada and 2 more
- Volunteers
- 48 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
Alpha 1-Antitrypsin Deficiency
4-PBA: Will it Increase the Level of Alpha 1-Antitrypsin(AAT) in Persons With AAT Deficiency?
The purpose of this study is to find out whether 4-PBA will increase the level of AAT in persons with AAT deficiency whether or not they have liver disease.
- Trial name
- NCT00067756
- Testing
- 4-PBA
- Where
- United States
- Volunteers
- 12 people
- Sponsor
- University of Florida
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency.
- Trial name
- NCT00261833
- Testing
- Alpha1-proteinase inhibitor
- Where
- 28 sites in United States, Australia and 11 more
- Volunteers
- 180 people
- Sponsor
- CSL Behring
Liver Fibrosis, Alpha-1 Antitrypsin Deficiency
Liver Fibrosis in Alpha-1 Antitrypsin Deficiency (Liver AATD)
We hypothesize that individuals with Alpha-1 Antitrypsin (AAT) deficiency have ongoing liver injury which is not detected by the usual blood tests used to look at liver function.
- Conditions
- Liver Fibrosis, Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD
- Trial name
- NCT01810458
- Testing
- Abdominal ultrasound, History and physical, Intravenous catheter
- Where
- United States
- Volunteers
- 109 people
- Sponsor
- University of Florida
Emphysema, Chronic Obstructive Pulmonary Disease (COPD)
Ex-vivo Perfusion and Ventilation of Lungs Recovered From Non-Heart-Beating Donors to Assess Transplant Suitability
The purpose of this research study is to learn about the safety of transplanting lungs obtained from non-heart-beating donors (NHBDs) that have been ventilated (attached to a breathing machine or ventilator to deliver oxygen) and…
- Conditions
- Emphysema, Chronic Obstructive Pulmonary Disease (COPD), Cystic Fibrosis, Pulmonary Fibrosis
- Trial name
- NCT01615484
- Testing
- Transplantation of lungs obtained from Non-Heart-Beating Donors (NHBDs) after ex-vivo perfusion w/ STEEN Solution™, STEEN Solution™
- Where
- 2 sites in United States
- Volunteers
- 24 people
- Sponsor
- University of North Carolina, Chapel Hill
Alpha-1 Anti-trypsin Deficiency
AL1TER™: Alpha-1 Therapy, Evaluation, and Research Patient Registry
This is a longitudinal, observational, non-interventional registry study, designed to collect both retrospective and prospective real world data, on patients receiving Alpha-1 augmentation therapy in the home through Coram…
- Trial name
- AL1TER (NCT01974830)
- Where
- United States
- Volunteers
- 120 people
- Sponsor
- Coram Clinical Trials
Alpha 1-Antitrypsin Deficiency
Experimental Gene Transfer Procedure to Treat Alpha 1-Antitrypsin Deficiency
Individuals with a deficiency of the Alpha 1-antitrypsin (AAT) protein are at risk for developing emphysema and liver damage. Researchers have developed a way to introduce normal AAT genes into muscle cells so that the AAT…
- Trial name
- NCT00377416
- Testing
- rAAV2-CB-hAAT Gene Vector
- Where
- 2 sites in United States
- Volunteers
- 12 people
- Sponsor
- University of Massachusetts, Worcester