Clinical trials: Alpha1-Antitrypsin Deficiency
Alpha1-Antitrypsin Deficiency
A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency
The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC…
- Trial name
- NCT04722887
- Testing
- Alpha-1 15%, Liquid Alpha1-Proteinase Inhibitor (Human)
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Grifols Therapeutics LLC
alpha1 Anti-trypsin, Pre-Eclampsia
Maternal alpha1 Antitrypsin as a Marker of Intrauterine Growth Restriction in Pre-eclamptic Women
Intrauterine growth restriction (IUGR) is defined as a velocity of fetal growth less than the normal fetus growth potential for a specific neonate as per the race and gender.
- Conditions
- alpha1 Anti-trypsin, Pre-Eclampsia, Intrauterine Growth Restriction
- Trial name
- NCT05622838
- Testing
- Alpha1 -Antitrypsin level
- Volunteers
- 160 people, healthy volunteers welcome
- Sponsor
- Assiut University
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
Epigenetic Regulation of Immunity in Alpha-1 Anti-trypsin Deficiency
The investigators hypothesize that environmentally influenced histone modifications regulate AM mediated inflammation, contributing to a variable clinical course of AATD, and may also influence or be influenced by the activity of…
- Trial name
- AATD_Epi (NCT02691611)
- Where
- United States
- Volunteers
- 39 people, healthy volunteers welcome
- Sponsor
- National Jewish Health
Alpha 1-Antitrypsin Deficiency, Emphysema or COPD
PiMZ Longitudinal Cohort (PiMZ Logic)
Alpha-1 Anti-trypsin Deficiency (AATD) is a genetic disease with lung and liver disease presentations. Presentations are variable in the heterozygous population, the most predominant genotype being PiMZ.
- Trial name
- NCT06505603
- Where
- 5 sites in United States
- Volunteers
- 80 people
- Sponsor
- Columbia University
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, Alpha 1-Antitrypsin Deficiency
Functional and Structural Lung Imaging in Chronic Obstructive Pulmonary Disease
This study aims to use novel proton-based MRI techniques to assess lung function and structure in healthy volunteers and patients with chronic obstructive pulmonary disease (COPD) and alpha-1-anti-trypsin deficiency (A1ATD).
- Trial name
- FASTCOPD (NCT04966221)
- Testing
- Lung MRI scan
- Where
- United Kingdom
- Volunteers
- 75 people, healthy volunteers welcome
- Sponsor
- University of Nottingham
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
Alpha-1 Antitrypsin Deficiency
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
The purpose of this open-label study is to assess the safety, tolerability, pharmacodynamics, and pharmacokinetics of WVE-006 in participants with alpha-1 antitrypsin deficiency (AATD) following Period 1 single ascending dose…
- Trial name
- NCT06405633
- Testing
- WVE-006
- Where
- 9 sites in Australia, Canada and 4 more
- Volunteers
- 48 people
- Sponsor
- Wave Life Sciences USA, Inc.
Lung Diseases, Emphysema
Alpha1-antitrypsin Deficiency Registry
To collect data from the 37 participating clinical centers on patients with alpha1-antitrypsin deficiency, including those who received replacement therapy with an intravenous preparation of alpha1-proteinase inhibitor (A1Pi)…
- Conditions
- Lung Diseases, Emphysema, Alpha-1 Antitrypsin Deficiency, Chronic Obstructive Pulmonary Disease
- Trial name
- NCT00005292
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Chronic Obstructive Pulmonary Disease, Alpha-1 Antitrypsin Deficiency
Environment Effect on Six-Minute Walk Test Performance
The purpose of this research study is to determine the effect of the environment on six-minute walk test performance in individuals with chronic obstructive pulmonary disease due to alpha-1 antitrypsin deficiency.
- Trial name
- 6MWTAATD (NCT02502201)
- Testing
- six-minute walk study indoors, six-minute walk study outdoors
- Where
- United States
- Volunteers
- 27 people
- Sponsor
- University of Florida
Emphysema, Alpha 1-Antitrypsin Deficiency
Efficacy/Safety of HA Inhalation Solution for Hereditary Emphysema in Patients With Alpha-1 Antitrypsin Deficiency
The purpose of the study is to evaluate the safety and efficacy of administering repeated doses of Hyaluronic Acid Inhalation Solution to subjects with Emphysema that have Alpha-1-Antitrypsin deficiency
- Trial name
- NCT03114020
- Testing
- Hyaluronic Acid Inhalation Solution
- Where
- 6 sites in United States, Canada
- Volunteers
- 27 people
- Sponsor
- Gerard Turino
Alpha 1-Antitrypsin Deficiency
Safety Dose Finding Study of ADVM-043 Gene Therapy to Treat Alpha-1 Antitrypsin (A1AT) Deficiency
The ADVANCE study is being conducted by Adverum Biotechnologies, Inc. as an open-label, multicenter, dose-escalation study in order to assess the safety and protein expression of ADVM-043 following a single intravenous or…
- Trial name
- ADVANCE (NCT02168686)
- Testing
- ADVM-043
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Adverum Biotechnologies, Inc.
Alpha 1-Antitrypsin Deficiency
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1…
- Trial name
- Serpentine-1 (NCT06049082)
- Testing
- KB408 (Nebulization)
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Krystal Biotech, Inc.
Alpha-1 Antitrypsin Deficiency
A Study of ARC-AAT in Healthy Volunteer Subjects and Patients With Alpha-1 Antitrypsin Deficiency (AATD)
The purpose of the study is to determine the safety and tolerability of escalating doses of ARC-AAT and to evaluate the pharmacokinetics of ARC-AAT and the effect of ARC-AAT on circulating levels of alpha-1 antitrypsin (AAT).
- Trial name
- NCT02363946
- Testing
- ARC-AAT Injection, Diphenhydramine
- Where
- 4 sites in Australia, Germany and 2 more
- Volunteers
- 65 people, healthy volunteers welcome
- Sponsor
- Arrowhead Pharmaceuticals
HIV Disease
Therapy to Elevate CD4 Counts in HIV-1 Disease
For more than 20 years, alpha-1-proteinase inhibitor therapy has been the standard treatment for patients who have inherited alpha-1-proteinase inhibitor deficiency.
- Trial name
- NCT01370018
- Testing
- alpha-1-Proteinase Inhibitor
- Where
- United States
- Volunteers
- 4 people, healthy volunteers welcome
- Sponsor
- Institute for Human Genetics and Biochemistry
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
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
Efficacy and Safety Study of Augmentation Therapy With ARALAST Fraction IV-1 (Human Alpha 1 - Proteinase Inhibitor)
The purpose of this study is to evaluate the effects of weekly augmentation therapy with ARALAST Fraction IV-1 (Fr IV-1) on epithelial lining fluid (ELF) alpha 1-proteinase inhibitor levels and other ELF analytes and to assess…
- Trial name
- NCT00396006
- Testing
- Alpha1-Proteinase Inhibitor
- Where
- 5 sites in Australia, New Zealand
- Volunteers
- 21 people
- Sponsor
- Baxalta now part of Shire