Clinical trials: Alpha-1 Protease Inhibitor Deficiency
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, AAT Deficiency
Lung Disease and Its Affect on the Work of White Blood Cells in the Lungs
The purpose of this study is to look at how Alpha-1-antitrypsin (AAT) deficiency and Cystic Fibrosis (CF) affect white blood cells in the lungs, called macrophages, and their ability to work.
- Conditions
- Alpha-1 Antitrypsin Deficiency, AAT Deficiency, AATD, Cystic Fibrosis (CF)
- Trial name
- NCT01851642
- Testing
- History and physical exam., Blood draw., Pulmonary function testing.
- Where
- United States
- Volunteers
- 220 people, healthy volunteers welcome
- Sponsor
- University of Florida
HIV Disease
Safety and Efficacy of (α1Proteinase Inhibitor, α1PI) in HIV Disease
Our primary objective is to further characterize the mechanism by which alpha-1PI regulates CD4 counts. HIV-1 infected patients will be initiated on PROLASTIN®-C (Alpha-1 Proteinase Inhibitor [Human], Grifols Biotherapeutics…
- Trial name
- NCT01731691
- Testing
- α1 Proteinase Inhibitor
- Where
- United States
- Volunteers
- 12 people, healthy volunteers welcome
- Sponsor
- Institute for Human Genetics and Biochemistry
GVHD, Bone Marrow Transplant Failure
α1-antitrypsin (AAT) Levels and Functions in Allogeneic Bone Marrow Transplantation and Throughout Progression Into GVHD
Create a personalized time and context curve of patient circulating α1-antitrypsin (AAT) levels and functions before hematopoietic stem cell transplantation and throughout progression into GVHD. PRIMARY ENDPOINT 1.
- Conditions
- GVHD, Bone Marrow Transplant Failure, Alpha 1-Antitrypsin
- Trial name
- AATGVHD-MARK (NCT03188601)
- Where
- 4 sites in Israel
- Volunteers
- 27 people, healthy volunteers welcome
- Sponsor
- Rambam Health Care Campus
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
Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1
Baby Detect : Genomic Newborn Screening
Newborn screening (NBS) is a global initiative of systematic testing at birth to identify babies with pre-defined severe but treatable conditions.
- Conditions
- Congenital Adrenal Hyperplasia, Familial Hyperinsulinemic Hypoglycemia 1, Phosphoglucomutase 1 Deficiency, Maturity Onset Diabetes of the Young
- Trial name
- BabyDetect (NCT05687474)
- Where
- Belgium
- Volunteers
- 6,824 people, healthy volunteers welcome
- Sponsor
- Centre Hospitalier Universitaire de Liege
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
Healthy, Pulmonary Fibrosis
Identification of Genes Associated With Lung Disease in Patients With Rheumatoid Arthritis
Pulmonary fibrosis (PF) is a condition in which the lungs of a patient become scarred and fibrous. It has been known to occur in as many as 40% of patients diagnosed with rheumatoid arthritis (RA).
- Conditions
- Healthy, Pulmonary Fibrosis, Rheumatoid Arthritis
- Trial name
- NCT00001885
- Where
- United States
- Volunteers
- 400 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
Alpha 1-antitrypsin Deficiency (AATD)
Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction
Brief Summary: This study aims to find out if a genetic condition called Alpha-1 Antitrypsin Deficiency (AATD) is more common in people who have shortness of breath and signs of airway obstruction on their breathing tests.
- Trial name
- AATD-SAO (NCT07152834)
- Testing
- Blood Sampling
- Where
- Turkey (Türkiye)
- Volunteers
- 734 people, healthy volunteers welcome
- Sponsor
- Muğla Sıtkı Koçman University
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
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
Alpha1-antitrypsin Deficiency, Healthy Volunteers
GLASSIA Infusion Rate Study
The purpose of this study was to generate sufficient safety and tolerability information in support of an increase in the infusion rate of intravenous GLASSIA in the prescribing information from 0.04 to 0.2 mL/kg/min.
- Trial name
- NCT01651351
- Testing
- Alpha1-proteinase inhibitor
- Where
- United States
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Baxalta now part of Shire
alpha1-antitrypsin Deficiency
Liver Disease in Patients With alpha1-antitrypsin Deficiency
Alpha1-antitrypsin deficiency (AATD) is the third most common genetic disorder leading to death worldwide. Apart from lung disease, AATD also leads to liver involvement in up to 50% of patients.
- Trial name
- NCT02929940
- Testing
- No intervention
- Where
- Germany
- Volunteers
- 500 people, healthy volunteers welcome
- Sponsor
- RWTH Aachen University
Asthma, Chronic Obstructive Pulmonary Disease
Exploring the Utility of Hyperpolarized 129Xe MRI in Healthy Volunteers and Patients With Lung Disease
This is a single centre exploratory study that aims to apply hyperpolarized xenon-129 (129Xe) magnetic resonance imaging (MRI) methods and measurements in individual patients with and without lung disease to better understand…
- Conditions
- Asthma, Chronic Obstructive Pulmonary Disease, Bronchiectasis, Emphysema
- Trial name
- NCT03455686
- Testing
- Hyperpolarized 129Xe MRI
- Where
- Canada
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- McMaster University
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, Lung Diseases, Obstructive
Characterization of the Pathobiology of Early Lung Destruction in Alpha 1-Antitrypsin Deficient Individuals
Alpha 1-antitrypsin-deficient individuals develop severe destructive lung disease much earlier and their lung function declines faster than the general population of individuals with chronic obstructive lung disease.
- Conditions
- Emphysema, Lung Diseases, Obstructive, Alpha 1-Antitrypsin Deficiency
- Trial name
- NCT00001462
- Where
- United States
- Volunteers
- 60 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
Alpha-1 Antitrypsin Deficiency
Microbioma in Sputa From COPD With Alpha-1 Antitrypsin Deficiency
In the context of the increasing evidence of the pathogenetic role of microbiome in COPD, our aim is to determine the total and specific bacterial and viral load in sputa from patients with COPD due to AATD and to correlate these…
- Trial name
- AATD (NCT02547532)
- Testing
- collection of sputum
- Volunteers
- 100 people, healthy volunteers welcome
- Sponsor
- Fondazione Salvatore Maugeri
Alpha-1 Antitrypsin Deficiency (AATD)
A Phase 1 Research Study to Evaluate Safety, Tolerability, and Pharmacokinetics of WVE-006 in Healthy Participants With Wild-type AAT Expression (RestorAATion-1)
This study is the first study in the RestorAATion clinical program. The purpose of this first-in human (FIH), double-blind, randomized, placebo-controlled, single ascending dose (SAD) and multiple-dose Phase 1 study is to assess…
- Trial name
- RestorAATion-1 (NCT06186492)
- Testing
- WVE-006
- Where
- United Kingdom
- Volunteers
- 47 people, healthy volunteers welcome
- Sponsor
- Wave Life Sciences USA, Inc.
Antitrypsin Deficiency Liver Disease
A Study of an Investigational Drug, ALN-AAT, in Healthy Adult Subjects and Patients With ZZ Type Alpha-1 Antitrypsin Deficiency Liver Disease
The purpose of this study is to evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Subcutaneously Administered ALN-AAT in Healthy Adult Subjects and Patients with ZZ Type Alpha-1 Antitrypsin Deficiency…
- Trial name
- NCT02503683
- Testing
- ALN-AAT, Sterile Normal Saline (0.9% NaCl)
- Where
- United Kingdom
- Volunteers
- 26 people, healthy volunteers welcome
- Sponsor
- Alnylam Pharmaceuticals