Clinical trials: Alpha-1 Antitrypsin Deficiency
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.
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, 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
Cystic Fibrosis, Pulmonary Fibrosis
Role of Genetic Factors in the Development of Lung Disease
This study is designed to evaluate the genetics involved in the development of lung disease by surveying genes involved in the process of breathing and examining the genes in lung cells of patients with lung disease.
- Conditions
- Cystic Fibrosis, Pulmonary Fibrosis, Tuberous Sclerosis, Asthma
- Trial name
- NCT00001532
- Where
- 2 sites in United States
- Volunteers
- 3,500 people, healthy volunteers welcome
- Sponsor
- National Heart, Lung, and Blood Institute (NHLBI)
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
The Alpha-1 Foundation's and University of Florida's Alpha-1 Coded Testing (ACT) Study
The Alpha-1 Coded Testing (ACT) Study was established to study genetic testing and outcomes of individuals at risk for alpha-1 antitrypsin deficiency.
- Trial name
- ACT (NCT00500123)
- Testing
- Alpha-1 Antitrypsin Genotype
- Where
- United States
- Volunteers
- 50,000 people, healthy volunteers welcome
- Sponsor
- University of Florida
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
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
Alpha 1-Antitrypsin Deficiency
Study of DCR-A1AT in Healthy Adult Volunteers
This is a research study to test an experimental study drug (belcesiran, also known as DCR-A1AT). This drug is being tested to see if it helps people with a rare condition known as Alpha-1 Antitrypsin Deficiency, or A1ATD.
- Trial name
- NCT04174118
- Testing
- belcesiran
- Where
- 2 sites in New Zealand, Sweden
- Volunteers
- 30 people, healthy volunteers welcome
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
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
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
Alpha 1-Antitrypsin Deficiency
A Phase 1, First-in-human Study of VX-634
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-634 at various doses.
- Trial name
- NCT05579431
- Testing
- VX-634
- Where
- 5 sites in United States
- Volunteers
- 127 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
Chronic Liver Disease
An Observational Study Evaluating Patients With Chronic Liver Diseases Associated With Hepatic Steatosis
Hepatic steatosis may cause inflammation and fibrosis within the liver potentially leading to end-stage liver disease cirrhosis, liver failure and death.
- Trial name
- FALL (NCT05335603)
- Where
- Denmark
- Volunteers
- 335 people, healthy volunteers welcome
- Sponsor
- University of Copenhagen
Alpha-1 Antitrypsin Deficiency
A Phase 1, First-in-human Study of VX-668
The purpose of this study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of VX-668 at various doses.
- Trial name
- NCT05727800
- Testing
- VX-668
- Where
- 5 sites in United States
- Volunteers
- 114 people, healthy volunteers welcome
- Sponsor
- Vertex Pharmaceuticals Incorporated
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
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
COPD, Lung Function Decreased
Permian Health Women's Lung Health Study
The goal of this cross-sectional observational study is to estimate the prevalence of lung function impairment as measured by spirometry in a population of Gambian women aged 15 and older.
- Conditions
- COPD, Lung Function Decreased, Alpha 1-Antitrypsin
- Trial name
- WLHS (NCT06029543)
- Where
- The Gambia
- Volunteers
- 350 people, healthy volunteers welcome
- Sponsor
- Permian Health Lung Institute
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
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
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