Clinical trials: Alpha 1 Antitrypsin Deficiency, active
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)
Alpha 1 Antitrypsin Deficiency
A Non-inferiority Pharmacokinetic and Safety/Tolerability Study of Two Different Doses of Weekly SC Alpha1-PI 15% Compared With Corresponding Standard IV Alpha1-PI in Participants With Alpha1-Antitrypsin Deficiency (AATD)
This study is designed to compare two different weekly doses of a medicine called Alpha1-Proteinase Inhibitor given by injection under the skin with the standard doses of the same medicine given through a vein.
- Trial name
- SWIFT-SC (NCT07555483)
- Testing
- Alpha-1 15%, Liquid Alpha1-PI
- Where
- 22 sites in United States, Denmark and 7 more
- Volunteers
- 40 people
- Sponsor
- Grifols Therapeutics LLC
Alpha 1 Antitrypsin Deficiency
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1, open-label, single ascending dose (SAD) and multiple dose (MD) study of AIR-001 in participants with alpha-1 antitrypsin deficiency (AATD) due to PiZZ genotype.
- Trial name
- RepAIR1 (NCT07431112)
- Testing
- AIR-001 SAD dose level 1, AIR-001 SAD dose level 2, AIR-001 SAD dose level 3
- Where
- 13 sites in Australia, Georgia and 7 more
- Volunteers
- 54 people
- Sponsor
- AIRNA Corporation
Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease
A Study of the Safety and Efficacy of Prime Editing (PM647a) in Participants With Alpha-1 Antitrypsin Deficiency
The purpose of this study is to evaluate the safety, tolerability, biological activity, and initial efficacy of PM647a, an investigational Prime Editing therapy, in adults and adolescents with Alpha-1 Antitrypsin Deficiency…
- Conditions
- Alpha-1 Antitrypsin Deficiency, Alpha-1 Antitrypsin Deficiency-associated Lung Disease, Alpha-1 Antitrypsin Deficiency (AATD), AATD
- Trial name
- PM647a (NCT07887347)
- Testing
- PM647a
- Volunteers
- 54 people
- Sponsor
- Prime Medicine, Inc.
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.
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
Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency
Phase 4, Double-blind Study Evaluating the Response on Computed Tomography (CT) Lung Density Decline Rates of Respreeza / Zemaira Weekly for 3 Years in Adults With alpha1 Antitrypsin Deficiency (AATD)
This is a multicenter, parallel-group, double-blind, randomized phase 4 study designed to identify the optimal dose of CE1226 (2 active doses) to slow disease progression as assessed by reduced rates of annual lung density…
- Conditions
- Alpha1 Antitrypsin Deficiency, Alpha1-Proteinase Inhibitor Deficiency, Emphysema
- Trial name
- NCT07326592
- Testing
- CE1226
- Where
- 2 sites in Denmark
- Volunteers
- 270 people
- Sponsor
- CSL Behring
Alpha 1-Antitrypsin Deficiency
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1/2, multicenter, open-label study conducted in 3 parts (Part A, Part B, and Part C) to evaluate the safety, tolerability, PK/PD, and efficacy of BEAM-302 in adult patients with AATD-associated lung disease and/or…
- Trial name
- NCT06389877
- Testing
- BEAM-302
- Where
- 12 sites in United States, Australia and 4 more
- Volunteers
- 192 people
- Sponsor
- Beam Therapeutics Inc.
Alpha-1 Antitrypsin Deficiency (AATD)
A Trial Evaluating the Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of CTX460 in Adults With Emphysema Associated With Severe Alpha-1 Antitrypsin Deficiency (AATD)
A Study of CTX460 in Adults with Emphysema Associated with Severe Alpha-1 Antitrypsin Deficiency (AATD).
- Trial name
- NCT07850076
- Testing
- CTX460
- Volunteers
- 68 people
- Sponsor
- CRISPR Therapeutics
Alpha-1 Antitrypsin Deficiency (AATD)
A Study of TSRA-196 in Adults With PiZZ Alpha-1 Antitrypsin Deficiency (AATD)
This is a Phase 1/2, open-label, multi-center, dose escalation (Part 1), dose expansion (Part 2), and single repeat dose (Part 3) study to evaluate the safety, tolerability, efficacy, and PK/PD parameters of TSRA-196 in adults…
- Trial name
- NCT07227207
- Testing
- TSRA-196
- Where
- 8 sites in United States, Australia and 1 more
- Volunteers
- 72 people
- Sponsor
- Tessera Therapeutics, Inc.
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
Alpha1-Antitrypsin Deficiency
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
The liver produces a protein called alpha-1 antitrypsin (AAT). AAT is normally released into the bloodstream. In some people, the liver makes an abnormal version of AAT, called Z-AAT.
- Trial name
- ALPHATUDE (NCT06512454)
- Testing
- No Intervention
- Where
- 8 sites in United States, Austria and 4 more
- Volunteers
- 500 people
- Sponsor
- Takeda
Alpha-1-antitrypsin Deficiency, Emphysema
Development of a Prediction Score for the Occurrence of Death or Lung Transplantation in Patients With Emphysema Secondary to Alpha-1-anti-tripsin Deficiency
Emphysema linked to alpha-1-antitrypsin deficiency (DAAT): towards a better prediction of risks Emphysema caused by alpha-1-antitrypsin deficiency (DAAT) is a rare genetic disorder that can lead to serious complications, such as…
- Conditions
- Alpha-1-antitrypsin Deficiency, Emphysema, Artificial Intelligence (AI), Predictive Learning Models
- Trial name
- FLARE (NCT07715617)
- Where
- 4 sites in France
- Volunteers
- 230 people
- Sponsor
- University Hospital, Bordeaux
Irritable Bowel Syndrome (IBS)
IBS-Oriented CDED Versus Low FODMAP Diet for Irritable Bowel Syndrome
Background: Irritable Bowel Syndrome (IBS) affects approximately 4.1% of the global population and imposes a significant personal and healthcare burden.
- Trial name
- IBS-CDED (NCT07707635)
- Testing
- Modified Crohn's Disease Exclusion Diet (CDED), Low FODMAP Diet
- Volunteers
- 150 people
- Sponsor
- Ariel University
Pulmonary Emphysema in Alpha-1 Antitrypsin Deficiency
Long Term Safety of Alpha1-Proteinase Inhibitor in Subjects With Alpha1 Antitrypsin Deficiency
This is a 2-year open-label, multicenter extension of the double-blind, placebo-controlled GTi1201 study. The purpose of this study is to obtain an additional 2 years of safety data for intravenously administered Alpha1-MP 60…
- Trial name
- SPARTA-OLE (NCT02796937)
- Testing
- Alpha-1 MP
- Where
- 28 sites in United States, Australia and 10 more
- Volunteers
- 290 people
- Sponsor
- Grifols Therapeutics LLC
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
Alpha1-Antitrypsin Deficiency
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
The main aim of this study is to learn if fazirsiran reduces liver scarring (fibrosis) compared to placebo. Other aims are to learn if fazirsiran slows down the disease worsening in the liver, to get information on how fazirsiran…
- Trial name
- NCT05677971
- Testing
- Fazirsiran Injection
- Where
- 89 sites in United States, Australia and 17 more
- Volunteers
- 160 people
- Sponsor
- Takeda
Alpha 1-Antitrypsin Deficiency
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
The goal of this clinical trial is to assess the safety and tolerability of a single oral dose of BMN 349 in participants with PiZZ or PiMZ/MASH.
- Trial name
- NCT06738017
- Testing
- BMN 349
- Where
- 7 sites in United States, United Kingdom
- Volunteers
- 6 people
- Sponsor
- BioMarin Pharmaceutical
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
Alpha 1-Antitrypsin Deficiency, Emphysema
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
Phase 2 open label extension study to evaluate SAR447537 (INBRX-101) in adults with AATD emphysema
- Trial name
- ELEVAATE OLE (NCT05897424)
- Testing
- SAR447537
- Where
- 40 sites in United States, Australia and 7 more
- Volunteers
- 185 people
- Sponsor
- Sanofi