Clinical trials: Alpha-1 Protease Inhibitor Deficiency
Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations
Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency
This is a Phase 2, multicenter, double-blind, randomized (1:1), placebo-controlled, 12-week, proof-of-concept study to evaluate the safety and tolerability as well as the mechanistic effect of oral administration of alvelestat…
- Conditions
- Alpha-1 Antitrypsin Deficiency (AATD), Pi*ZZ, Pi*SZ, Pi*Null, Another Rare Phenotype/Genotype Known to be Associated With Either Low or Functionally Impaired AAT Including F or I Mutations, Emphysema or COPD
- Trial name
- ATALANTa (NCT03679598)
- Testing
- Alvelestat (MPH966)
- Where
- 10 sites in United States
- Volunteers
- 63 people
- Sponsor
- University of Alabama at Birmingham
Alpha 1-Antitrypsin Deficiency
An Extension Study of Belcesiran in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATLD)
This is a Phase 2, multicenter, open-label extension of Study DCR-A1AT-201, designed to evaluate the long-term safety and further characterize the pharmacodynamics (PD) of belcesiran in adult patients with PiZZ AATLD.
- Trial name
- NCT05146882
- Testing
- Belcesiran
- Where
- New Zealand
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
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
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
Alpha 1 Antitrypsin Deficiency
Effect of Double Dose of Alpha 1-antitrypsin Augmentation Therapy on Lung Inflammation.
The current treatment of individuals with alpha-1 antitrypsin deficiency (AATD) who develop lung disease (COPD) is the administration of intravenous purified alpha-1 antitrypsin (augmentation therapy) at a fixed dose of 60 mg/kg…
- Trial name
- NCT01669421
- Testing
- Alpha-1 Antitrypsin (human)
- Where
- United States
- Volunteers
- 10 people
- Sponsor
- Michael Campos, MD
Alpha 1 Antitrypsin Deficiency, AATD
Genomic Research in Alpha-1 Antitrypsin Deficiency
This project is designed to examine the interaction between the microflora in the lower airway and the concentration of a serum protein called alpha-1 antitrypsin.
- Trial name
- GRADS Alpha-1 (NCT01832220)
- Where
- 9 sites in United States
- Volunteers
- 140 people
- Sponsor
- University of Pittsburgh
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
Type 1 Diabetes Mellitus
Study of Human Plasma-Derived Alpha1-Proteinase Inhibitor in Subjects With New-Onset Type 1 Diabetes Mellitus
This is a multicenter, randomized, partial-blinded, five-arm, placebo-controlled study of human plasma-derived alpha1-proteinase inhibitor (alpha1-PI) in children (ages 6-11 years old) and teens/adults (ages 12-35 years old) with…
- Trial name
- NCT02093221
- Testing
- 180 mg/kg Alpha1-PI, 90 mg/kg Alpha1-PI
- Where
- 36 sites in United States
- Volunteers
- 76 people
- Sponsor
- Grifols Therapeutics LLC
Alpha 1-Antitrypsin Deficiency
Safety, Tolerability and Pharmacodynamic Effect of Fazirsiran (TAK-999, ARO-AAT)
The purpose of AROAAT2001 (SEQUOIA) is to evaluate the safety, efficacy and tolerability of multiple doses of the investigational product, Fazirsiran Injection, administered subcutaneously to participants with alpha-1 antitrypsin…
- Trial name
- SEQUOIA (NCT03945292)
- Testing
- Fazisiran Injection
- Where
- 21 sites in United States, Germany and 4 more
- Volunteers
- 40 people
- Sponsor
- Arrowhead Pharmaceuticals
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
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
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
Hereditary Emphysema (Alpha 1-antitrypsin Deficiency)
Endoscopic Lung Volume Reduction in Patients With Advanced Emphysema Due to alpha1 Antitrypsin Deficiency
Patients with advanced heterogeneous emphysema due to alpha1 antitrypsin deficiency might benefit from endoscopic implantation of intrabronchial valves.
- Trial name
- NCT01357460
- Testing
- Implantation of intrabronchial valves (IBV) (Spiration IBV)
- Where
- Germany
- Volunteers
- 18 people
- Sponsor
- Heidelberg University
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
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
Alpha 1-Antitrypsin Deficiency
Alpha-1-Antitrypsin (AAT) To Treat Emphysema In AAT-Deficient Patients (EXACTLE)
The goal of this trial was to explore the utility of evaluating emphysema progression through CT scans measuring lung density during a 2 year period of weekly infusions of either placebo or human alpha-1-antitrypsin (AAT…
- Trial name
- NCT00263887
- Testing
- Alpha1-Proteinase Inhibitor (Human), Albumin (Human) 20%, United States Pharmacopeia (USP)
- Where
- 3 sites in Denmark, Sweden and 1 more
- Volunteers
- 77 people
- Sponsor
- Grifols Therapeutics LLC
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
Alpha-1 Antitrypsin Deficiency (AATD)
Effect Of Pulmonary Rehabilitation in Patients With Alpha-1 Antitrypsin Deficiency
This single-center, longitudinal, observational, prospective study aims to assess the applicability, adherence, and clinical impact of the Active Cycle of Breathing Technique (ACBT) with augmented reality support in patients with…
- Trial name
- RRALFA1 (NCT07242079)
- Testing
- Pulmonary Rehabilitation
- Where
- Italy
- Volunteers
- 50 people
- Sponsor
- University of Parma
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
Liver Diseases, Alagille Syndrome
Longitudinal Study of Genetic Causes of Intrahepatic Cholestasis (LOGIC)
Cholestasis is a condition in which bile is not properly transported from the liver to the small intestine. Cholestasis can be caused by an array of childhood diseases, including the genetic diseases Alagille syndrome (ALGS)…
- Conditions
- Liver Diseases, Alagille Syndrome, Alpha 1-Antitrypsin Deficiency
- Trial name
- LOGIC (NCT00571272)
- Where
- 17 sites in United States, Canada
- Volunteers
- 1,675 people
- Sponsor
- Arbor Research Collaborative for Health