Clinical trials: Alpha-1 Protease Inhibitor Deficiency
Alpha1-Antitrypsin Deficiency
Safety and Pharmacokinetics of Alpha-1 MP (Alpha1-proteinase Inhibitor (Human), Modified Process) in Participants With Alpha1-Antitrypsin Deficiency
This study is a multicenter, open-label trial to evaluate the safety and pharmacokinetics of weekly intravenous infusions of 60 mg/kg of Alpha-1 MP (alpha1-proteinase inhibitor (human), modified process) for 8 weeks.
- Trial name
- NCT02870309
- Testing
- Alpha-1 MP
- Where
- 2 sites in Japan
- Volunteers
- 4 people
- Sponsor
- Grifols Therapeutics LLC
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
Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease
Early Access Program Using Alpha 1 Antitrypsin Infusion for Patients With Steroid Refractory Acute GvHD After Hematopoietic Stem Cell Transplantation (HSCT)
An Early Access Program for patients with steroid refractory acute GvHD after hematopoietic stem cell transplantation. This Program is available for female and male who are recipients of allogenic HSCT and who have been newly…
- Conditions
- Acute-graft-versus-host Disease, Steroid Refractory Acute Graft Versus Host Disease, Graft-versus-host-disease, Graft Vs Host Disease
- Trial name
- NCT03172455
- Testing
- Glassia
- Sponsor
- Impatients N.V. trading as myTomorrows
Alpha₁-Antitrypsin Deficiency
A Study to Assess Safety and PK of Liquid Alpha₁-Proteinase Inhibitor (Human) in Treating Alpha₁-Antitrypsin Deficiency
Grifols Therapeutics Inc. conducted a multi-center, randomized, double-blind, crossover study to evaluate the safety, immunogenicity, and pharmacokinetics (PK) of Liquid Alpha₁-PI compared to the currently licensed product…
- Trial name
- NCT02282527
- Testing
- Liquid Alpha₁-PI, Prolastin-C
- Where
- 6 sites in United States
- Volunteers
- 32 people
- Sponsor
- Grifols Therapeutics LLC
Alpha-1 Antitrypsin Deficiency
Respreeza® Self-administration and Learning Program (AmAREtTI Study)
According to the Respreeza® Summary of Product Characteristics, the initial infusions must be administered under the supervision of a health professional experienced in the treatment of alpha-1 antitrypsin deficiency, although…
- Trial name
- AmAREtTI (NCT04262284)
- Where
- 11 sites in France
- Volunteers
- 15 people
- Sponsor
- CSL Behring
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
Emphysema, Alpha 1-proteinase Inhibitor Deficiency
Extension Study of Zemaira® i.v. Administration in Subjects With Emphysema Due to alpha1-proteinase Inhibitor Deficiency.
This study is a continuation of the placebo-controlled study CE1226_4001 (NCT00261833) to evaluate the efficacy and safety of Zemaira® intravenous (i.v).
- Trial name
- NCT00670007
- Testing
- Alpha1- proteinase inhibitor [human]
- Where
- 21 sites in Australia, Canada and 9 more
- Volunteers
- 140 people
- Sponsor
- CSL Behring
Alpha 1-Antitrypsin Deficiency
Phase III, Efficacy and Safety of "Kamada-AAT for Inhalation"
The goal of this clinical trial is to learn if AAT for inhalation, at a dose of 80 mg/day can slow the progression of lung disease in people who have lung disease caused by severe genetic deficiency in Alpha 1 Antitrypsin (AATD).
- Trial name
- InnovAATe (NCT04204252)
- Testing
- Alpha 1-Antitrypsin
- Where
- 9 sites in Belgium, Finland and 4 more
- Volunteers
- 98 people
- Sponsor
- Kamada, Ltd.
Alpha 1-Antitrypsin Deficiency
Pharmacokinetic Study of ARALAST (Human Alpha1- PI)
The primary purpose of this study is to characterize the pharmacokinetic profile of intravenous Aralast Fraction (Fr.) IV-1, a sterile, stable, lyophilized preparation of functionally intact human Alpha1- Proteinase Inhibitor…
- Trial name
- NCT00242385
- Testing
- Dose of 60 mg/kg Fraction IV-1 Alpha1-Proteinase Inhibitor, Dose of 60 mg/kg alpha1-proteinase inhibitor
- Where
- 7 sites in Australia, New Zealand
- Volunteers
- 25 people
- Sponsor
- Baxalta now part of Shire
Alpha1-Antitrypsin Deficiency
Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)
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 the AAT protein, called Z-AAT.
- Trial name
- NCT06165341
- Testing
- Fazirsiran Injection
- Where
- 41 sites in United States, Austria and 11 more
- Volunteers
- 50 people
- Sponsor
- Takeda
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
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
Study of Fazirsiran (TAK-999, ARO-AAT) in Patients With Alpha-1 Antitrypsin Deficiency Associated Liver Disease (AATD)
The purpose of this study is to evaluate the the safety and efficacy of the investigational product, fazirsiran (TAK-999, ARO-AAT), administered subcutaneously to patients with alpha-1 antitrypsin deficiency associated liver…
- Trial name
- NCT03946449
- Testing
- ARO-AAT
- Where
- 4 sites in Austria, Germany and 1 more
- Volunteers
- 16 people
- Sponsor
- Arrowhead Pharmaceuticals
Alpha1-proteinase Inhibitor Deficiency, Emphysema
Zemaira in Subjects With Emphysema Due to Alpha1-Proteinase Inhibitor Deficiency
This is a randomized, placebo-controlled, double-blind, multicenter phase III/IV study to compare the efficacy and safety of Zemaira® with placebo in subjects with emphysema due to alpha1-proteinase inhibitor deficiency.
- Trial name
- NCT00261833
- Testing
- Alpha1-proteinase inhibitor
- Where
- 28 sites in United States, Australia and 11 more
- Volunteers
- 180 people
- Sponsor
- CSL Behring
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
Evaluation of the Efficacy and Safety of VX-864 in Subjects With the PiZZ Genotype
This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-864 in PiZZ subjects.
- Trial name
- NCT04474197
- Testing
- VX-864
- Where
- 30 sites in United States, Canada and 4 more
- Volunteers
- 44 people
- Sponsor
- Vertex Pharmaceuticals Incorporated
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.
Pulmonary Emphysema in Alpha-1 PI Deficiency
Efficacy and Safety of Alpha1-Proteinase Inhibitor (Human), Modified Process (Alpha-1 MP) in Subjects With Pulmonary Emphysema Due to Alpha1 Antitrypsin Deficiency (AATD)
This is a multi-center, randomized, placebo-controlled, double blind clinical study to assess the efficacy and safety of two separate dose regimens of Alpha-1 MP versus placebo for 156 weeks (i.e., 3 years) using computed…
- Trial name
- SPARTA (NCT01983241)
- Testing
- Alpha-1 MP, 0.9% Sodium Chloride for Injection, USP
- Where
- 52 sites in United States, Argentina and 15 more
- Volunteers
- 345 people
- Sponsor
- Grifols Therapeutics LLC
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, Emphysema
A 12-week Study Treating Participants Who Have alpha1-antitrypsin-related COPD With Alvelestat (MPH966) or Placebo.
The purpose of this study is to investigate the effect of alvelestat (an oral neutrophil elastase inhibitor) on blood and sputum biomarkers in patients with PiZZ, null or rare variant phenotype/genotype alpha-1 anti-trypsin…
- Conditions
- Alpha 1-Antitrypsin Deficiency, Emphysema, COPD
- Trial name
- ASTRAEUS (NCT03636347)
- Testing
- Alvelestat oral tablet - dose 1, Alvelestat oral tablet - dose 2
- Where
- 26 sites in United States, Belgium and 6 more
- Volunteers
- 99 people
- Sponsor
- Mereo BioPharma