Clinical trials: Alpha-1 Proteinase Inhibitor
Alpha 1-Antitrypsin Deficiency
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
The Sponsor is developing KB408, a replication-defective, non-integrating herpes simplex virus type 1 (HSV-1)-derived vector engineered to deliver functional full-length human SERPINA1 to the airways of people with alpha-1…
- Trial name
- Serpentine-1 (NCT06049082)
- Testing
- KB408 (Nebulization)
- Where
- 3 sites in United States
- Volunteers
- 15 people
- Sponsor
- Krystal Biotech, Inc.
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
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
ARALAST NP Alpha-1 Lung Density Chronic Obstructive Pulmonary Disease-Emphysema (COPD-E) Study
The purpose of this study is to evaluate the efficacy of ARALAST NP A1PI augmentation therapy 120 milligrams per kilogram (mg/kg) body weight (BW)/week compared with an external placebo comparator on the loss of emphysematous…
- Trial name
- NCT04440488
- Testing
- ARALAST NP
- Sponsor
- Shire
Cystic Fibrosis
Safety and Tolerability Trial of Inhaled Alpha1-Proteinase Inhibitor (Human), Hydrophobic Chromatography Process (Alpha-1 HC) in Subjects With Cystic Fibrosis
This was a randomized, double-blind, placebo-controlled, dose escalation study to assess the safety and tolerability of 100 mg and 200 mg of inhaled Alpha-1 HC administered once a day for three weeks in subjects aged 18 years and…
- Trial name
- NCT01684410
- Testing
- Alpha-1 HC 100 mg, Alpha-1 HC 200 mg
- Where
- 6 sites in United States
- Volunteers
- 30 people
- Sponsor
- Grifols Therapeutics LLC
Cystic Fibrosis
Phase II Study of the Safety and Efficacy of Inhaled Alpha-1 Antitrypsin (AAT ) in Cystic Fibrosis Patients
Cystic Fibrosis (CF) is an inherited disorder in which mucus-secreting glands in the lungs produce considerable quantity of thick, sticky secretions that clog the airways, promote bacterial growth and lead to chronic obstruction…
- Trial name
- NCT00499837
- Testing
- Aerosolized, human, plasma-derived Alpha-1 Antitrypsin
- Where
- Israel
- Volunteers
- 21 people
- Sponsor
- Kamada, Ltd.
Alpha-1 Antitrypsin Deficiency
Safety & Efficacy Study of rAAV1-CB-hAAT for Alpha-1 Antitrypsin Deficiency
Assessment of the safety and efficacy of intramuscular (IM) administration of a recombinant adenoassociated virus (rAAV) alpha-1 antitrypsin (AAT) vector (rAAV1-CB-hAAT) in AAT-deficient adults at three dosage levels [6.0 ×…
- Trial name
- NCT01054339
- Testing
- rAAV1-CB-hAAT
- Where
- 4 sites in United States, Ireland
- Volunteers
- 9 people
- Sponsor
- Beacon Therapeutics
Alpha 1-Antitrypsin Deficiency, Emphysema
Study of SAR447537 (INBRX-101) Compared to Plasma-derived A1PI Therapy in Adults With AATD Emphysema
Phase 2 study to compare SAR447537 (INBRX-101) to plasma derived A1PI therapy in adults with AATD emphysema
- Trial name
- ELEVAATE (NCT05856331)
- Testing
- SAR447537, Zemaira
- Where
- 65 sites in United States, Australia and 6 more
- Volunteers
- 99 people
- Sponsor
- Sanofi
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
Alpha1-antitrypsin Deficiency
GLASSIA Safety, Immunogenicity, and Bronchoalveolar Lavage Study
The purpose of the study is 2-fold: (1) to evaluate the safety and potential immunogenicity of GLASSIA following intravenous (IV) administration via in-line filtration; and (2) to assess the effects of GLASSIA augmentation…
- Trial name
- NCT02525861
- Testing
- GLASSIA
- Where
- 18 sites in United States, Canada
- Volunteers
- 34 people
- Sponsor
- Baxalta now part of Shire
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
Safety, Tolerability and Effect of ARC-AAT Injection on Circulating and Intrahepatic Alpha-1 Antitrypsin Levels
Patients with Alpha-1 Antitrypsin Deficiency (AATD) will be enrolled to receive multiple doses of ARC-AAT Injection. All subjects will require a pre-dose biopsy and a biopsy post last dose.
- Trial name
- NCT02900183
- Testing
- ARC-AAT Injection
- Where
- 4 sites in Canada, Ireland and 2 more
- Sponsor
- Arrowhead Pharmaceuticals
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
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
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
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
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
A Study of Belcesiran in Patients With AATLD
This is a multiple dose, randomized, placebo-controlled, double-blind study of belcesiran to evaluate the safety, tolerability, PK, and PD in adult patients with PiZZ AATD-associated liver disease (AATLD).
- Trial name
- ESTRELLA (NCT04764448)
- Testing
- Belcesiran
- Where
- 23 sites in United States, Australia and 12 more
- Volunteers
- 16 people
- Sponsor
- Dicerna Pharmaceuticals, Inc., a Novo Nordisk company
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
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
Graft Versus Host Disease
A Phase 2/3 Study of GLASSIA for the Treatment of Acute GvHD
The purpose of the study is to evaluate the safety and efficacy of GLASSIA as an add-on biopharmacotherapy to standard-of-care steroid treatment as the first-line treatment in participants with acute GvHD with lower GI…
- Trial name
- NCT02956122
- Testing
- GLASSIA, methylprednisolone or equivalent steroid, Albumin
- Where
- United States
- Volunteers
- 1 people
- Sponsor
- Baxalta now part of Shire