Clinical trials: Alpha-1 Protease Inhibitor Deficiency
- Condition Alpha-1 Protease Inhibitor Deficiency
- Funder type Industry (pharma, biotech, devices)
- Has posted results yes
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 (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.
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
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
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
Chronic Obstructive Pulmonary Disease, Alpha1-antitrypsin Deficiency
Stage 1 Study of ARALAST NP and GLASSIA in A1PI Deficiency
The purpose of this study is to conduct a pilot study to evaluate the safety and efficacy of weekly administration of Alpha1-Proteinase Inhibitor (A1PI) augmentation therapy in subjects with A1PI deficiency and emphysema/ chronic…
- Trial name
- NCT02722304
- Testing
- ARALAST NP 60 mg/kg, ARALAST NP 120 mg/kg, GLASSIA 60 mg/kg
- Where
- 17 sites in United States, Australia and 1 more
- Volunteers
- 7 people
- Sponsor
- Baxalta now part of Shire
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
Alpha1-Antitrypsin Deficiency
A Study to Evaluate Safety, Tolerability and Pharmacokinetics of Two Different Doses of Alpha1-Proteinase Inhibitor Subcutaneous (Human) 15% in Participants With Alpha1-Antitrypsin Deficiency
The purpose of this study is to evaluate the safety and tolerability of 72 milligrams per kilogram (mg/kg) and 180 mg/kg Alpha-1 15%, administered as a single-dose subcutaneous (SC) infusion and subsequently as weekly SC…
- Trial name
- NCT04722887
- Testing
- Alpha-1 15%, Liquid Alpha1-Proteinase Inhibitor (Human)
- Where
- 5 sites in United States
- Volunteers
- 17 people
- Sponsor
- Grifols Therapeutics LLC
Alpha1-antitrypsin Deficiency
Aralast alpha1-proteinase Inhibitor Surveillance Study
The primary objectives of this Phase 4, open label, prospective U.S. surveillance study are to evaluate the health outcomes of Alpha 1-Antitrypsin (AAT)-deficient subjects who are initiating treatment with ARALAST on…
- Trial name
- NCT00313144
- Testing
- ARALAST Alpha1-Proteinase Inhibitor
- Where
- United States
- Volunteers
- 127 people
- Sponsor
- Baxalta now part of Shire
Alpha 1-Antitrypsin Deficiency
Comparison of Pharmacokinetic, Safety, Tolerability of Alpha-1 MP and Prolastin In Alpha1-antitrypsin Deficient Adults
The purpose of this clinical study (ChAMP - Comparability pharmacokinetics of Alpha-1 Modified Process) is to compare the pharmacokinetic, safety and tolerability of Alpha-1 Proteinase Inhibitor (Human), modified process (Alpha-1…
- Trial name
- ChAMP (NCT00295061)
- Testing
- Alpha-1 MP, alpha-1 proteinase inhibitor (human)
- Where
- 8 sites in United States
- Volunteers
- 24 people
- Sponsor
- Grifols Therapeutics LLC
Alpha1-Antitrypsin Deficiency
Long-term Safety of Alpha1-Proteinase Inhibitor (Human) in Japanese Subjects With Alpha1 Antitrypsin Deficiency (GTI1401-OLE)
This is a multi-center, open-label study to evaluate the long-term safety of weekly intravenous (IV) infusions of 60 mg/kg alpha1-PI (human), modified process (Alpha-1 MP) in adult participants with Alpha1 Antitrypsin Deficiency…
- Trial name
- NCT02870348
- Testing
- Alpha-1 MP
- Where
- 3 sites in Japan
- Volunteers
- 4 people
- Sponsor
- Grifols Therapeutics LLC
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
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
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
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
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
Emphysema, Alpha 1-antitrypsin Deficiency (AATD)
Safety and Pharmacokinetics of Alpha-1 Proteinase Inhibitor in Subjects With Alpha1-Antitrypsin Deficiency
This is a study to assess the safety and pharmacokinetics of weekly infusions of 120 mg/kg of Prolastin-C (alpha1-proteinase inhibitor [alpha1-PI] [Human]), compared to weekly infusions of 60 mg/kg of Prolastin-C in patients with…
- Trial name
- SPARK (NCT01213043)
- Testing
- Prolastin-C, 60 mg/kg, Prolastin-C, 120 mg/kg
- Where
- 5 sites in United States
- Volunteers
- 30 people
- Sponsor
- Grifols Therapeutics LLC
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
Alpha-1 Antitrypsin Deficiency
Phase II, Safety and Efficacy Study of Kamada-alpha-1-antitrypsin (AAT) for Inhalation"
To evaluate different doses of "Kamada-AAT for Inhalation" on the levels of alpha 1-proteinase inhibitor and other analytes in epithelial lining fluid (ELF) and serum and to assess the safety of the treatment in subjects with AAT…
- Trial name
- NCT02001688
- Testing
- Kamada-AAT for Inhalation, 80mg, Kamada-AAT for Inhalation, 160mg
- Where
- 2 sites in United States
- Volunteers
- 36 people
- Sponsor
- Kamada, Ltd.
Alpha-1 Antitrypsin Deficiency
Long-Term Follow-up Study of ADVM-043
ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 (NCT02168686) multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin…
- Trial name
- NCT03804021
- Testing
- No Intervention
- Where
- 2 sites in United States
- Volunteers
- 6 people
- Sponsor
- Adverum Biotechnologies, Inc.