View clinical trials related to Alpha 1-Antitrypsin Deficiency.
Filter by:This study will evaluate the efficacy, safety and pharmacokinetics (PK) of VX-814 in PiZZ subjects.
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. The Registry was established to facilitate research initiatives and promote the development of improved treatments and a cure for Alpha-1.
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.
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 deficiency (AATD).
This will be a prospective study examining serum levels of MMP-13 and alpha-1 antitrypsin as well as other biomarkers as well as urine biomarkers of smoking status and collagen degradation in the COPD patient population. Serum and urine biomarkers at baseline and after COPD exacerbations will be assessed against change in lung function as measured by pulmonary function testing.
This is an open-label, 2-part, dose-escalating, Phase 1 study of INBRX-101 (rhAAT-Fc). Part 1 will consist of single ascending dose (SAD) administration of INBRX-101 and Part 2 will consist of multiple ascending dose (MAD) administrations of INBRX-101. The planned dosing schedule is IV every 3 to 4 weeks.
This study is a Phase 2/3 prospective, double-blind, randomized, multi-center, placebo-controlled study for prevention of acute GVHD (aGVHD) in subjects undergoing an allogeneic hematopoietic cell transplant (HCT).
ADVM-043-03 is a long-term follow-up (LTFU) study of subjects who participated in the ADVM-043-01 multi-center gene therapy clinical study (ADVANCE) that evaluated ADVM-043 for the treatment of Alpha-1 Antitrypsin (A1AT) deficiency.
Pulmonary rehabilitation (PR) including exercise training is highly effective by improving health-related quality of life, exercise capacity and symptoms in patients with chronic obstructive pulmonary disease (COPD). Therefore, PR is a main component in the management of COPD. In a former study patients with Alpha-1 Antitrypsin deficiency (A1ATD)-related COPD (genotype PiZZ) have been found to show smaller improvements in exercise capacity after a 3-week inpatient PR program compared to COPD patients without A1ATD (genotype PiMM)[1]. These between-group differences were mirrored by missing adaptations of the fatigue-resistant skeletal muscle fibre type I in A1ATD patients. This was in contrast to COPD patients without A1ATD who increased the proportion of this fibre type after PR. Myofibre type I is crucial because it enables patients for physical endurance activities (walking, cycling etc.) during their daily life. The aim of this study is to compare the effects of an exercise Training program with high vs. moderate Training intensity in order to find a Training modality which improves Training effects in A1ATD patients.
The purpose of this study is to evaluate the safety and tolerability of single or multiple doses of ALN-AAT02. The study will be conducted in 2 sequential phases in which Part A will be a single-ascending dose (SAD) phase in healthy participants, and Part B will be a multiple-ascending dose (MAD) phase in participants with ZZ type alpha-1 antitrypsin deficiency (PiZZ) and biopsy-proven alpha-1 antitrypsin (AAT) deficiency-associated liver disease.