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Alpha 1 Antitrypsin Deficiency Clinical Trials

A listing of Alpha 1 Antitrypsin Deficiency medical research trials actively recruiting patient volunteers. Search for closest city to find more detailed information on a research study in your area.


Found (8) clinical trials

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.


Alvelestat (MPH966) for the Treatment of ALpha-1 ANTitrypsin Deficiency

Alpha-1 antitrypsin deficiency (AATD) is the most common genetic cause of chronic obstructive pulmonary disease (COPD) and early-onset emphysema. AATD is characterized by low AAT levels; leading to excessive neutrophil elastase (NE) mediated lung destruction. Current treatment requires the periodic infusion of pooled AAT derived from human plasma, but this …


COPD Exacerbation Blood and Urine Biomarkers Study

Chronic obstructive pulmonary disease (COPD), a term describing emphysema and chronic bronchitis, is the third leading cause of death in the United States, with approximately 24 million US adults estimated to have the disease and over 130,000 US adults dying each year due to COPD. Acute exacerbations of chronic obstructive …

Phase N/A

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 deficient lung disease. Change in a number of different blood and sputum biomarkers related to lung damage, …


Safety Tolerability and Effect on Liver Histologic Parameters of ARO-AAT

In Part A, participants will be enrolled to receive multiple doses of ARO-AAT or placebo at varying dose levels. Once Part A is complete, a single dose level for Part B will be selected based on safety and pharmacodynamic parameters from Part A. Patients enrolled into Part A will roll …


Assessment of Changes in a Novel Histological Activity Scale in Response to ARO-AAT

Approximately 12 participants will be enrolled to receive multiple subcutaneous injections of ARO-AAT. All eligible participants will require a pre-dose biopsy completed as part of the study within the screening window. All participants will undergo an End of Study (EOS) biopsy. Treated participants will be offered the opportunity to continue …


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 subsequent infusions may be administered at home by the person responsible for care or by the patient. Clinical data on …

Phase N/A

EARCO REGISTRY. History Of Patients With Alpha-1 Antitrypsin

EARCO takes advantage of existing AATD registries that have been developed at the national level. Several countries have established registries in which AATD patients are included and followed-up with clinical and biological data collected. However, these registries differ in terms of inclusion criteria, data collected and frequency and extent of …

Phase N/A