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Clinical trial

A First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency a Genetically Linked Orphan Disease

NCT03679598 is registered in ClinicalTrials.gov with University of Alabama at Birmingham as lead sponsor.

NCT NCT03679598
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Registry record

As filed in ClinicalTrials.gov.

Title
A First in Class Disease Modifying Therapy to Treat Alpha-1 Antitrypsin Deficiency a Genetically Linked Orphan Disease
Lead sponsor
University of Alabama at Birmingham
Phase
Phase 2
Overall status
Completed
Start date
April 8, 2019
Completion date
December 1, 2023
Enrollment
63 (actual)
Sites
10

Conditions

As the registry lists them; a condition with its own page is linked.

alpha-1 antitrypsin deficiency (aatd), emphysema or copd, pi*zz, pi*sz, pi*null, another rare phenotype/genotype known to be associated with either low or functionally impaired aat including f or i mutations