Showing 20 of 59 recruiting trials for “alpha-delta-granule-deficiency”
A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)
RecruitingNCT06906367 ↗
A Study of Patients With Fabry Disease (US Specific)
A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants
RecruitingNCT07235709 ↗
Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study
A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease
Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT
Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency
RecruitingNCT07152834 ↗
Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction
A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes
RecruitingNCT06505603 ↗
PiMZ Longitudinal Cohort (PiMZ Logic)
RecruitingNCT06941025 ↗
Maternal and Postnatal Outcomes Study (MOS): A Global Observational Registry Assessing the Safety of Elfabrio® in Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease
the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease
Enrolling by InvitationNCT06880250 ↗
Efficacy and Safety of Enzyme Replacement Therapy in Patients With Fabry Disease
👨⚕️ Sergey V. Moiseev, MD, Sechenov First Moscow State Medical University (Sechenov University)📍 23 sites📅 Started Mar 2025View details ↗
RecruitingNCT04252066 ↗
A Global Prospective Observational Study of Women With Fabry Disease and Their Infants During Pregnancy and Breastfeeding
Gene Therapy for Alpha 1- Antitrypsin Deficiency
👨⚕️ Ronald G Crystal, MD, Weill Medical College of Cornell University📍 1 site📅 Started Feb 2025View details ↗
Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants
Preparation of IPSC for Cell Gene Editing for the Treatment of AATD
RecruitingNCT06884358 ↗
Functional Capacity in Anderson-Fabry Disease Patients
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