Showing 20 of 59 recruiting trials for “alpha-delta-granule-deficiency”
RecruitingNCT06663358 ↗
A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients
Evaluate the Safety and Preliminary Efficacy of EXG110 in Subjects With Fabry Disease
👨⚕️ Jianhua Mao, PhD, Children's Hospital, Zhejiang University School of Medicine📍 2 sites📅 Started Oct 2024View details ↗
Study of the Quality of Life of Patients With Fabry Disease Aged 65 and Over With and Without Specific Treatment
👨⚕️ Wladimir MAUHIN, Doctor, Groupe Hospitalier Diaconesses Croix Saint-Simon📍 1 site📅 Started Oct 2024View details ↗
RecruitingNCT06512454 ↗
A Study in Adults to Learn About Inherited Alpha-1 Antitrypsin Deficiency (AATD) and AATD Related Liver Problems
A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD
Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema
A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)
Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease
👨⚕️ Arian Pano, MD, MPH, Clinical Development and Progam Lead, uniQure Biopharma, B.V.📍 8 sites📅 Started Jun 2024View details ↗
Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease
Study to Learn About the Safety of Fazirsiran and if it Can Help People With Alpha-1 Antitrypsin Liver Disease With Mild Liver Scarring (Fibrosis)
Evaluation of the Safety, Tolerability and Efficacy of a Gene Therapy Drug for the Treatment of Pediatric Fabry Disease
👨⚕️ Hong Xu, MD,PhD, Children's Hospital of Fudan University📍 1 site📅 Started Feb 2024View details ↗
A Study of KB408 for the Treatment of Alpha-1 Antitrypsin Deficiency
👨⚕️ David Sweet, MD, PhD, Director of Clinical Development📍 3 sites📅 Started Feb 2024View details ↗
Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease
German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa
Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease
T1 Mapping in Fabry Disease
Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease
RecruitingNCT06610019 ↗
Cardiovascular Multimodality Imaging Study
Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
Study to Check the Safety of Fazirsiran and Learn if Fazirsiran Can Help People With Liver Disease and Scarring (Fibrosis) Due to an Abnormal Version of Alpha-1 Antitrypsin Protein
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