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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

🏥 Chiesi Farmaceutici S.p.A.📍 10 sites📅 Started Nov 2024View details ↗
NARecruitingNCT06539624

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 ↗
NARecruitingNCT07277361

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

👨‍⚕️ Study Director, Takeda📍 2 sites📅 Started Sep 2024View details ↗
Phase 1, PHASE2RecruitingNCT06405633

A Phase 1b/2a, Open-label Single Ascending Doses and Multiple Ascending Doses Study in Participants With Pi*ZZ AATD

👨‍⚕️ Cynthia Caracta, MD, Wave Life Sciences📍 9 sites📅 Started Jul 2024View details ↗
Phase 2RecruitingNCT05897424

Long-term, Open-label Study of SAR447537 (INBRX-101) in Adults With Alpha-1 Antitrypsin Deficiency Emphysema

👨‍⚕️ Clinical Sciences & Operations, Sanofi📍 40 sites📅 Started Jun 2024View details ↗
Phase 1, PHASE2RecruitingNCT06389877

A Study to Evaluate the Safety and Efficacy of BEAM-302 in Adult Patients With Alpha-1 Antitrypsin Deficiency (AATD)

👨‍⚕️ Medical Information, Beam Therapeutics📍 11 sites📅 Started Jun 2024View details ↗
Phase 1RecruitingNCT06270316

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 ↗
NARecruitingNCT06325488

Fibrosis, Inflammation, Oxygenation of Renal Tissue In FabrY Disease

👨‍⚕️ Caroline M Kistorp, Professor, Rigshospitalet, Denmark📍 1 site📅 Started Jun 2024View details ↗
Phase 3RecruitingNCT06165341

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)

👨‍⚕️ Study Director, Takeda📍 41 sites📅 Started Mar 2024View details ↗
EARLY_Phase 1RecruitingNCT06207552

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 ↗
Phase 1RecruitingNCT06049082

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 ↗
Phase 2RecruitingNCT06114329

Study of the Safety and Biologic Activity of AL01211 in Treatment Naive Males With Classic Fabry Disease

👨‍⚕️ Nan Chen, Dr., Ruijin Hospital📍 6 sites📅 Started Oct 2023View details ↗
NARecruitingNCT06095713

German Observational Multicenter Study of Patients With Fabry Disease Under Enzyme Replacement Therapy With Pegunigalsidase-alfa

👨‍⚕️ Eva Brand, MD, PhD, Universitätsklinikum Münster📍 8 sites📅 Started Oct 2023View details ↗
NARecruitingNCT05698901

Biomarkers and Cardiac Imaging Diagnostic Assay for Monitoring Patients With Fabry Disease

👨‍⚕️ Charles Jia-Yin Hou, Mackay Memorial Hospital📍 1 site📅 Started Sep 2023View details ↗
NARecruitingNCT05923788

T1 Mapping in Fabry Disease

👨‍⚕️ SANDRINE LEMOINE, PU-PH📍 2 sites📅 Started Aug 2023View details ↗
Phase 2, PHASE3RecruitingNCT05710692

Study to Evaluate the Safety, PK, PD, and Efficacy of PRX-102 in Japanese Patients With Fabry Disease

🏥 Chiesi Farmaceutici S.p.A.📍 10 sites📅 Started Aug 2023View details ↗
RecruitingNCT06610019

Cardiovascular Multimodality Imaging Study

👨‍⚕️ Leandro Slipczuk, MD, PhD, Montefiore Medical Center📍 1 site📅 Started May 2023View details ↗
Phase 3RecruitingNCT06081062

Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease

🏥 ISU Abxis Co., Ltd.📍 3 sites📅 Started Apr 2023View details ↗
Phase 3RecruitingNCT05677971

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

👨‍⚕️ Study Director, Takeda📍 89 sites📅 Started Mar 2023View details ↗
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