🧪 UPDATED DAILY FROM CLINICALTRIALS.GOVSynced Apr 1

Find Clinical Trials for Your Rare Disease

59+ recruiting trials across all rare diseases. Filter by phase, location, and condition.

Clinical trial records are synced from ClinicalTrials.gov.
Report missing data
Clear

Showing 20 of 59 recruiting trials for “alpha-delta-granule-deficiency

Phase 1RecruitingNCT07431112

A Study of AIR-001 in Adults With Alpha-1 Antitrypsin Deficiency (AATD)

👨‍⚕️ Clinical Information, AIRNA Corporation📍 2 sites📅 Started Feb 2026View details ↗
RecruitingNCT06906367

A Study of Patients With Fabry Disease (US Specific)

👨‍⚕️ Clinical Research, Amicus Therapeutics📍 7 sites📅 Started Feb 2026View details ↗
Phase 3RecruitingNCT06904261

A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants

🏥 Amicus Therapeutics📍 9 sites📅 Started Jan 2026View details ↗
RecruitingNCT07235709

Effect of Agalsidase Alfa on Cardiac Inflammation in Patients With Fabry Disease: A [18F]-FDG PET-CMR Study

🏥 Yonsei University📍 1 site📅 Started Nov 2025View details ↗
NARecruitingNCT07187440

A Study of Agalsidase Alfa Enyzme Replacement Therapy in Chinese Children and Adults With Fabry Disease

👨‍⚕️ Study Director, Takeda📍 18 sites📅 Started Nov 2025View details ↗
NAEnrolling by InvitationNCT07495410

Anderson-Fabry Disease Fitness Improvement Training: A-FAD-FIT

🏥 General University Hospital, Prague📍 1 site📅 Started Oct 2025View details ↗
Phase 4RecruitingNCT07135427

Genetic Variation in IgG in Alpha 1 Antitrypsin Deficiency

🏥 University of Alabama at Birmingham📍 1 site📅 Started Sep 2025View details ↗
RecruitingNCT07152834

Screening for Alpha-1 Antitrypsin Deficiency in Patients With Airway Obstruction

🏥 Muğla Sıtkı Koçman University📍 1 site📅 Started Aug 2025View details ↗
Phase 2, PHASE3RecruitingNCT06328608

A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease

🏥 Chiesi Farmaceutici S.p.A.📍 12 sites📅 Started Jul 2025View details ↗
NARecruitingNCT07382128

Myocardial Perfusion CMR for Differentiating and Characterizing Hypertrophic Cardiomyopathy Phenotypes

👨‍⚕️ Luigi Lovato📍 1 site📅 Started Jun 2025View details ↗
RecruitingNCT06505603

PiMZ Longitudinal Cohort (PiMZ Logic)

👨‍⚕️ Monica Goldklang, MD, Columbia University📍 5 sites📅 Started May 2025View details ↗
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

🏥 Chiesi Farmaceutici S.p.A.📍 5 sites📅 Started May 2025View details ↗
Phase 1RecruitingNCT06858397

A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease

👨‍⚕️ GC Biopharma📍 10 sites📅 Started May 2025View details ↗
NARecruitingNCT06776419

the Role of cArdiac Inflammation, endoThelial Dysfunction, and FIbrosis in fabrY Disease

👨‍⚕️ Caroline Kistorp, Professor, Rigshospitalet, Denmark📍 1 site📅 Started May 2025View details ↗
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

🏥 Amicus Therapeutics📍 1 site📅 Started Mar 2025View details ↗
Phase 1RecruitingNCT06996756

Gene Therapy for Alpha 1- Antitrypsin Deficiency

👨‍⚕️ Ronald G Crystal, MD, Weill Medical College of Cornell University📍 1 site📅 Started Feb 2025View details ↗
Phase 1RecruitingNCT06738017

Study of BMN 349 Single Dose in PiZZ and PiMZ/MASH Adult Participants

👨‍⚕️ Medical Director, MD, BioMarin Pharmaceutical📍 8 sites📅 Started Feb 2025View details ↗
NAEnrolling by InvitationNCT06892236

Preparation of IPSC for Cell Gene Editing for the Treatment of AATD

🏥 Fondazione IRCCS Policlinico San Matteo di Pavia📍 1 site📅 Started Jan 2025View details ↗
RecruitingNCT06884358

Functional Capacity in Anderson-Fabry Disease Patients

🏥 IRCCS Policlinico S. Donato📍 5 sites📅 Started Nov 2024View details ↗
Page 1 of 3Next →

Get alerts when new trials open

Create a free account to follow conditions and receive trial notifications.

Create free account →