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Condition: Lysosomal Diseases Synced from ClinicalTrials.gov · July 29, 2026

Recruiting studies: Lysosomal Diseases

Studies currently listed as recruiting on the public registry for this search term. Each page explains the study in plain language, with locations and the study team's contact details. Clinicians: generate a printable list for your patient →

DFT383 in Pediatric Participants With Nephropathic Cystinosis
Phase 1/Phase 2 · 4 location(s) · Novartis Pharmaceuticals · NCT06910813Natural History and Longitudinal Clinical Assessments in NCL / Batten Disease, the International DEM-CHILD Database
Phase N/A · 1 location(s) · Universitätsklinikum Hamburg-Eppendorf · NCT04613089Italian Anderson Fabry Disease Cardiovascular Registry
Phase N/A · 54 location(s) · IRCCS Azienda Ospedaliero-Universitaria di Bologna · NCT06806813A Gaucher Disease Gene Therapy Trial With FLT201
Phase 3 · 33 location(s) · Spur Therapeutics · NCT07223944Precision Diagnosis and Risk Stratification of Rare Cardiomyopathies Based on Novel Cardiac Magnetic Resonance Technique
Phase N/A · 1 location(s) · Chinese Academy of Medical Sciences, Fuwai Hospital · NCT07336394A Study of the Safety and Preliminary Efficacy of LY-M001 Injection in the Treatment of Adult Patients With Gaucher Dise
NA · 1 location(s) · He Huang · NCT06162338A Study Evaluating the Impact of Regular FODMAP-targeting Digestive Enzyme Blend Use on Gastrointestinal Symptoms in Ind
NA · 1 location(s) · Kiwi Health Inc · NCT07591584A Multi-cohort Study of Safety, Efficacy, PK and PD of GNR-055 in Patients With Mucopolysaccharidosis Type II
Phase 2/Phase 3 · 5 location(s) · AO GENERIUM · NCT05208281Phrenic Nerve and Diaphragm Electrophysiology in Pompe Disease
Phase N/A · 1 location(s) · IRCCS National Neurological Institute "C. Mondino" Foundatio · NCT07664930A Study to Determine the Efficacy and Safety of Tividenofusp Alfa (DNL310) vs Idursulfase in Pediatric and Young Adult P
Phase 2/Phase 3 · 32 location(s) · Denali Therapeutics Inc. · NCT05371613An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambrox
Phase 2/Phase 3 · 1 location(s) · Ozlem Goker-Alpan · NCT06614894Fabry Disease Registry & Pregnancy Sub-registry
Phase N/A · 284 location(s) · Genzyme, a Sanofi Company · NCT00196742Analysis of Velmanase Alfa (Lamzede®)'s Effects in the Body of Children With Alpha-Mannosidosis Under the Age 3
Phase N/A · 2 location(s) · Chiesi Farmaceutici S.p.A. · NCT06184503Natural History of Pompe Disease
Phase N/A · 1 location(s) · Assistance Publique - Hôpitaux de Paris · NCT03564561A Pilot Study for Systematic Neonatal Screening for Lysosomal Storage Diseases Using Tandem Mass Spectrometry
Phase N/A · 2 location(s) · University Hospital, Rouen · NCT04393701Natural History Study of Participants With Sanfilippo Syndrome Type IIIC
Phase N/A · 2 location(s) · Phoenix Nest · NCT05825131Evaluation of the Safety and Efficacy of Late-onset Pompe Disease Gene Therapy Drug
Phase 1/Phase 2 · 1 location(s) · GeneCradle Inc · NCT06391736Comparing Efficacy of 8-Week and 12-Week Faricimab Initial Follow-Up Treatment Intervals
Phase 4 · 1 location(s) · Faculty Hospital Kralovske Vinohrady · NCT06875245A Clinical Trial Evaluating the Safety and Efficacy of a New Light Combination Therapy Addressing Intermediate AMD
NA · 1 location(s) · Oculox Technologies SA · NCT06557369Natural History and Structural Functional Relationships in Fabry Renal Disease Treatment Outcomes(Changes)in Fabry Renal
Phase N/A · 2 location(s) · University of Minnesota · NCT01581424Evaluate the Safety and Efficacy of Fabagal® (Agalsidase Beta) in Patients With Fabry Disease
Phase 3 · 3 location(s) · ISU Abxis Co., Ltd. · NCT06081062A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset P
Phase 1 · 3 location(s) · Denali Therapeutics Inc. · NCT07354724Investigations of Juvenile Neuronal Ceroid Lipofuscinosis
Phase N/A · 1 location(s) · Eunice Kennedy Shriver National Institute of Child Health an · NCT03307304Longitudinal Study of Neurodegenerative Disorders
Phase N/A · 1 location(s) · University of Pittsburgh · NCT03333200Non-invasive Functional Assessment and Pathogenesis of Morquio A
Phase N/A · 1 location(s) · Nemours Children's Clinic · NCT05284006Decoding the Genetic Landscape of Skeletal Diseases
Phase N/A · 1 location(s) · Karolinska Institutet · NCT05876416Use of Cysteamine in the Treatment of Cystinosis
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT00359684A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease
Phase 2/Phase 3 · 12 location(s) · Chiesi Farmaceutici S.p.A. · NCT06328608A Study of Replagal in Children and Adults With Fabry Disease in India
Phase 4 · 3 location(s) · Shire · NCT05067868Natural History of Glycosphingolipid Storage Disorders and Glycoprotein Disorders
Phase N/A · 1 location(s) · National Human Genome Research Institute (NHGRI) · NCT00029965
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This page is independently generated by Eichor from the public ClinicalTrials.gov record and re-synced daily. It is not the sponsor's official website unless claimed. Nothing here is medical advice; eligibility is always determined by the study team — talk to your own doctor first.