| NCT # (link) |
Study Title | Status | Conditions | Intervention | Sponsors | Study Type |
|---|---|---|---|---|---|---|
| NCT07054515 AZA-001-301 | A Study to Evaluate the Safety and Efficacy of Oral Nizubaglustat (AZ-3102) in Late-infantile and Juvenile Forms of Niemann-Pick Type C Disease, GM1 Gangliosidosis or GM2 Gangliosidosis | RECRUITING | Niemann-Pick Type C Disease GM1 Gangliosidosis GM2 Gangliosidosis | DRUG: AZ-3102 DRUG: Placebo | Azafaros B.V. | INTERVENTIONAL |
| NCT06858397 GC1134A_FD_P1201 | A proof-of Concept Study to Assess Safety and Tolerability of HM15421/GC1134A in Patients With Fabry Disease | RECRUITING | Fabry Disease | DRUG: HM15421/GC1134A | GC Biopharma Corp | INTERVENTIONAL |
| NCT06904261 AT1001-033 | A Study of Migalastat in Pediatric Subjects (2 to <12 Yrs) With Fabry Disease and Amenable GLA Variants | RECRUITING | Fabry Disease | DRUG: Migalastat HCl 20 mg | Amicus Therapeutics | INTERVENTIONAL |
| NCT06270316 CT-AMT-191-01 | Safety, PK/PD, and Exploratory Efficacy Study of AMT-191 in Classic Fabry Disease | RECRUITING | Fabry Disease | DRUG: AMT-191 | UniQure Biopharma B.V. | INTERVENTIONAL |
| NCT06328608 CLI-06657AA1-01 | A Study to Learn About the Safety and Effects of the Study Drug PRX-102 in Children and Adolescents With Fabry Disease | RECRUITING | Fabry Disease | DRUG: PRX-102 1 mg/kg every two weeks | Chiesi Farmaceutici S.p.A. | INTERVENTIONAL |
| NCT07223944 FLT201-03 | A Gaucher Disease Gene Therapy Trial With FLT201 | RECRUITING | Gaucher Disease Type 1 | GENETIC: FLT201 | Spur Therapeutics | INTERVENTIONAL |
| NCT07123155 2405N1221 | Study of S-606001 as an Add-on to Enzyme Replacement Therapy (ERT) in Participants With Late-onset Pompe Disease (LOPD) | RECRUITING | Pompe Disease | DRUG: S-606001 DRUG: Placebo | Shionogi | INTERVENTIONAL |
| NCT07354724 DNLI-J-0001 | A Study to Evaluate the Safety, Pharmacokinetics, and Pharmacodynamics of DNL952 in Adult Participants With Late-Onset Pompe Disease | RECRUITING | Late-onset Pompe Disease | DRUG: DNL952 | Denali Therapeutics Inc. | INTERVENTIONAL |
| NCT07640984 JR-446-201 | A Phase I/II Trial of JR-446 in Mucopolysaccharidosis Type IIIB (MPS IIIB) | RECRUITING | Mucopolysaccharidosis IIIB | DRUG: JR-446 | JCR Pharmaceuticals Co., Ltd. | INTERVENTIONAL |
| NCT06614894 24-LDRTC-01 | An Open Label Dose Escalation Study to Assess the Safety, Tolerability, and Pharmacologic Properties of High Dose Ambroxol Hydrochloride in Adult (≥ 18 Years of Age) Subjects With MPS III | RECRUITING | Sanfilippo Syndrome MPS3 | DRUG: Ambroxol Hydrochloride 30 mg tablet - 9 mg/kg/day DRUG: Ambroxol Hydrochloride 30 mg tablet - 18 mg/kg/day DRUG: Ambroxol Hydrochloride 30 mg tablet - 27 mg/kg/day | Ozlem Goker-Alpan | INTERVENTIONAL |
| NCT06906367 AT1001-030X | A Study of Patients With Fabry Disease (US Specific) | RECRUITING | Fabry Disease | DRUG: migalastat HCl DRUG: ERT | Amicus Therapeutics | OBSERVATIONAL |
| NCT06663358 CLI-06657AA1-10 | A Multi-Country Observational Study of Safety and Effectiveness of Elfabrio® in Fabry Patients | RECRUITING | Fabry Disease | DRUG: Pegunigalsidase-alfa | Chiesi Farmaceutici S.p.A. | OBSERVATIONAL |
| NCT06121011 POM-005 | A Global Prospective Observational Registry of Patients With Pompe Disease | RECRUITING | Pompe Disease | BIOLOGICAL: Cipaglucosidase alfa DRUG: Miglustat BIOLOGICAL: Alglucosidase alfa or Avalglucosidase alfa OTHER: Untreated | Amicus Therapeutics | OBSERVATIONAL |
| NCT05705674 22-LDRTC-01 | The Natural History Study of Patients With Sanfilippo Disease(s) (MPS3) | RECRUITING | Sanfilippo Syndrome MPS3 | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | OBSERVATIONAL | |
| NCT03812055 18-LDRTC-02 | Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders | RECRUITING | Lysosomal Storage Diseases | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | OBSERVATIONAL | |
| NCT02000310 13-CFCT-07 | Molecular and Cellular Mechanisms of Lysosomal Storage Diseases | RECRUITING | Lysosomal Storage Disorders | O & O Alpan LLC | OBSERVATIONAL | |
| Not yet on CT.gov | FD24-LDRTC-03 | RECRUITING | Fabry Disease | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | ||
| Not yet on CT.gov | GD25-LDRTC-01 | RECRUITING | Gaucher Disease | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | ||
| Not yet on CT.gov | PD25-LDRTC-02 | RECRUITING | Pompe Disease | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | ||
| NCT05206773 EFC17045 | A Study to Evaluate the Effect of Venglustat Tablets on Neuropathic and Abdominal Pain in Male and Female Participants ≥16 Years of Age With Fabry Disease | ACTIVE NOT RECRUITING | Fabry Disease | DRUG: Venglustat (GZ402671) DRUG: Placebo | Sanofi | INTERVENTIONAL |
| NCT05280548 EFC16158 | A Study to Evaluate the Effect of Venglustat Tablets on Left Ventricular Mass Index in Male and Female Adult Participants With Fabry Disease | ACTIVE NOT RECRUITING | Fabry Disease | DRUG: Venglustat (GZ402671) DRUG: Agalsidase alfa DRUG: Agalsidase beta (GZ419828) DRUG: Migalastat | Sanofi | INTERVENTIONAL |
| NCT03737214 ID-069A302 | A Study to Evaluate the Long-term Safety and Tolerability of Lucerastat in Adult Subjects With Fabry Disease | ACTIVE NOT RECRUITING | Fabry Disease | DRUG: Lucerastat | Idorsia Pharmaceuticals Ltd. | INTERVENTIONAL |
| NCT05487599 J3Z-MC-OJAE | A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) | ACTIVE NOT RECRUITING | Gaucher Disease Gaucher Disease, Type 1 | GENETIC: LY3884961 | Prevail Therapeutics | INTERVENTIONAL |
| NCT02843035 PDY13949 | Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension | ACTIVE NOT RECRUITING | Gaucher Disease Type 1 Gaucher Disease Type 3 | DRUG: venglustat (GZ402671) DRUG: imiglucerase | Genzyme, a Sanofi Company | INTERVENTIONAL |
| NCT05222906 EFC17215 | Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 | ACTIVE NOT RECRUITING | Gaucher's Disease Type III | DRUG: Venglustat DRUG: imiglucerase | Sanofi | INTERVENTIONAL |
| NCT06545136 FLT201-02 | Long Term Follow-up Study of Type-1 Gaucher Subjects Post FLT201 Dose (GALILEO-2) | ACTIVE NOT RECRUITING | Gaucher Disease, Type 1 | GENETIC: FLT201 | Spur Therapeutics | INTERVENTIONAL |
| NCT04860960 CTD-TCNPC-301 | Phase 3 Study to Evaluate Intravenous Trappsol(R) Cyclo(TM) in Pediatric and Adult Patients With Niemann-Pick Disease Type C1 | ACTIVE NOT RECRUITING | Niemann-Pick Disease, Type C1 | DRUG: Hydroxypropyl-beta-cyclodextrin DRUG: Placebo | Cyclo Therapeutics, Inc. | INTERVENTIONAL |
| NCT06150820 AT845-02 | A Study About Antibody Levels and Biomarkers in the Blood in People With Late-onset Pompe Disease | ACTIVE NOT RECRUITING | Pompe Disease (Late-onset) | OTHER: No Intervention | Astellas Gene Therapies | INTERVENTIONAL |
| NCT05039866 ST-920-LT01 | Long-Term Follow-up of Subjects Who Were Treated With ST-920 | ACTIVE NOT RECRUITING | Fabry Disease Fabry Disease, Cardiac Variant | BIOLOGICAL: ST-920 | Sangamo Therapeutics | OBSERVATIONAL |
| NCT06116071 23-LDRTC-02 | Biomarkers Related to Bone in Pediatric Gaucher Disease | ACTIVE NOT RECRUITING | Gaucher Disease Bone Diseases | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | OBSERVATIONAL | |
| NCT06109948 ABX1100-1001 | Study to Assess the Safety, Tolerability, PK and PD of ABX1100 | COMPLETED | Healthy Late Onset Pompe Disease | DRUG: ABX1100 injection for IV infusion DRUG: Placebo injection for IV infusion | Aro Biotherapeutics | INTERVENTIONAL |
| NCT06065605 23-LDRTC-01 | Assess Urine Biomarkers to Predict Nephropathy in Fabry Disease | CLOSED | Fabry Disease | Lysosomal and Rare Disorders Research and Treatment Center, Inc. | OBSERVATIONAL |
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