Ongoing

3

110

Europe

Venglustat, SAR402671, GZ402671 or GZ/SAR402671, Tablet, Concentrate for solution for infusion, Powder for concentrate for solution for infusion, Capsule, hard, Replagal, Fabrazyme, Galafold

sanofi-aventis recherche et développement, sanofi-aventis recherche et développement

Fabry’s disease Enfermedad de Fabry, Fabry’s disease Enfermedad de Fabry, Diseases [C] - Congenital, Hereditary, and Neonatal Diseases and Abnormalities [C16]

 

 

Active, not recruiting

3

104

Europe, Canada, Japan, US, RoW

Venglustat (GZ402671), Agalsidase alfa, Replagal®, Agalsidase beta (GZ419828), Fabrazyme®, Migalastat, Galafold®

Sanofi, sanofi-aventis recherche et développement

Fabry Disease

05/26

12/27

Completed

3

16

Europe, US

migalastat HCl 150 mg, migalastat, AT1001

Amicus Therapeutics

Fabry Disease

11/24

Available

N/A

NA

migalastat HCl 150 mg, AT1001, migalastat, migalastat HCl 20 mg

Amicus Therapeutics

Fabry Disease

 

 

Recruiting

N/A

30

Europe

Wuerzburg University Hospital, Charite University, Berlin, Germany, University Hospital Muenster, Health Care Center Dr. Markus Cybulla, Muellheim

Rare Diseases, Fabry Disease, Adherence, Medication, Quality of Life

12/23

12/23

Recruiting

N/A

20

RoW

Echocardiography

Yonsei University

Fabry Disease

01/25

04/25

Recruiting

N/A

20

US

migalastat

Amicus Therapeutics

Fabry Disease

08/29

02/30

Not yet recruiting

3

286

Europe

Repagermanium, DMX-200, Capsule

Dimerix Bioscience Pty Ltd, Dimerix Bioscience Pty Ltd

Focal segmental glomerulosclerosis, Focal segmental glomerulosclerosis, Diseases [C] - Immune System Diseases [C20]

 

 

Recruiting

3

20000

Europe, Canada, Japan, US, RoW

Ceftriaxone, Moxifloxacin or Levofloxacin, Piperacillin-tazobactam, Ceftaroline, Amoxicillin-clavulanate, Standard course macrolide, Extended course macrolide, No systemic corticosteroid, Fixed-duration Hydrocortisone, Shock-dependent hydrocortisone, Fixed-duration higher dose Hydrocortisone, No antiviral agent for influenza, Five-days oseltamivir, Ten-days oseltamivir, No antiviral agent for COVID-19, Lopinavir / Ritonavir, Hydroxychloroquine, Hydroxychloroquine + lopinavir/ritonavir, Ivermectin, No immune modulation for COVID-19, Interferon beta-1a, IFN-β1a, Anakinra, Tocilizumab, Sarilumab, Local standard venous thromboprophylaxis, Therapeutic dose anticoagulation, Conventional low dose thromboprophylaxis, Intermediate dose thromboprophylaxis, Continuation of therapeutic dose anticoagulation, No immunoglobulin, Convalescent plasma, Delayed administration of convalescent plasma, No vitamin C, Vitamin C, No antiplatelet, Aspirin, acetylsalicylic acid, P2Y12 inhibitor, Clopidogrel, Prasugrel, Ticagrelor, No simvastatin, Simvastatin, Eritoran, Apremilast, Clinician-preferred mechanical ventilation strategy, Protocolised mechanical ventilation strategy, No renin-angiotensin system inhibitor, Angiotensin converting enzyme inhibitor, Ramipril, Lisinopril, Perindopril, Enalapril, Trandolapril, Captopril, Angiotensin Receptor Blockers, Losartan, Valsartan, Candesartan, Irbesartan, Telmisartan, Olmesartan, ARB + DMX-200, No cysteamine, Cysteamine, Fixed-duration dexamethasone, Baloxavir Marboxil, Five-days oseltamivir + baloxavir marboxil, Ten-days oseltamivir + baloxavir marboxil, No endothelial modulator, Imatinib, No Immune Modulator for Influenza, Baricitinib, Nirmatrelvir/ritonavir, Paxlovid, Remdesivir, Nirmatrelvir/ritonavir + remdesivir

UMC Utrecht, Australian and New Zealand Intensive Care Research Centre, Medical Research Institute of New Zealand, Unity Health, Berry Consultants, Global Coalition for Adaptive Research, University of Pittsburgh Medical Center, Intensive Care National Audit & Research Centre, St. Marianna University School of Medicine, Nat Intensive Care Surveillance - MORU, National University Hospital, Singapore

Community-acquired Pneumonia, Influenza, COVID-19

02/26

02/28

Not yet recruiting

1/2

13

Europe

scAAV9.CB.CLN6, AT-GTX-501, Solution for injection

Amicus Therapeutics, Inc., Amicus Therapeutics, Inc.

Patients with variant late infantile neuronal ceroid lipofuscinosis associated with mutation(s) in the CLN6 gene (vLINCL6 disease), a subset of patients with CLN6 Batten disease., Patients with a mutation in the genetic material resulting CLN6 Batten disease that primarily affect the brain and retina., Diseases [C] - Nervous System Diseases [C10]

 

 

Completed

3

119

Europe, Canada, Japan, US, RoW

AT2221, Miglustat, ATB200

Amicus Therapeutics

Pompe Disease (Late-onset)

12/24

12/24

Recruiting

3

36

Europe, US, RoW

Cipaglucosidase alfa, ATB200, Miglustat, AT2221

Amicus Therapeutics

Glycogen Storage Disease Type II Infantile Onset

04/27

04/27

Available

N/A

NA

ATB200, AT2221

Amicus Therapeutics

Pompe Disease

 

 

Recruiting

N/A

500

Europe, US

Cipaglucosidase alfa, ATB200, Pombiliti, Miglustat, AT2221, Opfolda, Alglucosidase alfa or Avalglucosidase alfa, Myozyme, Lumizyme, Nexviazyme, Nexviadyme, Untreated

Amicus Therapeutics

Pompe Disease

12/34

12/34

Recruiting

1

10

US

Aldurazyme (laronidase), Elaprase (idursulfase), Vimizim (elosulfase alfa), Naglazyme (galsulfase), Mepsevii (vestronidase alfa-vjbk), Lumizyme (alglucosidase alfa), Kanuma (sebelipase alfa)

University of California, San Francisco, Duke University

MPS I, MPS II, MPS IVA, MPS VI, Mps VII, Gaucher Disease, Type 2, Gaucher Disease, Type 3, Pompe Disease Infantile-Onset, Wolman Disease

07/31

07/32

Available

N/A

NA

Mepsevii, UX003, recombinant human beta-glucuronidase, rhGUS, vestronidase alfa

Ultragenyx Pharmaceutical Inc

MPS VII, Mucopolysaccharidosis VII, Sly Syndrome