Activities Report / InformationREPORT & INFORMATION
2024.04.01
Medical and disease
information
- #Lysosomal storage diseases
- #Clinical trial for regulatory submission
- #Clinical trial
Research and Development of Lysosomal Storage Diseases
Information access date: Feb 16, 2024 (ClinicalTrials.gov)
*Please note the following:
- ●
This page indicates
when there is a Phase 1, 2, or 3 clinical trial listed on ClinicalTrials.gov that has the status of “Not yet recruiting” or “Recruiting” when the disease name is searched. Please check the various clinical studies for details.
- ● The clinical trials include not only medication treatments but also hematopoietic stem cell transplants, symptomatic therapies and other related treatments.
- ● If a clinical trial is not being conducted for a specific disease,
is indicated (e.g., a clinical trial of hematopoietic stem cell transplantation for multiple metabolic disorders). - ● Existing medications may be approved only in certain countries or for certain disease types. For example, medications for the treatment of metachromatic leukodystrophy are approved only in Europe and medications for the treatment of mannosidosis are approved only in Europe and the US (as of December 2023).
- ● Please refer to “Introduction to the Clinical Trial Database” for how to view trial information.
For an overview of each disease, please refer to the following website.
Information Center for Specific Pediatric Chronic Diseases > List of diseases > List of diseases by disease group > List of congenital metabolic disorders
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To the external sitesGo to external site
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To the external sites- Lipid metabolism disorders (sphingolipidosis, lipidosis)
-
Lipid metabolism disorders are a general term for diseases in which lipids accumulate in the body. In the following lysosomal storage diseases, glycolipids such as globotriaosylceramide (Fabry disease) and glucosylceramide (Gaucher disease) accumulate due to the defective function of lysosomal enzyme.
= Those with approved medication.- Disease name
- Clinical trial available
- Gaucher Disease
-
Clinical trial
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To the external sites - Fabry Disease
-
Clinical trial
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To the external sites - Acid Sphingomyelinase Deficiency
-
No clinical trial
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To the external sites - Farber Disease
-
No clinical trial
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To the external sites - GM1 Gangliosidosis
-
Clinical trial
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To the external sites - GM2 Gangliosidosis
-
Clinical trial
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To the external sites - Metachromatic Leukodystrophy
-
Clinical trial
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To the external sites - Multiple Sulfatase Deficiency
-
No clinical trial
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To the external sites - Krabbe Disease
-
Clinical trial
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To the external sites - Saposin Deficiency
-
No clinical trial
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To the external sites - Lysosomal Acid Lipase Deficiency
-
No clinical trial
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To the external sites
- Mucopolysaccharidoses
-
Mucopolysaccharidoses are a general term for diseases in which mucopolysaccharides (glycosaminoglycans: GAGs) accumulate in the body. In the following lysosomal storage diseases, deficiency of lysosomal enzymes results in the accumulation of mucopolysaccharides such as dermatan sulfate and heparan sulfate.
= Those with approved medication.- Disease name
- Clinical trial available
- Mucopolysaccharidosis I / Hurler, Scheie Syndrome
-
Clinical trial
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To the external sites - Mucopolysaccharidosis II / Hunter Syndrome
-
Clinical trial
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To the external sites - Mucopolysaccharidosis III / Sanfilippo Syndrome
-
Clinical trial
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To the external sites - Mucopolysaccharidosis IV / Morquio Syndrome
-
Clinical trial
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To the external sites - Mucopolysaccharidosis VI / Maroteaux Lamy Syndrome
-
Clinical trial
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To the external sites - Mucopolysaccharidosis VII / Sly Syndrome
-
No clinical trial
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To the external sites
- Glycoprotein metabolism disorders
-
Glycoprotein metabolism disorders are a general term for diseases in which glycoproteins accumulate in the body. In the following lysosomal storage diseases, deficiency of lysosomal enzymes results in the accumulation of glycoproteins such as mannose-containing oligosaccharides (mannosidosis) and sialic acid-containing oligosaccharides (sialidosis).
= Those with approved medication.- Disease name
- Clinical trial available
- Fucosidosis
-
No clinical trial
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To the external sites - Mannosidosis*only α⁻Mannosidosis
-
No clinical trial
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To the external sites - Aspartylglucosaminuria
-
No clinical trial
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To the external sites - Galactosialidosis
-
No clinical trial
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To the external sites - Mucolipidosis type II, type III
-
No clinical trial
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To the external sites - Sialidosis
-
No clinical trial
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To the external sites - Kanzaki Disease
-
No clinical trial
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To the external sites
- Others
-
= Those with approved medication.
- Disease name
- Clinical trial available
- Pompe Disease
-
Clinical trial
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To the external sites - Neuronal Ceroid Lipofuscinosis*only CLN2
-
Clinical trial
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To the external sites - Niemann-Pick Disease type C
-
Clinical trial
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To the external sites - Cystinosis
-
Clinical trial
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To the external sites - Free Sialic Acid Storage Disease
-
No clinical trial
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To the external sites - Cathepsin K Deficiency
-
No clinical trial
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To the external sites - Cobalamin F-type Deficiency
-
No clinical trial
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Please note that the linked websites are not operated by MEDIPAL Group.
To the external sites - Danon Disease
-
Clinical trial
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To the external sites
Disclaimer
- ● The information contained herein is current as of the date it was obtained. Please note that there is a possibility that the information may have been updated or that the URL links may have changed.
- ● These materials and the publicly available information in the websites presented within are not intended to provide medical advice. If you have any medically related questions, concerns, or other inquiries, including participation in a clinical trial, be sure to consult with your physician.
- ● This information is not intended to encourage participation in a corporate-sponsored clinical trial.
Reference materials
- Yoshikatsu Eto Edition. Lysosomal Storage Disease - Recent Advances in Pathophysiology, Diagnosis, and Treatment - Revised 2nd Edition. SHINDAN TO CHIRYO SHA, Inc. 2023.
- ClinicalTrials.gov. (https://www.clinicaltrials.gov/) (Accessed Nov 20, 2023)
(2024.01.22 release／2024.04.01 update)
