Lysosomal Storage Diseases
Information
- Disease name
- Lysosomal Storage Diseases
- Disease ID
- Description
Disease area statistics
[No Data.]
Chromosome band
[No Data.]
Annotation
| Genes | Mutation | Description | Source | Links |
|---|
| NCT ID | Status | Phase | Summary | Start date | Completion date |
|---|---|---|---|---|---|
| NCT04943991 | Active, not recruiting | N/A | Fabry Disease in High-risk Patients With Left Ventricular Hypertrophy: Prevalence and Implementation of a Clinical Score | July 1, 2021 | October 1, 2024 |
| NCT04283227 | Active, not recruiting | Phase 3 | OTL-200 in Patients With Late Juvenile Metachromatic Leukodystrophy (MLD) | January 17, 2022 | March 31, 2031 |
| NCT04093349 | Active, not recruiting | Phase 1/Phase 2 | A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE) | October 1, 2020 | April 2032 |
| NCT03897361 | Active, not recruiting | Phase 1/Phase 2 | Stem Cell Gene Therapy for Cystinosis | July 8, 2019 | November 2024 |
| NCT02416661 | Completed | Lyso-Gb1 as a Long-term Prognostic Biomarker in Gaucher Disease | August 27, 2018 | January 15, 2021 | |
| NCT04455230 | Completed | Phase 1/Phase 2 | A Long Term Follow-Up Study of Fabry Disease Subjects Treated With FLT190 | September 8, 2020 | September 28, 2023 |
| NCT02363153 | Completed | N/A | Diet and Exercise in Pompe Disease | November 6, 2017 | May 12, 2021 |
| NCT03893240 | Completed | N/A | Neutralizing Antibody Seroprevalence Study With a Retrospective Component in Participants With Late-Onset Pompe Disease | June 12, 2019 | October 27, 2020 |
| NCT06130228 | Not yet recruiting | Phase 2 | Nutritional Therapy in Late-onset Pompe Disease | April 1, 2024 | April 1, 2025 |
| NCT03333200 | Recruiting | Longitudinal Study of Neurodegenerative Disorders | January 11, 2012 | January 2035 | |
| NCT04399694 | Suspended | Identification and Characterization of Novel Non-Coding Variants That Contribute to Genetic Disorders | March 3, 2020 | April 2026 | |
| NCT00215527 | Terminated | Phase 1 | Intrathecal Enzyme Replacement Therapy for Spinal Cord Compression in Mucopolysaccharidosis (MPS) I | November 2005 | October 2011 |
| NCT04040049 | Terminated | Phase 1/Phase 2 | A Fabry Disease Gene Therapy Study | July 8, 2019 | May 2, 2023 |
| NCT01963650 | Terminated | Natural History Study of Children With Metachromatic Leukodystrophy | November 2, 2015 | April 8, 2016 | |
| NCT03853876 | Terminated | A Natural History Study of Aspartylglucosaminuria | April 18, 2019 | March 17, 2022 | |
| NCT04393701 | Unknown status | N/A | A Pilot Study for Systematic Neonatal Screening for Lysosomal Storage Diseases Using Tandem Mass Spectrometry | March 8, 2021 | March 2024 |
| NCT03812055 | Unknown status | Cellular Pharmacodynamics of Small Molecules in Lysosomal Storage Disorders | July 6, 2018 | July 2020 | |
| NCT03812042 | Unknown status | Screening of Lysosomal Storage Disorders Diseases in Minority Groups | March 17, 2016 | December 2019 | |
| NCT01003912 | Withdrawn | Phase 1 | Fetal Umbilical Cord Blood (UCB) Transplant for Lysosomal Storage Diseases | October 2009 | March 2017 |
| NCT04189601 | Withdrawn | Complement Activation in the Lysosomal Storage Disorders | September 30, 2020 | April 30, 2021 |
- MeSH unique ID (MeSH (Medical Subject Headings))
- D016464