Gaucher Disease, Type 1
Information
- Disease name
- Gaucher Disease, Type 1
- 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 |
|---|---|---|---|---|---|
| NCT00954460 | Approved for marketing | Treatment Protocol of Velaglucerase Alfa for Patients With Type 1 Gaucher Disease | |||
| NCT00364858 | Completed | Phase 4 | Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease | December 2001 | February 2007 |
| NCT00430625 | Completed | Phase 3 | A Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Gaucher Disease | February 15, 2007 | April 1, 2009 |
| NCT00433147 | Completed | Phase 2 | A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy | March 23, 2007 | February 19, 2008 |
| NCT00446550 | Completed | Phase 2 | A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease | June 11, 2008 | August 20, 2009 |
| NCT00553631 | Completed | Phase 3 | Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease | January 29, 2008 | May 5, 2009 |
| NCT00635427 | Completed | Phase 3 | An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease | March 13, 2008 | December 28, 2012 |
| NCT00813865 | Completed | Phase 2 | A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients | May 11, 2009 | May 1, 2012 |
| NCT00891202 | Completed | Phase 3 | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE) | November 2009 | January 2016 |
| NCT00943111 | Completed | Phase 3 | A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE) | September 2009 | June 2015 |
| NCT00358150 | Completed | Phase 2 | A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients | June 2006 | December 2015 |
| NCT02770625 | Completed | Phase 3 | Phase III Study of ISU302 in Patients With Type 1 Gaucher Disease | September 2011 | August 2014 |
| NCT03333447 | Completed | Retrospective and Prospective Observational Study of MRI Changes in Bone and Visceral Lesions of Patients With Type 1 Gaucher Disease Treated With VPRIV® (Velaglucerase Alfa) | January 17, 2017 | December 27, 2018 | |
| NCT03950050 | Completed | Phase 2 | Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy | March 1, 2019 | December 30, 2022 |
| NCT04120506 | Completed | Phase 4 | Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV) | January 10, 2016 | January 1, 2022 |
| NCT04353466 | Completed | N/A | Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs | January 1, 2017 | July 31, 2021 |
| NCT05253560 | Recruiting | Prodromal Parkinsonian Features in GBA1 Mutation Carriers | May 16, 2017 | December 31, 2025 | |
| NCT05687474 | Recruiting | Baby Detect : Genomic Newborn Screening | September 1, 2022 | August 31, 2025 | |
| NCT05324943 | Recruiting | Phase 1 | A Gene Therapy Study in Patients With Gaucher Disease Type 1 | April 15, 2022 | January 31, 2025 |
| NCT05447494 | Recruiting | Phase 1/Phase 2 | Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease | July 11, 2022 | December 30, 2024 |
| NCT05487599 | Recruiting | Phase 1/Phase 2 | A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED) | December 20, 2022 | October 2, 2030 |
| NCT03746587 | Terminated | Phase 2 | Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3 | June 6, 2018 | October 15, 2021 |
| NCT00795197 | Withdrawn | A Screening Study Evaluating Disease Status of Gaucher Type I Patients | December 2008 | December 2009 |