Genetics
Protocol:VTS-270-303
Condition or Disease: Niemann-Pick Disease, Type C
Study Type: Interventional (Clinical Trial)
Status: Opening Mid 2019
Study Title: An Open-label, Multicenter Safety and Tolerability Study of VTS-270 (2-hydroxypropyl-ë_-cyclodextrin) in Pediatric Subjects Aged < 4 Years With Neurologic Manifestations of Niemann-Pick Type C (NPC) Disease
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03687476
Genetics
Protocol:VTS-270-001
Condition or Disease: Niemann-Pick Disease, Type C
Study Type: Expanded Access
Status: Open for enrollment
Study Title: An Open-label Expanded Access Treatment Protocol for VTS-270 (2-hydroxypropyl-ë_-cyclodextrin) in Patients With Neurologic Manifestations of Niemann-Pick Type C Disease (NPC)
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT03643562
Genetics
Protocol:VTS-270 IND
Condition or Disease: Niemann-Pick Disease, Type C
Study Type: Expanded Access
Status: Open for enrollment
Study Title: Intrathecal 2-Hydroxypropyl-ë_-cyclodextrin for Neurological Decline in Patients with Niemann-Pick Disease Type C1
Principal I investigator: Alan Ikeda, MD
Genetics
Protocol:110-504
Condition or Disease: Mucopolysaccharidosis Type IVA
Study Type: Observational
Status: Open for enrollment
Study Title: Morquio A Registry Study (MARS)
Principal I investigator: Angela Berg, DNP, APRN, CPN
Genetics
Protocol:DIREGC07009
Condition or Disease: Gaucher
Study Type:
Status: Open for enrollment
Study Title: Gaucher Registry
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT00358943
Genetics
Protocol:DIREGC07008
Condition or Disease: MPSI
Study Type: Observational
Status: Open for enrollment
Study Title: MPS 1 Registry
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT00144794
Genetics
Protocol:DIREGC07006
Condition or Disease: Fabry
Study Type: Observational
Status: Open for enrollment
Study Title: Faby Registry
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT00196742
Genetics
Protocol:DIREGC07005
Condition or Disease: Pompe
Study Type: Observational
Status: Open for enrollment
Study Title: Pompe Registry
Principal I investigator: Alan Ikeda, MD
More Info: //clinicaltrials.gov/ct2/show/NCT00231400
