Gaucher Disease, Type III (DBCOND0109869)

Identifiers

Synonyms

Gaucher's disease type III / Gaucher Disease Type 3 / Gaucher Disease, Type 3 / Type 3 Gaucher disease / Subacute neuronopathic Gaucher's disease (disorder)

Associated Data

Indicated Drugs and Targets

Drug	Description	Targets
Imiglucerase	A form of recombinant human beta-glucocerebrosidase enzyme used to replace the deficient endogenous enzyme in the treatment of Gaucher disease.	Glucocerebrosidetarget
Taliglucerase alfa	A hydrolytic lysosomal glucocerebroside specific enzyme, used for long term enzyme replacement therapy in patients with Type 1 Gaucher disease.	Glucocerebrosidetarget

Clinical Trials

Identifier	Title	Drug(s)	Purpose	Phase	Status
NCT05222906	Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3	Imiglucerase Venglustat	treatment	3	recruiting
NCT03485677	Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3	Eliglustat Imiglucerase	treatment	3	active_not_recruiting
NCT03519646	Eliglustat on Gaucher Disease Type IIIB	Eliglustat	treatment	Not Available	completed
NCT03240653	Gaucherite - A Study to Stratify Gaucher Disease	No drug interventions	Not Available	Not Available	recruiting
NCT06627543	Effect of Enzyme Replacement Therapy on Cardiac Function in Children With Gaucher Disease Type 3	No drug interventions	Not Available	Not Available	enrolling_by_invitation
NCT02528617	The Effect of Velaglucerase Alfa (Vpriv) on Skeletal Development in Pediatric Gaucher Disease	Velaglucerase alfa	treatment	4	withdrawn
NCT02843035	Venglustat in Combination With Cerezyme in Adult Patients With Gaucher Disease Type 3 With Venglustat Monotherapy Extension	Imiglucerase Venglustat	treatment	2	active_not_recruiting
NCT04532047	In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases	Alglucosidase alfa Elosulfase alfa Galsulfase Idursulfase Laronidase Sebelipase alfa Vestronidase alfa	treatment	1	recruiting
NCT04002830	A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease	Benzalkonium Taliglucerase alfa	treatment	4	completed
NCT05447494	Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease	No drug interventions	treatment	1 / 2	recruiting
NCT05815004	An Efficacy and Safety Study of AVR-RD-02 Compared to Enzyme Replacement Therapy for Treatment of Gaucher Disease Type 3	AVR-RD-02 Imiglucerase Taliglucerase alfa	treatment	2 / 3	withdrawn
NCT05586243	MAGNETIC RESONANCE SPECTROSCOPY BIOMARKERS IN TYPE 3 GAUCHER DISEASE (GD3)	No drug interventions	Not Available	Not Available	recruiting
NCT03746587	Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3	Arimoclomol	treatment	2	terminated
NCT01685216	Efficacy and Safety Study of Velaglucerase Alfa in Children and Adolescents With Type 3 Gaucher Disease	Velaglucerase alfa	treatment	1 / 2	completed