Gaucher Disease, Type 1 Clinical Trial
Official title:
A Multicenter, Randomized, Double-Blind, Parallel Group, Two-Dose Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) Enzyme Replacement Therapy in Patients With Type 1 Gaucher Disease
Gaucher disease is a rare lysosomal storage disorder caused by the deficiency of the enzyme glucocerebrosidase (GCB). Due to this deficiency of functional GCB, glucocerebroside accumulates within macrophages leading to cellular engorgement, organomegaly, and organ system dysfunction. The purpose of this study is to evaluate the efficacy of every other week dosing of Gene-Activated® Human Glucocerebrosidase (GA-GCB, velaglucerase alfa) at doses of 45 and 60 U/kg in treatment-naïve patients with type 1 Gaucher disease.
Type 1 Gaucher disease, the most common form, accounts for more than 90% of all cases and does not involve the Central Nervous System (CNS). Typical manifestations of type 1 Gaucher disease include hepatomegaly, splenomegaly, thrombocytopenia, bleeding tendencies, anemia, hypermetabolism, skeletal pathology, growth retardation, pulmonary disease, and decreased quality of life. Gene-Activated® human glucocerebrosidase (GA-GCB; velaglucerase alfa) is produced in a continuous human cell line using proprietary gene-activation technology and has an identical amino acid sequence to the naturally occurring human enzyme. Velaglucerase alfa contains terminal mannose residues that target the enzyme to the macrophages-the primary target cells in Gaucher disease. This study was designed to determine the efficacy, safety and pharmacokinetics of GA-GCB in men, women, and children with Type 1 Gaucher disease. Each patients duration of treatment was 12 months. ;
Status | Clinical Trial | Phase | |
---|---|---|---|
Active, not recruiting |
NCT00943111 -
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease Who Have Reached Therapeutic Goals With Enzyme Replacement Therapy (ENCORE)
|
Phase 3 | |
Recruiting |
NCT05687474 -
Baby Detect : Genomic Newborn Screening
|
||
Active, not recruiting |
NCT00891202 -
A Study of Eliglustat Tartrate (Genz-112638) in Patients With Gaucher Disease (ENGAGE)
|
Phase 3 | |
Recruiting |
NCT05253560 -
Prodromal Parkinsonian Features in GBA1 Mutation Carriers
|
||
Completed |
NCT00813865 -
A Long-Term Extension Study of AT2101 (Afegostat Tartrate) in Type 1 Gaucher Patients
|
Phase 2 | |
Completed |
NCT00433147 -
A Study of AT2101 (Afegostat Tartrate) in Adult Patients With Type 1 Gaucher Disease Currently Receiving Enzyme Replacement Therapy
|
Phase 2 | |
Completed |
NCT00635427 -
An Open-Label Extension Study of GA-GCB ERT in Patients With Type 1 Gaucher Disease
|
Phase 3 | |
Completed |
NCT03950050 -
Ambroxol Therapy for Patients With Type 1 Gaucher Disease and Suboptimal Response to Enzyme Replacement Therapy
|
Phase 2 | |
Terminated |
NCT03746587 -
Study of Arimoclomol in Patients Diagnosed With Gaucher Disease Type 1 or 3
|
Phase 2 | |
Active, not recruiting |
NCT00358150 -
A Study of the Efficacy and Safety of Eliglustat Tartrate (Genz-112638) in Type 1 Gaucher Patients
|
Phase 2 | |
Completed |
NCT00446550 -
A Study of Oral AT2101 (Afegostat Tartrate) in Treatment-naive Patients With Gaucher Disease
|
Phase 2 | |
Recruiting |
NCT05447494 -
Phase 1/2 Study of CAN103 in Subjects With Gaucher Disease
|
Phase 1/Phase 2 | |
Completed |
NCT00553631 -
Study of Gene-Activated® Human Glucocerebrosidase (GA-GCB) ERT Compared With Imiglucerase in Type I Gaucher Disease
|
Phase 3 | |
Completed |
NCT03333447 -
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)
|
||
Completed |
NCT04120506 -
Long Term Impact of Rapid Intravenous Infusion of Velaglucerase Alfa (VPRIV)
|
Phase 4 | |
Completed |
NCT04353466 -
Assessing the Impact of Elelyso on Bone Involvement Currently Treated With Other ERTs
|
N/A | |
Withdrawn |
NCT00795197 -
A Screening Study Evaluating Disease Status of Gaucher Type I Patients
|
N/A | |
Completed |
NCT00364858 -
Safety and Efficacy of Cerezyme® Infusions Every 4 Weeks Versus Every 2 Weeks in Type 1 Gaucher Disease
|
Phase 4 | |
Recruiting |
NCT05487599 -
A Clinical Trial of PR001 (LY3884961) in Patients With Peripheral Manifestations of Gaucher Disease (PROCEED)
|
Phase 1/Phase 2 | |
Recruiting |
NCT05324943 -
A Gene Therapy Study in Patients With Gaucher Disease Type 1
|
Phase 1 |