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Found 26 clinical trials
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Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

PRV-GD2-101 is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose PR001 in infants diagnosed with Type 2 Gaucher disease (GD2). For each patient, the

type 1 gaucher disease
  • 0 views
  • 29 Sep, 2021
  • 5 locations
None
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3

Primary Objective: Evaluate the safety and pharmacokinetics of eliglustat in pediatric patients (2 to <18 years old). Secondary Objective: Evaluate the efficacy of eliglustat and quality of life in pediatric patients (2 to <18 years old).

imiglucerase
eliglustat
platelet count
cerezyme
deficiency
  • 77 views
  • 27 Jan, 2021
  • 37 locations
None
Phase 1/2a Clinical Trial of PR001A in Patients With Parkinson's Disease With at Least One GBA1 Mutation (PROPEL)

patients have mutations in at least one chromosomal copy of GBA1, while Gaucher disease patients have mutations in both chromosomal copies. Without enough GCase, glycolipids accumulate

  • 11 views
  • 18 Sep, 2020
  • 4 locations
None
A Multicenter Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease

This is a multicenter study to assess the safety and efficacy of taliglucerase alfa (60 units/kg) in previously untreated subjects of any age with Type 3 GD. Subjects will receive an infusion of taliglucerase alfa every 2 weeks for 12 months. Subjects who tolerate the infusions well, and who are …

  • 0 views
  • 27 Mar, 2021
  • 3 locations
None
Lentiviral Vector Gene Therapy - The GuardOne Trial of AVR-RD-02 for Subjects With Type 1 Gaucher Disease

Screening with a confirmed diagnosis of Type 1 Gaucher disease (based on clinical phenotype, genotyping, and deficient GCase enzyme activity in whole blood).

a hemoglobin
alglucerase
platelet count
type 1 gaucher disease
  • 33 views
  • 11 Sep, 2021
  • 5 locations
None
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

The study will provide information on outcomes in people with type 1 Gaucher disease when they are treated with velaglucarase alfa (also called VPRIV), under standard care. Standard care means

substrate reduction therapy
velaglucerase alfa
replacement therapy
vpriv
type 1 gaucher disease
  • 0 views
  • 22 May, 2021
  • 1 location
None
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type

Primary Objective To evaluate the efficacy on hematologic manifestations of imiglucerase treatment in Chinese patients who are diagnosed as Gaucher disease type To

  • 0 views
  • 21 Mar, 2021
  • 1 location
None
Getting Global Rare Disease Insights Through Technology Study

metabolic disorders such as Fabry disease, Pompe disease, and Gaucher disease, will provide access to Zamplo to patients free-of-charge to evaluate its utility in managing the symptoms of their disease

gaucher disease
  • 0 views
  • 08 Sep, 2021
  • 1 location
None
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases

The investigators aims to determine the the maternal and fetal safety and feasibility of in utero fetal enzyme replacement therapy in fetuses with Lysosomal Storage Diseases.

  • 0 views
  • 18 Apr, 2021
  • 1 location
None
GSL Synthetase Inhibitor or in Combination With Immune Checkpoint Inhibitor in Previously Treated Blood and Solid Tumor

Immune checkpoint blockade has made great but unsatisfied success in treating cancers. One important reason is the hijacked HLA-I antigen presentation. Eliglustat could inhibit glycosphingolipids synthesis and restore HLA-I antigen presentation and transform the immunogenicity of tumor cells. Therefore,GSL synthetase inhibitor eliglustat monotherapy or in combination with immune checkpoint inhibitor …

solid tumor
cancer
  • 0 views
  • 06 Oct, 2021
  • 1 location