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Found 28 clinical trials
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

J3Z-MC-OJAB is an open-label, Phase 1/2, multicenter study to evaluate the safety and efficacy of single-dose LY3884961 (formerly PR001) in infants diagnosed with Type 2 Gaucher disease (GD2

type 1 gaucher disease
  • 8 views
  • 04 Oct, 2022
  • 4 locations
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (LEAP2MONO)  

Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.

deficiency
platelet count
imiglucerase
replacement therapy
sterility
  • 6 views
  • 07 Oct, 2022
  • 1 location
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

cancer
  • 25 views
  • 13 May, 2022
  • 4 locations
A Gene Therapy Study in Patients With Gaucher Disease Type 1 (GALILEO-1)

This study is a first-in-human, phase 1/2, open-label, safety, tolerability, and efficacy study in adult patients with Gaucher disease Type 1. The aims are to investigate the safety/tolerability

substrate reduction therapy
replacement therapy
alglucerase
serum pregnancy test
type 1 gaucher disease
  • 0 views
  • 07 Oct, 2022
  • 1 location
Lentiviral Vector Gene Therapy - The Guard1 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).

alglucerase
a hemoglobin
platelet count
type 1 gaucher disease
  • 53 views
  • 16 Feb, 2022
  • 5 locations
Study to Evaluate the Efficacy and Safety of Venglustat in Adult and Pediatric Patients With Gaucher Disease Type 3 (LEAP2MONO)

Gaucher disease Type 3 (GD3) who have been treated with Enzyme Replacement Therapy (ERT) for at least 3 years.

deficiency
platelet count
imiglucerase
replacement therapy
sterility
  • 0 views
  • 07 Oct, 2022
  • 4 locations
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ (SEED)

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

imiglucerase
neuronopathic gaucher disease
cerezyme
  • 0 views
  • 04 Oct, 2022
  • 5 locations
Study to Evaluate Efficacy and Safety of Imiglucerase Treatment in Chinese Patients With Gaucher Disease Type Ⅲ (SEED)

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

cerezyme
neuronopathic gaucher disease
imiglucerase
  • 0 views
  • 07 Oct, 2022
  • 5 locations
In Utero Enzyme Replacement Therapy for Lysosomal Storage Diseases (IUERT)

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.

replacement therapy
  • 11 views
  • 15 May, 2022
  • 1 location
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 (Human Leukocyte Antigen) 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 …

solid tumor
cancer
  • 0 views
  • 14 May, 2022
  • 1 location