Filter By
Clear all Advanced
I am/have/had
added new label for I am/have/had
more
I am looking for
added new label for I am looking for
more
Advanced Filters
Found 30 clinical trials
Phase 1/2 Clinical Trial of PR001 in Infants With Type 2 Gaucher Disease (PROVIDE)

JRZ-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
  • 17 May, 2022
  • 4 locations
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (ELIKIDS)

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).

alglucerase
deficiency
imiglucerase
thrombocytopenia
eliglustat
  • 77 views
  • 20 Apr, 2022
  • 35 locations
Safety and Efficacy of Eliglustat With or Without Imiglucerase in Pediatric Patients With Gaucher Disease (GD) Type 1 and Type 3 (ELIKIDS)

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).

alglucerase
deficiency
platelet count
thrombocytopenia
imiglucerase
  • 0 views
  • 13 May, 2022
  • 23 locations
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

  • 23 views
  • 13 May, 2022
  • 4 locations
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
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
type 1 gaucher disease
a hemoglobin
platelet count
  • 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.

alglucerase
deficiency
imiglucerase
sterility
replacement therapy
  • 0 views
  • 09 May, 2022
  • 1 location
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.

alglucerase
deficiency
platelet count
imiglucerase
sterility
  • 6 views
  • 19 May, 2022
  • 1 location
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

vpriv
type 1 gaucher disease
substrate reduction therapy
velaglucerase alfa
replacement therapy
  • 0 views
  • 22 May, 2021
  • 1 location
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
  • 13 May, 2022
  • 5 locations