Clinical Trials

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Clinical Trial
  • Status: Not yet recruiting
  • Intervention Type: Other
  • Participants: 20
  • Start Date: February 28, 2021
Gaucher Disease During Infancy and Early Childhood and Experience With Enzyme Replacement Therapy (ERT) Using Velaglucerase Alfa (VPRIV): A Combined Retrospective and Prospective Cohort Study
Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 4
  • Intervention Type: Drug
  • Participants: 20
  • Start Date: February 26, 2021
A Multicenter, Interventional, Retrospective and Prospective Study of Enzyme Replacement Therapy (VPRIV) Clinical Outcomes and Safety in Gaucher Disease Type 1 Patients Previously Treated With Substrate Reduction Therapy
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Clinical Trial
  • Status: Not yet recruiting
  • Intervention Type: Drug
  • Participants: 35
  • Start Date: January 31, 2021
A Post Marketing Surveillance (PMS) Study for VPRIV (Velaglucerase Alfa) in India
Clinical Trial
  • Status: Not yet recruiting
  • Participants: 77
  • Start Date: January 2021
Prospective Study on the Impact of COVID-19 on the Equilibrium of Hereditary Metabolic Disease (MHM) in Patients With or Who Have Contracted COVID-19
Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 4
  • Intervention Type: Drug
  • Participants: 12
  • Start Date: December 31, 2020
A Single Arm, Prospective, Open Label, Multicenter Study to Evaluate Efficacy and Safety of One-year Maximum Dosage in Chinese Label of Imiglucerase Treatment in Chinese Patients Who Are Diagnosed as Gaucher Disease Type Ⅲ
Clinical Trial
  • Status: Not yet recruiting
  • Intervention Type: Diagnostic Test
  • Participants: 140
  • Start Date: September 30, 2020
Complement Activation in the Lysosomal Storage Disorders
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Drug, Biological
  • Participants: 15
  • Start Date: September 2020
An Open-label, Phase 1/2 Study to Evaluate the Safety and Efficacy of Single-dose PR001A in Infants With Type 2 Gaucher Disease
Clinical Trial
  • Status: Not yet recruiting
  • Participants: 300
  • Start Date: June 1, 2020
IIR REGISTRY for the Collection of Real World Data on the Safety and Efficacy of Ambroxol for Patients With Gaucher Disease or GBA Carriers With Parkinson Disease
Clinical Trial
  • Status: Recruiting
  • Phase: Phase 1/Phase 2
  • Intervention Type: Drug, Biological
  • Participants: 12
  • Start Date: January 3, 2020
A Phase 1/2a Open-Label Ascending Dose Study to Evaluate the Safety and Effects of PR001A in Patients With Parkinson's Disease With at Least One GBA1 Mutation
Clinical Trial
  • Status: Not yet recruiting
  • Phase: Phase 4
  • Intervention Type: Drug
  • Participants: 15
  • Start Date: November 20, 2019
A Multicenter, Safety and Efficacy Study of Taliglucerase Alfa in Subjects With Type 3 Gaucher Disease
Clinical Trial
  • Status: Recruiting
  • Participants: 24
  • Start Date: September 19, 2019
Canadian VPRIV® Non-Interventional Study in Patients Previously Treated With Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs)
Clinical Trial
  • Status: Recruiting
  • Participants: 180
  • Start Date: July 31, 2019
Natural History Study for Pediatric Patients With Early Onset of Either GM1 Gangliosidosis, GM2 Gangliosidoses, or Gaucher Disease Type 2
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