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Found 40 clinical trials
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A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients

A Phase 1/2, Open Label, Dose Ranging Study to Evaluate the Safety,Tolerability, Pharmacokinetics and Exploratory Efficacy Parameters of PRX-102 Administered by Intravenous Infusion Every 2 Weeks for 12 Weeks to Adult Fabry Patients

  • 184 views
  • 08 Nov, 2020
  • 1 location
None
RGX-121 Gene Therapy in Children 5 Years of Age and Over With MPS II (Hunter Syndrome)

RGX-121 is a gene therapy which is designed to deliver a functional copy of the iduronate-2-sulfatase (IDS) gene to the central nervous system. This study is a phase I/II study to determine whether RGX-121 is safe, well tolerated, and potentially effective in children five years of age and over who …

  • 8 views
  • 03 Oct, 2021
  • 2 locations
None
Clinical Study for IOPD Participants Less Than or Equal to 6 Months of Age to Evaluate Efficacy and Safety of Enzyme Replacement Therapy (ERT) With Avalglucosidase Alfa

This is a single group, treatment, Phase 3, open-label study to assess efficacy, safety, pharmacokinetic (PK), pharmacodynamics (PD) of avalglucosidase alfa in male and female participants less than or equal to 6 months of age with IOPD. Study details include: Study duration: Screening - up to 4 weeks; Primary Analysis …

deficiency
replacement therapy
  • 0 views
  • 12 Sep, 2021
  • 4 locations
None
A Gene Transfer Study for Late-Onset Pompe Disease (RESOLUTE)

receiving enzyme replacement therapy (ERT). Participants will be treated in sequential, dose-level cohorts.

replacement therapy
gene therapy
  • 29 views
  • 25 Jul, 2021
  • 21 locations
None
ZIP Study - A Study of the Safety Pharmacokinetics Efficacy Pharmacodynamics and Immunogenicity of ATB200/AT2221 in Pediatric Subjects Aged 0 to < 18 Years With Pompe Disease

This is a Phase 3, open-label, multicenter study to evaluate the safety, PK, efficacy, PD, and immunogenicity of ATB200/AT2221 treatment in enzyme replacement therapy (ERT)-experienced and ERT

replacement therapy
alglucosidase alfa
  • 17 views
  • 24 Oct, 2021
  • 14 locations
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
A Study of Enzyme Replacement Therapy (VPRIV) in People With Type 1 Gaucher Disease Who Were Previously Treated With Substrate Reduction Therapy

clinic will record what happens during the study. VPRIV is a type of enzyme replacement therapy (also known as ERT). Before starting the study, participants must either have switched from

velaglucerase alfa
substrate reduction therapy
type 1 gaucher disease
replacement therapy
vpriv
  • 0 views
  • 22 May, 2021
  • 1 location
None
Evaluation of Safety Tolerability and Efficacy of INZ-701 in Adults With ENPP1 Deficiency

The purpose of this study is to assess the safety and tolerability of INZ-701, an ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) enzyme replacement therapy, for the treatment of

  • 0 views
  • 30 Sep, 2021
  • 2 locations
None
Glycerol Phenylbutyrate Corrector Therapy For CF (Cystic Fibrosis)

We propose to test the effectiveness of the combination of CF pancreatic enzyme replacement therapy (PERT) on absorption of Ravicti and subsequent restoration of nasal epithelial cystic fibrosis

oximetry
nasal potential difference
antibiotics
spirometry
bronchodilator
  • 27 views
  • 17 Mar, 2021
  • 4 locations
None
FAB- GT Open-Label Study Of Efficacy and Safety Of AVR-RD-01 for Treatment -Naive Subjects With Classic Fabry Disease

postpubertal with a confirmed diagnosis of classic Fabry disease based on deficient AGA enzyme activity who have not previously received treatment with enzyme replacement therapy (ERT) and/or chaperone therapy

replacement therapy
alpha-galactosidase
  • 404 views
  • 26 Jan, 2021
  • 5 locations