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Report for D005776: Gaucher Disease NIH
(Synonyms: Gaucher Disease)
Developed by Shray Alag
Clinical Trial MeSH HPO Drug Gene SNP Protein Mutation
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There are 2 clinical trials
Clinical Trials
1 Gaucher Disease Outcome Survey (GOS)
The Gaucher Outcomes Survey (GOS) is an ongoing observational, international, multi-center, long-term Registry of Patients with Gaucher Disease irrespective of their treatment status or type of treatment received. No experimental intervention is involved. Patients undergo clinical assessments and receive care as determined by the patients' treating physician. The objectives of the registry include to evaluate the safety and long-term effectiveness of velaglucerase alfa, to characterize patients receiving velaglucerase alfa or other Gaucher Disease-specific treatments, to gain a better understanding of the natural history of GD and to serve as a database for evidence-based management of Gaucher Disease over time in real-life clinical practice.
NCT03291223 Gaucher Disease MeSH:Gaucher Disease
Primary Outcomes
Description: Treatment-emergent adverse events (TEAEs) are defined as adverse events (AEs) that either commenced or worsened following the first dose of VPRIV.
Measure: Number of Participants With Treatment-emergent Adverse Events (AEs) and Serious Adverse Events (SAEs) Time: Baseline to one year for up to 20 yearsDescription: An IRR is defined as an AE that has been assessed as at least possibly related to treatment with VPRIV and occurs during an infusion or up to 24 hours post-VPRIV infusion.
Measure: Number of Participants With Infusion-related Reactions (IRRs) Time: Baseline to one year for up to 20 yearsDescription: Hemoglobin concentration will be assessed.
Measure: Increase of Hemoglobin Concentration Time: Baseline to one year for up to 20 yearsDescription: Platelet count will be assessed.
Measure: Increase of Platelet Count Time: Baseline to one year for up to 20 yearsDescription: Liver volume will be assessed by abdominal imaging.
Measure: Decrease in Liver Volume Time: Baseline to one year for up to 20 yearsDescription: Spleen volume will be assessed by abdominal imaging.
Measure: Decrease in Spleen Volume Time: Baseline to one year for up to 20 yearsDescription: Bone mineral density will be assessed.
Measure: Increase in Bone Mineral Density (BMD) Time: Baseline to one year for up to 20 years2 Canadian VPRIV® Non-Interventional Study in Patients Previously Treated With Other Enzyme Replacement Therapies (ERTs)/Substrate Reduction Therapies (SRTs)
The purpose of the study is to assess safety of velaglucerase alfa (VPRIV) in participants affected with Gaucher disease type 1 (GD1) transitioning from other enzyme replacement therapies (ERTs)/ substrate reduction therapies (SRTs) to VPRIV in a Canadian real-world setting across all age groups.
NCT04094181 Gaucher Disease MeSH:Gaucher Disease
Primary Outcomes
Description: An AE is any untoward medical occurrence in a clinical investigation participant administered a pharmaceutical product and that does not necessarily have a causal relationship with this treatment. Number of participants transitioning from other ERTs/SRTs to VPRIV with AEs will be reported.
Measure: Number of Participants with Adverse Events (AEs) Following the Transition From Other ERTs/SRTs to VPRIV Time: Baseline up to 12 monthsSecondary Outcomes
Description: Change in use of Lyso-Gb1 by participants following the transition from other ERTs/SRTs to velaglucerase alfa (VPRIV) will be assessed.
Measure: Change From Baseline in Use of Glucosylspingosine (Lyso-Gb1) Biomarker Time: Baseline, Month 12Description: Gaucher disease questionnaire (PRO) scores will be calculated for each adult participant (greater than or equal to [>=] 18 years). The questionnaire asks how much the participant is affected by the Gaucher disease. A total summated score is calculated and ranges from 0 to 100 with higher scores indicating more severe impairment and worse quality of life.
Measure: Change From Baseline in Gaucher Disease Questionnaire Score at Month 12 Time: Baseline, Month 12