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LYSO-PROVE - Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease (LYSO-PROVE)

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ClinicalTrials.gov Identifier: NCT02416661
Recruitment Status : Recruiting
First Posted : April 15, 2015
Last Update Posted : April 30, 2019
Sponsor:
Information provided by (Responsible Party):
Centogene AG Rostock

Brief Summary:
This study should demonstrate the correlation and predictive value of lyso-Gb1 concentration with the clinical severity of naïve, initially non-ERT/SRT Gaucher disease type 1 and during the study ERT/SRT-newly started Gaucher type 1 patients

Condition or disease
Lysosomal Storage Diseases Gaucher Disease Sphingolipidoses

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Study Type : Observational
Estimated Enrollment : 250 participants
Observational Model: Cohort
Time Perspective: Prospective
Official Title: Lyso-Gb1 as Long-term Prognostic Biomarker in Gaucher Disease - an International Multicenter Epidemiological Study (LYSO-PROVE) to Determine the Prognostic Value of Lyso-Gb1 for Monitoring the Progress of Gaucher Disease
Study Start Date : March 2015
Estimated Primary Completion Date : April 2022
Estimated Study Completion Date : May 2022


Group/Cohort
Observation
Patients with genetically confirmed diagnosis of Gaucher disease type 1 without treatment prior to enrollment



Primary Outcome Measures :
  1. To demonstrate the correlation and predictive value of lyso-Gb1 concentration with the clinical severity of naïve, initially non-ERT/SRT Gaucher disease type 1 and during the study ERT/SRT-newly started Gaucher type 1 patients [ Time Frame: 48 month ]

Secondary Outcome Measures :
  1. To correlate lyso-Gb1 concentration with the clinical improvement of ERT or SRT treated Gaucher type 1 and the clinical course of non-treated patients based on GD-DS3,Quality of life measured with the SF-36 [ Time Frame: 48 month ]

Biospecimen Retention:   Samples With DNA
lyso-Gb1 as biomarker for Gaucher Disease For the purpose of measuring lyso-Gb1 one filter card is sent to the following laboratory, Centogene AG, Schillingallee 68, 18057 Rostock, Germany


Information from the National Library of Medicine

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Ages Eligible for Study:   6 Months and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Sampling Method:   Probability Sample
Study Population
male or female patients aged 6 months or older with genetically confirmed diagnosis of Gaucher disease type 1 without treatment prior to enrollment or no treatment for more than 24 months ago
Criteria

Inclusion Criteria:

  • Male or female patients aged 6 months or older
  • Patients with genetically confirmed diagnosis of Gaucher disease type 1
  • No prior treatment with enzyme replacement therapy or substrate reduction therapy ro no traetment for more than 24 months
  • Signed informed consent by parents/legal guardian and patient

Exclusion Criteria:

  • Male or female patients being younger than 6 months
  • Patients without genetically confirmed diagnosis of Gaucher disease type 1
  • Gaucher disease 2 or 3
  • Patient is currently undergoing enzyme replacement therapy or substrate reduction therapy
  • Missing signed informed consent

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT02416661


Contacts
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Contact: Volha Skrahina, Dr +4938180113594 ext 594 volha.skrahina@centogene.com

Locations
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Sponsors and Collaborators
Centogene AG Rostock
Investigators
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Principal Investigator: Arndt Rolfs, Prof Centogene AG Rostock

Additional Information:
Publications automatically indexed to this study by ClinicalTrials.gov Identifier (NCT Number):
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Responsible Party: Centogene AG Rostock
ClinicalTrials.gov Identifier: NCT02416661    
Other Study ID Numbers: LP 06-2018
First Posted: April 15, 2015    Key Record Dates
Last Update Posted: April 30, 2019
Last Verified: April 2019
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided
Keywords provided by Centogene AG Rostock:
Gaucher Disease type 1
Lymphatic Diseases
Lipid Metabolism, Inborn Errors
Lipid Metabolism Disorders
Lipidoses
Additional relevant MeSH terms:
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Gaucher Disease
Sphingolipidoses
Lysosomal Storage Diseases
Lysosomal Storage Diseases, Nervous System
Brain Diseases, Metabolic, Inborn
Brain Diseases, Metabolic
Brain Diseases
Central Nervous System Diseases
Nervous System Diseases
Metabolism, Inborn Errors
Genetic Diseases, Inborn
Lipidoses
Lipid Metabolism, Inborn Errors
Metabolic Diseases
Lipid Metabolism Disorders