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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

  Show Detailed Description

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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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Algeria
Pediatric department Debaghine's Hospital Universitary Hospital Center of Bab-El-Oued Recruiting
Algiers, Algeria, 16030
Contact: Souhila Melzi, MD         
Hôpital de Ben Aknoun BOUKHROUFA Abdelkader Service Rhumatologie Recruiting
Algiers, Algeria, 16306
Contact: Fadia Rahal, MD         
Universitary Hosspital Center,Bab-El-Oued Recruiting
Algiers, Algeria
Contact: Djennette Hakem, Prof.         
Contact: Abdelkrim Berrah, Prof.         
Pediatric practice Recruiting
Oran, Algeria, 31000
Contact: Houaria Cheriet, MD         
Principal Investigator: Houaria Cheriet, MD         
Service medicine interne EPH Bouguera Boulares Bekkaria Recruiting
Tébessa, Algeria, 12130
Contact: Souhlia Moundher, MD         
Egypt
Gene Lab Recruiting
Cairo, Egypt
Contact: Ekram Fateen, Prof.         
Germany
Klinikum rechts der Isar der TU Recruiting
München, Germany
Contact: Claudia Regenbogen, Dr.         
India
Amrita Institute of Medical Sciences and Research Centre Recruiting
Cochin, India
Contact: Sheela Nampoothiri, Prof.         
Rainbow Children Hospital Recruiting
Hyderabad, India, 500034
Contact: Radha Rama Devi, MD         
Iran, Islamic Republic of
Sarem Women Hospital - Metabolic and Genetics Department Recruiting
Tehran, Iran, Islamic Republic of
Contact: Zahra Hadipour, MD         
Contact: Fatemeh Hadipou, MD         
Israel
Emek Medical Center Recruiting
Afula, Israel
Contact: Efrat Shalev, Dr.         
Shaare Zedek Medical Center Recruiting
Jerusalem, Israel, 93722
Contact: Ari Zimran, MD         
Contact: Tama Dinur, MD         
Principal Investigator: Ari Zimran, MD         
Mexico
División de Genética Not yet recruiting
Guadalajara, Mexico, 44340
Contact: José Elias García-Ortiz, MD         
Morocco
Children hospital's Rabat Recruiting
Rabat, Morocco, 10100
Contact: Nezha Mouane, Prof.         
Contact: Yamna Kriouile, Prof.         
Hopital d'Enfant Recruiting
Rabat, Morocco
Contact: Yamna Kriouile, Prof         
Pakistan
The Children's Hospital and the Institute of Child Health Recruiting
Lahore, Pakistan
Contact: Huma Cheema, Prof.         
Russian Federation
National Research Center for Hematology Recruiting
Moscow, Russian Federation, 125167
Contact: Elena Lukina, Prof.         
Contact: Kira Lukina, MD         
Spain
FETEEG Recruiting
Zaragoza, Spain
Contact: Pilar Giraldo, Dr         
Sweden
Karolinska University, Hospital Huddinge, Hematology Center Not yet recruiting
Stockholm, Sweden, 141 86
Contact: Maciej Machaczka, Prof.         
Tunisia
Hospital Hedi Chaker Recruiting
Sfax, Tunisia, 1008
Contact: Hadji Mseddi Sondes         
Venezuela
Av. Pateón, Centro Clínico Profesional Caracas Not yet recruiting
Caracas, Venezuela
Contact: Liliana Vera, Prof.         
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     History of Changes
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