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

This study is currently recruiting participants.
Verified May 2017 by Prof. Dr. Arndt Rolfs, University of Rostock
Sponsor:
ClinicalTrials.gov Identifier:
NCT02416661
First Posted: April 15, 2015
Last Update Posted: May 5, 2017
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
Collaborator:
Centogene AG Rostock
Information provided by (Responsible Party):
Prof. Dr. Arndt Rolfs, University of Rostock
  Purpose
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
Lysosomal Storage Diseases Gaucher Disease Sphingolipidoses

Study Type: Observational
Study Design: 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

Resource links provided by NLM:


Further study details as provided by Prof. Dr. Arndt Rolfs, University of Rostock:

Primary Outcome Measures:
  • 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:
  • 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

Estimated Enrollment: 250
Study Start Date: March 2015
Estimated Study Completion Date: May 2019
Estimated Primary Completion Date: April 2019 (Final data collection date for primary outcome measure)
Groups/Cohorts
Observation
Patients with genetically confirmed diagnosis of Gaucher disease type 1 without treatment prior to enrollment

  Show Detailed Description

  Eligibility

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Ages Eligible for Study:   6 Months and older   (Child, Adult, Senior)
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
  Contacts and Locations
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
Contact: Arndt Rolfs, MD 49-381-494 ext 9540 arndt.rolfs@med.uni-rostock.de
Contact: Sabine Rösner 49-381-494 ext 4797 sabine.roesner@med.uni-rostock.de

Locations
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       ffadia_2007@yahoo.fr   
internal Medecine, Dr Mohammad-Lamine"sDebaghine, Universitary Hosspital Center,Bab-El-Oued Recruiting
Algiers, Algeria
Contact: Djennette Hakem, Prof.       hakem_dj@yahoo.fr   
Contact: Abdelkrim Berrah, Prof.         
Pediatric practice Recruiting
Oran, Algeria, 31000
Contact: Houaria Cheriet, MD       houaria26dz@yahoo.fr   
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.       efateen@yahoo.com   
Germany
Albrecht-Kossel-Institute for Neuroregeneration (AKos) Centre for Mental Health Disease University of Rostock Recruiting
Rostock, Germany, 18147
Contact: Arndt Rolfs, Prof.    +49 381 494 ext 9540    arndt.rolfs@med.uni-rostock.de   
Contact: Sabine Rösner    +49 381 494 ext 4797    sabine.roesner@med.uni-rostock.de   
Greece
Aristotle University of Thessaloniki-Department of Pediatrics Recruiting
Thessaloniki, Greece, 54622
Contact: Dimitrios Zafeiriou, MD    +30 2310 241 ext 845    jeff@med.auth.gr   
Principal Investigator: Dimitrios Zafeiriou, MD         
India
Senior Consultant Pediatrician Rainbow Children Hospital Recruiting
Hyderabad, India, 500034
Contact: Radha Rama Devi, MD       radharamadevi@gmail.com   
Kasturba Medical College, University of Manipal, 575001 Ambedkar (Jyothi) Circle Not yet recruiting
Munipal, India
Contact: Girisha Katta, MD       girish.katta@manipal.edu   
Principal Investigator: Girisha Katta, MD         
Division of Genetics, Department of Pediatrics, AIIMS,New Delhi,India Recruiting
New Delhi, India
Contact: Madhulika Kabra, MD       madhulikakabra@hotmail.com   
Contact: Neerja Gupta, MD       neerja17@gmail.com, neerja17aiims@gmail.com   
Principal Investigator: Madhulika Kabra, MD         
Iran, Islamic Republic of
Sarem Women Hospital - Metabolic and Genetics Department Recruiting
Tehran, Iran, Islamic Republic of
Contact: Zahra Hadipour, MD    +98 (21) 446 70 888 ext 1515    f.ashuo@yahoo.com   
Contact: Fatemeh Hadipou, MD    +98 (21) 446 70 888 ext 1515    f.ashuo@yahoo.com   
Israel
Shaare Zedek Medical Center Recruiting
Jerusalem, Israel, 93722
Contact: Ari Zimran, MD       azimran@gmail.com   
Contact: Tama Dinur, MD       dinurtama@gmail.com   
Principal Investigator: Ari Zimran, MD         
Mexico
División de Genética Recruiting
Guadalajara, Mexico, 44340
Contact: José Elias García-Ortiz, MD       jose.elias.garcia@gmail.com   
Morocco
CHU Averroès Casablanca Recruiting
Casablanca, Morocco
Contact: Mina Moudatir, MD       minamoudatir@gmail.com   
Children hospital's Rabat Recruiting
Rabat, Morocco, 10100
Contact: Nezha Mouane, Prof.       nezhamouane@hotmail.com   
Contact: Yamna Kriouile, Prof.       dr.kriouile@gmail.com   
Russian Federation
National Research Center for Hematology Recruiting
Moscow, Russian Federation, 125167
Contact: Elena Lukina, Prof.       elenalukina02@gmail.com   
Contact: Kira Lukina, MD       kira1404@gmail.com   
Sweden
Karolinska University, Hospital Huddinge, Hematology Center Recruiting
Stockholm, Sweden, 141 86
Contact: Maciej Machaczka, Prof.       maciej.machaczka@ki.se   
Tunisia
Hospital Hedi Chaker Recruiting
Sfax, Tunisia, 1008
Contact: Hadji Mseddi Sondes       mseddi_sondes@medecinesfax.org   
Venezuela
Av. Pateón, Centro Clínico Profesional Caracas Recruiting
Caracas, Venezuela
Contact: Liliana Vera, Prof.       lilive29@yahoo.com   
Sponsors and Collaborators
University of Rostock
Centogene AG Rostock
Investigators
Principal Investigator: Arndt Rolfs, MD University of Rostock, Albrecht-Kossel-Institute for Neuroregeneration
  More Information

Additional Information:
Responsible Party: Prof. Dr. Arndt Rolfs, Prof. Dr. med., University of Rostock
ClinicalTrials.gov Identifier: NCT02416661     History of Changes
Other Study ID Numbers: LP 03-2015
First Submitted: March 13, 2015
First Posted: April 15, 2015
Last Update Posted: May 5, 2017
Last Verified: May 2017
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Undecided

Keywords provided by Prof. Dr. Arndt Rolfs, University of Rostock:
Gaucher Disease type 1
Lymphatic Diseases
Lipid Metabolism, Inborn Errors
Lipid Metabolism Disorders
Lipidoses

Additional relevant MeSH terms:
Gaucher Disease
Lysosomal Storage Diseases
Sphingolipidoses
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