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Trial record 5 of 103 for:    Gaucher Disease

Auto-antibodies Prevalence and CD1 Role in Gaucher Disease

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ClinicalTrials.gov Identifier: NCT02650219
Recruitment Status : Completed
First Posted : January 8, 2016
Results First Posted : February 9, 2016
Last Update Posted : March 23, 2016
Sponsor:
Information provided by (Responsible Party):
Christine Serratrice, Hospital St. Joseph, Marseille, France

Study Type: Observational
Study Design: Observational Model: Case Control;   Time Perspective: Prospective
Condition: Gaucher Disease
Intervention: Genetic: genetic analyses

  Participant Flow

Recruitment Details
Key information relevant to the recruitment process for the overall study, such as dates of the recruitment period and locations
from January 2010 to April 2011, 40 GD1 patients and 20 healthy volunteers (control group) were included in the study in the 12 inclusions centers (medical clinic or hopsital)

Pre-Assignment Details
Significant events and approaches for the overall study following participant enrollment, but prior to group assignment
No text entered.

Reporting Groups
  Description
Gaucher Disease Type 1

Inclusion criteria:

  • Adult patients >= 18 years old
  • Gaucher disease type 1, proved by low betaglucosidase, with or without treatment
  • Patients must have read, understood and signed informed consent. intervention : genetic analyses

genetic analyses

Control

healthy subjects intervention: genetic analyses

genetic analyses


Participant Flow:   Overall Study
    Gaucher Disease Type 1   Control
STARTED   40   20 
COMPLETED   40   20 
NOT COMPLETED   0   0 



  Baseline Characteristics

Population Description
Explanation of how the number of participants for analysis was determined. Includes whether analysis was per protocol, intention to treat, or another method. Also provides relevant details such as imputation technique, as appropriate.
No text entered.

Reporting Groups
  Description
Gaucher Disease Type 1

Inclusion criteria:

  • Adult patients >= 18 years old
  • Gaucher disease type 1, proved by low betaglucosidase, with or without treatment
  • Patients must have read, understood and signed informed consent. intervention : genetic analyses

genetic analyses

Control

healthy subjects intervention: genetic analyses

genetic analyses

Total Total of all reporting groups

Baseline Measures
   Gaucher Disease Type 1   Control   Total 
Overall Participants Analyzed 
[Units: Participants]
 40   20   60 
Age 
[Units: Participants]
     
<=18 years   0   0   0 
Between 18 and 65 years   31   17   48 
>=65 years   9   3   12 
Age 
[Units: YEARS]
Mean (Standard Deviation)
 52.4  (13.4)   47.3  (15.4)   50.7  (14.2) 
Gender 
[Units: Participants]
     
Female   22   13   35 
Male   18   7   25 
Region of Enrollment 
[Units: Participants]
     
France   40   20   60 


  Outcome Measures

1.  Primary:   Number of Patients With GD Diagnosis Confirmed by : Enzyme Testing of acidβ-glucosidase Activity Activity <15% in Blood Leucocytes Completed When Necsssary by GB1 Mutation Analyses (Analyses From Samples)   [ Time Frame: baseline ]

2.  Secondary:   Number of Patients With : Splenectomy and/or Bone Events and/or Pulmonary Hypertension and/or Specific Treatment and Non-specific (Medical History,Physiological Parameters and Questionnaire)   [ Time Frame: Baseline ]

3.  Secondary:   Number of Patients With : Antinuclear and/or Anti-SSa and/or Anti-SSb and/or Anti-RNP and/or Anti-DNA and/or Anti-Sm and/or Anticardiolipid and/or Anti β2Gp1 and/or Antiganglioside Autoantibodies (Genetics Analyses From Blood Samples)   [ Time Frame: baseline ]

4.  Secondary:   Number of Patients With : Photosensitivity and/or Raynaud Phenomenon and/or Sicca Syndrome and/or Arthralgia and/or Arthritis and/or Thrombosis (Medical History and Questionnaire)   [ Time Frame: Baseline ]
Results not yet reported.   Anticipated Reporting Date:   No text entered.  


  Serious Adverse Events


  Other Adverse Events


  Limitations and Caveats

Limitations of the study, such as early termination leading to small numbers of participants analyzed and technical problems with measurement leading to unreliable or uninterpretable data
No text entered.


  More Information

Certain Agreements:  
Principal Investigators are NOT employed by the organization sponsoring the study.
There is NOT an agreement between Principal Investigators and the Sponsor (or its agents) that restricts the PI's rights to discuss or publish trial results after the trial is completed.


Results Point of Contact:  
Name/Title: Dr Christine Serratrice
Organization: St Joseph Hospital Marseille
phone: 0041798731474
e-mail: cserratrice@hotmail.fr



Responsible Party: Christine Serratrice, Hospital St. Joseph, Marseille, France
ClinicalTrials.gov Identifier: NCT02650219     History of Changes
Other Study ID Numbers: ID-RCB 2010-A00315-3
First Submitted: January 5, 2016
First Posted: January 8, 2016
Results First Submitted: January 7, 2016
Results First Posted: February 9, 2016
Last Update Posted: March 23, 2016