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Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease (MGMC)

Primary Purpose

Gaucher Disease

Status
Completed
Phase
Not Applicable
Locations
France
Study Type
Interventional
Intervention
Bone marrow and blood sampling
Sponsored by
Assistance Publique - Hôpitaux de Paris
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional basic science trial for Gaucher Disease focused on measuring Gaucher disease

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

  • age > 18 years
  • Gaucher disease
  • clinical examination written consent

Exclusion Criteria:

  • pregnancy
  • HIV seropositive
  • contraindication to bone marrow sampling

Sites / Locations

  • Bertrand Arnulf

Arms of the Study

Arm 1

Arm Type

Experimental

Arm Label

Bone marrow and blood sampling

Arm Description

Outcomes

Primary Outcome Measures

stem cells

Secondary Outcome Measures

Full Information

First Posted
June 22, 2011
Last Updated
May 22, 2014
Sponsor
Assistance Publique - Hôpitaux de Paris
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1. Study Identification

Unique Protocol Identification Number
NCT01439607
Brief Title
Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease
Acronym
MGMC
Official Title
Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells, Macrophages and Plasma Cell Biology in Gaucher Disease.
Study Type
Interventional

2. Study Status

Record Verification Date
April 2014
Overall Recruitment Status
Completed
Study Start Date
December 2009 (undefined)
Primary Completion Date
February 2012 (Actual)
Study Completion Date
March 2012 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Assistance Publique - Hôpitaux de Paris

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
Gaucher disease is an inherited autosomal recessive lysosomal storage disorder caused by the defective activity of the glucocerebrosidase, leading to accumulation of glucocerebroside particularly in cells of the macrophage lineage. Clinical manifestations associate hematological, neurological and bone disorders.
Detailed Description
Gaucher disease is an inherited autosomal recessive lysosomal storage disorder caused by the defective activity of the glucocerebrosidase, leading to accumulation of glucocerebroside particularly in cells of the macrophage lineage. Clinical manifestations associate hematological, neurological and bone disorders. In this project, the investigators aimed to study the cellular and molecular mechanisms governing the biology of various cell populations of the bone marrow and blood in these patients in order to define their potential role in the physiopathology of the disease. The main research topics the investigators plan will then be focused on characterization and functional analysis of hematopoietic stem cells, mesenchymal stem cells (with a special focus on osteogenic differentiation), macrophages and osteoclastic differentiation, B lymphocytes and plasma cells.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Gaucher Disease
Keywords
Gaucher disease

7. Study Design

Primary Purpose
Basic Science
Study Phase
Not Applicable
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
N/A
Enrollment
30 (Actual)

8. Arms, Groups, and Interventions

Arm Title
Bone marrow and blood sampling
Arm Type
Experimental
Intervention Type
Other
Intervention Name(s)
Bone marrow and blood sampling
Intervention Description
Bone marrow and blood sampling
Primary Outcome Measure Information:
Title
stem cells
Time Frame
day 1

10. Eligibility

Sex
All
Minimum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: age > 18 years Gaucher disease clinical examination written consent Exclusion Criteria: pregnancy HIV seropositive contraindication to bone marrow sampling
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Bertrabd Arnulf, MD
Organizational Affiliation
Assistance Publique - Hôpitaux de Paris
Official's Role
Principal Investigator
Facility Information:
Facility Name
Bertrand Arnulf
City
Paris
ZIP/Postal Code
75010
Country
France

12. IPD Sharing Statement

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Cellular and Molecular Mechanisms Governing Bone Marrow Stem Cells in Gaucher Disease

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