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Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children (XCGDinChildren)

Primary Purpose

Chronic Granulomatous Disease

Status
Completed
Phase
Phase 1
Locations
Switzerland
Study Type
Interventional
Intervention
retroviral SF71-gp91phox transduced CD34+ cells
Sponsored by
University of Zurich
About
Eligibility
Locations
Outcomes
Full info

About this trial

This is an interventional treatment trial for Chronic Granulomatous Disease focused on measuring Chronic Granulomatous Disease in children (=or< 18 years)

Eligibility Criteria

1 Year - 18 Years (Child, Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • x-linked Chronic Granulomatous Disease
  • history of life-threatening severe infections
  • no HLA-matched related or unrelated donor
  • therapy resistent life threatening infections/organ dysfunction
  • no other treatment options e.g. HSCT

Exclusion Criteria:

  • > 18 years of age
  • HIV infection
  • life expectancy > 2 years
  • infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)

Sites / Locations

  • University Children's Hospital

Outcomes

Primary Outcome Measures

eradication of pre-existing therapy refractory bacterial and/or fungal infections

Secondary Outcome Measures

Reconstitution of ROS production by peripheral blood cells

Full Information

First Posted
June 22, 2009
Last Updated
September 26, 2011
Sponsor
University of Zurich
Collaborators
Goethe University
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1. Study Identification

Unique Protocol Identification Number
NCT00927134
Brief Title
Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children
Acronym
XCGDinChildren
Official Title
Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children
Study Type
Interventional

2. Study Status

Record Verification Date
September 2011
Overall Recruitment Status
Completed
Study Start Date
June 2004 (undefined)
Primary Completion Date
December 2010 (Actual)
Study Completion Date
September 2011 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
University of Zurich
Collaborators
Goethe University

4. Oversight

Data Monitoring Committee
No

5. Study Description

Brief Summary
The aim of the study is to evaluate the side effects and risks after infusion of retroviral gene corrected autologous CD34+ cells of the peripheral blood of chemotherapy conditioned (busulphan) children with chronic granulomatous disease (CGD). Also gene corrected and functional active granulocytes in the peripheral blood and the engraftment in the bone marrow of the patients will be monitored an documented.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Chronic Granulomatous Disease
Keywords
Chronic Granulomatous Disease in children (=or< 18 years)

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Single Group Assignment
Masking
None (Open Label)
Allocation
Non-Randomized
Enrollment
2 (Actual)

8. Arms, Groups, and Interventions

Intervention Type
Genetic
Intervention Name(s)
retroviral SF71-gp91phox transduced CD34+ cells
Intervention Description
autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells
Primary Outcome Measure Information:
Title
eradication of pre-existing therapy refractory bacterial and/or fungal infections
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Reconstitution of ROS production by peripheral blood cells
Time Frame
1 month

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
1 Year
Maximum Age & Unit of Time
18 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: x-linked Chronic Granulomatous Disease history of life-threatening severe infections no HLA-matched related or unrelated donor therapy resistent life threatening infections/organ dysfunction no other treatment options e.g. HSCT Exclusion Criteria: > 18 years of age HIV infection life expectancy > 2 years infections treatable by conventional therapy (antibiotics, antimycotics, allogeneic granulocytes)
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Reinhard Seger, Prof Dr med
Organizational Affiliation
University Children's Hospital, Zurich
Official's Role
Principal Investigator
First Name & Middle Initial & Last Name & Degree
Janine Reichenbach, PD Dr med
Organizational Affiliation
University Children's Hospital, Zurich
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Ulrich Siler, Dr rer nat
Organizational Affiliation
University Children's Hospital, Zurich
Official's Role
Study Chair
First Name & Middle Initial & Last Name & Degree
Manuel Grez, Dr rer nat
Organizational Affiliation
Georg Speyer Research Institute, Frankfurt a.M.
Official's Role
Study Chair
Facility Information:
Facility Name
University Children's Hospital
City
Zürich
ZIP/Postal Code
CH-8032
Country
Switzerland

12. IPD Sharing Statement

Citations:
PubMed Identifier
16582916
Citation
Ott MG, Schmidt M, Schwarzwaelder K, Stein S, Siler U, Koehl U, Glimm H, Kuhlcke K, Schilz A, Kunkel H, Naundorf S, Brinkmann A, Deichmann A, Fischer M, Ball C, Pilz I, Dunbar C, Du Y, Jenkins NA, Copeland NG, Luthi U, Hassan M, Thrasher AJ, Hoelzer D, von Kalle C, Seger R, Grez M. Correction of X-linked chronic granulomatous disease by gene therapy, augmented by insertional activation of MDS1-EVI1, PRDM16 or SETBP1. Nat Med. 2006 Apr;12(4):401-9. doi: 10.1038/nm1393. Epub 2006 Apr 2.
Results Reference
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Gene Therapy for X-linked Chronic Granulomatous Disease (CGD) in Children

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