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

September 26, 2011 updated by: University of Zurich

Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children

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.

Study Overview

Status

Completed

Conditions

Intervention / Treatment

Study Type

Interventional

Enrollment (Actual)

2

Phase

  • Phase 2
  • Phase 1

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • Switzerland
      • Zürich, Switzerland, CH-8032
        • University Children's Hospital

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Ages Eligible for Study

1 year to 18 years (ADULT, CHILD)

Accepts Healthy Volunteers

No

Genders Eligible for Study

Male

Description

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)

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: TREATMENT
  • Allocation: NON_RANDOMIZED
  • Interventional Model: SINGLE_GROUP
  • Masking: NONE

What is the study measuring?

Primary Outcome Measures

Outcome Measure
Time Frame
eradication of pre-existing therapy refractory bacterial and/or fungal infections
Time Frame: 6 months
6 months

Secondary Outcome Measures

Outcome Measure
Time Frame
Reconstitution of ROS production by peripheral blood cells
Time Frame: 1 month
1 month

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

University of Zurich

Collaborators

Goethe University

Investigators

  • Principal Investigator: Reinhard Seger, Prof Dr med, University Children's Hospital, Zurich
  • Study Chair: Janine Reichenbach, PD Dr med, University Children's Hospital, Zurich
  • Study Chair: Ulrich Siler, Dr rer nat, University Children's Hospital, Zurich
  • Study Chair: Manuel Grez, Dr rer nat, Georg Speyer Research Institute, Frankfurt a.M.

Publications and helpful links

The person responsible for entering information about the study voluntarily provides these publications. These may be about anything related to the study.

General Publications

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start

June 1, 2004

Primary Completion (ACTUAL)

December 1, 2010

Study Completion (ACTUAL)

September 1, 2011

Study Registration Dates

First Submitted

June 22, 2009

First Submitted That Met QC Criteria

June 23, 2009

First Posted (ESTIMATE)

June 24, 2009

Study Record Updates

Last Update Posted (ESTIMATE)

September 27, 2011

Last Update Submitted That Met QC Criteria

September 26, 2011

Last Verified

September 1, 2011

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

  • PedsZürich_GT05

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

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