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

NCT00927134 · Status: COMPLETED · Phase: PHASE1/PHASE2 · Type: INTERVENTIONAL · Enrollment: 2

Last updated 2011-09-27

No results posted yet for this study

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.

Conditions

  • Chronic Granulomatous Disease

Interventions

GENETIC

retroviral SF71-gp91phox transduced CD34+ cells

autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells

Sponsors & Collaborators

  • Goethe University

    collaborator OTHER

  • University of Zurich

    lead OTHER

Principal Investigators

  • Reinhard Seger, Prof Dr med · University Children's Hospital, Zurich

  • Janine Reichenbach, PD Dr med · University Children's Hospital, Zurich

  • Ulrich Siler, Dr rer nat · University Children's Hospital, Zurich

  • Manuel Grez, Dr rer nat · Georg Speyer Research Institute, Frankfurt a.M.

Study Design

Allocation
NON_RANDOMIZED
Purpose
TREATMENT
Masking
NONE
Model
SINGLE_GROUP

Eligibility

Min Age
1 Year
Max Age
18 Years
Sex
MALE
Healthy Volunteers
No

Timeline & Regulatory

Start
2004-06-30
Primary Completion
2010-12-31
Completion
2011-09-30

Countries

  • Switzerland

Study Locations

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Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT00927134 on ClinicalTrials.gov