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

Overview

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.

Full Title of Study: “Phase I/II Gene Therapy Study for X-linked Chronic Granulomatous Disease in Children”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Single Group Assignment
    • Primary Purpose: Treatment
    • Masking: None (Open Label)
  • Study Primary Completion Date: December 2010

Interventions

  • Genetic: retroviral SF71-gp91phox transduced CD34+ cells
    • autologous ex-vivo transduced (SF71-gp91phox)CD34+ cells

Clinical Trial Outcome Measures

Primary Measures

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

Secondary Measures

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

Participating in This Clinical Trial

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)

Gender Eligibility: Male

Minimum Age: 1 Year

Maximum Age: 18 Years

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • University of Zurich
  • Collaborator
    • Goethe University
  • Provider of Information About this Clinical Study
    • Sponsor
  • Overall Official(s)
    • Reinhard Seger, Prof Dr med, Principal Investigator, University Children’s Hospital, Zurich
    • Janine Reichenbach, PD Dr med, Study Chair, University Children’s Hospital, Zurich
    • Ulrich Siler, Dr rer nat, Study Chair, University Children’s Hospital, Zurich
    • Manuel Grez, Dr rer nat, Study Chair, Georg Speyer Research Institute, Frankfurt a.M.

References

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.

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