Gene Therapy for Chronic Granulomatous Disease

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)patients 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”

Study Type

  • Study Type: Interventional
  • Study Design
    • Allocation: Non-Randomized
    • Intervention Model: Single Group Assignment
    • Masking: None (Open Label)

Interventions

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

Clinical Trial Outcome Measures

Primary Measures

  • safety, toxicity and feasibility
    • Time Frame: 2 years

Secondary Measures

  • Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit
    • Time Frame: 2 years

Participating in This Clinical Trial

Inclusion Criteria

  • x-linked Chronic Granulomatous Disease – history of life-threatening severe infections – no HLA-matched related or non-related donor – therapy resistent life threatening infections/organ dysfunction – no other treatment options e.g. BMT Exclusion Criteria:

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

Gender Eligibility: Male

Minimum Age: 18 Years

Maximum Age: N/A

Are Healthy Volunteers Accepted: No

Investigator Details

  • Lead Sponsor
    • Johann Wolfgang Goethe University Hospital
  • Collaborator
    • German Federal Ministry of Education and Research
  • Overall Official(s)
    • Dieter Hoelzer, MD, PhD, Principal Investigator, Goethe University

Citations Reporting on Results

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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