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Completed

NCT Number: NCT00927134

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

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Key information

Conditions

Chronic Granulomatous Disease Chronic Disease Congenital, Hereditary, and Neonatal Diseases and Abnormalities Disease Attributes Genetic Diseases, Inborn Genetic Diseases, X-Linked Granulomatous Disease, Chronic Hematologic Diseases Hemic and Lymphatic Diseases Immune System Diseases Immunologic Deficiency Syndromes Leukocyte Disorders Pathologic Processes Pathological Conditions, Signs and Symptoms Phagocyte Bactericidal Dysfunction

Age range

1 year–18 year

Sex eligibility

Male

Study type

Interventional

Phase

Phase 1 / Phase 2

Primary location

University Children's Hospital

Zurich, CH-8032, Switzerland

Who can participate

Healthy volunteers accepted: No

Only the study team can determine whether someone qualifies for participation.

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)

Treatment and study plan

retroviral SF71-gp91phox transduced CD34+ cells

Genetic

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

Primary outcomes

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

    Time frame: 6 months

Secondary outcomes

  1. Reconstitution of ROS production by peripheral blood cells

    Time frame: 1 month

Sponsors and collaborators

Lead sponsor

University of Zurich

Other

Collaborators

  • Goethe University

Registry information

Official study title

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

Acronym: XCGDinChildren

Important dates

Study start
2004
Primary completion
2010
Study completion
2011
First posted
Jun 24, 2009
Registry last updated
Sep 27, 2011

OpenTrials presents study information sourced from ClinicalTrials.gov. The official registry record should be consulted for the latest information.

View the official ClinicalTrials.gov record (opens in a new tab)

This listing is for discovery and informational purposes only. It is not medical advice, does not guarantee that a study is recruiting, and does not determine eligibility. Contact the study team and a qualified healthcare professional when considering participation.

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