Gene Therapy for Chronic Granulomatous Disease

Johann Wolfgang Goethe University Hospital2 enrolled

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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Masking

NONE

Enrollment

Conditions

Granulomatous Disease, Chronic

Interventions

retroviral SF71-gp91phox transduced CD34+ cellsDRUG

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

Eligibility

Sex: MALEMin age: 18 Years

Medical Language ↔ Plain English

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)

Locations (1)

University Hospital, Hematology

Frankfurt, Germany

Outcomes

Primary Outcomes

safety, toxicity and feasibility

Time frame: 2 years

Secondary Outcomes

Engraftment of gene corrected stem cells, functional reconstitution of respiratory burst, clinical benefit

Time frame: 2 years

Data from ClinicalTrials.gov

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