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

University of Zurich2 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) 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.

Study Type

INTERVENTIONAL

Allocation

NON_RANDOMIZED

Purpose

TREATMENT

Masking

NONE

Enrollment

Conditions

Chronic Granulomatous Disease

Interventions

retroviral SF71-gp91phox transduced CD34+ cellsGENETIC

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

Eligibility

Sex: MALEMin age: 1 YearMax 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 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)

Locations (1)

University Children's Hospital

Zurich, Switzerland

Outcomes

Primary Outcomes

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

Time frame: 6 months

Secondary Outcomes

Reconstitution of ROS production by peripheral blood cells

Time frame: 1 month

Data from ClinicalTrials.gov

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