Gene therapy with autologous genetically-modified CD34+ cells for X-linked Chronic Granulomatous Disease (X-CGD)

Conditions: X-linked Chronic Granulomatous Disease
MedDRA version: 14.1Level: PTClassification code 10008906Term: Chronic granulomatous diseaseSystem Organ Class: 10010331 - Congenital, familial and genetic disorders
Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Registration Number: EUCTR2012-000242-35-DE

Lead Sponsor: GENETHO

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: Authorised-recruitment may be ongoing or finished

Sex: Male

Target Recruitment: 20

Inclusion Criteria

-Male X-CGD patients > 23 months of age
-Molecular diagnosis confirmed by DNA sequencing and supported by laboratory evidence for absent or reduction > 70% of the biochemical activity of the NAHPD-oxidase
-At least one ongoing or resistant severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy
-No HLA-matched donor available after 3 months search
-No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBsAg positive) or hepatitis C virus (HCV RNA positive)
-Written informed consent for adult patient
-Parental/guardian and where appropriate child’s signed consent/assent

Are the trial subjects under 18? yes
Number of subjects for this age range: 15
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 5
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range 0

Exclusion Criteria

-10/10 HLA identical (A,B,C,DR,DQ) family or unrelated or cord blood donor unless there is deemed to be an unacceptable risk associated with an allogeneic procedure
-Contraindication for leukapheresis (anaemia Hb <8g/dl, cardiovascular instability, severe coagulopathy)
-Contraindication for administration of conditioning medication
-Administration of gammainterferon within 30 days before the infusion of transduced autologous CD34+ cells
-Participation in another experimental therapeutic protocol within 6 months prior to baseline and during the study period
-Any other condition that, in the opinion of the Investigator, may compromise the safety or compliance of the patient or would preclude the patient from successful completion of the study
-Patient/Parent/Guardian unable or unwilling to comply with the protocol requirements