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

Phase 1

Conditions: Chronic Granulomatous Disease (CGD) is a rare inherited disorder (1/250,000) of the phagocytes characterized by the inability of phagocytes (monocytes and neutrophils) to produce reactive oxygen species (ROS) in response to a stimulus, due to the absence of NADPH oxidase activity. Affected patients present an elevated susceptibility to bacterial and fungal infections, as well as an excessive inflammatory response that leads to granuloma formation.
Therapeutic area: Diseases [C] - Immune System Diseases [C20]

Registration Number: EUCTR2014-002222-12-FR

Lead Sponsor: Genethon

Brief Summary: Not available

Detailed Description: Not available

Recruitment & Eligibility

Status: ot Recruiting

Sex: Male

Target Recruitment: 5

Inclusion Criteria

-Male X-CGD patients >23 months of age. Youngest patients (>1 month and = 23 months) may be enrolled at physician’s appreciation
-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 or at high risk of relapse severe infection and/or inflammatory complications requiring hospitalisation despite conventional therapy
-No HLA-matched donor available after 3 months search, unless the risk of waiting for a potential match or for performing an allogeneic transplant is considered unacceptable
-No co-infection with Human Immunodeficiency Virus (HIV) or hepatitis B virus (HBs Ag 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: 4
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 1
F.1.3 Elderly (>=65 years) no
F.1.3.1 Number of subjects for this age range

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 and any component of the Investigational Medicinal Product (IMP) preparation
-Administration of gamma interferon 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