Chronic Granulomatous Disease (CGD) Gene Therapy Clinical Trial
Gene Therapy
Phone: 617-632-5064
Gene Transfer in Patients with Chronic
Granulomatous Disease (CGD)
A Phase I/II Open Label Study of Gene Transfer
(Lentiviral Vector Transduced CD34+ Cells) in Patients with Chronic
Granulomatous Disease (CGD)
Chronic
Granulomatous Disease (CGD) is an inherited immune system disorder that affects males. It occurs
when a type of white blood cell that usually helps the body fight infection,
called a phagocyte, does not work properly. In CGD, the phagocytes can’t kill
germs that are ingested, and so cannot protect the body from bacterial and
fungal infections.
This chronic granulomatous
disease (CGD) gene therapy clinical trial uses a safety-improved, third
generation self-inactivating lentiviral vector to transduce CD34+ selected
hematopoietic stem cells. Genetically modified cells are infused after busulfan
preparative conditioning. This vector was designed to address the leukemias
that have occurred in other past gene therapy trials using a gammaretroviral vector
containing strong intact long-term repeat regions.
Preclinical data
showed an excellent safety profile for this vector, leading to FDA approval and
NHLBI-appointed DSMB approval of this trial. The costs of research aspects of
the protocol will be provided for patients treated on the trial by the Gene
Therapy Resource Program, NHLBI and NIH.
Inclusion Criteria:
- Male X-CGD patients, ages 23 months or older
- Molecular
diagnosis confirmed by DNA sequencing and supported by laboratory evidence for
absent or reduction of more than 95% of the biochemical activity of the NAHPD-oxidase
- At
least one prior, ongoing or refractory severe infection and/or inflammatory
complications requiring hospitalization despite conventional therapy
- No
10/10 HLA-matched donor available after a search of the National Marrow Donor Program (NMDP) registries
- No
co-infection with Human Immunodeficiency Virus (HIV), hepatitis B virus
(HBsAg positive), hepatitis C virus (HCV RNA positive), CMV, adenovirus,
parvovirus B 19, or toxoplasmosis
Exclusion Criteria:
- Contraindication
to stem cell collection or to administration of conditioning medication
- Known
positive HIV serology or HIV nucleic acid testing
- Active
malignancy
- Known
myelodysplasia of the bone marrow or abnormal bone marrow cytogenetics
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