BPX-501 T Cells Infused Post Stem Cell Transplant in Pediatrics With Non-Malignant Disorders Ineligible for BPU004 Study

This is an expanded access protocol of BPX-501 T cells infused after T cell-depleted HSCT in
pediatric patients with non-malignant hematologic disorders eligible for treatment on the
BP-U-004 study.

The purpose of this protocol is to provide access to the CaspaCIDe system combination product
(BPX-501 gene modified T cells and rimiducid) to patients on a case by case basis who do not
meet the BP-U-004 protocol eligibility criteria. BPX-501 infusion can enhance immune
reconstitution with the potential for reducing the severity and duration of severe acute
GVHD.

Inclusion Criteria:

1. Males or females

2. Age < 21 years and > 3 months

3. Life expectancy > 10 weeks

4. Patients deemed eligible for allogeneic stem cell transplantation.

5. Non-malignant disorders including:

1. inherited metabolic disorders such as adrenal leukodystrophy;

2. lysosomal storage disorders such as Hurler syndrome or metachromatic
leukodystrophy

3. other inborn errors of metabolism

6. Lack of suitable conventional donor (HLA identical sibling or HLA phenotypically
identical relative evaluated using high resolution molecular typing).

7. A minimum genotypic identical match of 5/10 is required.

8. The donor and recipient must be identical, as determined by high resolution typing, at
least one allele of each of the following genetic loci: HLA-A, HLA-B, HLA-Cw, and HLA-
DRB1.

9. Lansky/Karnofsky score > 50

10. Signed written informed consent

3.2 Subject exclusion criteria

1. Age < 3 months or >21 years

2. Patients with non-malignant disorders eligible for treatment on the BP-U-004 study:

1. primary immune deficiencies,

2. severe aplastic anemia not responding to immune suppressive therapy,

3. osteopetrosis,

4. selected cases of hemoglobinopathies and

5. congenital/hereditary cytopenia, including Fanconi Anemia before any clonal
malignant evolution (MDS, AML)

3. Greater than Grade II acute GVHD or chronic extensive GVHD due to a previous allograft
at the time of inclusion

4. Patient receiving an immunosuppressive treatment for GVHD treatment due to a previous
allograft at the time of inclusion

5. Dysfunction of liver (ALT/AST > 5 times normal value, or bilirubin > 3 times normal
value), or of renal function (creatinine clearance < 30 ml / min)

6. Severe cardiovascular disease (arrhythmias requiring chronic treatment, congestive
heart failure or left ventricular ejection fraction < 40%)

7. Current active infectious disease (including positive HIV serology or viral RNA)

8. Serious concurrent uncontrolled medical disorder

9. Pregnant or breast feeding female patient

10. Lack of parents'/guardian's informed consent.