Treatment Use of the CliniMACS® CD34 TCR Alpha/Beta for Allogenic Transplant (Expanded Access Use)

Recipient Inclusion Criteria:

- Age less than 22 years of age

- No available genotypically matched related donor (sibling)

- Availability of a suitable donor and graft source

- Haploidentical related mobilized peripheral blood cells

- 9/10 or 10/10 allele matched (HLA-A, -B, -C, -DRB1, -DQB1) volunteer unrelated donor
mobilized peripheral blood cells

Donor Inclusion Criteria:

- Matching as described in the Recipient and Donor Suitability Criteria

- Size and vascular access appropriate by center standard for peripheral blood stem
cell (PBSC) collection. Donors with inadequate peripheral access will require
placement of an apheresis catheter for collection.

- Must meet appropriate screening/eligibility requirements:

- Haploidentical matched family members: screened by center health screens and
found to be eligible.

- Unrelated donors: meet suitability criteria to donate PBSC

- HIV negative

- Not pregnant or lactating

- Recipient must not have high-level donor specific anti-HLA antibodies according
to institutional practices.

- Must agree to donate PBSC

- Must give informed consent

Recipient Suitability:

- Adequate organ functioning as demonstrated by:

- MUGA ≥ 50% or ECHO >30%

- 24-hour Creatinine Clearance or Glofil ≥ 50 ml/min or Serum Creatinine ≤2 times the
upper limit of normal

- DLCO > 65% of predicted value. FEV1 >65% of predicted

- AST / ALT ≤ 2.5 and/or Bilirubin ≤ 1.5 times the upper limit of normal

- Pregnancy test negative

- HIV 1 / 2 (HIV screen) negative with a negative qualitative HIV PCR.

- HTLV I negative

- RPR or VDRL negative

- RVP (Respiratory Viral PCR) with a negative result

- Positive hepatitis testing does not make the patient ineligible. Hepatitis results
will be evaluated on a case-by-case basis by the transplant physician to determine
donor suitability/eligibility.

Donor Suitability:

- Allowed Donor Sources

- Fully matched sibling donors are not allowed.

- Unrelated donors. HLA typing of at least 10 alleles is required. Donor must be
matched at 9/10 or 10/10 alleles (HLA-A, -B, -C, -DRB1, -DQB1). Haploidentical
matched family members. Minimum match level full haploidentical (at least 5/10;
HLA-A, -B, -C, -DRB1, -DQB1 alleles), but use of haploidentical donors with extra
matches (e.g., 6, 7, or 8/10) encouraged.

- Cord blood is not allowed as a stem cell source on this IDE.

Recipient and Donor Exclusion Criteria:

- Pregnant or lactating females are ineligible as the risk to unborn children and
infants is unknown

- Presence of life-threatening, uncontrolled opportunistic infection (fungal,
bacterial, or viral infections). Patients with history of fungal disease during
induction therapy may proceed if they have a significant response to antifungal
therapy with no or minimal evidence of disease remaining by CT evaluation.

- HIV or HTLV I/II infection