Clinical Trial Details
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NCT01962415 : Reduced Intensity Conditioning in Patients Aged ?30 With Non-Malignant Disorders Undergoing Cord Blood Transplantation
PhasePhase 2
AgesMin: 2 Months Max: 30 Years
Inclusion Criteria:

1. Patient must be 2 months to 30 years (inclusive) of age for all diagnoses.

2. Patients must have a 4/6, 5/6 or 6/6 HLA matched related or unrelated UCB unit
available that will deliver a pre-cryopreservation total nucleated cell dose of > 3 x
107 cells/kg.

3. Patient must have adequate function of other organ systems as measured by:

1. Creatinine ? 2.0 mg/dl and creatinine clearance ? 50 ml/min/1.73 m2.

2. Hepatic transaminases (ALT/AST) ? 4 x normal.

3. Normal cardiac function by echocardiogram or radionuclide scan (shortening
fraction > 26% or ejection fraction > 40% or > 80% of normal value for age).

4. Pulmonary function testing demonstrating CVC or FEV1/FVC of > 50% of predicted
for age and/or resting pulse oximeter ? 92% on room air or clearance by the
pediatric or adult pulmonologist. For adult patients DLCO (corrected for
hemoglobin) should be > 50% of predicted.

4. Patient, parent, or legal guardian must have given written informed consent and/or
assent according to FDA guidelines.

5. Patients may not be pregnant or lactating and must have a current negative pregnancy
test if pubertal and/or menstruating.

6. Patients must have a minimum life expectancy of at least 6 months.

7. Patients must be HIV negative.

8. Patients must not have uncontrolled infections at the time of cytoreduction.

9. Patients should have a non-malignant disorder amenable to treatment by stem cell
transplantation, including but not limited to:

A. Primary Immunodeficiency syndromes including but not limited to:

- Severe Combined Immune Deficiency (SCID) with NK cell activity

- Omenn Syndrome

- Bare Lymphocyte Syndrome (BLS)

- Combined Immune Deficiency (CID) syndromes

- Combined Variable Immune Deficiency (CVID) syndrome

- Wiskott-Aldrich Syndrome

- Leukocyte adhesion deficiency

- Chronic granulomatous disease (CGD)

- X-linked Hyper IgM (XHIM) syndrome

- IPEX syndrome

- Chediak -Higashi Syndrome

- Autoimmune Lymphoproliferative Syndrome (ALPS)

- Hemophagocytic Lymphohistiocytosis (HLH) syndromes

- Lymphocyte Signaling defects

- Other primary immune defects where hematopoietic stem cell transplantation may
be beneficial

B. Congenital bone marrow failure syndromes including but not limited to:

- Dyskeratosis Congenita (DC)

- Congenital Amegakaryocytic Thrombocytopenia (CAMT)

- Osteopetrosis

C. Inherited Metabolic Disorders (IMD) including but not limited to:

- Mucopolysaccharidoses o Hurler syndrome (MPS I) o Hunter syndrome (MPS II)

o Sanfilippo syndrome (MPS II)

- Leukodystrophies

o Krabbe Disease, also known as globoid cell leukodystrophy

- Metachromatic leukodystrophy (MLD)

- X-linked adrenoleukodystrophy (ALD)

- Other inherited metabolic disorders

o alpha mannosidosis

- Other inheritable metabolic diseases where hematopoietic stem cell
transplantation may be beneficial.

D. Hereditary anemias

• Thalassemia major

• Sickle cell disease (SCD)

- Diamond Blackfan Anemia (DBA)

- other congenital transfusion dependent anemias

Patients with sickle disease must have one or more of the following:

• Overt or silent stroke

• Pain crises ? 2 episodes per year for past year

• One or more episodes of acute chest syndrome

• Osteonecrosis involving ? 1 joints

• Priapism

Exclusion Criteria:

1. UCB unit with a total nucleated cell count of < 3 x 107/kg or > 2 antigen mismatching.

2. Available HLA-matched related living donor unaffected with recipient's disease and able
to donate without previous UCB donation.

3. Allogeneic hematopoietic stem cell transplant within the previous 6 months.

4. Any active malignancy or MDS.

5. Severe acquired aplastic anemia.

7. Uncontrolled bacterial, viral or fungal infection (currently taking medication and with
progression of clinical symptoms).

8. Pregnancy or nursing mother.

9. HIV positive

10. Poorly controlled pulmonary hypertension.

11. Any condition that precludes serial follow-up.
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