Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant

• Register: ClinicalTrials.gov
• Last refreshed on: 7 June 2021
• Main ID: NCT01200017
• Date of registration: 09/09/2010
• Prospective Registration: No
• Primary sponsor: Christopher Dvorak
• Public title: Expanded Access Protocol (EAP) Using the CliniMACS® Device for Pediatric Haplocompatible Donor Stem Cell Transplant
• Scientific title: An Expanded Access Study of the Feasibility of Using the CliniMACS® Device for CD34+ Cell Selection and T Cell Depletion for Graft-versus-Host Disease Prophylaxis in Alternative Donor Stem Cell Transplant Recipients
• Date of first enrolment: September 2010
• Recruitment status: No longer available
• URL: https://clinicaltrials.gov/show/NCT01200017
• Study type: Expanded Access

Countries of recruitment

United States

Contacts

• Name: Christopher Dvorak, MD
• Affiliation: University of California, San Francisco

Key inclusion & exclusion criteria

Inclusion Criteria:

- >2 months - 30 years

- Patient must have a malignant or non-malignant disease that can benefit from
alternative stem cell transplantation according to standard practice guidelines for
including patients for transplant as outlined in UCSF Pediatric Bone Marrow Transplant
(BMT) Standard Operating Procedure (SOP) #206.04. Examples include acute and chronic
leukemias, myelodysplastic syndrome, lymphoma, severe acquired and congenital
cytopenias, white and red blood cell abnormalities, inborn errors of metabolism and
immunodeficiencies. Patient with Fanconi's Anemia will be eligible regardless of match
with donor.

- Patients with acute leukemia (AML excepted) or lymphoma must be in remission at the
time of transplant.

- Patients must lack a healthy human leukocyte antigen (HLA)-identical related donor.

- Recipient or authorized guardian must sign informed consent for this study.

- If recipient is female and of child-bearing age, negative pregnancy test.

- Patient must have a healthy, willing mismatched related or an unrelated donor who is:

- Able to receive Granulocyte colony-stimulating factor (G-CSF) +/- Plerixafor and
undergo apheresis either through placement of catheters in antecubital veins or a
temporary central venous catheter,

- For Related donor: sibling, half-sibling, parent, cousin, aunt, uncle or
grandparent will all be considered eligible.

- For Related donor: HLA antigen genotypic match = 4/8 and = 7/8 (haplocompatible).

- For unrelated donor: 6/8 or 7/8 HLA antigen match (if two mismatches, they must
be at different loci).

- Complete medical history, physical and screening for infectious diseases that are
acceptable for donation.

- If donor is female and of child-bearing age, negative pregnancy test.

- Absence of anti-HLA antibodies in recipient directed against donor antigens.

- Donor must be willing to sign informed consent for this study. If donor is < 18
years of age, donor must be willing to give assent and parents willing to sign
informed consent.

- Be suitable for an autologous gene-modified transplant:

- Using either bone marrow or cytokine-mobilized peripheral blood stem cells
(PBSC).

- Patient or authorized guardian must sign informed consent for this study.

- For unrelated donors: Per New Algorithm, Jan. 14, 2016, effective immediately,
National Marrow Donor Program (NMDP) "Be The Match" is adopting a revised algorithm
for determining if a donor is a research subject on their recipient's research
protocol: The NMDP will inform the donor of the activities and the use of donor's
apheresis product to be used in this study and obtain written consent from the donor.
Transplant centers are sent documentation of the donor consent to participate in the
research support activities. (The revised algorithm can be found on the "Be the Match
Clinical Network")

- Criteria to consider when choosing among related haplo donors:

- CMV positive donor is preferred over other factors. CMV negative donor can be
used only if recipient is CMV negative.

- HLA disparity i.e. 2 Ag mismatch preferred over 3 Ag mismatch; Drß1 match
preferred over class I match; HLA-C matched preferred over A and B match.

- killer immunoglobulin-like receptor (KIR) mismatch in GVH direction is preferred
for patients with malignant disorders

- ABO compatibility

- Age = 2 months

Exclusion Criteria:

- Patient with an anticipated life expectancy of < 1 month

- Active infectious hepatitis or cytomegalovirus (CMV) disease (organ involvement)

- Human immunodeficiency virus (HIV) or Human T-lymphotropic virus (HTLV-I/II) infection

- Cardiac ejection fraction < 45%; can be lower if patient is not in clinical cardiac
failure and a reduced intensity conditioning regimen is used.

- Creatinine clearance <60 ml/min/1.72 m2; can be lower if a reduced intensity
conditioning regimen is used.

- Pulmonary diffusion capacity (corrected for hemoglobin), forced expiratory volume in
one second (FEV1), or forced vital capacity (FVC) <60% of predicted or oxygen
saturation (O2 sat) > 94% on room air if unable to perform pulmonary function tests
(PFTs); can be lower if a reduced intensity conditioning regimen is used.

- Serum alanine aminotransferase (ALT) > 5 x upper limit of normal (can be up to 10x
upper limit of normal if a reduced intensity conditioning regimen is used) or
bilirubin > 2.

- Performance score (Lansky/Karnofsky) < 50

- Any condition that compromises compliance with the procedures of this protocol, as
judged by the principal investigator.



Age minimum: 2 Months
Age maximum: 30 Years
Gender: All

Health Condition(s) or Problem(s) studied

Leukocyte Disorders

Bone Marrow Failure

Chronic Myeloid Leukemia

Lymphomas

Acute Myeloid Leukemia

Hemoglobinopathy

Acute Lymphoblastic Leukemia

Anemia Due to Intrinsic Red Cell Abnormality

Myelodysplastic Syndrome

Osteopetrosis

Cytopenias

Immune Deficiency

Intervention(s)

Biological: CD34+ enriched, T Cell Depleted donor stem cell product

Secondary ID(s)

10082

NCI-2020-02478

Source(s) of Monetary Support

Please refer to primary and secondary sponsors