Expanded Access Protocol Using Alpha/Beta T and CD19+ Depleted PBSC

Sponsor

Children's Hospital of Philadelphia (Other)

Overall Status

Available

CT.gov ID

NCT03145545

Collaborator

(none)

Location

Study Details

Study Description

Brief Summary

The primary objective of this protocol is to expand access for patients who lack a fully HLA (Human leukocyte antigen) matched sibling donor, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-threatening disease for which HSCT is indicated. These patients are not eligible for other Children's Hospital of Philadelphia Institutional Review Board (IRB) approved protocols that utilize CliniMACs technology for T depletion.

Condition or Disease	Intervention/Treatment	Phase
Leukemia Bone Marrow Failure Syndrome Immunodeficiencies	Device: Apha/beta T and CD19+ cell depletion using CliniMACS device

Detailed Description

Only 25-30% of patients who may benefit from HSCT have a matched related donor. An unrelated cord blood may not be available due to size or matching criteria, or if a reduced intensity regiment is recommended. The risk of severe graft vs. host disease (GVHD) and other complications is higher with unrelated donors, or partially matched related donors. At the Children's Hospital of Philadelphia (CHOP) there is extensive experience using mismatched unrelated donors or partially matched related donors with complete or partial T depletion to reduce the risk of severe GVHD.

Study Design

Study Type:

Expanded Access

Official Title:

Expanded Access Protocol Using TCR Alpha/Beta T Cell/CD19+ Depleted Unrelated Donor or Partially Matched Related Donor Peripheral Stem Cells

Outcome Measures

Primary Outcome Measures

Eligibility Criteria

Criteria

Ages Eligible for Study:

1 Month and Older

Sexes Eligible for Study:

All

PATIENT AND DONOR ELIGIBILITY

Patients who lack an HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but do not meet criteria for current open institutional protocols using ClinMACs device for β T/CD19+ depletion.

Patients with the following transplantable diseases:

Non-malignant diseases:

Metabolic storage diseases correctable by HSCT
Bone marrow failure syndromes
Immunodeficiencies/immune dysregulation syndromes
Sickle cell disease with severe central nervous system (CNS) vasculopathy
Other hemoglobinopathies requiring HSCT

Malignant diseases:

Acute leukemias
Chronic leukemias
Lymphomas
Myelodyplastic syndrome

Organ function criteria:

It is important to note that the conditioning prescribed to the patient will be determined based on the disease and organ status and will be regimens considered standard. Appropriate combinations of chemotherapy, immunotherapy and/or radiation will be determined on an individual basis.

Patient eligibility will be assessed as per our institutional standard operating procedures:

Lansky or Karnofsky performance >60
Renal function: will be determined based on serum creatinine as per our Institutional SOP
Hepatic: Transaminases will be assessed as per current institutional SOP
Cardiac shortening fraction >27% as per institutional SOP
Bilirubin <2.5x normal (unless elevation due to Gilberts disease) as per Institutional SOP
No active untreated infection
Signed informed consent
No fully HLA matched sibling donor available.
Females of childbearing potential must have negative pregnancy test.

Donor Eligibility Patients must have an identified living donor

Donor selection will comply with 21 Code of Federal Regulations (CFR) 1271*
Unrelated donor that meets the matching criteria of the NMDP: Unrelated donors that may be up to a one antigen mismatch at A, B or DRB1. donor
Related donor mismatched at one to five antigens (haploidentical)
Donor suitable for mobilization of peripheral stem cells and apheresis and fulfills infectious disease criteria as per our institutional SOP, including HIV, Hepatitis B (HepB), Hepatitis C (HepC) polymerase chain reaction (PCR) negative.
CHOP bone marrow transplant (BMT) procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is Foundation for the Accreditation of Cellular Therapy (FACT) accredited.
Unrelated donor identified through the National Marrow Donor Program (NMDP) and fulfills the NMDP criteria for donation. Unrelated donor willing and able to undergo mobilization of peripheral stem cells and apheresis
The donors selected for this investigational new drug (IND) will either be unrelated donors identified through the National Marrow Donor Program (NMDP) or related donors. Regarding the unrelated donors; NMDP procedures for determining donor eligibility include donor screening and testing for relevant communicable disease agents and diseases.

Exclusion criteria:

Uncontrolled bacterial, viral or fungal infections
Fully HLA matched sibling donor
Donor unable to donate peripheral stem cells
Pregnant Females

Contacts and Locations

Locations

	Site	City	State	Country	Postal Code
1	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania	United States	19104

Sponsors and Collaborators

Children's Hospital of Philadelphia

Investigators

None specified.

Study Documents (Full-Text)

None provided.

More Information

Publications

None provided.

Responsible Party:

Nancy Bunin, Director, Blood and Marrow Transplant, Children's Hospital of Philadelphia

ClinicalTrials.gov Identifier:

NCT03145545

Other Study ID Numbers:

16-013527

First Posted:

May 9, 2017

Last Update Posted:

Nov 3, 2021

Last Verified:

Jul 1, 2021

Additional relevant MeSH terms:

Bone Marrow Failure Disorders

Pancytopenia

Study Results

No Results Posted as of Nov 3, 2021