Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Brief Title

Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Official Title

Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells

Brief Summary

      The goal 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 IRB approved protocols that utilize CliniMACs technology for T
      depletion.
    

Detailed Description

      Only 25-30% of patients who may benefit from HSCT have a matched related donor. There is a
      higher rate of complications using cells from an unrelated or partially matched related
      donor. T cells within the donor cells may cause a complication called graft vs. host disease
      (GVHD). The goal of this study is to use the CliniMACs device to remove the T cells that
      cause GVHD, called T cell depletion.
    

Study Phase

Early Phase 1

Study Type

Interventional


Primary Outcome

Overall Survival

Secondary Outcome

 Graft versus Host Disease

Condition

Leukemia

Intervention

Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)

Study Arms / Comparison Groups

 Expanded access to CliniMACs device for T cell depletion
Description:  access for patients who lack a fully HLA matched sibling, and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT). These patients have a serious or immediately life-open protocols that utilize CliniMACs technology for T depletion. Subjects will undergo transplant of stem cells with CD3+/CD19+ depletion.

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Device

Estimated Enrollment

100

Start Date

December 2013

Completion Date

January 2025

Primary Completion Date

January 2023

Eligibility Criteria

        Inclusion Criteria:

          1. Patients who lack a fully 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 CD3+/CD19+ depletion.

          2. Patients with the following transplantable diseases:

             Non-malignant diseases:

             Metabolic storage diseases correctable by HSCT

             Bone marrow failure syndromes

             Immunodeficiencies/immune dysregulation syndromes

             Malignant diseases:

             Acute leukemias

             Chronic leukemias

             Lymphomas

             Myelodyplastic syndrome

          3. Organ function criteria:

             Lansky or Karnofsky performance ≥60

             Serum creatinine ≤3xupper limit of normal for age

             Hepatic: Transaminases ≤10xnormal

             Cardiac shortening fraction ≥27%

             Bilirubin <2.5x normal (unless elevation due to Gilberts disease)

          4. No active untreated infection

          5. Signed informed consent

          6. No fully HLA matched sibling donor available

          7. Females of childbearing potential must have negative pregnancy test

        Exclusion Criteria:

          1. Uncontrolled bacterial, viral or fungal infections

          2. Fully HLA matched sibling donor

          3. Donor unable to donate peripheral stem cells

          4. Pregnant Females
      

Gender

All

Ages

N/A - 30 Years

Accepts Healthy Volunteers

No

Contacts

Nancy J Bunin, MD, 215-590-4029, [email protected]

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT02356653

Organization ID

13-010286

Secondary IDs

12BT125

Responsible Party

Principal Investigator

Study Sponsor

Children's Hospital of Philadelphia


Study Sponsor

Nancy J Bunin, MD, Principal Investigator, Children's Hospital of Philadelphia


Verification Date

November 2021