Expanded Access Protocol Using CD3+/CD19+ Depleted PBSC

Leukemia Clinical Trial

— ExpMACs  

Official title:

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

Clinical Trial Details — Status: Recruiting

Administrative data

• NCT number: NCT02356653
• Other study ID #: 13-010286
• Secondary ID: 12BT125
• Status: Recruiting
• Phase: Early Phase 1
• Start date: December 2013
• Est. completion date: January 2027

Study information

• Verified date: December 2023
• Source: Children's Hospital of Philadelphia
• Contact: Patricia Hankins, BSN, RN, CCRC
• Phone: 215-590-5168
• Email: hankinsp[@]chop.edu
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Recruiting
• Enrollment: 100
• Est. completion date: January 2027
• Est. primary completion date: January 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: N/A to 30 Years

Eligibility

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

Related Conditions & MeSH terms

• Bone Marrow Failure Disorders
• Bone Marrow Failure Syndromes
• Immunodeficiencies
• Immunodysregulation Polyendocrinopathy Enteropathy X-linked Syndrome
• Inborn Errors of Metabolism
• Leukemia
• Metabolism, Inborn Errors
• Pancytopenia
• Syndrome

Intervention

Device:
• Transplant of stem cells with CD3+/CD19+ depletion (CliniMACs)
Processing of stem cells using the CliniMACs device to selectively deplete specific T cells to decrease risk of graft versus host disease when using donor stem cells which are not fully matched.

Locations

Country	Name	City	State
United States	Children's Hospital of Philadelphia	Philadelphia	Pennsylvania

Sponsors (1)

Lead Sponsor	Collaborator
Children's Hospital of Philadelphia

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Overall Survival		1 year
Secondary	Graft versus Host Disease		1 year
Secondary	Graft Failure		100 days