← Back to Search

Stem Cell Transplantation

Stem Cell Transplant for Bone Marrow Failure Syndrome (ExpMACs Trial)

Phase < 1

Recruiting

Led By Timothy Olson, MD, PhD

Research Sponsored by Children's Hospital of Philadelphia

Eligibility Criteria Checklist

Specific guidelines that determine who can or cannot participate in a clinical trial

Must have

Patients with organ function criteria including 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), and no active untreated infection

Patients with malignant diseases including acute leukemias, chronic leukemias, lymphomas, and myelodyplastic syndrome

Must not have

Donor unable to donate peripheral stem cells

Pregnant Females

Timeline

Screening 3 weeks

Treatment Varies

Follow Up up to 5 years

Awards & highlights

No Placebo-Only Group

Summary

This trial is for children with a serious disease who are not eligible for other stem cell transplant protocols. The goal is to expand access for patients who lack a fully HLA matched sibling donor.

Who is the study for?

This trial is for patients without a fully matched sibling donor who need a stem cell transplant due to serious conditions like leukemia, metabolic diseases correctable by HSCT, bone marrow failure, or immune system problems. They must meet specific health criteria and not be pregnant or have uncontrolled infections.

What is being tested?

The trial provides access to a special stem cell transplant using CliniMACs technology to deplete certain cells (CD3+/CD19+) from the donated material. It's aimed at those who can't participate in other trials but need this potentially life-saving procedure.

What are the potential side effects?

While specific side effects are not listed here, stem cell transplants can generally cause reactions such as fatigue, infection risk increase due to immune suppression, graft-versus-host disease where the new cells attack the body, and organ complications.

Eligibility Criteria

Inclusion Criteria

You may be eligible if you check “Yes” for the criteria below

Select...

My organ functions meet the required levels, and I don't have an untreated infection.

Select...

I have a type of blood cancer such as leukemia, lymphoma, or myelodysplastic syndrome.

Select...

I have a non-cancerous condition treatable by stem cell transplant.

Exclusion Criteria

You may be eligible for the trial if you check “No” for criteria below:

Select...

My donor cannot give stem cells through their bloodstream.

Select...

I am currently pregnant.

Select...

I do not have any ongoing serious infections.

Select...

I have a sibling donor who is a complete HLA match.

Timeline

Screening ~ 3 weeks3 visits

Treatment ~ Varies

Follow Up ~ up to 5 years

Screening ~ 3 weeks

Treatment ~ Varies

Follow Up ~up to 5 years

This trial's timeline: 3 weeks for screening, Varies for treatment, and up to 5 years for reporting.

Treatment Details

Awards & Highlights

No Placebo-Only Group

All patients enrolled in this study will receive some form of active treatment.

Trial Design

1Treatment groups

Experimental Treatment

Group I: Expanded access to CliniMACs device for T cell depletionExperimental Treatment1 Intervention

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.

0 / 7Eligibility criteria met