Access to stem cell transplant for patients without matched donors

Is this trial a fit for you?

A plain-English reading of the study's rules. Only the study team can confirm whether you qualify.

What this trial studies

This protocol aims to provide access to allogeneic hematopoietic stem cell transplant (HSCT) for patients who do not have a fully HLA matched sibling donor and are not eligible for existing protocols. The study utilizes the CliniMACs device to deplete T cells from donor stem cells, which can cause graft vs. host disease (GVHD). By removing these T cells, the study seeks to reduce complications associated with HSCT and improve outcomes for patients with serious or life-threatening conditions. Eligible patients include those with various non-malignant and malignant diseases that can be treated with HSCT.

Why it matters

Who can join: the full rules

Inclusion Criteria:

1. Patients who lack a fully HLA matched sibling and who are candidates for allogeneic hematopoietic stem cell transplant (HSCT) but are not deemed suitable candidates per their treating clinical team 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/including HLH, Hemoglobinopathies correctable and requiring HSCT, and Other diseases treated with HSCT/Other non-malignant blood, metabolic, or immune disorders for which HSCT has been recommended

   Malignant diseases:

   Acute leukemias, Chronic leukemias, Lymphomas, Myelodyplastic syndrome
3. Signed informed consent
4. Lansky or Karnofsky performance ≥60
5. Hematologic and Organ Function per current institutional SOP.
6. Infectious Evaluation as per current institutional SOP.
7. Participants of childbearing potential must have a negative pregnancy test as per institutional SOP
8. In cases that are deemed clinical emergencies (primary or secondary graft failure, severe marrow suppression), the above status criteria will be waived.
9. Patients must have an identified living donor

   * Donor selection will comply with 21 CFR 1271
   * Unrelated donor that meets the matching criteria of the NMDP with allele matching at HLA -A, -B, -C, -DRB1, and -DQB1: Unrelated donors may be a 10/10 match, a 9/10 match, or an 8/10 match if one of the mismatches is at DQB1
   * Related donor suitable for mobilization infectious disease criteria as per SOP, including HIV, HepB, HepC PCR negative.
   * CHOP BMT procedures apply for determining donor eligibility, including donor screening and testing for relevant communicable disease agents and diseases. Our donor collection program is 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 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:

1. Uncontrolled bacterial, viral or fungal infections
2. Suitable, fully HLA matched sibling donor
3. Donor unable to donate peripheral stem cells
4. Pregnant participants

Where it's running

• Children's Hospital of Philadelphia
  Philadelphia, Pennsylvania, United States Recruiting

Study contacts

• Principal investigator: Timothy Olson, MD, PhD — Children's Hospital of Philadelphia
• Study coordinator: Megan Atkinson
• Email: cttsbmtintake@chop.edu
• Phone: 215-590-2820

Similar studies

Other studies for the same condition or treatment. Recruiting studies are listed first.

• Using CD45RA Depleted Stem Cells to Prevent Infections After Transplant

  This study is testing whether using special stem cells can help patients with high-risk leukemia recover their immune system faster and prevent infections after a stem cell transplant.

  Recruiting Phase 1/2 Children's Hospital of Philadelphia · Philadelphia, Pennsylvania · NCT06839456

• Genetics of Blood Cancers in the U.S. population

  This study tests how genetic factors relate to blood cancers by collecting saliva DNA and online health surveys from at least 10,000 U.S. adults.

  Recruiting Rutgers, The State University of New Jersey · Piscataway, New Jersey · NCT07714044

• Dibotatug in adults with bone marrow failure syndromes

  This study tests dibotatug (DR-01) to see if it is safe and helps people with bone marrow failure syndromes like severe aplastic anemia who have not responded to standard therapies.

  Recruiting at 2 sites Phase 1/2 Dren Bio · Duarte, California and 9 other locations · NCT07712562

• Strength Training for Kids with Acute Lymphoblastic Leukemia and Lymphoblastic L lymphoma

  Researchers are testing a structured strength-training program for children and teens recently diagnosed with ALL or lymphoblastic lymphoma to see if it helps with weight gain and physical function during treatment.

  Recruiting UNC Lineberger Comprehensive Cancer Center · Chapel Hill, North Carolina · NCT07634653

• Cord blood transplant for children and young adults with blood cancers.

  This study will test whether a cord blood transplant given as a first or second transplant can help children and young adults with leukemia, myelodysplasia, or lymphoma who lack a matched donor.

  Recruiting Phase 2 Memorial Sloan Kettering Cancer Center · New York, New York · NCT07566377

• Cohort for people with chronic myelomonocytic leukemia

  This project will collect blood and bone marrow samples from adults with chronic myelomonocytic leukemia at diagnosis and during disease changes to create an anonymized biobank researchers can use to test new questions.

  Recruiting Gustave Roussy, Cancer Campus, Grand Paris · Amiens and 23 other locations · NCT07512882

How to take part

1. Review the eligibility criteria above with your treating physician.
2. Check the official listing on ClinicalTrials.gov for the latest contact details and recruitment status.
3. Contact the study coordinator or principal investigator to ask for pre-screening. Pre-screening is free and never obligates you to enroll.