Access to stem cell transplant for patients without matched donors
Expanded Access Protocol Using CD3+/CD19+ Depleted Unrelated Donor or Related Donor Peripheral Stem Cells
This study is testing a new way to help patients without a matched donor get a stem cell transplant by using a special device to reduce complications and improve their chances of recovery.
Quick facts
| Phase | Early Phase 1 |
|---|---|
| Study type | Interventional |
| Enrollment | 100 (estimated) |
| Ages | N/A to 30 Years |
| Sex | All |
| Sponsor | Children's Hospital of Philadelphia Academic / other |
| Locations | 1 site (Philadelphia, Pennsylvania) |
| Trial ID | NCT02356653 on ClinicalTrials.gov |
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.
Who should consider this trial
Good fit: Ideal candidates include patients with leukemia, metabolic disorders, bone marrow failure syndromes, and immunodeficiencies who lack a fully matched sibling donor.
Not a fit: Patients who have a fully matched sibling donor or are not candidates for HSCT will not benefit from this study.
Why it matters
Potential benefit: If successful, this approach could significantly improve transplant outcomes and reduce complications for patients lacking matched donors.
How similar studies have performed: Other studies utilizing T cell depletion techniques have shown promise, indicating potential for success in this approach.
Eligibility criteria
Show full inclusion / exclusion criteria
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 this trial is running
Philadelphia, Pennsylvania
- 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
How to participate
- Review the eligibility criteria above with your treating physician.
- Visit the official trial page on ClinicalTrials.gov for the most current contact information and recruitment status.
- Contact the listed study coordinator or principal investigator to request pre-screening. Pre-screening is free and never obligates you to enroll.