A Phase 2 interventional study of ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs in Bone Marrow Failure, Hemoglobinopathies and Primary Immune Deficiency (PID), sponsored by Fred Hutchinson Cancer Center. Not yet recruiting at 2 sites in United States. Open to participants aged 6 Months to 50 Years. Per ClinicalTrials.gov, last updated 2026-06-22.
Sponsored by Fred Hutchinson Cancer Center · Phase 2, Interventional, and Treatment
This phase II trial investigates how well a naive T cell depleted graft work for the reduction of graft versus host disease in patients with non-malignant diseases requiring hematopoietic cell transplantation. Giving chemotherapy and total-body irradiation before a donor peripheral blood stem cell transplant helps stop the growth of cells in the bone marrow, including normal blood-forming cells (stem cells) and cancer cells. It may also stop the patient's immune system from rejecting the donor's stem cells. When the healthy stem cells from a donor are infused into the patient, they may help the patient's bone marrow make stem cells, red blood cells, white blood cells, and platelets. The donated stem cells may also replace the patient's immune cells and help destroy any remaining cancer cells.
OUTLINE: Patients will receive CD34+ enriched CD45RA-depleted donor T-lymphocytes IV on day 0. For conditioning, patients receive cyclophosphamide by IV on day -8, fludarabine by IV on day -7 to day -3, thiotepa IV on day -7 and day -6, and undergo total-body irradiation (TBI) for 2 doses on day -2 and day -1.
GVHD Prophylaxis:
All patients also receive tacrolimus IV continuously starting on day -1, and mycophenolate mofetil (MMF) starting day 0 through day 35. If there is no evidence of grade II-IV acute GVHD on or prior to day 100, tacrolimus is tapered.
All patients also undergo bone marrow aspiration/biopsy and collection of blood samples throughout the trial.
After completion of study treatment, patients are followed up at days 7, 14, 21, 28, 56, 80, 180, and 270 and at 1, 1.5, and 2 years.
91 studies on the registry are indexed under Bone Marrow Failure Disorders; 29 are open to participants now.
This study's planned enrollment of 40 is above the median of 26 across 69 interventional studies indexed under Bone Marrow Failure Disorders.
Browse Bone Marrow Failure Disorders studies →Fred Hutchinson Cancer Center is the lead sponsor of 547 studies on the registry; 80 are open to participants now.
Of its 59 completed or terminated interventional studies of FDA-regulated products, 46 (78%) have results posted.
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Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Allogeneic and autologous transplant
Stem cell (bone marrow) transplants. Allogeneic uses cells from a donor; autologous uses your own cells, collected earlier and given back.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Autoimmune disease
A condition in which the immune system attacks the body’s own tissues, such as rheumatoid arthritis, lupus, psoriasis, type 1 diabetes or inflammatory bowel disease.
Immunotherapy studies often leave out active autoimmune disease, and some name exceptions, such as thyroid disease on replacement hormone. Check the exceptions this study lists.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Informed consent
Before you join, the study team explains what taking part involves — its purpose, what happens, the risks and possible benefits — and answers your questions, so you can decide freely. Consent is usually recorded on a form (the informed consent form, ICF) signed by you or, where the law allows, by someone legally authorised to decide for you.
You can keep asking questions during the study, you are told of new information that matters, and you can choose to leave at any time.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
HIV
The human immunodeficiency virus, which weakens the immune system.
Many studies now accept people whose HIV is controlled on treatment, judged by the viral load and the CD4 count. Check the limits this study sets.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraindication
A reason a treatment, test or procedure should not be given to a particular person, such as an allergy, another illness or a medicine they take.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
GVHD (graft-versus-host disease)
After a transplant from a donor, the donor’s immune cells attack the recipient’s body, often the skin, gut or liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Hypersensitivity
An allergy, or another reaction of the immune system, to a substance. “Hypersensitivity to the study drug” means a known allergic reaction to it — not every side effect a medicine can have.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Conditioning regimen for HLA-Haploidentical or mismatched unrelated donor consisting of Cyclophosphamide (50 mg/kg x1 day), Fludarabine 35 mg/m2/day x 5 days, Thiotepa (5 mg/kg/day x 2 days), TBI (200 cGy x 2). Tacrolimus starting Day -1, MMF D0-35.
Biological: ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Conditioning regimen for HLA-matched related or matched unrelated donor consisting of Cyclophosphamide (50 mg/kg x1 day), Fludarabine (30 mg/m2/day x 5 days), Thiotepa (5 mg/kg/day x 2 days), TBI (200 cGy x 2). Tacrolimus starting Day -1, MMF D0-35.
Biological: ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Drug page: Allogeneic CD34+-enriched and CD45RA-depleted PBSCs
CD34-selected graft with CD45RA- depleted peripheral blood stem cells given to patients with Non-Malignant Diseases
GVHD-free Survival
Free of grade III-IV acute and NIH chronic (moderate-severe) GVHD requiring systemic immunosuppression
Time frame: 1 year post-transplant
Overall survival
Time frame: 1 year post-transplant
Transplant related mortality
Time frame: Day 100 post-transplant and 1 year post-transplant
Graft failure
Graft failure defined as failure to achieve an ANC ≥ 0.5 × 109/L before death or second HCT, or decrease to ANC \<0.1 × 109/L for 14 consecutive days (date of graft failure defined as the 14th day) after an established donor graft despite daily administration of G-CSF (SC or IV) and ≤ average 20% bone marrow cellularity on bone marrow aspirate or biopsy any time in the first 2 years following HCT. If a patient dies from organ toxicity and/or infection prior to day 28 without ANC ≥ 0.5 × 109/L this will not be considered graft failure. If the graft failure is attributed to viral infection, multi-organ failure or drug effect it will still be considered graft failure if it meets the definition of graft failure specified above.
Time frame: Day 42 post-transplant
Graft rejection
Graft rejection defined as \<5% donor CD3 T cell and CD33 myeloid chimerism
Time frame: Day 100 post-transplant
Incidence of chronic GVHD
Defined and graded based on NIH criteria and graded operationally as the occurrence of compatible symptoms
Time frame: At 1 year and 2 years post transplant
Prednisone for GVHD
Number of participants alive and off prednisone (or equivalent systemic corticosteroid) for the treatment of GVHD
Time frame: Every 3 months post-transplant for the first 2 years
Incidence of opportunistic infections requiring treatment
Proportion of participants who experience viral infections/reactivations. * of participants with \>/= grade 1-3 viral infections * of participants with CMV grade 2-3 * of participants with EBV grade 2-3 * of participants with ADV, HHV-6 or BK \>/= grade 2 * of participants with definite (confirmed organism) viral respiratory tract infection requiring hospital admission for \>/= 7 days or causing death between day 90 and day 365 post-transplant
Time frame: First year post-transplant
Peripheral blood donor chimerism
Full donor chimerism will be defined as CD33 and CD3 ≥95%. Mixed chimerism will be defined as CD33 OR CD3 \<95% but \>5%. Actual chimerism values will be reported and summarized as well as dichotomized according to the 'full chimerism' and 'mixed chimerism' labels
Time frame: 2 years post-transplant
Plan to share: No
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Studies of the drug tested here: Allogeneic CD34+-enriched and CD45RA-depleted PBSCs
Bone Marrow Failure Disorders →
Fred Hutchinson Cancer Center →