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AzTrials

Bone Marrow FailurePhase 2NCT07660783

Naive T Cell Deplete Grafts for GVHD Prevention in Non-Malignant Diseases

A Phase 2 study testing ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs in people with Bone Marrow Failure and Hemoglobinopathies, run by Fred Hutchinson Cancer Center at 2 sites in United States.

Not yet recruiting

Opening soon. The study is approved but enrolment has not started yet. Sites may already take enquiries.

Sites and contacts
Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 6 months to 50 years
What is being tested?
ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Could I receive a placebo?
No, everyone receives an active treatment
How many people
40 participants
Where
2 sitesUnited States
Study dates
Sep 2026to Sep 2035
Phase
Phase 2
Are there results?
Not yet: the study is under way

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 6 months to 50 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • Considered appropriate candidate for allogeneic HCT following low dose (4Gy) TBI containing-conditioning and have one of the following diagnoses: A) BMF B)Hemoglobinopathies C)PID D) Autoimmune cytopenias E) Immune dysregulation F) HLH G) Other NMD treatable by HCT and NMD that is not clearly defined (a patient with a NMD for whom genetic testing has been done and a genetic mutation responsible for their NMD phenotype has not been identified) are eligible for the study following discussion with and approval by the protocol PI
  • Patients aged 6 months- 5 years old (inclusive) at the time of informed consent
  • Patient with suitable HCT donor (see inclusion criteria below)
  • Recipient informed consent/assent (13 years and older), and/or legal guardian permission must be obtained

Who cannot join

  • Patient with aplastic anemia
  • Patients with severe combined immunodeficiency (SCID)
  • Fanconi anemia
  • Dyskeratosis congenita
  • Patient weight > 100 kg
  • Patients who are positive for HIV-1, HIV-2
Show all 14Show fewer
  • Patients with current neoplastic disorders
  • Patients with uncontrolled infections for whom HCT is considered contraindicated by the consulting infectious disease physician.
  • Patients with organ dysfunction including A) Renal insufficiency B) Impaired cardiac function C)Impaired pulmonary function D) Liver dysfunction
  • Patients who are pregnant or breast-feeding
  • Patients on other experimental protocols for prevention of GVHD
  • Patients of childbearing age who are presumed to be fertile and are unwilling to use an effective birth control method or refrain from sexual intercourse during and for 12 months post-HCT
  • Patients with any other significant medical conditions that would make them unsuitable for transplantation, as determined by the PI
  • Patients with a known hypersensitivity to tacrolimus or MMF

Trial locations and contacts

2 sites in 1 country. Contact the study team or a site near you to ask about taking part.

Madhavi Lakkaraja, MD, MPH contact

206-667-7166

mlakkara@fredhutch.org

United States2 sites

  • Seattle Children's Hospital

    Seattle, Washington, 98105

  • Fred Hutchinson Cancer Center

    Seattle, Washington, 98109

Other trials in United States

About this study

Official title: A Phase II Prospective Study Evaluating Selective Depletion of CD45RA+ (Naïve) T Cells (TND) From Peripheral Blood Stem Cell (PBSC) Grafts for Prevention of Graft Versus Host Disease (GVHD) in Non-Malignant Diseases (NMDs)

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.

Read the detailed description

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.

Being tested
ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Medical subject headings
Bone Marrow Failure Disorders, Hemoglobinopathies, Primary Immunodeficiency Diseases, Lymphohistiocytosis, Hemophagocytic

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCsBiological

    CD34-selected graft with CD45RA- depleted peripheral blood stem cells given to patients with Non-Malignant Diseases

Study groups in NCT07660783
Study groupRoleWhat they receive
Arm A (HLA-Haploidentical or mismatched unrelated donor)OtherConditioning 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.
Receives: Biological: ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs
Arm B (HLA-matched related or matched unrelated donor )OtherConditioning 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.
Receives: Biological: ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. GVHD-free Survival

    Free of grade III-IV acute and NIH chronic (moderate-severe) GVHD requiring systemic immunosuppression

    Measured: 1 year post-transplant

Secondary outcomes (8)

  1. Overall survival

    Measured: 1 year post-transplant

  2. Transplant related mortality

    Measured: Day 100 post-transplant and 1 year post-transplant

  3. 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.

    Measured: Day 42 post-transplant

  4. Graft rejection

    Graft rejection defined as <5% donor CD3 T cell and CD33 myeloid chimerism

    Measured: Day 100 post-transplant

  5. Incidence of chronic GVHD

    Defined and graded based on NIH criteria and graded operationally as the occurrence of compatible symptoms

    Measured: At 1 year and 2 years post transplant

  6. Prednisone for GVHD

    Number of participants alive and off prednisone (or equivalent systemic corticosteroid) for the treatment of GVHD

    Measured: Every 3 months post-transplant for the first 2 years

  7. 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

    Measured: First year post-transplant

  8. 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

    Measured: 2 years post-transplant

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Jun 2026
  2. Study startSep 2026 (estimated)
  3. Primary completionSep 2031 (estimated)
  4. Study completionSep 2035 (estimated)

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Non-randomised
Intervention model
Parallel groups
Masking
None (open label)
Enrolment
40 participants (estimated)
IDs, dates and publications
ClinicalTrials.gov ID
NCT07660783
Sponsor's protocol ID
20798
Other IDs
RG1126016
Status last verified
Jun 2026
First submitted
16 Jun 2026
Last updated
22 Jun 2026
Results posted
No
Data monitoring committee
Yes
FDA-regulated drug
Yes
FDA-regulated device
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing ALLOGENEIC CD34+ ENRICHED AND CD45RA- DEPLETED PBSCs in people with Bone Marrow Failure and Hemoglobinopathies, run by Fred Hutchinson Cancer Center at 2 sites in United States.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 4 inclusion and 14 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: opening soon, as of the sponsor's last update on 22 Jun 2026. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 2 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

This study is still under way. Results are not available in the source record yet.

How can a doctor refer a patient or contact the research team?

Contact Madhavi Lakkaraja, MD, MPH (206-667-7166, mlakkara@fredhutch.org), or a site near the patient. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

GVHD-free Survival (time frame: 1 year post-transplant). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT07660783.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Jun 2026.

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