New stem cell filtering method could make transplants safer for kids
NCT ID NCT04806347
First seen Jun 27, 2026 · Last updated Sep 03, 2026 · Updated 6 times
Summary
This early-phase study tests a new way to do stem cell transplants for children and young adults with blood diseases. Doctors will take stem cells from a family member or closely matched unrelated donor, filter out certain immune cells using a special machine, and then give them to the patient. The goal is to see if this method helps the new stem cells grow without causing severe graft-versus-host disease, a common and dangerous complication.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- TCRαβ+ and CD19+ depleted hematopoietic stem cells
- What this could lead to
- If successful, this approach could offer a safer stem cell transplant option for children with blood diseases who lack a perfect donor match, reducing severe side effects.
- What could go wrong
- This is a very early (Phase 1) and small (12 participants) study, so results may not apply broadly. The procedure carries risks like graft failure, severe GVHD, or infection.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
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Phase 1
The first testing in people. Mainly checks safety and dose, usually in a small group.
- Participants
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About 12 people
The number the study aims to enrol. It can still change while the study runs.
- Expected to start
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Sep 2026
An estimate. Start dates often move.
- Expected to finish
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Jun 2030
An estimate. End dates often move.
- Lead sponsor
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Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
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3 months to 25 years
- Sex
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Anyone
- Healthy volunteers
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Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: * No Human leukocyte antigen (HLA) identical sibling available AND * NO HLA matched unrelated donor available OR urgent need of HSCT precludes time necessary to search for suitable HLA matched unrelated donor AND * Haploidentical donor OR closely matched unrelated donor available and willing to undergo mobilization and apheresis * If subject has genetically confirmed inherited bone marrow failure, related donor must be evaluated for this disorder and testing must be negative. * If subject has sickle cell disease, donor must be unaffected or have only sickle cell trait * Patient must be diagnosed with one of the following diseases or disorders: * Hemoglobinopathies * Sickle Cell Disease for patients ≤ 21 years of age for whom hydroxyurea has been trialed for at least six months, and failed * Thalassemia Major for patients ≤ 21 years of age * Acquired Bone Marrow Failure Syndromes * Paroxysmal Nocturnal Hemoglobinuria with bone marrow failure * Myelodysplastic Syndromes (lower risk) * Severe acquired aplastic anemia * Inherited Bone Marrow Failure Syndromes * Fanconi Anemia * Diamond Blackfan Anemia * Dyskeratosis Congenita and related telomere disorders * Congenital Thrombocytopenia Syndromes * Severe Congenital Neutropenia * Shwachman-Diamond Syndrome * Inborn Errors of Immunity (IEI) or Primary Immune Regulatory Disorders (PIRD) * Wiskott-Aldrich syndrome (WAS) * Chronic granulomatous disease (CGD) * Severe combined immunodeficiency (SCID) * Primary hemophagocytic lymphohistiocytosis (HLH) * Age ≤ 25 years (except patients with hemoglobinopathies) * Life Expectancy ≥ 3 months * Karnofsky (patients \> 16 years)/Lansky (patients ≤ 16 years) index ≥ 60 * Organ Function Requirements * Renal Function * Creatinine clearance or radioisotope Glomerular Filtration Rate (GFR) greater than or equal to 60 ml/min/1.73m\^2 * Liver Function * Total bilirubin \< 3 mg/dL * Alanine aminotransferase (ALT)/ Serum glutamic-pyruvic transaminase; synonymous with ALT (SCPT) ≤ 3 x Upper Limit of Normal (ULN) for age * Cardiac Function * Ejection fraction of \> 40% by Multiple gated acquisition scan (MUGA) or echocardiogram * Pulmonary Function * No evidence of dyspnea at rest * No supplemental oxygen requirement * If measured, carbon monoxide diffusion capacity (DLCO) \> 50% * Willing to use effective birth control method if patient is of reproductive potential * Informed consent obtained (patient or legal representative) Exclusion Criteria: * Pregnant * HIV infection * Uncontrolled, serious active infection at screening * Significant serious intercurrent illnesses * Enrollment in any other treatment study that would interfere with the endpoints of this study according to judgement of Principal Investigator (or PI designee).
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Genom att skicka in godkänner du våra Användarvillkor
Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
How to take part
Only the study team decides who joins. These are the ways to reach them.
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The study's own enquiry address
This study publishes an address for enquiries. See it below .
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The official record
ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.
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A doctor treating you
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Contacts and locations
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Genom att skicka in godkänner du våra Användarvillkor
Study contacts
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Contact
Email: •••••@•••••
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