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New stem cell filtering method could make transplants safer for kids

NCT ID NCT04806347

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting This study
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

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

Phase 1

The first testing in people. Mainly checks safety and dose, usually in a small group.

Participants

About 12 people

The number the study aims to enrol. It can still change while the study runs.

Expected to start

Sep 2026

An estimate. Start dates often move.

Expected to finish

Jun 2030

An estimate. End dates often move.

Lead sponsor

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

3 months to 25 years

Sex

Anyone

Healthy volunteers

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

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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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.

  1. The study's own enquiry address

    This study publishes an address for enquiries. See it below .

  2. The official record

    ClinicalTrials.gov lists the study team's own contact details, including names and phone numbers. We don't republish those.

    Open the record ↗

  3. A doctor treating you

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Contacts and locations

Study contacts

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