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New stem cell transplant option for patients without a perfect donor match

NCT ID NCT02356653

Disease control Sponsor: Children's Hospital of Philadelphia Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now This study
This trial is taking on new participants right now.
Not yet recruiting
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 24, 2026 · Last updated Jun 27, 2026 · Updated 1 time

Summary

This expanded access program offers a stem cell transplant using donor cells that have been specially processed to remove certain immune cells, reducing the risk of graft-versus-host disease. It is for patients with serious blood disorders, immune deficiencies, or metabolic diseases who lack a fully matched sibling donor and cannot join other clinical trials. Up to 100 participants will receive the transplant, and researchers will track survival, graft success, and complications.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

Active substance
Donor stem cells with T-cell depletion (CliniMACs)
What this could lead to
If successful, this approach could provide a treatment option for patients with life-threatening blood or immune disorders who lack a fully matched donor.
What could go wrong
This is an early-phase expanded access protocol, not a controlled trial. Risks include graft failure, graft-versus-host disease, and infection. Results may not apply broadly.

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

Early phase 1

The earliest testing in people: a first look at safety, in a very small group.

Participants

About 100 people

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

Start date

Dec 2013

Expected to finish

Jan 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

Up to 30 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: 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

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Conditions

The condition(s) this trial relates to.

acute leukemia Bone Marrow Failure Disorders bone marrow failure syndrome chronic leukemia hemoglobinopathy immune dysregulation-polyendocrinopathy-enteropathy-X-linked syndrome immunodeficiency disease inborn error of immunity inborn errors of metabolism leukemia lymphoma myelodysplastic syndrome

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Bone marrow failure syndromes Immunodeficiencies Immunodysregulation polyendocrinopathy enteropathy X-linked syndrome Inborn errors of metabolism Leukemia

How to take part

Only the study team decides who joins. These are the ways to reach them.

  1. The places running it

    1 site. The list below names each one and where it is.

  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

    A doctor who knows your case can contact a study site on your behalf, and can tell you whether this study is worth pursuing at all.

Contacts and locations

Locations

  • Children's Hospital of Philadelphia

    RECRUITING

    Philadelphia, Pennsylvania, 19104, United States

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Other studies related to the condition(s) this trial covers.