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First step toward gene therapy for rare anemia: can stem cells be harvested?

NCT ID NCT07186179

Knowledge-focused Sponsor: Northwell Health 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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 27, 2026 · Last updated Jun 27, 2026

Summary

This study checks if people with Diamond Blackfan Anemia (ages 3-30) can produce enough blood stem cells for future gene therapy. Participants receive two drugs to move stem cells from bone marrow into the blood, then researchers measure the cell count. No actual stem cell collection is done—this is a feasibility test to see if enough cells can be mobilized.

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.

Participants

About 10 people

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

Expected to start

Jun 2026

An estimate. Start dates often move.

Expected to finish

Apr 2027

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.

Who is studied

Patients with Diamond Blackfan Anemia Syndrome (DBAS) ages 3-30 with red blood cell transfusion dependence

Ages

3 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. Diamond Blackfan anemia syndrome as defined by the known criteria with a known gene mutation 2. Male or female patients of all ethnic background, greater than or equal to 3 years of age and weighing at least 10 kg, and less than or equal to 30 years of age 3. Enrolled in Diamond Blackfan Anemia Registry of North America (DBAR) 4. Chronically red blood cell transfusion dependent for at least 6 months 5. Performance scale (Lansky Play-performance Scale for Pediatric Functional Status for age \<16 years; Karnofsky Performance Scale for age ≥16 years) ≥ 70 6. Must sign informed consent Exclusion Criteria: 1. Receiving prednisone therapy for treatment of DBAS (this does not include patients receiving physiologic steroid replacement for adrenal insufficiency) 2. Known history of myelodysplasia or presence of a hematopoietic clone 3. Current malignancy or previous treatment for malignancy 4. Pregnancy or breast-feeding mother 5. Known history of severe iron overload as defined by a liver iron concentration (LIC) \> 15 mg Fe/ g dry liver weight 6. Significant cytopenias, defined as: * Platelet count \<100,000/mcL * Absolute neutrophil count \<750/mCL 7. Any GCSF use in the 3 months prior to enrollment 8. Liver dysfunction: aspartate aminotransferase (AST), alanine aminotransferase (ALT), or direct bilirubin values \>3 x the upper limit of normal (ULN) 9. Kidney dysfunction: baseline estimated glomerular filtration rate (GFR) \<70 mL/min/1.73 m2

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Conditions

The condition(s) this trial relates to.

Diamond-Blackfan anemia

As listed by the trial registrant

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

Diamond Blackfan anemia

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

  • Cohen Children's Medical Center

    RECRUITING

    New Hyde Park, New York, 11040, United States

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