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New transplant approach aims to cure blood failure without harming lungs or liver

NCT ID NCT01659606

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

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
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 This study
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 Results may not be published yet.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This status has not been confirmed recently, so it may be out of date.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 tests a bone marrow transplant method that avoids harsh chemotherapy and radiation for people with dyskeratosis congenita, a genetic disorder causing bone marrow failure. The goal is to fix the blood system without worsening lung or liver disease or raising cancer risk. About 40 participants will receive this gentler transplant to see if it leads to successful engraftment and survival.

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 2

Tests whether the treatment actually works, and watches for side effects, in a larger group.

Participants

About 40 people

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

Started

Jul 2012

Expected to finish

Dec 2034

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

30 days to 65 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: * Bone marrow hypocellular for age * Moderate or severe aplastic anemia defined by one of the following: peripheral blood neutrophils \< 0.5 x 10\^9/L; platelets \< 30 x 10\^9/L or platelet transfusion dependence; reticulocytes \< 50 x 10\^9/L in anemic patients or red cell transfusion dependence * Diagnosis of dyskeratosis congenita based on clinical triad of abnormalities of skin pigmentation, nail dystrophy, oral leukoplakia; OR one of clinical triad and presence of two or more associated features; OR a pathogenic mutation in DKC1,TERC, TERT, NOP10, NHP2, TCAB1, TINF2, CTC1, PARN, RTEL1, ACD, NAF1, STN1, or ZCCHC8, as reported by a CLIA-approved laboratory; OR age-adjusted mean telomere length \< 1%ile in peripheral blood lymphocytes as reported by a CLIA-approved laboratory; OR Hoyeraal-Hreidarsson syndrome; OR Revesz syndrome * Availability of a related or unrelated donor with a 7/8 or 8/8 match for HLA-A, B, C, and DRB1. * Patient and/or legal guardian must be able to sign informed consent. * Donor must provide a marrow allograft. * Diagnosis of Fanconi anemia must be excluded by mitomycin C or diepoxybutane chromosomal breakage testing on peripheral blood at a CLIA-approved laboratory (not required for patients with a genetic mutation consistent with DC) * Adequate renal function with glomerular filtration rate equal to or greater than 30 ml/min/1.73 m2 Exclusion Criteria: * Clonal cytogenetic abnormalities associated with MDS or AML on bone marrow examination. * Karnofsky/Lansky performance status \< 40. * Uncontrolled bacterial, viral or fungal infections. * Positive test for the human immunodeficiency virus (HIV). * Pregnancy or breastfeeding. * Known severe or life-threatening allergy or intolerance to fludarabine, alemtuzumab, mycophenolate mofetil or both cyclosporine and tacrolimus. * Positive patient anti-donor HLA antibody, which is deemed clinically significant. * Prior allogeneic marrow or stem cell transplantation. * Prior solid organ transplantation.

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Conditions

The condition(s) this trial relates to.

aplastic anemia Bone Marrow Failure Disorders dyskeratosis congenita Hoyeraal-Hreidarsson syndrome Revesz syndrome

As listed by the trial registrant

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

Aplastic anemia Dyskeratosis congenita Hoyeraal hreidarsson syndrome Revesz syndrome

Contacts and locations

Locations

  • Baylor College of Medicine

    Houston, Texas, 77030, United States

  • Boston Children's Hospital (pediatric patients)

    Boston, Massachusetts, 02115, United States

  • Children's Hospital of Philadelphia

    Philadelphia, Pennsylvania, 19104, United States

  • Children's Mercy Hospital Kansas City

    Kansas City, Missouri, 64108, United States

  • Cincinnati Children's Hospital Medical Center

    Cincinnati, Ohio, 45229, United States

  • Dana-Farber Cancer Institute (adult patients)

    Boston, Massachusetts, 02115, United States

  • Fred Hutch/University of Washington/Seattle Children's Cancer Consortium

    Seattle, Washington, 98109, United States

  • Hackensack University Medical Center

    Hackensack, New Jersey, 07601, United States

  • Karolinska University Hospital

    Stockholm, Sweden

  • Massachusetts General Hospital

    Boston, Massachusetts, 02114, United States

  • Oslo University Hospital

    Oslo, Norway

  • University of Chicago

    Chicago, Illinois, 60637, United States

  • University of Wisconsin Hospital and Clinics

    Madison, Wisconsin, 53792, United States

More trials for these conditions

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