ClinicalTrials.gov record NCT04528355

Data Collection Study of Patients With Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT With RIC

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Jul 27, 2026

A Prospective Outcomes Study of Pediatric and Adult Patients With Non-Malignant Disorders Undergoing Umbilical Cord Blood, Bone Marrow, or Peripheral Blood Stem Cell Transplantation With a Reduced-Intensity Conditioning Regimen (PRO-RIC)

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
Age2 months old to 60 years old
SexAll
LocationUnited States
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This prospective data-collection study follows children and people with a range of non-malignant disorders who receive umbilical cord blood, bone marrow, or peripheral blood stem cell transplants with a reduced-intensity conditioning regimen that includes alemtuzumab. Investigators will abstract medical-record data to track engraftment, graft-versus-host disease, immune recovery, immunosuppression use, donor leukocyte infusions, and survival for up to five years. The study observes and records outcomes; it does not assign treatments.

What it is studying. The study is collecting medical-record data to describe outcomes after reduced-intensity conditioning hematopoietic stem cell transplantation using alemtuzumab-containing regimens. It aims to characterize rates and severity of graft-versus-host disease, donor engraftment (chimerism), tempo of immune reconstitution, discontinuation of systemic immunosuppression, use of donor leukocyte infusion, and overall survival.

What the researchers hope to learn. To describe engraftment, incidence and severity of graft-versus-host disease, the tempo of immune reconstitution, probability of stopping systemic immunosuppression, use of donor leukocyte infusions, and overall survival after alemtuzumab-based reduced-intensity conditioning HSCT. To provide outcome data that may serve as a reference for emerging gene-therapy strategies.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

This is a data collection study that will examine the general diagnostic and treatment data associated with the reduced-intensity chemotherapy-based regimen paired with simple alemtuzumab dosing strata designed to prevented graft failure and to aid in immune reconstitution following hematopoietic stem cell transplantation.

Interventions being studied

Being studied in this trial does not mean the treatment or procedure is approved or available for this condition.

Drug
  • data collection

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Patient, parent, or legal guardian must have given written informed consent.
  • Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
  • Patients should have a non-malignant disorder amenable to treatment by stem cell transplantation, including but not limited to the following:
  • A. Primary Immunodeficiency Syndromes
  • Severe Combined Immune Deficiency (SCID) with NK cell activity
  • Omenn Syndrome
  • Bare Lymphocyte Syndrome (BLS)
  • Combined Immune Deficiency (CID) syndromes
  • Combined Variable Immune Deficiency (CVID) syndrome
  • Wiskott-Aldrich Syndrome
  • Leukocyte adhesion deficiency
  • Chronic granulomatous disease (CGD)
  • Hyper IgM (XHIM) syndrome
  • IPEX syndrome
  • Chediak-Higashi Syndrome
  • Autoimmune Lymphoproliferative Syndrome (ALPS)
  • Hemophagocytic Lymphohistiocytosis (HLH) syndromes
  • Lymphocyte Signaling defects
  • B. Congenital Bone Marrow Failure Syndromes
  • Congenital Amegakaryocytic Thrombocytopenia (CAMT)
  • Osteopetrosis
  • C. Inherited Metabolic Disorders (IMD)
  • Mucopolysaccharidoses
  • Hurler syndrome (MPS I)
  • Hunter syndrome (MPS II)
  • Leukodystrophies
  • Krabbe Disease, also known as globoid cell leukodystrophy
  • Metachromatic leukodystrophy (MLD)
  • X-linked adrenoleukodystrophy (ALD)
  • Other inherited metabolic disorders
  • Alpha Mannosidosis
  • Gaucher Disease
  • Other inheritable metabolic diseases where HSCT may be beneficial
  • D. Hereditary Anemias
  • Thalassemia major
  • Sickle cell disease (SCD)
  • Diamond Blackfan Anemia (DBA)
  • E. Inflammatory Conditions
  • Crohn's Disease or Inflammatory Bowel Disease
  • IPEX or IPEX-like Syndromes
  • Rheumatoid Arthritis
  • Other inflammatory conditions where HSCT may be beneficial
  • Subjects receive either umbilical cord blood, bone marrow, or peripheral blood stem cell transplant with an alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen, according to clinical practice at UPMC Children's Hospital of Pittsburgh.
  • There are no

Exclusion criteria

  • .

Study description and what participation involves

The study's detailed description, as published on ClinicalTrials.gov.

Detailed description from the registry

Hematopoietic stem cell transplantation (HSCT) from a healthy donor can cure or alleviate a broad spectrum of non-malignant disorders (NMD). Although reduced-intensity conditioning (RIC) regimens promise decreased treatment-related morbidity and mortality, graft failure and infections are limiting the use of RIC in chemotherapy-naive patients. Dr. Szabolcs have completed several trials to evaluate a novel RIC regimen of alemtuzumab, hydroxyurea, fludarabine, melphalan, and thiotepa. The last trial at UPMC Children's Hospital of Pittsburgh of a highly effective and biologically rational chemotherapy-based RIC regimen paired with simple alemtuzumab dosing strata was tested and resulted in outstanding survival and remarkably low rates of graft failure. The favorable outcome described may serve as a toxicity and efficacy reference for emerging gene therapy strategies as well. This prospective collection of clinical data will allow the investigators to further assess engraftment, GVHD, immunosuppressant use and overall survival in this patient population.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

After giving written consent, study staff will abstract and review your existing medical records and test results related to the transplant and follow-up care. Data collected will include outcomes such as graft-versus-host disease, survival status, donor chimerism (engraftment), use and discontinuation of systemic immunosuppression (checked at about 6, 9, and 12 months), tempo of immune reconstitution over the first year, and any use of donor leukocyte infusion; some outcomes may be followed by record review for up to 5 years. The transplant and conditioning drugs are given as part of clinical care per the treating team; the registry record describes medical-record data collection rather than assigning additional study-only treatments or procedures.

How the study is structured. A prospective observational cohort study. No treatments are assigned by the study; participants receive clinical care per their treating team. Study staff will abstract data from participants' medical records and follow outcomes (including engraftment, GVHD, immune recovery, immunosuppression use, DLI, and survival) for up to five years after transplant.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in United States. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on Hunter syndrome

This trial is also listed on this site under alpha-mannosidosis, Gaucher disease, hemophagocytic lymphohistiocytosis, Hurler syndrome, Krabbe disease and metachromatic leukodystrophy.

Clinical trials10 trials listed for Hunter syndromeEvery trial the registry lists for Hunter syndrome, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in Hunter syndrome researchLatest monthly update: September 2026. Includes: Brain-targeted enzyme. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Aug 20, 2020
Primary completion
Dec 31, 2027 (estimated)
Study completion
Jun 30, 2028 (estimated)
Target enrollment
50 participants (estimated)
Last updated on registry
Jan 13, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Paul Szabolcs

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (1)

  • UPMC Children's Hospital of PittsburghRecruiting

    Pittsburgh, Pennsylvania, 15224 · United States

Terms used on this page · explanations by MedicalUpdates.ai

legal guardian
A person who is legally allowed to make decisions, including consent to research, on behalf of someone who cannot consent for themselves.
written informed consent
The process of explaining a study, including its risks and what participation involves, so that a person can decide freely whether to take part. Consent is usually documented in writing, and a participant can choose to leave the study later.
HSCT
A procedure that replaces blood-forming cells after high-dose treatment. 'Autologous' uses the person's own cells collected beforehand; 'allogeneic' uses cells from a donor.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
exclusion criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.
immunosuppressant
A medicine that reduces the activity of the immune system. Studies of autoimmune conditions often require a stable dose, a prior failure, or a washout of such medicines.
overall survival
The length of time from the start of the study or treatment that participants are alive, whatever the cause of death.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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