Crohn'sRecruiting

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

Observational · No treatment assigned · 1 site · Paul Szabolcs

Official record & contacts on ClinicalTrials.gov ↗

In plain English

What this trial is testing

Condition(s) listed: Primary Immunodeficiency (PID), Congenital Bone Marrow Failure Syndromes and Inherited Metabolic Disorders (IMD). This is an observational study: researchers collect information (for example questionnaires, samples, or scans) but the study does not assign a treatment.

Who can join

Ages 2 Months to 60
Any sex

Some of the main requirements, copied from the record:

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:

Plus 41 more requirement(s). Only the trial team can confirm whether you qualify.

What's involved

About 50 participants are expected to take part.
Start date: 2020-08-20.
Main results expected around 2027-12-31 (estimate from the record).
1 study site listed (1 marked recruiting) in United States.

Main thing being measured

incidence of acute graft versus host disease (GVHD) (up to 5 years)

In the researchers’ words

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.

This is a short automatic summary built from the official record. A fuller plain-English card is coming; until then, please read the official record.

Questions to ask your doctor and the trial team

Am I likely to qualify, given my health history?
What would I have to do, and how often would I need to visit?
Could I get a placebo? What happens to my current treatment?
What are the possible risks and benefits?
Are travel or other costs covered?
What happens after the trial ends?

Official details

Status	Recruiting
Phase	Observational
Study type	Observational
Conditions	Primary Immunodeficiency (PID); Congenital Bone Marrow Failure Syndromes; Inherited Metabolic Disorders (IMD); Hereditary Anemias; Inflammatory Conditions
Lead sponsor	Paul Szabolcs
Enrollment	50 (estimated)
Start date	2020-08-20
Primary completion (est.)	2027-12-31
First posted	2020-08-27
Last updated	2026-01-13
Official title	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)
ClinicalTrials.gov ID	NCT04528355

Interventions

data collection (Drug)
Study subjects will receive alemtuzumab, melphalan, thiotepa, fludarabine and hydroxyurea-based, reduced-intensity conditioning regimen in accordance with clinical practice at UPMC Children's Hospital of Pittsburgh at the discretion of the treating physician. Medical data will be abstracted from subject's medical charts once the patient signs the informed consent.

Full eligibility criteria (from the record)

Inclusion Criteria:

1. Patient, parent, or legal guardian must have given written informed consent.
2. Patient must be 2 months to 60 years (inclusive) of age at time of consent for all diagnoses.
3. 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
4. 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.

Locations (1)

Site	City	Country	Site status
UPMC Children's Hospital of Pittsburgh	Pittsburgh, Pennsylvania	United States	Recruiting

For contacts at each site, use the official record.

Information only, not medical advice. Talk to your doctor and the trial team before making any decision about a clinical trial.