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)

Study point of contact

Shawna McIntyre, RN
412-692-5552
[email protected]
Paul Szabolcs, MD
412-692-5427
[email protected]

Locations

1 United States site

Age

2 to 60 Years

Study type

Observational

Gender

All

Interventions

Drug

Compensation

Unknown

About the study

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.

participation requirements

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

participation restrictions

.

Locations

  • Pittsburgh, Pennsylvania, United States, UPMC Children's Hospital of Pittsburgh, 15224 [Recruiting]