Actively Recruiting

Phase 2
Age: 2Months - 55Years
All Genders
NCT01962415

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Led by Paul Szabolcs · Updated on 2025-12-15

100

Participants Needed

1

Research Sites

716 weeks

Total Duration

On this page

AI-Summary

What this Trial Is About

The objective of this study is to evaluate the efficacy of using a reduced-intensity condition (RIC) regimen with umbilical cord blood transplant (UCBT), double cord UCBT, matched unrelated donor (MUD) bone marrow transplant (BMT) or peripheral blood stem cell transplant (PBSCT) in patients with non-malignant disorders that are amenable to treatment with hematopoietic stem cell transplant (HSCT). After transplant, subjects will be followed for late effects and for ongoing graft success.

CONDITIONS

Official Title

Reduced Intensity Conditioning for Non-Malignant Disorders Undergoing UCBT, BMT or PBSCT

Who Can Participate

Age: 2Months - 55Years
All Genders

Eligibility Criteria

Eligible

You may qualify if you...

  • A 4/6, 5/6 or 6/6 HLA matched related or unrelated umbilical cord blood unit available with a minimum total nucleated cell dose of ≥ 3 x 10e7 cells/kg, or double cord blood units each delivering at least 2 x 10e7 cells/kg, OR an 8 of 8 or 7 of 8 HLA allele matched unrelated donor bone marrow or peripheral blood progenitor graft.
  • Adequate organ function as shown by: creatinine ≤ 2.0 mg/dL and creatinine clearance ≥ 50 mL/min/1.73 m2; hepatic transaminases (ALT/AST) ≤ 4 times the upper limit of normal; cardiac function with shortening fraction > 26% or ejection fraction > 40% or > 80% of normal for age; pulmonary function with CVC or FEV1/FVC ≥ 50% predicted for age and/or resting oxygen saturation ≥ 92% on room air, or pulmonologist clearance; for adults DLCO ≥ 50% if available.
  • Written informed consent and/or assent according to FDA guidelines.
  • Negative pregnancy test if pubertal and/or menstruating.
  • HIV negative.
  • Diagnosis of a non-malignant disorder eligible for stem cell transplant, including primary immunodeficiency syndromes, congenital bone marrow failure syndromes, inherited metabolic disorders, hereditary anemias, and inflammatory conditions such as Crohn's disease or inflammatory bowel disease.
Not Eligible

You will not qualify if you...

  • Allogeneic hematopoietic stem cell transplant within the previous 6 months.
  • Any active malignancy or myelodysplastic syndrome (MDS).
  • Severe acquired aplastic anemia.
  • Uncontrolled bacterial, viral, or fungal infection with progressive symptoms despite treatment.
  • Pregnancy or nursing mother.
  • Poorly controlled pulmonary hypertension.
  • Any condition that prevents serial follow-up visits.

AI-Screening

AI-Powered Screening

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Trial Site Locations

Total: 1 location

1

UPMC Children's Hospital of Pittsburgh

Pittsburgh, Pennsylvania, United States, 15224

Actively Recruiting

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Research Team

P

Paul Szabolcs, MD

CONTACT

S

Shawna McIntyre, RN

CONTACT

How is the study designed?

Study Type

INTERVENTIONAL

Masking

NONE

Allocation

NON_RANDOMIZED

Model

PARALLEL

Primary Purpose

TREATMENT

Number of Arms

2

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