Status:
COMPLETED
Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
Lead Sponsor:
Baylor College of Medicine
Collaborating Sponsors:
Center for Cell and Gene Therapy, Baylor College of Medicine
Conditions:
Congenital Pediatric Disorders
Eligibility:
All Genders
Up to 17 years
Phase:
NA
Brief Summary
The purpose of this study is to determine the safety and effectiveness of Umbilical Cord Blood Transplant (UCBT) to treat the patient's disease, and to see if this treatment can decrease the incidence...
Detailed Description
Patients will be examined to make sure that they meet the requirements of this study. There will be tests of the heart and of the lungs. X-rays will be taken of the lungs and other organs, depending o...
Eligibility Criteria
Inclusion
- INCLUSION CRITERIA:
- Patients less than 18 years of age.
- Patients with a congenital or acquired immunologic, hematological, or metabolic pediatric disease (including SCID) in which stem cell transplantation has been beneficial.
- Related or Unrelated Umbilical Cord Blood Unit with 0-1 antigen mismatch, 5-6 HLA- A and B (at low to intermediate resolution) and DRB1 (at high resolution).
- Total cryopreserved HSC graft cell dose must be 5 x 10\^7 or greater nucleated cells per kilogram recipient body weight.
- Lansky/Karnofsky scores 60 or greater.
- Patient has DLCO \> 50% predicted or FEV1 \> 50%, if applicable.
- Written informed consent and/or signed assent line from patient, parent or guardian.
- EXCLUSION CRITERIA:
- Patients with uncontrolled infections as assessed by the principal investigator only. For bacterial infections, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to starting conditioning. For fungal infections patients must be receiving definitive systemic antifungal therapy and have no signs of progressing infection for 1 week prior to enrollment. Progressing infection is defined as hemodynamic instability attributable to sepsis or new symptoms, worsening physical signs or radiographic findings attributable to infection. Persisting fever without other signs or symptoms will not be interpreted as progressing infection.
- Severe renal disease (creatinine \> 3X normal for age).
- Severe hepatic disease (direct bilirubin \> 3 mg/dL or SGOT \> 500).
- Patients with symptomatic cardiac failure unrelieved by medical therapy or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction \< 20%).
- HIV positive.
Exclusion
Key Trial Info
Start Date :
September 1 2009
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
February 4 2021
Estimated Enrollment :
40 Patients enrolled
Trial Details
Trial ID
NCT00950846
Start Date
September 1 2009
End Date
February 4 2021
Last Update
October 30 2023
Active Locations (1)
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1
Texas Children's Hospital
Houston, Texas, United States, 77030