Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
UCB
1 other identifier
interventional
40
1 country
1
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 of GVHD. This study is for patients that were born with a disease that affects their body's metabolism or immune system. The doctor plans to treat the patient for this illness with a stem cell transplant. While improved medical care has allowed many people with these diseases to live longer, the only way to truly cure the diseases is by means of a stem cell transplant from a donor who does not have the disease. A stem cell transplant will replace sick cells with new healthy donor cells. Stem cells grow into different types of blood cells that people need, including red blood cells, white blood cells, and platelets. In a stem cell transplant, the patients own stem cells would be killed by chemotherapy drug and then replaced by stem cells from the donor. Stem cells can be collected from the bone marrow, peripheral blood or umbilical cords. In this study, umbilical cords will be the source of the stem cells. Currently, large inventories of umbilical cord blood units are available in public banks for transplantation in those lacking bone marrow donors. UCB transplants offer several advantages over adult bone marrow or peripheral blood stem cell transplants, including:
- 1.Rapid availability,
- 2.Absence of donor risk,
- 3.Low risk of transmissible infectious diseases,
- 4.Low risk of acute GvHD (as compared to recipients of unrelated donor marrow and peripheral blood cells).
Trial Health
Trial Health Score
Automated assessment based on enrollment pace, timeline, and geographic reach
participants targeted
Target at P25-P50 for not_applicable
Started Sep 2009
Longer than P75 for not_applicable
1 active site
Health score is calculated from publicly available data and should be used for screening purposes only.
Trial Relationships
Click on a node to explore related trials.
Study Timeline
Key milestones and dates
First Submitted
Initial submission to the registry
July 30, 2009
CompletedFirst Posted
Study publicly available on registry
August 3, 2009
CompletedStudy Start
First participant enrolled
September 1, 2009
CompletedPrimary Completion
Last participant's last visit for primary outcome
July 21, 2020
CompletedStudy Completion
Last participant's last visit for all outcomes
February 4, 2021
CompletedResults Posted
Study results publicly available
October 30, 2023
CompletedOctober 30, 2023
October 1, 2023
10.9 years
July 30, 2009
July 7, 2023
October 6, 2023
Conditions
Keywords
Outcome Measures
Primary Outcomes (3)
Overall Survival at 100 Days After Umbilical Cord Blood Transplant in Pediatric Patients.
To determine the overall survival rate at 100 days after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
100 days
Overall Survival at 1 Year After Umbilical Cord Blood Transplant in Pediatric Patients.
To determine the overall survival rate at 1 year after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
1 year
Overall Survival at 3 Years After Umbilical Cord Blood Transplant in Pediatric Patients.
To determine the overall survival rate at 3 years after umbilical cord blood transplant in pediatric patients with myeloid hematological malignancies.
3 years
Secondary Outcomes (5)
Number of Participants With Platelet Engraftment
Day 42
Incidence of Severe Grade III-IV Acute GvHD at Day 100.
Day 100
Number of Participants With Chronic GvHD
1 year
Number of Participants With Donor Engraftment After Transplant.
100 days, 6 months and 12 months
Number of Participants With Neutrophil Engraftment
Day 42
Study Arms (1)
Umbilical Cord Blood Transplant Treatment Plan
EXPERIMENTALBusulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Interventions
Day -9, -8, -7 and -6 Patients less than or equal to 12 kg: 1.1 mg/kg/dose IV every 6 hours for 16 doses total; patients \>12 kg: 0.8 mg/kg/dose IV every 6 hours for 16 doses.
(50 mg/kg/dose) will be given IV on Days -5, - 4, -3, and -2 over 2 hours (can be given over 1 to 4 hours as determined by the treating physician). The total dose to be given over 4 days is 200 mg/kg.
40 mg/m2/day IV over 1 hour for patients greater than 10 kg, or 1.3 mg/kg/day for patients less than or equal to 10 kg.
The cord blood stem cells will be infused on Day 0.
Eligibility Criteria
You may qualify if:
- 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.
You may not qualify if:
- 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.
Contact the study team to confirm eligibility.
Sponsors & Collaborators
Study Sites (1)
Texas Children's Hospital
Houston, Texas, 77030, United States
Related Publications (1)
Martinez C, Aguayo-Hiraldo P, Chaimowitz N, Forbes L, Rider N, Nicholas S, Seeborg F, Chinen J, Chinn I, Davis C, Roseblatt H, Noroski L, Omer B, John T, Yassine K, Naik S, Craddock J, Bhar S, Allen C, Ahmed N, Sasa G, Steffin D, Doherty E, George A, Salem B, Friend B, Hegde M, Brenner MK, Heslop HE, Leen A, Pena A, Wu M, Hanson IC, Krance RA. Cord blood transplantation for nonmalignant disorders: early functional immunity and high survival. Blood Adv. 2023 May 9;7(9):1823-1830. doi: 10.1182/bloodadvances.2022009038.
PMID: 36453638DERIVED
MeSH Terms
Conditions
Interventions
Condition Hierarchy (Ancestors)
Intervention Hierarchy (Ancestors)
Results Point of Contact
- Title
- Dr. Caridad A. Martinez
- Organization
- Baylor College of Medicine/Texas Children's Hospital
Study Officials
- PRINCIPAL INVESTIGATOR
Caridad Martinez, MD
Baylor College of Medicine
Publication Agreements
- PI is Sponsor Employee
- Yes
Study Design
- Study Type
- interventional
- Phase
- not applicable
- Allocation
- NA
- Masking
- NONE
- Purpose
- TREATMENT
- Intervention Model
- SINGLE GROUP
- Sponsor Type
- OTHER
- Responsible Party
- PRINCIPAL INVESTIGATOR
- PI Title
- Assistant Professor, Pediatric Hematology/Oncology, Center for Cell and Gene Therapy
Study Record Dates
First Submitted
July 30, 2009
First Posted
August 3, 2009
Study Start
September 1, 2009
Primary Completion
July 21, 2020
Study Completion
February 4, 2021
Last Updated
October 30, 2023
Results First Posted
October 30, 2023
Record last verified: 2023-10