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Umbilical Cord Blood Transplant for Congenital Pediatric Disorders (UCB)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details. Identifier: NCT00950846
Recruitment Status : Active, not recruiting
First Posted : August 3, 2009
Last Update Posted : January 27, 2021
Center for Cell and Gene Therapy, Baylor College of Medicine
Information provided by (Responsible Party):
Caridad Martinez, Baylor College of Medicine

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).

The two main causes of death after umbilical cord blood transplantation for disorders for these kinds of patients, are graft failure and infection.

In this study we are trying to address these two problems by using different drugs to prepare patients for the transplant.

To help improve engraftment (cells begin to grow), we will include the drug Fludarabine to the usually used Busulfan and Cytoxan that the study patients will receive before their transplant.

We will try to decrease the chance of developing graft-versus-host disease (GvHD) by using Cyclosporin A (CSA) and Mycophenolate Mofetil (MMF), instead of Anti-Thymocyte Globulin (ATG) which is normally used.

Condition or disease Intervention/treatment Phase
Congenital Pediatric Disorders Drug: Busulfan Drug: Cytoxan Drug: Fludarabine Procedure: Cord Blood Stem Cell Infusion Not Applicable

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 on the disease. An MRI and consultations with different specialists will also be conducted.

Patients also must have a negative pregnancy test before entering this study if they are a woman of childbearing potential. The blood will be tested for viruses and to look at the functioning of the liver and kidneys. The examination also includes HIV testing. If the patient has HIV, they will not be able to be treated on this protocol.

After we have determined that the patient is eligible for treatment on this study and a suitable UCB stem donor has been found, they will have a central line placed.

After placement of the central line, the following chemotherapy will be given to after admission to the hospital:

  • 9 days before the infusion through 6 days before the infusion: Busulfan every 6 hours for 16 total doses.
  • 5 days before the infusion through 2 days before the infusion: Cytoxan given daily for 4 days over 2 hours. (It can be given over 1 to 4 hours if needed as decided by the physician). Mesna will be given per standards.
  • 4 days before the infusion through 1 day before the infusion: Fludarabine given daily for 4 days over 1 hour.

Stem cell transplant (infusion of the UCB stem cells) - defined as Day 0 of the treatment. All other "numbered" days relate to this infusion date. For example, Day 1 is the first day after the stem cell transplant.

Standard Therapy: Phenytoin will be given according to the standards of the TCH formulary.

Cyclosporin A (CSA) will be given starting 2 days prior to the stem cell infusion. It will be given daily over 2 hours every 12 hours, and then tapered if no GvHD is present.

Administration of Mycophenolate Mofetil (MMF) will start on the day the stem cell infusion is completed, and will continue daily for 45 days unless the patient develops GvHD.

Intravenous Immunoglobulins (IVIG) will be given as per CAGT SOP for infections prophylaxis.

Granulocyte Colony-Stimulating Factor (GCSF) will be given daily starting at Day +7 until ANC is greater than 2,500 for three consecutive days.

Study Evaluation: Patients will have various study evaluations, including blood samples, before and after the transplant.

Follow-Up: After year 1, the patients will be asked to return to the clinic once a year for consultations. These consultations with specialists will be similar to the ones the patients had before their transplant.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 40 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Umbilical Cord Blood Transplant for Congenital Pediatric Disorders
Actual Study Start Date : September 2009
Estimated Primary Completion Date : January 2025
Estimated Study Completion Date : January 2025

Resource links provided by the National Library of Medicine

Drug Information available for: Busulfan

Arm Intervention/treatment
Experimental: Umbilical Cord Blood Transplant Treatment Plan
Busulfan, Cytoxan, Fludarabine, Cord Blood Stem Cell Infusion
Drug: Busulfan

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.

Other Name: Busulfex

Drug: Cytoxan
(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.
Other Name: Cyclophosphamide

Drug: Fludarabine
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.
Other Name: Fludera

Procedure: Cord Blood Stem Cell Infusion
The cord blood stem cells will be infused on Day 0.

Primary Outcome Measures :
  1. Overall survival at 100 days, 1 year, 3 years, and 5 years after umbilical cord blood transplant in pediatric patients with congenital diseases. [ Time Frame: 5 years ]

Secondary Outcome Measures :
  1. Neutrophil and platelet count recovery at Day 42. [ Time Frame: Day 42 ]
  2. Incidence of severe Grade III-IV acute GvHD at Day 100. [ Time Frame: Day 100 ]
  3. Incidence of chronic GvHD at 1 year. [ Time Frame: 1 year ]
  4. Engraftment of donor cells at 100 days, 6 months, and 12 months after transplant. [ Time Frame: 100 days, 6 months and 12 months ]

Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.

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Ages Eligible for Study:   up to 17 Years   (Child)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No


  • 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.


  • 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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its identifier (NCT number): NCT00950846

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United States, Texas
Texas Children's Hospital
Houston, Texas, United States, 77030
Sponsors and Collaborators
Baylor College of Medicine
Center for Cell and Gene Therapy, Baylor College of Medicine
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Principal Investigator: Caridad Martinez, MD Baylor College of Medicine
  Study Documents (Full-Text)

Documents provided by Caridad Martinez, Baylor College of Medicine:
Informed Consent Form  [PDF] September 5, 2018

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Responsible Party: Caridad Martinez, Assistant Professor, Pediatric Hematology/Oncology, Center for Cell and Gene Therapy, Baylor College of Medicine Identifier: NCT00950846    
Other Study ID Numbers: 25064-UCB
First Posted: August 3, 2009    Key Record Dates
Last Update Posted: January 27, 2021
Last Verified: January 2021
Keywords provided by Caridad Martinez, Baylor College of Medicine:
Congenital Pediatric Disorders
Umbilical Cord Blood Transplant
Congenital Abnormalities
Cord Blood Stem Cell Transplantation
Additional relevant MeSH terms:
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Pathologic Processes
Immunosuppressive Agents
Immunologic Factors
Physiological Effects of Drugs
Antirheumatic Agents
Antineoplastic Agents, Alkylating
Alkylating Agents
Molecular Mechanisms of Pharmacological Action
Antineoplastic Agents
Myeloablative Agonists