Umbilical Cord Blood Transplant for Children With Lymphoid Hematological Malignancies

NCT01247688 | Withdrawn DMC

• 1 other identifier: 26336-UCALL
• Study Type: interventional
• Target: N/A
• Locations: 0 countries
• Sites: N/A

Brief Summary

The purpose of this study is to determine the safety and effectiveness of UCBT to treat patients with lymphoid hematological diseases and to see if this treatment can decrease the incidence of leukemia relapse, GVHD and infections. These patients have a type of blood cell disorder that is very hard to cure. This treatment that is being used in this trial is known as a stem cell transplant. This treatment might help the patient live longer without the disease. It uses much stronger doses of drugs and radiation to kill the diseased cells that could be given without the transplant. We also think that the healthy cells from the donor may help fight any diseased cells left after the transplant. For the transplant to take place, we will administer stem cells from a 'donor' whose cells best 'match' the patient's. In this study umbilical cords will be the source of the stem cells. Before the transplant, two very strong drugs plus total body irradiation will be given to as preconditioning. This treatment will kill most of your blood-forming cells in the bone marrow. The patient will then get then healthy stem cells. If the patient has the disease in the central nervous system (CNS), they will receive radiation to the head and spine before starting the conditioning. This is to try to get disease control in the CNS. Radiation will not be given for children under 2 yrs old. Currently, many umbilical cord blood units are available in public banks for transplantation in patients lacking bone marrow donors. UCB transplants (UCBT) may offer several advantages over adult bone marrow or peripheral blood stem cell transplants, including:

1. 1.rapid availability,
2. 2.absence of donor risk,
3. 3.low risk of transmissible infectious diseases,
4. 4.low risk of acute GvHD (Graft vs. Host Disease)

Conditions

Lymphoid Hematological Malignancies; Umbilical Cord Blood Transplant

Keywords

Lymphoid; hematologic malignancy; UCB; Umbilical Cord Blood Transplant; Total Body Irradiation; Cytoxan; Cyclophosphamide; Fludarabine

Outcome Measures

Primary Outcomes (1)

• Overall Survival at 1 year after UCB transplant in pediatric patients

  To determine the survival rate at 1 year after umbilical cord blood transplant in pediatric patients with lymphoid hematological malignancies

  1 year

Secondary Outcomes (7)

• Assessment of Platelet Count Recovery

  Day 42

• Number of participants with severe acute GVHD grade III-IV as an assessment of safety

  Day 100

• Number of participants with chronic GvHD as an assessment of safety

  1 year

• Assess relapse rate after transplant

  1 and 3 years

• Assessment of neutrophil count recovery

  Day 42

Study Arms (1)

Umbilical Cord Blood Transplant Treatment Plan

EXPERIMENTAL

Cytoxan, Fludarabine, Total Body Irradiation (TBI), Cord Blood Stem Cell Infusion

Drug: Cytoxan; Drug: Fludarabine; Radiation: Total Body Irradiation (TBI); Procedure: Cord Blood Stem Cell Infusion

Interventions

Cytoxan DRUG

50 mg/kg/dose will be given IV on day -5,-4, -3, -2 over 1 hour. The total dose to be given over 4 days is 200 mg/kg.

Also known as: Cyclophophamide

Umbilical Cord Blood Transplant Treatment Plan

Fludarabine DRUG

40mg/m2/day will be given IV over 1 hour on day -4, -3, -2

Also known as: Fludara

Umbilical Cord Blood Transplant Treatment Plan

Total Body Irradiation (TBI) RADIATION

1.75Gy/fraction will be given on day -9, -8, -7, -6

Umbilical Cord Blood Transplant Treatment Plan

Cord Blood Stem Cell Infusion PROCEDURE

The cord blood stem cells will be infused on day 0

Umbilical Cord Blood Transplant Treatment Plan

Eligibility Criteria

• Sex: all
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• Potential recipients of umbilical cord blood transplantation (i.e. HLA (human leukocyte antigen) matched or mismatched / related or unrelated) with a lymphoid hematologic malignancy (acute lymphoblastic leukemia, hodgkin/non-hodgkin lymphoma) unlikely to be cure by standard chemotherapy. This includes patients who have relapsed after standard chemotherapy treatments and patients in first remission with unfavorable prognostics features.
• Related or Unrelated Umbilical Cord Blood Unit with 0-1 antigen mismatch, Total Nucleated cell dose of greater than or equal to 4 x 10\^7/kg.
• Lansky/Karnofsky scores greater than or equal to 60
• Negative pregnancy test (if applicable)
• Written informed consent and/or signed assent line from patient, parent or guardian.

You may not qualify if:

• Patients with uncontrolled infections. For bacterial infections, patients must be receiving definitive therapy and have no signs of progressing infection for 72 hours prior to enrollment. 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 \> x 3 normal for age)
• Severe hepatic disease (direct bilirubin \> 3 mg/dl or SGOT (Serum glutamic oxaloacetic transaminase) \> 500)
• Patient has DLCO (Diffusing Capacity of the Lung for Carbon Monoxide) \< 50% predicted or FEV1 (Forced expiratory volume ) \< 50% of predicted, if applicable
• Patients with symptomatic cardiac failure unrelieved by medical therapy or evidence of significant cardiac dysfunction by echocardiogram (shortening fraction\<20%).
• HIV positive

Sponsors & Collaborators

• Baylor College of Medicine: lead
• Center for Cell and Gene Therapy, Baylor College of Medicine: collaborator

MeSH Terms

Conditions

Hematologic Neoplasms

Interventions

Cyclophosphamide; fludarabine; fludarabine phosphate; Whole-Body Irradiation

Condition Hierarchy (Ancestors)

Neoplasms by Site; Neoplasms; Hematologic Diseases; Hemic and Lymphatic Diseases

Intervention Hierarchy (Ancestors)

Phosphoramide Mustards; Nitrogen Mustard Compounds; Mustard Compounds; Hydrocarbons, Halogenated; Hydrocarbons; Organic Chemicals; Phosphoramides; Organophosphorus Compounds; Radiotherapy; Therapeutics; Investigative Techniques

Study Officials

• Caridad A Martinez, MD

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

• Robert A Krance, MD

  Baylor College of Medicine

  PRINCIPAL INVESTIGATOR

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: November 17, 2010
• First Posted: November 24, 2010
• Study Start: November 1, 2010
• Primary Completion: December 1, 2013
• Study Completion: December 1, 2013
• Last Updated: December 12, 2013

Record last verified: 2013-12