Health TrialsbyHealthPanel

Umbilical Cord Blood Transplantation From Unrelated Donors

ConditionsAcute LeukemiaImmune Deficiency DisorderCongenital Hematological DisorderMetabolism DisorderAplastic AnemiaMyelodysplastic SyndromesChronic LeukemiaLymphomaMultiple MyelomaSolid Tumor
Trial statusRecruiting
Trial phaseNot listed
Trial typeObservational
Biological sexAll
Age2-75
SponsorUniversity of Rochester

About this trial

This study is being done to determine how long it takes for the engraftment (recovery of blood cell counts) of umbilical cord stem cells and also how often engraftment of umbilical cord stem cells transplanted from an unrelated donor fails. Another purpose will be to document the rate of disease-free survival and the rate of relapse (a return of your disease or syndrome) as well as the incidence and severity of graft versus host disease (GvHD) following cord blood stem cell transplantation. GvHD is a complication of stem cell transplants in which white blood cells from the transplanted tissue (graft) attack the transplant recipient's body (host).

Eligibility criteria

This trial does not accept healthy volunteers

Qualifiers

Immunodeficiency disorders (e.g. Severe Combined Immunodeficiency, Wiskott-Aldrich Syndrome)

Congenital hematopoietic stem cell defects (e.g. Chediak-Higashi Syndrome, Congenital Osteopetrosis, Osteogenesis Imperfecta)

Metabolic disorders (e.g. Hurler's Syndrome)

Severe aplastic anemia

Disqualifiers

Availability of 10/10 or 9/10 HLA-matched related or unrelated donor within a reasonable timeframe dictated by the clinical urgency of the transplant

Autologous HSCT < 6 months prior to proposed UCB transplant

Pregnant or breast feeding

Current uncontrolled infection

Trial design

Design model

Cohort

Time perspective

Prospective

Treatments tested in this trial

  • Total Body Irradiation 1200 cGy

    Radiation

    Total Body Irradiation 1200 cGy in 8 fractions

  • Total Body Irradiation 200 cGy

    Radiation

    Total Body Irradiation 200 cGy in one fraction

  • Cyclophosphamide

    Drug

    50 mg/kg or 60 mg/kg

  • Mesna

    Drug

    50 mg/kg or 60 mg/kg plus 10% loading dose

  • Cord Blood Infusion

    Procedure/Surgery

    Intravenous infusion of cord blood stem cells

  • Busulfan

    Drug

    0.8 mg/kg x 16 doses

  • Fludarabine

    Drug

    30 mg/m2/day x 5 or 40 mg/m2/day x 5

  • Melphalan

    Drug

    140 mg/m2

Treatment groups

30 Participants
are divided into 4 treatment groups
Group A: Full Intensity, TBI-based Conditioning4 interventions
Group B: Full Intensity, Chemo-based Conditioning4 interventions
Group C: Reduced Intensity Chemotherapy3 interventions
Group D: Non-Myeloablative Conditioning4 interventions

Trial outcomes

Primary outcomes

1

Engraftment of ANC and Platelets

The date of engraftment of ANC is the first of 3 consecutive days of ANC of 500 or higher based on daily CBC and Differential Counts. The date of engraftment of platelets is the first of three consecutive days of platelet counts of 20,000 or higher in the absence of platelet transfusions for a t least 7 days prior.

Time frame
42 days following the infusion of stem cells for ANC [If engraftment of ANC does not occur within 42 days, a subsequent transplant will be performed if a donor is available.]

Secondary outcomes

1

Rate of non-engraftment and of secondary graft failure

The percentage of patients who fail to initially engraft ANC will be tabulated as well as the percentage of patients who have primary engraftment of ANC but whose graft fails as evidenced by pancytopenia, failure of bone marrow function and loss of donor chimerism following initial engraftment of ANC.

Time frame
At 30 days, 100 days, 6 months and yearly from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.
2

Incidence of acute graft-versus-host disease

Routine physical exams, liver function tests, and clinical history of diarrhea and upper GI symptoms will be used to assess the presence of, the maximum severity of and the date of onset of Acute GvHD based on the criteria published by Przepioka et al., Bone Marrow Transplant 1995; 15(6):825-8 as used by the Center for International Blood \& Marrow Transplant Research. The percentage of patients developing symptoms of acute graft-versus-host disease will be tabulated.

Time frame
At 30 days and 100 days after transplant from the date of transplant until the date of documented acute GvHD.
3

Incidence of chronic graft-versus-host disease

Assess the presence of and the maximum severity of and the date of onset of chronic GvHD based on Sullivan KM, Blood 1981;57-267 as used by the Cneter for International Blood \& Marrow Transplant Research.

Time frame
At 100 days, 6 months and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.
4

Disease-free survival

Document and update the length of time a subject survives without recurrence of the disease for which they were transplanted at 30 days, 100 days, 6 months and yearly following the infusion of cord blood stem cells for as long as the subjects survive and remain disease-free.

Time frame
At 30 days, 100 days, 6 months and yearly after transplant from the date of transplant until the date of documented graft failure or the subject's death up to 120 months.

Other outcomes

Sponsors and contacts

Click on the lead sponsor to view all of their trials.

University of Rochester

Lead sponsor

Source ClinicalTrials.gov
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