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Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Haematological Diseases Using a Reduced Intensity Conditioning Regimen

Phase 2
2 Years
60 Years
Open (Enrolling)
Hematopoietic/Lymphoid Cancer

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Trial Information

Transplantation of Umbilical Cord Blood From Unrelated Donors in Patients With Haematological Diseases Using a Reduced Intensity Conditioning Regimen


- To assess the safety and efficacy of unrelated-donor umbilical cord blood
transplantation (UCBT) using a nonmyeloablative preparative regimen in patients with
hematological disease, in a multi-institution UK setting.

- To confirm that unrelated-donor UCBT following nonmyeloablative conditioning is
associated with consistent and durable engraftment in these patients.

- To assess transplant-related mortality at day 100 associated with nonmyeloablative UCBT
in these patients.

- To assess the incidence of grades II-IV and III-IV acute graft-vs-host disease (GVHD)
in these patients.

- To assess the risk of relapse and progressive disease in these patients at 1 year post
transplant after nonmyeloablative UCBT.

- To assess overall and progression-free survival of these patients at 1 year after
nonmyeloablative UCBT.

- To assess immune reconstitution at 1, 2, 3, 6, 12, and 24 months after transplant as
measured by quantitative recovery of B, T, and NK cells (flow cytometry), qualitative
recovery of T cells (TREC and spectratyping), in vivo functional T-cell responses (EBV
and CMV tetramers), and quantitative immunoglobulins.

OUTLINE: This is a multicenter study.

- Reduced-intensity conditioning regimen: Patients receive cyclophosphamide IV over 2
hours on day -6 and fludarabine phosphate IV over 1 hour on days -6 to -2. Patients
undergo a single fraction of total-body irradiation on day -1.

- Umbilical cord blood (UCB) transplantation: Patients undergo umbilical cord blood
transplantation on day 0.

- Graft-vs-host disease prophylaxis: Patients receive cyclosporine IV or orally on days
-3 to 100 followed by taper and mycophenolate mofetil IV or orally on days -3 to 35
followed by taper.

Blood and bone marrow samples are collected periodically for analysis.

After completion of study treatment, patients are followed up every 3 months in year 1,
every 4 months in year 2, every 6 months until 5 years, and then annually thereafter.

Peer Reviewed and Funded or Endorsed by Cancer Research UK.

Inclusion Criteria


- Diagnosis of high-risk, advanced or poorly responding hematological disease for which
a reduced-intensity hemopoietic stem cell transplantation is likely to be effective

- Disease status is such that there is no alternative therapy likely to achieve a
cure or provide a significant prolongation of disease-free survival

- No chronic myelogenous leukemia in first chronic phase responding to imatinib or
refractory blast crisis

- No acute leukemia in morphological relapse/persistent disease (defined as > 5% blasts
in normocellular bone marrow)

- No malignant disease that is refractory to or progressive on salvage therapy

- No myelofibrosis

- Donor must be matched at HLA-A and -B at antigen level and HLA-DRB1 at allelic level

- No available 5-6/6 HLA-A, -B, -DRB1 matched sibling donor OR 10/10 unrelated
volunteer donor


- Karnofsky performance status (PS) 60-100% OR Lansky PS 50-100% (pediatrics)

- Transaminases < 5 times upper limit of normal (ULN)

- Bilirubin < 3 times ULN

- Creatinine clearance > 50 mL/min

- DLCO > 50% predicted

- No supplemental oxygen requirements

- Not pregnant or nursing

- Negative pregnancy test

- No HIV or HTLV (I and II) antibody positivity or evidence of infection

- No acquired aplastic anemia

- No decompensated congestive heart failure or uncontrolled arrhythmia and left
ventricular ejection fraction ≥ 35%

- No current active serious infection, in particular uncontrolled fungal infection

- No congenital immune deficiencies


- See Disease Characteristics

- More than 6 months since prior exposure to combination chemotherapy OR only 1 course
of induction combination chemotherapy for myelodysplastic syndromes or acute myeloid
leukemia (please discuss with study coordinator/s if this course contained

- At least 6 months since prior myeloablative bone marrow transplantation

- No prior irradiation that precludes the safe administration of an additional dose of
200 cGy of total-body irradiation

- No prior autograft

Type of Study:


Study Design:

Allocation: Non-Randomized, Masking: Open Label, Primary Purpose: Treatment

Outcome Measure:

Non-relapse mortality at day 100

Safety Issue:


Principal Investigator

Rachael Hough, MD

Investigator Role:

Principal Investigator

Investigator Affiliation:

University College London Hospitals



Study ID:




Start Date:

January 2009

Completion Date:

Related Keywords:

  • Hematopoietic/Lymphoid Cancer
  • hematopoietic/lymphoid cancer
  • Hematologic Diseases