NCT00152139

Brief Summary

Childhood leukemias which cannot be cured by chemotherapy alone may be effectively treated by allogeneic bone marrow transplantation. Moreover, for patients with chronic myelogenous leukemia (CML), allogeneic hematopoietic stem cell transplantation (HSCT) is the only proven curative modality of treatment. Patients who have received hematopoietic stem cells from an HLA matched sibling donor have proven to be less at risk for disease relapse and regimen related toxicity. However, about 70% of patients in need of HSCT do not have an HLA matched sibling donor. This necessitates the search for alternative donors, which may increase the risk of a poor outcome. The nature of the hematopoietic stem cell graft has been implicated as a primary factor determining these outcomes. The standard stem cell graft has been unmanipulated bone marrow, but recently several advantages of T-lymphocyte depleted bone marrow and mobilized peripheral blood progenitor cells (PBPC) have been demonstrated. However, T-cell depletion may increase the risk of infectious complications and leukemic recurrence while an unmanipulated stem cell graft may increase the risk of graft vs. host disease (GVHD). A key element in long range strategies in improving outcomes for patients undergoing matched unrelated donor (MUD) HSCT is to provide the optimal graft. The primary objective of this clinical trial is to estimate the incidence of acute GVHD in pediatric patients with hematologic malignancies who receive HSCT with an unmanipulated marrow graft. The results of this study can be used as the foundation for future trials related to engineering unrelated donor graft.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
33

participants targeted

Target at below P25 for phase_3

Timeline
Completed

Started May 2002

Longer than P75 for phase_3

Geographic Reach
1 country

1 active site

Status
completed

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

Study Start

First participant enrolled

May 1, 2002

Completed
3.1 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

June 1, 2005

Completed
3 months until next milestone

First Submitted

Initial submission to the registry

September 8, 2005

Completed
1 day until next milestone

First Posted

Study publicly available on registry

September 9, 2005

Completed
3.3 years until next milestone

Study Completion

Last participant's last visit for all outcomes

January 1, 2009

Completed
Last Updated

January 29, 2009

Status Verified

January 1, 2009

Enrollment Period

3.1 years

First QC Date

September 8, 2005

Last Update Submit

January 28, 2009

Conditions

Acute Lymphoblastic LeukemiasAcute Myelocytic LeukemiaChronic Myeloid LeukemiaJuvenile Myelomonocytic LeukemiaMyelodysplastic SyndromeHemoglobinuria, ParoxysmalNon-Hodgkin Lymphoma

Keywords

High risk hematologic malignanciesAllogeneic stem cell transplantMatched unrelated donor transplantationUnmanipulated stem cell graftBone marrow transplantation

Outcome Measures

Primary Outcomes (1)

  • To estimate the occurrence of acute graft versus host disease in patients who have received an unmanipulated hematopoietic stem cell transplant from a matched unrelated donor

    July 2005

Study Arms (1)

1

OTHER
Procedure: Allogeneic Stem Cell TransplantationDrug: Chemotherapy and antibodies

Interventions

Allogeneic Stem Cell TransplantationPROCEDURE

An infusion of HLA matched unrelated bone marrow or peripheral blood stem cells.

Also known as: Allogeneic stem cell transplant, Matched unrelated donor stem cell transplant, Bone marrow stem cell transplant
1
Chemotherapy and antibodiesDRUG

Participants received a standard conditioning regimen consisting of total body irradiation, cyclophosphamide, thiotepa and ATG. GVHD prophylaxis consisted of cyclosporine and Methotrexate.

Also known as: Graft versus host disease
1

Eligibility Criteria

Age2 Years - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • All patients lacking an HLA identical sibling for whom an unrelated donor matched at 6/6 HLA loci is formally requested within about 3 months of search initiation
  • Age greater than or equal to 24 months, but less than 21 years for new patients.
  • Diagnosis of one of the following high risk hematological malignancies:
  • Acute lymphoblastic leukemia (in second or subsequent remission or high risk in first remission)
  • Acute myeloid leukemia (in relapse or remission)
  • Secondary AML/MDS
  • Chronic myeloid leukemia
  • Juvenile myelomonocytic leukemia
  • Myelodysplastic syndrome
  • Paroxysmal nocturnal hemoglobinuria
  • Non-Hodgkin's lymphoma (in second or subsequent remission)

You may not qualify if:

  • Patients with symptomatic cardiac disease, or evidence of significant cardiac disease by echocardiogram, or cardiac shortening fraction below 25%
  • Patients with renal creatinine clearance \< 40ml/min/1.73m\^2
  • Patients with FVC \< 40% predicted or pulse oximetry less than or equal to 92% on room air if unable to perform pulmonary function tests
  • Patients with direct bilirubin \> 3 mg/dl
  • Patients with SGPT \> 500 U/L
  • Patients with a Karnofsky or Lansky performance score \< 70
  • Patients who have received a previous allogeneic stem cell transplant
  • Patients with a known allergy to rabbit or murine products
  • Patients with isolated extramedullary leukemic relapse, including isolated CNS or testicular recurrence

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

St. Jude Children's Research Hospital

Memphis, Tennessee, 38105, United States

Location

Related Links

MeSH Terms

Conditions

Precursor Cell Lymphoblastic Leukemia-LymphomaLeukemia, Myeloid, AcuteLeukemia, Myelogenous, Chronic, BCR-ABL PositiveLeukemia, Myelomonocytic, JuvenileMyelodysplastic SyndromesHemoglobinuria, ParoxysmalLymphoma, Non-Hodgkin

Interventions

Drug TherapyAntibodies

Condition Hierarchy (Ancestors)

Leukemia, LymphoidLeukemiaNeoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesLeukemia, MyeloidMyeloproliferative DisordersBone Marrow DiseasesChronic DiseaseDisease AttributesPathologic ProcessesPathological Conditions, Signs and SymptomsMyelodysplastic-Myeloproliferative DiseasesAnemia, HemolyticAnemiaLymphoma

Intervention Hierarchy (Ancestors)

TherapeuticsImmunoglobulinsImmunoproteinsBlood ProteinsProteinsAmino Acids, Peptides, and ProteinsSerum GlobulinsGlobulins

Study Officials

  • Gregory Hale, M.D.

    St. Jude Children's Research Hospital

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 3
Allocation
NON RANDOMIZED
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER

Study Record Dates

First Submitted

September 8, 2005

First Posted

September 9, 2005

Study Start

May 1, 2002

Primary Completion

June 1, 2005

Study Completion

January 1, 2009

Last Updated

January 29, 2009

Record last verified: 2009-01

Locations

US(1)