NCT01050439

Brief Summary

Unrelated matched donor (cord blood, bone marrow or peripheral blood) allogeneic stem cell transplantation (UDAlloSCT) with either myeloablative or reduced intensity conditioning will be well tolerated and result in a high degree of engraftment in patients with selected malignant and non malignant disorders.

Trial Health

57
Monitor

Trial Health Score

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

Enrollment
22

participants targeted

Target at P25-P50 for phase_2 leukemia

Timeline
Completed

Started Nov 2002

Typical duration for phase_2 leukemia

Geographic Reach
1 country

1 active site

Status
terminated

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

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Study Timeline

Key milestones and dates

Study Start

First participant enrolled

November 1, 2002

Completed
5.6 years until next milestone

First Submitted

Initial submission to the registry

June 3, 2008

Completed
1.6 years until next milestone

First Posted

Study publicly available on registry

January 15, 2010

Completed
1.2 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

April 1, 2011

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

April 1, 2011

Completed
Last Updated

April 15, 2015

Status Verified

April 1, 2015

Enrollment Period

8.4 years

First QC Date

June 3, 2008

Last Update Submit

April 14, 2015

Conditions

LeukemiaLymphomaBone Marrow Failure SyndromesImmunodeficienciesHistiocytosis

Keywords

Allogeneic Stem Cell TransplantUnrelated donorCord blood donor

Outcome Measures

Primary Outcomes (1)

  • Incidence of toxicity related to myeloablative therapy

    To determine the safety and toxicity of myeloablative therapy (TBI + Melphalan) and unrelated donor alloSCT in selected patients with malignant and non-malignant disorders.

    Up to 10 years from start of study

Secondary Outcomes (3)

  • Incidence of toxicity related to reduced intensity therapy

    Up to 10 years from start of study

  • Percentage of donor chimerism

    Up to 10 years from start of study

  • Prevalence of progression free survival

    Up to 10 years from start of study

Study Arms (1)

UDAlloSCT + Therapy

EXPERIMENTAL

This is a non-randomized study to test the safety and response of unrelated matched donor allogeneic stem cell transplantation (UDAlloSCT) with either myleoablative (full intensity) or reduced intensity conditioning therapy in patients with selected malignant and non-malignant disorders. UDAlloSCT has been performed in both adults and children as an alternative transplant for patients who lack and HLA-matched family donor in both malignant and non-malignant disease with varying degrees of response.

Procedure: UDAlloSCTOther: Therapy

Interventions

UDAlloSCTPROCEDURE

unrelated matched donor allogeneic stem cell transplantation (UDAlloSCT)

UDAlloSCT + Therapy
TherapyOTHER

Full Intensity Therapy (myeloablative) (TBI + Thiotepa + Cyc) OR Reduced Intensity Therapy (Fludarabine, Busulfan, and Alemutuzumab (FBA))

UDAlloSCT + Therapy

Eligibility Criteria

AgeUp to 55 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • Adequate renal function defined as: serum creatinine 2.0 x normal, or creatinine clearance or radioisotope GFR \> 40 ml/min/m2 or \> 40 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range.
  • Adequate liver function defined as: total bilirubin \< 2.5 x normal; or SGOT (AST) or SGPT (ALT) \< 5.0 x normal.
  • Adequate cardiac function defined as: shortening fraction of \> 25% by echocardiogram, or ejection fraction of \> 40% by radionuclide angiogram or echocardiogram.
  • Adequate pulmonary function defined as: DLCO \> 35% by pulmonary function test. For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \> 94% in room air.
  • Diseases:
  • CML (CP, AP or BC)
  • AML/MDS/JCML
  • ALL
  • Lymphoma (Hodgkin's and non-Hodgkin's)
  • Non-malignant disorders
  • Bone Marrow Failure Syndromes: Patients with the following diagnoses are eligible:
  • Severe Aplastic Anemia:
  • Fanconi Anemia
  • Severe Congenital Neutropenia (Kostmann's Syndrome)
  • Amegakaryocytic Thrombocytopenia
  • +23 more criteria

You may not qualify if:

  • Women who are pregnant and/or breast feeding are ineligible

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Columbia University Medical Center

New York, New York, 10032, United States

Location

MeSH Terms

Conditions

LeukemiaLymphomaBone Marrow Failure DisordersImmunologic Deficiency SyndromesHistiocytosis

Interventions

Therapeutics

Condition Hierarchy (Ancestors)

Neoplasms by Histologic TypeNeoplasmsHematologic DiseasesHemic and Lymphatic DiseasesLymphoproliferative DisordersLymphatic DiseasesImmunoproliferative DisordersImmune System DiseasesBone Marrow Diseases

Study Officials

  • Mitchell S Cairo, MD

    Columbia University

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
interventional
Phase
phase 2
Allocation
NA
Masking
NONE
Purpose
TREATMENT
Intervention Model
SINGLE GROUP
Sponsor Type
OTHER
Responsible Party
SPONSOR

Study Record Dates

First Submitted

June 3, 2008

First Posted

January 15, 2010

Study Start

November 1, 2002

Primary Completion

April 1, 2011

Study Completion

April 1, 2011

Last Updated

April 15, 2015

Record last verified: 2015-04

Locations

US(1)