Clinical trial NCT00801931: Double Cord Blood Transplant for Patients With Malignant and Non-malignant Disorders
Official title: Double Umbilical Cord Blood Transplantation for Patients With Malignant and Non-Malignant Disorders
Study type: INTERVENTIONAL
Overall status: TERMINATED
Phase: PHASE1
PHASE2
Conditions: Leukemia
Lymphoma
Neuroblastoma
Immunodeficiencies
Anemia
Keywords: Cord Blood Transplant
Allogeneic Stem Cell Transplant
Lead sponsor: Columbia University
Sponsor class: OTHER
First posted: 2008-12-04
Enrollment: 1
Sex: ALL
Maximum age (years): 30
Standard ages: CHILD
ADULT
Accepts healthy volunteers: no
Allocation: NON_RANDOMIZED
Intervention model: PARALLEL
Primary purpose: TREATMENT

Brief summary: The purpose of this study is to determine the safety and toxicity and feasibility of double umbilical cord blood transplantation (DUCBT) in patients with selected malignant and non-malignant, and to quantify the percentage and donor sources of mixed donor chimerism following DUCBT in patients with selected malignant and non-malignant disorders.

Detailed description: Allogeneic stem cell transplantation from an human leukocyte antigen (HLA) matched related family donor is the treatment of choice for a wide variety of malignant and non-malignant disorders. Unfortunately, only 25% of potential recipients have an HLA matched related family donor, leaving approximately 75% of potential recipients requiring alternative sources of HLA matched allogeneic stem cells. One potential source of HLA matched allogeneic stem cells is from unrelated adult donors that have been identified in the national and international donor registries. However, several limitations restrict the uniform utilization of unrelated allogeneic adult donors including ethnic background of the recipient, acuity and timing of planned allogeneic transplant, availability of donor, and high risk of severe acute graft-versus-host disease (GVHD) (III/IV), among others. The investigators have recently identified a new alternative source of allogeneic stem cells, unrelated cryopreserved placental/cord blood stem cells.

Eligibility criteria: Inclusion Criteria:

* Patients will be eligible for double cord blood stem cell transplant (TNC ≥ 4x107/kg of two combined units) if available single cord blood has TNC ≤4.0 x 107/kg and they lack a matched (5-6/6) family donor, a 10/10 unrelated adult donor, and/or if their disease status required emergent stem cell transplant and they could not wait 2-3 months for searching for a matched unrelated adult donor.
* Adequate renal function defined as:Serum creatinine \<1.5 x normal, or Creatinine clearance or radioisotope glomerular filtration rate (GFR) \>60 ml/min/m2 or \>60 ml/min/1.73 m2 or an equivalent GFR as determined by the institutional normal range.
* Adequate liver function defined as:Total bilirubin \<1.5 x normal, or serum glutamic-oxaloacetic transaminase (SGOT) (aspartate aminotransferase (AST)) or serum glutamic pyruvic transaminase (SGPT) (alanine aminotransferase (ALT)) \<3.0 x normal
* Adequate cardiac function defined as:Shortening fraction \>27% by echocardiogram, or Ejection fraction \>47% by radionucleotide angiogram or echocardiogram.
* Adequate pulmonary function defined as:Uncorrected diffusing capacity of the lungs for carbon monoxide (DLCO) 50% by pulmonary function test.For children who are uncooperative, no evidence of dyspnea at rest, no exercise intolerance, and a pulse oximetry \>94% on room air.

Eligibility for Moderate Intensity, Reduced Intensity Regimen and Fanconi's Anemia (Regimens C, D and E)

* 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 \>40% by radionucleotide angiogram or echocardiogram.
* Adequate pulmonary function defined as:Uncorrected 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% on room air.

Exclusion Criteria:

* Females who are pregnant or breast-feeding
* Patients with documented uncontrolled infection at the time of study entry

Primary outcomes:
measure: Graft Failure Rate
timeFrame: Up to 2 years
measure: Response Rate (Complete and Partial Response)
timeFrame: Up to 2 years
measure: Overall Survival (OS)
timeFrame: Up to 2 years
measure: Disease Free Survival (DFS)
timeFrame: Up to 2 years
Secondary outcomes:
Other outcomes:
Locations:
city: New York
country: United States
facility: Columbia Presbyterian Medical Center
state: New York
Study officials:
affiliation: Columbia University
name: Prakash Satwani, MD
role: PRINCIPAL_INVESTIGATOR
References: