Allogenic Transplantation of Ex-vivo Expanded Cord Blood (CB)

Phase 1

Withdrawn

• Conditions: Stem Cell Transplantation; Umbilical Cord Blood; Hematological Malignancies
• Interventions: Biological: ex vivo expansion

• Registration Number: NCT01235468
• Lead Sponsor: Dr. Avichai Shimoni MD

Brief Summary

The aim of this study is to evaluate the safety profile and tolerability of infusion of cord blood cells expanded in the lab and to evaluated whether through the infusion of expanded cells it is possible to expedite engraftment time after transplantation.

Detailed Description

Stem cell transplantation is a curative approach for patients with hematological malignancies. Umbilical cord blood is a source of stem cells for transplantation in patients with no related donor. However, in adults, the number of stem cells in a single unit, may be too low to allow engraftment, and the time to engraftment may be prolonged, increasing the risks of the transplant. In this study, we expand part of the cord blood unit, in the lab, trying to increase unit size, such that it would be suitable for adults, and would allow safe engraftment.

Study Timeline

• Study First Submitted: 2010-11-02
• Study First Submitted QC: 2010-11-04
• Study First Posted: 2010-11-05
• Study Start: 2012-06
• Primary Completion: 2015-11
• Study Completion: 2015-11
• Status Verified: 2015-12
• Last Update Submitted: 2015-12-01
• Last Update Posted: 2015-12-03

Recruitment & Eligibility

• Status: WITHDRAWN
• Sex: All
• Target Recruitment: Not specified

Inclusion Criteria

• age > 18 years
• hematological malignancy
• standard indication for allogeneic transplantation
• expected survival time over 12 weeks
• no related or unrelated donor
• availability of a cord blood unit of compatible placental blood cryopreserved in at least 2 different bags; one of the two units should have a cellular content at least equal to the minimum dose of 2 x 107 nucleated cells per kilogram of body weight

Exclusion Criteria

• Eastern Cooperative Oncology Group (ECOG) performance status >2
• Prior allogeneic transplantation
• Pregnant or nursing women
• Positive serology for hepatitis B or C
• HIV positive
• Left ventricular ejection fraction < 50%
• DLCO < 50%
• Psychiatric, addictive, or any disorder/disease which compromises ability to give informed consent for participation in this study
• Treatment with other investigational drugs within 4 weeks of enrolling in this protocol

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
CB expnasion	ex vivo expansion	ex-vivo expansion of cord blood for transplantation

Primary Outcome Measures

Name	Time	Method
GVHD	1 year	Number of patients with acute and chronic GVHD after transplantation
treatment-related toxicity	3 months	Type and severity of adverse events after transplantation using the NCI CTC scale
Safety	1 month	Number of patients with adverse events during infusion
engraftment	3 months	Time to engraftment

Secondary Outcome Measures

Name	Time	Method
relapse	5 years	rate of disease recurrence
Immunological reconstruction	5 years	immunological reconstruction after transplantation measured by lymphocyte number and function