Intrabone Infusion of Umbilical Cord Blood Stem Cells

This study is currently recruiting participants.

Verified November 2012 by St. Justine's Hospital

Sponsor:

Collaborators:

Michel Duval, MD

Pierre Teira, MD

Sonia Cellot, MD, PhD

Isabelle Louis, PhD

Elie Haddad, MD, PhD

Marie-France Vachon, MScN

Marion Cortier, PhD

Information provided by (Responsible Party):

Henrique Bittencourt, MD, PhD, St. Justine's Hospital

ClinicalTrials.gov Identifier:

NCT01711788

First received: October 18, 2012

Last updated: November 8, 2012

Last verified: November 2012

History of Changes

Purpose

The purpose of this study is to determine if the method of intrabone infusion of hematologic stem cells can increase and accelerate hematopoietic reconstitution after umbilical cord blood transplantation in pediatric patients.

Condition	Intervention	Phase
Hematopoietic Stem Cell Transplantation	Procedure: Intrabone infusion of umbilical cord blood stem cells	Phase 2

Study Type:	Interventional
Study Design:	Endpoint Classification: Safety/Efficacy Study Intervention Model: Single Group Assignment Masking: Open Label Primary Purpose: Treatment
Official Title:	A New Approach to Improve Long-term Hematopoietic Recovery After Allogeneic Umbilical Cord Blood Transplantation in Children - Intrabone Infusion of Umbilical Cord Blood Stem Cells

Further study details as provided by St. Justine's Hospital:

Primary Outcome Measures:

Platelet recovery rate [ Time Frame: at 100 days post- transplantation ] [ Designated as safety issue: No ]
First of seven days of untransfused platelet count higher than 20 x 10^9/L

Secondary Outcome Measures:

Neutrophil recovery rate [ Time Frame: at 60 days post- transplantation ] [ Designated as safety issue: No ]
First of three days of absolute neutrophil count equal or higher than 0.5 x 10^9/L
Immunological reconstitution [ Time Frame: at 30, 60, 100, 180, and 360 days post- transplantation ] [ Designated as safety issue: No ]
Total number of T cells (and subpopulations), B and NK (natural killer) cells in peripheral blood at different time-points
Donor chimerism rate [ Time Frame: at 30, 60,100, and 180 days post-transplantation ] [ Designated as safety issue: No ]
Percentage of donor(s) cells in peripheral blood at different time-points
Acute GVHD (grade 2-4) rate [ Time Frame: at 180 days ] [ Designated as safety issue: No ]
Incidence of grade II-IV acute GVHD (Graft versus Host Disease)
Infection rate (bacterial, viral, fungal and parasitic) [ Time Frame: at 180 days post-transplantation ] [ Designated as safety issue: No ]
Clinical and microbiological documented infections will be reported according to anatomic site, date of onset and microorganism
Event-free and overall survival [ Time Frame: at 2 years ] [ Designated as safety issue: No ]
Event-free survival is defined as the time interval between transplantation and relapse, graft rejection, death or last follow-up, whichever occurs first; Overall survival is defined as the time between transplantation and death or last follow-up
Adverse infections (grade and frequency) [ Time Frame: at one month post-transplantation ] [ Designated as safety issue: No ]
Toxicity will be assessed using the Common Terminology Criteria for Adverse Events v4.0
chronic GVHD [ Time Frame: at 2 years post-transplantation ] [ Designated as safety issue: No ]
Incidence of chronic GVHD (Graft versus Host Disease) will be scored according to NIH consensus on chronic GVHD

Estimated Enrollment:	21
Study Start Date:	November 2012
Estimated Study Completion Date:	December 2015
Estimated Primary Completion Date:	December 2015 (Final data collection date for primary outcome measure)

Arms	Assigned Interventions
Experimental: Intrabone umbilical cord blood tranplant Intrabone infusion of umbilical cord blood stem cells	Procedure: Intrabone infusion of umbilical cord blood stem cells

Detailed Description:

Umbilical cord blood transplantation (UCBT) has been increasingly used to treat malignant and non-malignant haematological, immunodeficiency and some metabolic diseases. UCBT offers the advantages of easy procurement, no risk to donors, a reduced risk of transmitting infections, immediate availability of cryopreserved units, and acceptable partial HLA mismatches. However, patients treated with UCBT show delayed hematopoietic and immunological recoveries, have higher rates of infection, and relapse from the original malignant disease, which can all lead to life threatening problems. UCBT can also result in a higher rate of graft failure compared to other hematopoietic stem cell transplantation (HSCT) sources. The problem of a slower hematopoietic recovery post-UCBT has been addressed using a number of different approaches in adult patients.In adults, use of intrabone injection of cord blood results in a faster hematopoietic recovery in a phase II study. However, there is no clinical trial in pediatric patients.

This study is addressed to determine if a change in the cord blood stem cell infusion method can increase and accelerate hematopoietic reconstitution after UCBT in pediatric patients.

Eligibility

Ages Eligible for Study:	1 Year to 21 Years
Genders Eligible for Study:	Both
Accepts Healthy Volunteers:	No

Criteria

Inclusion Criteria:

One to 21 years of age;
More than 10 kg in weight;
Diagnosis of hematopoietic disorders (malignant or not) with an indication for hematopoietic stem cell transplantation;
Absence of an HLA-identical related donor;
Availability of a single cord blood (CB) with at least 3 x 10^7 nucleated cells (NCs)/kg (if HLA identical or 1 HLA-mismatch) or at least 4 x 10^7 NCs/kg (if a 2 HLA-mismatch) at freezing. Use of two CB units ("double cord transplant") will be allowed provided that: 1) a single CB unit fulfilling the above criteria is not available; 2) a maximum of 2 HLA mismatch is present for each CB unit; and 3) a minimum of 4 x 10^7 NCs/kg (as the sum for both CB units) is present at freezing.
A myeloablative-conditioning regimen;
A Lansky (for patients less than 16 years of age) or Karnofsky (for patients more than 16 years of age) score equal to or higher than 70%.
Adequate organ function as follows:
Cardiac (ejection fraction > 50%);
Renal (serum creatinine within the normal range for age, and creatinine clearance or a GFR > 70 ml/min/1.73m2);
Hepatic (AST or ALT < 5 x upper limit of normal for age);
Pulmonary (FEV1, FVC, and DLCO ≥ 50% by pulmonary function tests or, in children unable to cooperate, no sign of dyspnea at rest, no exercise intolerance, no supplementary oxygen therapy, and a normal pulmonary radiography or pulmonary scan);
No sign of uncontrolled systemic bacterial, fungal or viral infection;
Written informed consent by the patient or his/her legal guardian

Exclusion Criteria:

Non-myeloablative conditioning;
Pregnancy or breastfeeding;
HIV positive serology;
Bone disease (e.g. osteopetrosis, osteogenesis imperfecta)
Previous autologous or allogeneic hematopoietic stem cell transplantation performed up to one year before enrolment, except in the case of non-engraftment or early rejection of a previous allogeneic stem cell transplantation.
Active skin infection at the site of intrabone injection.
History of intolerance/allergy to sedation medications or local anesthetics.
Contraindication to sedation

Contacts and Locations

Please refer to this study by its ClinicalTrials.gov identifier: NCT01711788

Contacts

Contact: Henrique Bittencourt, MD, PhD

514-345-4969

henrique.bittencourt.hsj@ssss.gouv.qc.ca

Locations

Canada, Quebec
Centre Hospitalier Universitaire Sainte-Justine	Recruiting
Montreal, Quebec, Canada, H3C1T5

Sponsors and Collaborators

Henrique Bittencourt, MD, PhD

Michel Duval, MD

Pierre Teira, MD

Sonia Cellot, MD, PhD

Isabelle Louis, PhD

Elie Haddad, MD, PhD

Marie-France Vachon, MScN

Marion Cortier, PhD

Investigators

Principal Investigator:

Henrique Bittencourt, MD, PhD

St. Justine's Hospital

More Information

No publications provided

Responsible Party:	Henrique Bittencourt, MD, PhD, Hematologist - Oncologist, St. Justine's Hospital
ClinicalTrials.gov Identifier:	NCT01711788 History of Changes
Other Study ID Numbers:	IB-UCBT
Study First Received:	October 18, 2012
Last Updated:	November 8, 2012
Health Authority:	Canada: Ethics Review Committee

Keywords provided by St. Justine's Hospital:

Hematopoietic stem cells
Umbilical cord blood transplantation
Intrabone infusion

Platelet recovery
Neutrophil recovery
Pediatric patients

ClinicalTrials.gov processed this record on May 16, 2013

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