NCT01711788

Brief Summary

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.

Trial Health

87
On Track

Trial Health Score

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

Enrollment
15

participants targeted

Target at below P25 for phase_2

Timeline
Completed

Started Nov 2012

Longer than P75 for phase_2

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

First Submitted

Initial submission to the registry

October 18, 2012

Completed
4 days until next milestone

First Posted

Study publicly available on registry

October 22, 2012

Completed
10 days until next milestone

Study Start

First participant enrolled

November 1, 2012

Completed
4.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

October 3, 2017

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

October 3, 2017

Completed
Last Updated

October 5, 2017

Status Verified

October 1, 2017

Enrollment Period

4.9 years

First QC Date

October 18, 2012

Last Update Submit

October 3, 2017

Conditions

Hematopoietic Stem Cell Transplantation

Keywords

Hematopoietic stem cellsUmbilical cord blood transplantationIntrabone infusionPlatelet recoveryNeutrophil recoveryPediatric patients

Outcome Measures

Primary Outcomes (1)

  • Platelet recovery rate

    First of seven days of untransfused platelet count higher than 20 x 10\^9/L

    at 100 days post- transplantation

Secondary Outcomes (8)

  • Neutrophil recovery rate

    at 60 days post- transplantation

  • Immunological reconstitution

    at 30, 60, 100, 180, and 360 days post- transplantation

  • Donor chimerism rate

    at 30, 60,100, and 180 days post-transplantation

  • Acute GVHD (grade 2-4) rate

    at 180 days

  • Infection rate (bacterial, viral, fungal and parasitic)

    at 180 days post-transplantation

  • +3 more secondary outcomes

Study Arms (1)

Intrabone umbilical cord blood tranplant

EXPERIMENTAL

Intrabone infusion of umbilical cord blood stem cells

Procedure: Intrabone infusion of umbilical cord blood stem cells

Interventions

Intrabone infusion of umbilical cord blood stem cellsPROCEDURE
Intrabone umbilical cord blood tranplant

Eligibility Criteria

Age1 Year - 21 Years
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64)

You may qualify if:

  • 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

You may not qualify if:

  • 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

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Centre Hospitalier Universitaire Sainte-Justine

Montreal, Quebec, H3C1T5, Canada

Location

Related Publications (1)

  • Vairy S, Louis I, Vachon MF, Richer J, Teira P, Cellot S, Villeneuve E, Haddad E, Duval M, Bittencourt H. Intrabone infusion for allogeneic umbilical cord blood transplantation in children. Bone Marrow Transplant. 2021 Aug;56(8):1937-1943. doi: 10.1038/s41409-021-01275-0. Epub 2021 Apr 6.

    PMID: 33824433DERIVED

Study Officials

  • Henrique Bittencourt, MD, PhD

    St. Justine's Hospital

    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 INVESTIGATOR
PI Title
Hematologist - Oncologist

Study Record Dates

First Submitted

October 18, 2012

First Posted

October 22, 2012

Study Start

November 1, 2012

Primary Completion

October 3, 2017

Study Completion

October 3, 2017

Last Updated

October 5, 2017

Record last verified: 2017-10

Locations

CA(1)