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Hematopoietic Stem Cell TransplantationPhase 2NCT01711788

Intrabone Infusion of Umbilical Cord Blood Stem Cells

A Phase 2 study testing Intrabone infusion of umbilical cord blood stem cells in people with Hematopoietic Stem Cell Transplantation, run by Henrique Bittencourt, MD, PhD at 1 site in Canada.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial 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. Read more about this study

Who is it for?
Ages 1 year to 21 years
Conditions the study is for
Hematopoietic Stem Cell Transplantation
What is being tested?
Intrabone infusion of umbilical cord blood stem cells
Could I receive a placebo?
No, everyone receives an active treatment
How many people
15 participants
Where
1 siteCanada
Study dates
Nov 2012to 3 Oct 2017
Phase
Phase 2
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 1 year to 21 years
  • All sexes
  • No healthy volunteers
  • Child, Adult

Who may be able to join

  • 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;
Show all 14Show fewer
  • 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

Who cannot join

  • 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

Trial locations and contacts

1 site in 1 country.

Canada1 site

  • Centre Hospitalier Universitaire Sainte-Justine

    Montreal, Quebec, H3C1T5

Other trials in Canada

About this study

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

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.

Read the 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.

Being tested
Intrabone infusion of umbilical cord blood stem cells
Keywords
Hematopoietic stem cells, Umbilical cord blood transplantation, Intrabone infusion, Platelet recovery, Neutrophil recovery, Pediatric patients

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Intrabone infusion of umbilical cord blood stem cellsProcedure or surgery

Study groups in NCT01711788
Study groupRoleWhat they receive
Intrabone umbilical cord blood tranplantExperimentalIntrabone infusion of umbilical cord blood stem cells
Receives: Procedure: Intrabone infusion of umbilical cord blood stem cells

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Platelet recovery rate

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

    Measured: at 100 days post- transplantation

Secondary outcomes (8)

  1. Neutrophil recovery rate

    First of three days of absolute neutrophil count equal or higher than 0.5 x 10^9/L

    Measured: at 60 days post- transplantation

  2. Immunological reconstitution

    Total number of T cells (and subpopulations), B and NK (natural killer) cells in peripheral blood at different time-points

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

  3. Donor chimerism rate

    Percentage of donor(s) cells in peripheral blood at different time-points

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

  4. Acute GVHD (grade 2-4) rate

    Incidence of grade II-IV acute GVHD (Graft versus Host Disease)

    Measured: at 180 days

  5. Infection rate (bacterial, viral, fungal and parasitic)

    Clinical and microbiological documented infections will be reported according to anatomic site, date of onset and microorganism

    Measured: at 180 days post-transplantation

  6. Event-free and overall survival

    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

    Measured: at 2 years

  7. Adverse infections (grade and frequency)

    Toxicity will be assessed using the Common Terminology Criteria for Adverse Events v4.0

    Measured: at one month post-transplantation

  8. chronic GVHD

    Incidence of chronic GVHD (Graft versus Host Disease) will be scored according to NIH consensus on chronic GVHD

    Measured: at 2 years post-transplantation

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted22 Oct 2012 (estimated)
  2. Study startNov 2012
  3. Primary completion3 Oct 2017
  4. Study completion3 Oct 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2
Primary purpose
Treatment
Allocation
Not applicable
Intervention model
Single group
Masking
None (open label)
Enrolment
15 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT01711788
Sponsor's protocol ID
IB-UCBT
Status last verified
Oct 2017
First submitted
18 Oct 2012
Last updated
5 Oct 2017
Results posted
No
Data monitoring committee
Yes

Publications

  • 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.

    PubMed 33824433

Questions about this trial

What is this clinical trial studying?

A Phase 2 study testing Intrabone infusion of umbilical cord blood stem cells in people with Hematopoietic Stem Cell Transplantation, run by Henrique Bittencourt, MD, PhD at 1 site in Canada.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 5 Oct 2017. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in Canada. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Platelet recovery rate (time frame: at 100 days post- transplantation). It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT01711788, which also lists 1 linked publication.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 5 Oct 2017.

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