- ICH GCP
- US Clinical Trials Registry
- Klinisk utprøving NCT01711788
Intrabone Infusion of Umbilical Cord Blood Stem Cells
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
Studieoversikt
Status
Intervensjon / Behandling
Detaljert beskrivelse
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.
Studietype
Registrering (Faktiske)
Fase
- Fase 2
Kontakter og plasseringer
Studiesteder
-
-
Quebec
-
Montreal, Quebec, Canada, H3C1T5
- Centre Hospitalier Universitaire Sainte-Justine
-
-
Deltakelseskriterier
Kvalifikasjonskriterier
Alder som er kvalifisert for studier
Tar imot friske frivillige
Kjønn som er kvalifisert for studier
Beskrivelse
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
Studieplan
Hvordan er studiet utformet?
Designdetaljer
- Primært formål: Behandling
- Tildeling: N/A
- Intervensjonsmodell: Enkeltgruppeoppdrag
- Masking: Ingen (Open Label)
Våpen og intervensjoner
Deltakergruppe / Arm |
Intervensjon / Behandling |
---|---|
Eksperimentell: Intrabone umbilical cord blood tranplant
Intrabone infusion of umbilical cord blood stem cells
|
Hva måler studien?
Primære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
---|---|---|
Platelet recovery rate
Tidsramme: at 100 days post- transplantation
|
First of seven days of untransfused platelet count higher than 20 x 10^9/L
|
at 100 days post- transplantation
|
Sekundære resultatmål
Resultatmål |
Tiltaksbeskrivelse |
Tidsramme |
---|---|---|
Neutrophil recovery rate
Tidsramme: at 60 days post- transplantation
|
First of three days of absolute neutrophil count equal or higher than 0.5 x 10^9/L
|
at 60 days post- transplantation
|
Immunological reconstitution
Tidsramme: at 30, 60, 100, 180, and 360 days post- transplantation
|
Total number of T cells (and subpopulations), B and NK (natural killer) cells in peripheral blood at different time-points
|
at 30, 60, 100, 180, and 360 days post- transplantation
|
Donor chimerism rate
Tidsramme: at 30, 60,100, and 180 days post-transplantation
|
Percentage of donor(s) cells in peripheral blood at different time-points
|
at 30, 60,100, and 180 days post-transplantation
|
Acute GVHD (grade 2-4) rate
Tidsramme: at 180 days
|
Incidence of grade II-IV acute GVHD (Graft versus Host Disease)
|
at 180 days
|
Infection rate (bacterial, viral, fungal and parasitic)
Tidsramme: at 180 days post-transplantation
|
Clinical and microbiological documented infections will be reported according to anatomic site, date of onset and microorganism
|
at 180 days post-transplantation
|
Event-free and overall survival
Tidsramme: at 2 years
|
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
|
at 2 years
|
Adverse infections (grade and frequency)
Tidsramme: at one month post-transplantation
|
Toxicity will be assessed using the Common Terminology Criteria for Adverse Events v4.0
|
at one month post-transplantation
|
chronic GVHD
Tidsramme: at 2 years post-transplantation
|
Incidence of chronic GVHD (Graft versus Host Disease) will be scored according to NIH consensus on chronic GVHD
|
at 2 years post-transplantation
|
Samarbeidspartnere og etterforskere
Sponsor
Samarbeidspartnere
Etterforskere
- Hovedetterforsker: Henrique Bittencourt, MD, PhD, St. Justine's Hospital
Publikasjoner og nyttige lenker
Studierekorddatoer
Studer hoveddatoer
Studiestart
Primær fullføring (Faktiske)
Studiet fullført (Faktiske)
Datoer for studieregistrering
Først innsendt
Først innsendt som oppfylte QC-kriteriene
Først lagt ut (Anslag)
Oppdateringer av studieposter
Sist oppdatering lagt ut (Faktiske)
Siste oppdatering sendt inn som oppfylte QC-kriteriene
Sist bekreftet
Mer informasjon
Begreper knyttet til denne studien
Nøkkelord
Andre studie-ID-numre
- IB-UCBT
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