- ICH GCP
- Registro de ensayos clínicos de EE. UU.
- Ensayo clínico 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
Descripción general del estudio
Estado
Condiciones
Intervención / Tratamiento
Descripción detallada
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.
Tipo de estudio
Inscripción (Actual)
Fase
- Fase 2
Contactos y Ubicaciones
Ubicaciones de estudio
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Quebec
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Montreal, Quebec, Canadá, H3C1T5
- Centre Hospitalier Universitaire Sainte-Justine
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Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
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
Plan de estudios
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: N / A
- Modelo Intervencionista: Asignación de un solo grupo
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
---|---|
Experimental: Intrabone umbilical cord blood tranplant
Intrabone infusion of umbilical cord blood stem cells
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
---|---|---|
Platelet recovery rate
Periodo de tiempo: at 100 days post- transplantation
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First of seven days of untransfused platelet count higher than 20 x 10^9/L
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at 100 days post- transplantation
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
---|---|---|
Neutrophil recovery rate
Periodo de tiempo: at 60 days post- transplantation
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First of three days of absolute neutrophil count equal or higher than 0.5 x 10^9/L
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at 60 days post- transplantation
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Immunological reconstitution
Periodo de tiempo: at 30, 60, 100, 180, and 360 days post- transplantation
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Total number of T cells (and subpopulations), B and NK (natural killer) cells in peripheral blood at different time-points
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at 30, 60, 100, 180, and 360 days post- transplantation
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Donor chimerism rate
Periodo de tiempo: at 30, 60,100, and 180 days post-transplantation
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Percentage of donor(s) cells in peripheral blood at different time-points
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at 30, 60,100, and 180 days post-transplantation
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Acute GVHD (grade 2-4) rate
Periodo de tiempo: at 180 days
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Incidence of grade II-IV acute GVHD (Graft versus Host Disease)
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at 180 days
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Infection rate (bacterial, viral, fungal and parasitic)
Periodo de tiempo: at 180 days post-transplantation
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Clinical and microbiological documented infections will be reported according to anatomic site, date of onset and microorganism
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at 180 days post-transplantation
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Event-free and overall survival
Periodo de tiempo: at 2 years
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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
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at 2 years
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Adverse infections (grade and frequency)
Periodo de tiempo: at one month post-transplantation
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Toxicity will be assessed using the Common Terminology Criteria for Adverse Events v4.0
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at one month post-transplantation
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chronic GVHD
Periodo de tiempo: at 2 years post-transplantation
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Incidence of chronic GVHD (Graft versus Host Disease) will be scored according to NIH consensus on chronic GVHD
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at 2 years post-transplantation
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Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Henrique Bittencourt, MD, PhD, St. Justine's Hospital
Publicaciones y enlaces útiles
Fechas de registro del estudio
Fechas importantes del estudio
Inicio del estudio
Finalización primaria (Actual)
Finalización del estudio (Actual)
Fechas de registro del estudio
Enviado por primera vez
Primero enviado que cumplió con los criterios de control de calidad
Publicado por primera vez (Estimar)
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
Última actualización enviada que cumplió con los criterios de control de calidad
Última verificación
Más información
Términos relacionados con este estudio
Palabras clave
Otros números de identificación del estudio
- IB-UCBT
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