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- Ensayo clínico NCT01484470
Umbilical Cord Transplantation for the Elderly Population
19 de agosto de 2019 actualizado por: Patrick Stiff, Loyola University
While cord blood transplants have been performed safely in elderly patients, many still relapse.
The investigators propose to intensify the preparative regimen for this patient group in an attempt to decrease relapses, and combine this with an ex vivo expanded Umbilical Cord Blood (UCB) unit.
Descripción general del estudio
Estado
Terminado
Condiciones
Intervención / Tratamiento
Descripción detallada
Allogeneic stem cell transplantation is a life saving procedure in selected high-risk or recurrent hematologic malignancies and marrow failure syndromes.
However its wide application is limited by availability of suitably HLA matched adult donors.
Umbilical Cord Blood (UCB) has been increasingly used as an alternative hematopoietic stem cell source for these patients.
To date, over 10,000 UCB transplants have been performed in both children32-38 and adults.35,39-44
Its advantages include easier procurement, decreased risk to donors, reduced risk of transmitting infections, the immediate availability of cryopreserved units, and acceptable HLA mismatches.
The transplantation of UCB allows a greater degree of HLA mismatching without an unacceptably high incidence of graft versus host disease (GVHD).
Adult patients receiving myeloablative cord blood transplants have a 90% chance of engraftment, but carry a 50% rate of transplant related mortality.
Tipo de estudio
Intervencionista
Inscripción (Actual)
18
Fase
- Fase 2
Contactos y Ubicaciones
Esta sección proporciona los datos de contacto de quienes realizan el estudio e información sobre dónde se lleva a cabo este estudio.
Ubicaciones de estudio
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Illinois
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Maywood, Illinois, Estados Unidos, 60153
- Loyola University Medical Center
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Criterios de participación
Los investigadores buscan personas que se ajusten a una determinada descripción, denominada criterio de elegibilidad. Algunos ejemplos de estos criterios son el estado de salud general de una persona o tratamientos previos.
Criterio de elegibilidad
Edades elegibles para estudiar
55 años a 73 años (Adulto, Adulto Mayor)
Acepta Voluntarios Saludables
No
Géneros elegibles para el estudio
Todos
Descripción
Inclusion Criteria:
- Ages 55-73
Patients will have one of the following malignancies:
- Acute myelogenous leukemia (AML) deNovo in first CR with adverse cytogenetic abnormalities, M0, M6, M7 subtypes, extramedullary disease in remission or high CD34+ disease (> 50%)
- AML in early relapse (5-10% blasts on bone marrow aspirate or biopsy), or beyond CR-1 with no circulating blasts
- AML at any time if resulting from a previous myelodysplasia
- Acute lymphocytic leukemia or lymphoblastic lymphoma (ALL) in first CR with adverse prognostic features: t (9; 22), extra medullary disease, or mature B cell phenotype
- Acute lymphoid leukemia or lymphoblastic lymphoma in early relapse (5- 10% blasts on aspirate), or beyond CR-1
- Acute Undifferentiated Leukemia or biphenotypic leukemia in CR1 or CR2
- Transfusion dependent myelodysplastic syndrome (MDS) or refractory anemia with excess blasts (RAEB) or RAEB-in transition, CMMOL, or any myelodysplasia with 7q-, 5q-, 7-, 5- or resulting from prior anti cancer therapy.
- Relapsed Non-Hodgkin's Lymphoma (NHL), including those that have relapsed after an autologous marrow/blood stem cell transplant
- Chronic lymphocytic leukemia (CLL) patient who has had fludarabine and either failed or relapsed. Prior autologous transplant patients are eligible.
- Patients with adequate organ function and performance status criteria
Subject must have at least one or the following back-up stem cell sources in case of engraftment failure:
- Subject is willing to undergo BM harvest or peripheral blood progenitor cells (PBPC) collection for use in case of engraftment failure (when clinically applicable).
- Subject has a second CBU as a possible back up.
- Subject's haploidentical family member has been identified and agreed (by signing a written informed consent) to donate hematopoietic stem cells in case of engraftment failure.
- Evaluation by social service/psychologist
- Subject signs the written informed consent after being aware of the nature of the subject's disease and willingly consents to the treatment program after being informed of alternative treatments, potential risks, benefits and discomforts.
- Ability to understand and agree to compliance with strict evaluation, isolation,and medication schedules
- Designated primary care giver.
- Dental evaluation/treatment completed.
- ENT evaluation/treatment completed.
- All patient who survive to day 90 are eligible for measurement of T and B cell function and lymphocyte subset numbers to determine immune reconstitution post UCB transplantation with or without StemEx®
Exclusion Criteria:
- Patient with suitable related donor as defined per institutional guidelines
- Chemotherapy resistant or active AML, ALL, AUL, biphenotypic leukemia
- AML evolved from myelofibrosis
- MDS with 20% or greater bone marrow blasts at pre-transplant workup. Patients may receive therapy and if in remission, are eligible
- Prior allogeneic hematopoeitic stem cell transplant at any time
- Less than twenty-one days have elapsed since the subject's last radiation or chemotherapy prior to conditioning (except for hydroxyurea)
- Uncontrolled bacterial, fungal or viral infection at the time of study enrollment
- Seropositive or NAT positive for HIV, HTLV-1 and Hepatitis C
- Subjects with signs and symptoms of active central nervous system (CNS) disease
- Females who are pregnant or breastfeeding
- Allergy to bovine proteins or to aminoglycoside antibiotics (e.g. gentamicin) or to any product, which may interfere with the treatment.
- Patient unable to give informed consent or unable to comply with the treatment protocol including appropriate supportive care, follow-up and research tests.
- Enrolled in another clinical trial or received an investigational treatment during the last 30 days, unless approved by the primary investigator.
Plan de estudios
Esta sección proporciona detalles del plan de estudio, incluido cómo está diseñado el estudio y qué mide el estudio.
¿Cómo está diseñado el estudio?
Detalles de diseño
- Propósito principal: Tratamiento
- Asignación: No aleatorizado
- Modelo Intervencionista: Asignación paralela
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
|---|---|
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Sin intervención: Unmanipulated arm
Participants that do not meet criteria for StemEx®, will be registered into the unmanipulated UCB arm and receive the standard conditioning regimen.
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Experimental: Stemx Arm
StemEx is a stem/progenitor cell-based product of ex-vivo expanded allogeneic UCB, which is administered to the subject in combination with the non-manipulated portion of the same cord blood unit (CBU).
The CBU must be cryopreserved in two portions of which the larger (or equal) CBU portion contains at least 1.5 x 107 total nucleated cells (TNC)/Kg.
This portion remains unmanipulated and is transplanted on Day 0. StemEx is derived from the smaller (or equal) CBU portion, which is expanded ex vivo for 21 days starting pre-transplant in the presence of cytokines TPO, IL-6, Flt-3L and SCF at a concentration of 50ng/ml and 5μM tetraethylenepentamine (TEPA)
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For patients allocated to StemEx® arm:
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Efficacy of StemEx
Periodo de tiempo: 100 days
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The primary endpoint is to demonstrate the efficacy of StemEx® vs. unmanipulated UCB transplantation in the elderly population (>55years of age) following RIC regimen by demonstrating engraftment with full donor chimerism (>98%) by Day 100 of more than 60% of the patients who received transplants expanded by the StemEx method.
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100 days
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
|---|---|---|
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Time to engraftment
Periodo de tiempo: 42 days
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The day of neutrophil engraftment is defined as the first day of 3 consecutive days of an ANC greater than 500/microliter.
The platelet recovery is the first of 3 consecutive measurements tested on different days of a platelet count greater than or equal to 20,000 without requiring platelet transfusions in the previous 7 days.
Patients will be monitored for donor cell engraftment as evidenced by neutrophil recovery and donor chimerism in the marrow and/or peripheral blood at serial time points post transplant.
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42 days
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Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Patrick Stiff, MD, Loyola Universtiy Medical Center
Fechas de registro del estudio
Estas fechas rastrean el progreso del registro del estudio y los envíos de resultados resumidos a ClinicalTrials.gov. Los registros del estudio y los resultados informados son revisados por la Biblioteca Nacional de Medicina (NLM) para asegurarse de que cumplan con los estándares de control de calidad específicos antes de publicarlos en el sitio web público.
Fechas importantes del estudio
Inicio del estudio
1 de enero de 2010
Finalización primaria (Actual)
21 de noviembre de 2018
Finalización del estudio (Actual)
21 de noviembre de 2018
Fechas de registro del estudio
Enviado por primera vez
30 de noviembre de 2011
Primero enviado que cumplió con los criterios de control de calidad
1 de diciembre de 2011
Publicado por primera vez (Estimar)
2 de diciembre de 2011
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
21 de agosto de 2019
Última actualización enviada que cumplió con los criterios de control de calidad
19 de agosto de 2019
Última verificación
1 de agosto de 2019
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- 202041
Esta información se obtuvo directamente del sitio web clinicaltrials.gov sin cambios. Si tiene alguna solicitud para cambiar, eliminar o actualizar los detalles de su estudio, comuníquese con register@clinicaltrials.gov. Tan pronto como se implemente un cambio en clinicaltrials.gov, también se actualizará automáticamente en nuestro sitio web. .