CD4^LVFOXP3 en participantes con IPEX
2 de noviembre de 2026 actualizado por: Jessie L. Alexander, MD, Bacchetta, Rosa, MD
Estudio de fase 1 de CD4^LVFOXP3 autólogo en participantes con síndrome de desregulación inmunitaria poliendocrinopatía enteropatía ligada al cromosoma X (IPEX)
This first-in-human, Phase 1 clinical trial will test the feasibility of the manufacturing and the safety of the administration of CD4^LVFOXP3 in up to 30 evaluable human participants with IPEX and evaluate the impact of the CD4^LVFOXP3 infusion on the disease.
Descripción general del estudio
Descripción detallada
Treatment with CD4^LVFOXP3 is expected to replace the defective Treg cells of the participants, and restore control of the immune system and therefore ameliorate symptoms of IPEX.
We expect to learn the following from this study:
- That CD4^LVFOXP3 can be consistently produced and be of expected quality to be used in humans,
- That CD4^LVFOXP3 are safe in children and young adults with IPEX, and determine its effects, both good and bad,
- That CD4^LVFOXP3 can improve overall health and allow reduction of medication/s.
This Phase 1 (feasibility and safety) trial will gather data about CD4^LVFOXP3 in vivo persistency and early signs of impact on symptoms of IPEX.
Tipo de estudio
Intervencionista
Inscripción (Actual)
16
Fase
- Fase 1
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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California
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Palo Alto, California, Estados Unidos, 94305
- Lucile Packard Children's Hospital
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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
4 meses a 35 años (Niño, Adulto)
Acepta Voluntarios Saludables
No
Géneros elegibles para el estudio
Masculino
Descripción
Inclusion Criteria:
- Body weight greater than 8 kg, unless assessed as able to tolerate leukapheresis
- FOXP3 gene mutation
- Medical history of progressive symptoms of IPEX with persistency of some symptoms and/or signs requiring immune suppressive medication. The participant may or may not be on immunosuppression at time of starting the study.
- Uncontrolled IPEX disease but unable to tolerate immune suppressive medication
- Recurrent IPEX symptoms, requiring immune suppressive medications, in participants who have had prior allogeneic (allo) blood stem cell transplantation (HSCT).
- ≥ 50% Performance rating on Lansky/Karnofsky Scale
- Organ and marrow function within acceptable levels of function
- Absence of ongoing infections
- Must be able to consent if an adult
Exclusion Criteria:
- Medical instability
- Less than 6 months life expectancy
- Inability to meet limits for steroid dosing
- Eligible for an HLA matched sibling or matched unrelated donor blood stem cell transplant, and be willing to undergo transplant.
- Unrelated or comorbid disease
- Allergy to any study medication, product, or intervention
- Currently receiving another experimental treatment
- History of malignancy, unless disease free for at least 2 years, with the exception of non melanoma skin cancer or carcinoma in situ
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 Secuencial
- Enmascaramiento: Ninguno (etiqueta abierta)
Armas e Intervenciones
Grupo de participantes/brazo |
Intervención / Tratamiento |
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Experimental: Cohort A (≥12 years)
The first participant in Dose Level 1 will be administered 1.0 x 10^6 CD4^LVFOXP3 /kg (± 20%). If there is no toxicity observed in the first participant, the following participants in Dose Level 1 will be administered the same dose of 1.0 x 10^6 CD4^LVFOXP3 /kg (± 20%). If there is no toxicity observed in any participants in Dose Level 1, participants will be enrolled into Dose Level 2 and administered 3 x 10^6 CD4^LVFOXP3 /kg (± 20%). If there is no toxicity observed in any participants in Dose Level 2, participants will be enrolled into Dose Level 3 and administered 10 x 10^6 CD4^LVFOXP3 /kg (± 20%). If in any dose level 1 of 2 participants show toxicity, that dose level will be expanded to 6 participants. If there is no toxicity observed in any participants in Dose Level 3, participants will then be administered 10 x 10^6 CD4^LVFOXP3 /kg (± 20%) at two time points 21-42 days apart. |
Infusion of autologous CD4+ T cells that have undergone lentiviral-mediated gene transfer of: i) healthy human FOXP3 gene leading to persistent high FOXP3 expression and acquisition of Treg-like cell function; and ii) human CD271 surface marker gene that allows tracking and quantification of the CD4^LVFOXP3 in the blood.
Otros nombres:
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Experimental: Cohort B (<12 years)
Participants in Cohort B will always follow treatment of participants in Cohort A for the same dose level. Cohort B will start at Dose Level 2 and be administered 3 x 10^6 CD4^LVFOXP3 /kg (± 20%). If there is no toxicity observed in any participants in Dose Level 2, participants will be enrolled into Dose Level 3 and administered 10 x 10^6 CD4^LVFOXP3 /kg (± 20%). If in any dose level 1 of 2 participants show toxicity, that dose level will be expanded to 6 participants. If there is no toxicity observed in any participants in Dose Level 3, participants will then be administered 10 x 10^6 CD4^LVFOXP3 /kg (± 20%) at two time points 21-42 days apart. |
Infusion of autologous CD4+ T cells that have undergone lentiviral-mediated gene transfer of: i) healthy human FOXP3 gene leading to persistent high FOXP3 expression and acquisition of Treg-like cell function; and ii) human CD271 surface marker gene that allows tracking and quantification of the CD4^LVFOXP3 in the blood.
Otros nombres:
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¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Meet target cell number for dose manufacturing
Periodo de tiempo: Time at release from manufacturing (by Day 0 [infusion day] for each participant)
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No more than two products fail the target cell dose and established release criteria. |
Time at release from manufacturing (by Day 0 [infusion day] for each participant)
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Find the safe maximum tolerated dose
Periodo de tiempo: Up to 60 days post-infusion for each participant
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No more than 1 out of 6 participants may experience a related dose limiting toxicity or treatment emergent adverse events. |
Up to 60 days post-infusion for each participant
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Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
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Cambio en los percentiles de altura específicos de la edad
Periodo de tiempo: Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3) hasta después de la infusión (Mes 12)
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Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3) hasta después de la infusión (Mes 12)
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Cambio en los percentiles de peso corporal específicos de la edad
Periodo de tiempo: Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 1, 2, 3, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9, 12)
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Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 1, 2, 3, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9, 12)
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Cambio en los niveles de bilirrubina
Periodo de tiempo: Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Cambio en la enzima hepática: alanina transaminasa (ALT)
Periodo de tiempo: Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Cambio en la enzima hepática: aspartato transaminasa (AST)
Periodo de tiempo: Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Cambio en la enzima hepática: fosfatasa alcalina (ALP)
Periodo de tiempo: Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Cambio en la enzima hepática: gamma glutiltranspeptidasa (GGT)
Periodo de tiempo: Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Línea de base (hasta 60 días antes de la infusión de CD4^LVFOXP3), antes de la infusión hasta después de la infusión (Día 2, Semana 1, 2, 3, 4; Mes 2, 3, 6, 9 y 12)
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Cambio en el péptido C: diabetes tipo 1
Periodo de tiempo: Línea base tomada 60-30 días antes de la infusión de CD4^LVFOXP3; Post-infusión (Semana 4, Mes 3, 6 y 12).
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Línea base tomada 60-30 días antes de la infusión de CD4^LVFOXP3; Post-infusión (Semana 4, Mes 3, 6 y 12).
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Cambio en HbA1c - Diabetes tipo 1 Pre-inicio
Periodo de tiempo: Línea base tomada 60-30 días antes de la infusión de CD4^LVFOXP3; Post-infusión (Semana 4, Mes 3, 6 y 12).
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Línea base tomada 60-30 días antes de la infusión de CD4^LVFOXP3; Post-infusión (Semana 4, Mes 3, 6 y 12).
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Cambio en la Escala de Bienestar General PedsQL - Calidad de Vida
Periodo de tiempo: Pre-Infusión; Mes 6, 12
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Pre-Infusión; Mes 6, 12
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Cambio en la escala básica genérica PedsQL - Calidad de vida
Periodo de tiempo: Pre-Infusión; Mes 6, 12
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Pre-Infusión; Mes 6, 12
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Change in Diarrhea incidence
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (daily for the first month followed by monthly at Month 2, 3, 6, 9, 12)
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Stool Diary records - extent of diarrhea as measured by frequency and volume of stools, and the presence or absence of blood and/or mucus, and stool studies at specified time points (for all ages). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (daily for the first month followed by monthly at Month 2, 3, 6, 9, 12)
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Change in GI Symptoms - Gastrointestinal Symptoms Rating Scale
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3) through post-infusion (Week 4, Month 6, Month 12)
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Gastrointestinal Symptoms Rating Scale (GSRS) (for patients ≥12 years old). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3) through post-infusion (Week 4, Month 6, Month 12)
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Change in Body Mass Index (BMI)
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3, Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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BMI measured as kg/m^2. |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3, Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Change in INR level
Periodo de tiempo: Baseline/screening (up to 60 days before infusion of CD4^LVFOXP3) through post-infusion (Week 4; Month 3, 6, 12)
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International normalized ratio (INR) to determine prothrombin time. |
Baseline/screening (up to 60 days before infusion of CD4^LVFOXP3) through post-infusion (Week 4; Month 3, 6, 12)
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Skin Disease (EASI) - Changes from Baseline/ Pre-infusion
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Scoring of areas of involvement in each anatomical region (area), calculation of intensity using Eczema Area and Severity Index (EASI). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Skin Disease (POEM) - Changes from Baseline/ Pre-infusion
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Scoring of areas of involvement in each anatomical region (area), calculation of intensity using Patient oriented eczema measure (POEM). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Skin Disease (PASI) - Changes from Baseline/ Pre-infusion
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Changes in the extent (%) and severity of skin lesions and their complications (i.e. infections, atrophy, itching) using Psoriasis Area and Severity Index (PASI). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Skin Disease (MTLSS) - Changes from Baseline/ Pre-infusion
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Changes in the extent (%) and severity of skin lesions and their complications (i.e. infections, atrophy, itching) using Modified Total Lesional Sign Score (MTLSS). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Change in skin barrier function
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Biophysical Skin Evaluation: Skin measurement of transepidermal water loss to monitor skin barrier function and erythema |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), through post-infusion (Day 1; Week 1, 2, 3, 4; Month 3, 6 and 12)
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Change in Hemolytic Anemia (RBC)
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3; Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Measurement of the number of red blood cells (Complete Blood Counts with Differential [CBCD]). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3; Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Change in Hemolytic Anemia (Reticulocyte)
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3; Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Measurement of the number of reticulocytes. |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3; Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Change in Thrombocytopenia
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3; Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Measure the number of platelets (Complete Blood Counts with Differential (CBCD)). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3; Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Change in Neutropenia
Periodo de tiempo: Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3; Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Measure the number of neutrophils (Complete Blood Counts with Differential [CBCD]). |
Baseline (up to 60 days before infusion of CD4^LVFOXP3), pre-infusion through post-infusion (Day 1, 2, 3; Week 1, 2, 3, 4; Month 2, 3, 6, 9, 12)
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Change in Daily insulin requirement - Type 1 diabetes monitoring
Periodo de tiempo: Baseline taken 60-30 days before infusion of CD4^LVFOXP3 and post-infusion (over 7 consecutive days preceding each study visit);
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Mean daily insulin use recorded over 7 consecutive days preceding each evaluation timepoint for patients with Type 1 Diabetes. |
Baseline taken 60-30 days before infusion of CD4^LVFOXP3 and post-infusion (over 7 consecutive days preceding each study visit);
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Change in hyper-/hypo-glycemic events - Type 1 diabetes monitoring
Periodo de tiempo: Baseline taken 60-30 days before infusion of CD4^LVFOXP3 and post-infusion (over 7 consecutive days preceding each study visit);
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Continuous glucose monitoring (CGM) metrics to log episodes of hyper/hypoglycemic events. |
Baseline taken 60-30 days before infusion of CD4^LVFOXP3 and post-infusion (over 7 consecutive days preceding each study visit);
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Change in Autoantibody Profile
Periodo de tiempo: Screening/ Baseline, Post-infusion (Month 6 and 12)
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Measure autoantibodies to organs involved in the disease (participant-specific): anti-insulin (IAA), anti-islet antigens (IA), anti-glutamic acid decarboxylase (GAD), anti-zinc transporter8 (ZNT8), anti-islet cells (ICA), anti-liver kidney microsome (LKM), anti-thyroperoxidase (TPO), anti-thyroglobulin (TG), anti-enterocytes, anti-SMA |
Screening/ Baseline, Post-infusion (Month 6 and 12)
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Change in Creatinine as a measure of Kidney Function
Periodo de tiempo: Baseline taken 60-30 days before infusion of CD4^LVFOXP3, Pre-infusion, Week 1, 2, 3, 4; Month 2, 3, 6, 9 and 12
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Measure kidney functional parameters, i.e., creatinine, in the blood and urine. |
Baseline taken 60-30 days before infusion of CD4^LVFOXP3, Pre-infusion, Week 1, 2, 3, 4; Month 2, 3, 6, 9 and 12
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Change in PedsQL Gastrointestinal Symptoms Scale - Quality of Life
Periodo de tiempo: Pre-Infusion; Month 6, 12
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Measured with Gastrointestinal Symptoms Scale: minimum value = 0 (never a problem), maximum value = 4 (almost always a problem). Higher scores mean a worse outcome. |
Pre-Infusion; Month 6, 12
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Disease-free Survival - Changes from Baseline
Periodo de tiempo: Up to 15 years
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The length of time from cell infusion to the point at which the participant survives without any new or worsening of existing signs or symptoms of disease. The data will be compared with historical disease-free survival probability. Probability of disease-free survival will be computed with the use of Kaplan-Meier estimator. |
Up to 15 years
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Overall Survival - Changes from Baseline
Periodo de tiempo: Up to 15 years
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Participant survival |
Up to 15 years
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Colaboradores e Investigadores
Aquí es donde encontrará personas y organizaciones involucradas en este estudio.
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Jessie Alexander, MD, Stanford University
Publicaciones y enlaces útiles
La persona responsable de ingresar información sobre el estudio proporciona voluntariamente estas publicaciones. Estos pueden ser sobre cualquier cosa relacionada con el estudio.
Publicaciones Generales
- Marek-Trzonkowska N, Mysliwiec M, Dobyszuk A, Grabowska M, Derkowska I, Juscinska J, Owczuk R, Szadkowska A, Witkowski P, Mlynarski W, Jarosz-Chobot P, Bossowski A, Siebert J, Trzonkowski P. Therapy of type 1 diabetes with CD4(+)CD25(high)CD127-regulatory T cells prolongs survival of pancreatic islets - results of one year follow-up. Clin Immunol. 2014 Jul;153(1):23-30. doi: 10.1016/j.clim.2014.03.016. Epub 2014 Apr 1.
- Bluestone JA, Buckner JH, Fitch M, Gitelman SE, Gupta S, Hellerstein MK, Herold KC, Lares A, Lee MR, Li K, Liu W, Long SA, Masiello LM, Nguyen V, Putnam AL, Rieck M, Sayre PH, Tang Q. Type 1 diabetes immunotherapy using polyclonal regulatory T cells. Sci Transl Med. 2015 Nov 25;7(315):315ra189. doi: 10.1126/scitranslmed.aad4134.
- Brunstein CG, Miller JS, McKenna DH, Hippen KL, DeFor TE, Sumstad D, Curtsinger J, Verneris MR, MacMillan ML, Levine BL, Riley JL, June CH, Le C, Weisdorf DJ, McGlave PB, Blazar BR, Wagner JE. Umbilical cord blood-derived T regulatory cells to prevent GVHD: kinetics, toxicity profile, and clinical effect. Blood. 2016 Feb 25;127(8):1044-51. doi: 10.1182/blood-2015-06-653667. Epub 2015 Nov 12.
- Hori S, Nomura T, Sakaguchi S. Control of regulatory T cell development by the transcription factor Foxp3. Science. 2003 Feb 14;299(5609):1057-61. doi: 10.1126/science.1079490. Epub 2003 Jan 9.
- Allan SE, Alstad AN, Merindol N, Crellin NK, Amendola M, Bacchetta R, Naldini L, Roncarolo MG, Soudeyns H, Levings MK. Generation of potent and stable human CD4+ T regulatory cells by activation-independent expression of FOXP3. Mol Ther. 2008 Jan;16(1):194-202. doi: 10.1038/sj.mt.6300341. Epub 2007 Nov 6.
- Amendola M, Venneri MA, Biffi A, Vigna E, Naldini L. Coordinate dual-gene transgenesis by lentiviral vectors carrying synthetic bidirectional promoters. Nat Biotechnol. 2005 Jan;23(1):108-16. doi: 10.1038/nbt1049. Epub 2004 Dec 26.
- Barzaghi F, Passerini L, Gambineri E, Ciullini Mannurita S, Cornu T, Kang ES, Choe YH, Cancrini C, Corrente S, Ciccocioppo R, Cecconi M, Zuin G, Discepolo V, Sartirana C, Schmidtko J, Ikinciogullari A, Ambrosi A, Roncarolo MG, Olek S, Bacchetta R. Demethylation analysis of the FOXP3 locus shows quantitative defects of regulatory T cells in IPEX-like syndrome. J Autoimmun. 2012 Feb;38(1):49-58. doi: 10.1016/j.jaut.2011.12.009. Epub 2012 Jan 20.
- Barzaghi F, Amaya Hernandez LC, Neven B, Ricci S, Kucuk ZY, Bleesing JJ, Nademi Z, Slatter MA, Ulloa ER, Shcherbina A, Roppelt A, Worth A, Silva J, Aiuti A, Murguia-Favela L, Speckmann C, Carneiro-Sampaio M, Fernandes JF, Baris S, Ozen A, Karakoc-Aydiner E, Kiykim A, Schulz A, Steinmann S, Notarangelo LD, Gambineri E, Lionetti P, Shearer WT, Forbes LR, Martinez C, Moshous D, Blanche S, Fisher A, Ruemmele FM, Tissandier C, Ouachee-Chardin M, Rieux-Laucat F, Cavazzana M, Qasim W, Lucarelli B, Albert MH, Kobayashi I, Alonso L, Diaz De Heredia C, Kanegane H, Lawitschka A, Seo JJ, Gonzalez-Vicent M, Diaz MA, Goyal RK, Sauer MG, Yesilipek A, Kim M, Yilmaz-Demirdag Y, Bhatia M, Khlevner J, Richmond Padilla EJ, Martino S, Montin D, Neth O, Molinos-Quintana A, Valverde-Fernandez J, Broides A, Pinsk V, Ballauf A, Haerynck F, Bordon V, Dhooge C, Garcia-Lloret ML, Bredius RG, Kalwak K, Haddad E, Seidel MG, Duckers G, Pai SY, Dvorak CC, Ehl S, Locatelli F, Goldman F, Gennery AR, Cowan MJ, Roncarolo MG, Bacchetta R; Primary Immune Deficiency Treatment Consortium (PIDTC) and the Inborn Errors Working Party (IEWP) of the European Society for Blood and Marrow Transplantation (EBMT). Long-term follow-up of IPEX syndrome patients after different therapeutic strategies: An international multicenter retrospective study. J Allergy Clin Immunol. 2018 Mar;141(3):1036-1049.e5. doi: 10.1016/j.jaci.2017.10.041. Epub 2017 Dec 11.
- Battaglia M, Stabilini A, Roncarolo MG. Rapamycin selectively expands CD4+CD25+FoxP3+ regulatory T cells. Blood. 2005 Jun 15;105(12):4743-8. doi: 10.1182/blood-2004-10-3932. Epub 2005 Mar 3.
- Bluestone JA, Tang Q. Treg cells-the next frontier of cell therapy. Science. 2018 Oct 12;362(6411):154-155. doi: 10.1126/science.aau2688. No abstract available.
- Bonini C, Grez M, Traversari C, Ciceri F, Marktel S, Ferrari G, Dinauer M, Sadat M, Aiuti A, Deola S, Radrizzani M, Hagenbeek A, Apperley J, Ebeling S, Martens A, Kolb HJ, Weber M, Lotti F, Grande A, Weissinger E, Bueren JA, Lamana M, Falkenburg JH, Heemskerk MH, Austin T, Kornblau S, Marini F, Benati C, Magnani Z, Cazzaniga S, Toma S, Gallo-Stampino C, Introna M, Slavin S, Greenberg PD, Bregni M, Mavilio F, Bordignon C. Safety of retroviral gene marking with a truncated NGF receptor. Nat Med. 2003 Apr;9(4):367-9. doi: 10.1038/nm0403-367. No abstract available.
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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 (Actual)
22 de marzo de 2022
Finalización primaria (Estimado)
1 de diciembre de 2026
Finalización del estudio (Estimado)
1 de octubre de 2027
Fechas de registro del estudio
Enviado por primera vez
12 de enero de 2022
Primero enviado que cumplió con los criterios de control de calidad
4 de febrero de 2022
Publicado por primera vez (Actual)
15 de febrero de 2022
Actualizaciones de registros de estudio
Última actualización publicada (Actual)
5 de noviembre de 2026
Última actualización enviada que cumplió con los criterios de control de calidad
2 de noviembre de 2026
Última verificación
1 de noviembre de 2026
Más información
Términos relacionados con este estudio
Palabras clave
Términos MeSH relevantes adicionales
Otros números de identificación del estudio
- IRB-63373
- DRU-2020-7764 (Otro identificador: FDA Office of Orphan Products Development)
- RPD-2020-470 (Otro identificador: FDA Office of Orphan Products Development)
- CLIN2-13259 (Otro número de subvención/financiamiento: California Institute of Regenerative Medicine (CIRM))
- 1R01FD007540 (Subvención/Contrato de la FDA de EE. UU.)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
NO
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Sí
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
No
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. .