- ICH GCP
- Registro de ensayos clínicos de EE. UU.
- Ensayo clínico NCT01034592
Pilot Lenalidomide in Adult Diamond-Blackfan Anemia Patients w/ RBC Transfusion-Dependent Anemia
A Pilot Study of Lenalidomide in Adult Diamond-Blackfan Anemia Patients With Red Blood Cell Transfusion-Dependent Anemia
This is a single-center, single arm, open-label study of oral lenalidomide monotherapy administered to red blood cell (RBC) transfusion dependent adult subjects with Diamond-Blackfan Anemia (DBA).
Primary Objective: To evaluate the erythroid response rate as measured by rate of red blood cell transfusion independence [MDS International Working Group (IWG) 2000 Criteria will be applied].
Secondary Objective: 1)To evaluate the tolerability and safety profile of lenalidomide in patients with DBA and other inherited marrow failure syndromes 2) To correlate response to lenalidomide with biologic surrogates of DBA including ribosomal protein mutation status, ex vivo erythroid colony growth, and microarray gene expression
Descripción general del estudio
Estado
Intervención / Tratamiento
Descripción detallada
Tipo de estudio
Inscripción (Actual)
Fase
- Fase 1
Contactos y Ubicaciones
Ubicaciones de estudio
-
-
California
-
Stanford, California, Estados Unidos, 94305
- Stanford University School of Medicine
-
-
Criterios de participación
Criterio de elegibilidad
Edades elegibles para estudiar
Acepta Voluntarios Saludables
Géneros elegibles para el estudio
Descripción
INCLUSION CRITERIA
- Understand and voluntarily sign an informed consent form
- Diagnosis of DBA
- Age ≥ 18 years at the time of signing the informed consent form.
- Able to adhere to the study visit schedule and other protocol requirements.
- Red blood cell transfusion-dependent with a requirement of at least one unit of RBCs per month for the 2 months prior to study enrollment (eg, 2 units/8 weeks)
- If applicable, ongoing therapy with a stable or decreasing dose of prednisone ≤ 60 mg/d or corticosteroid equivalent, for which there has been no treatment-related improvement in RBC transfusion requirements for at least 2 months prior to study entry
- Eastern Cooperative Oncology Group (ECOG) performance status of ≤ 2 at study entry.
Laboratory test results within these ranges:
- Absolute neutrophil count (ANC) ≥ 1500/uL
- Platelet (Plt) count ≥ 100,000/uL
- Serum creatinine ≤ 2.0 mg/dL
- Direct bilirubin ≤ 1.5 mg/dL
- Aspartate aminotransferase (AST) ≤ 2.5 x upper limit of normal (ULN)
- Alanine aminotransferase (ALT) ≤ 2.5 x ULN
- Disease-free of prior malignancies for ≥ 5 years with exception of currently treated basal cell, squamous cell carcinoma of the skin, or carcinoma "in-situ" of the cervix or breast
- Females of childbearing potential (FCBP) must have a negative serum or urine pregnancy test with a sensitivity of ≥ 50 milli-International Units (MIU)/mL within 10 to 14 days prior to and again within 24 hours of prescribing lenalidomide (prescriptions must be filled within 7 days) and must either commit to continued abstinence from heterosexual intercourse or begin 2 acceptable methods of birth control, one highly effective method and one additional effective method at the same time, at least 28 days before she starts taking lenalidomide. FCBP must also agree to ongoing pregnancy testing. Men must agree to use a latex condom during sexual contact with a FCBP even if they have had a successful vasectomy
- Able to take aspirin (81 to 325 mg) daily as prophylactic anticoagulation (patients intolerant to aspirin may use warfarin or low molecular weight heparin)
EXCLUSION CRITERIA
- Any serious medical condition, laboratory abnormality, or psychiatric illness that would prevent the subject from signing the informed consent form.
- Pregnant or breast feeding females. (Lactating females must agree not to breast feed while taking lenalidomide)
- Any condition, including the presence of laboratory abnormalities, which places the subject at unacceptable risk if he/she were to participate in the study or confounds the ability to interpret data from the study
- Use of any other experimental drug or therapy (excluding steroids) specifically used for DBA within 28 days of baseline including metoclopramide, leucine, danazol, or other hormonal therapy
- Clinically significant anemia due to factors such as iron, B12, folate deficiencies, autoimmune or hereditary hemolysis, or gastrointestinal bleeding.
- Known hypersensitivity to thalidomide
- The development of erythema nodosum if characterized by a desquamating rash while taking thalidomide or similar drugs
- Any prior use of lenalidomide
- Concurrent use of other anti-cancer agents or treatments
- Known positive for HIV or infectious hepatitis, type A, B or C
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: Lenalidomide
Subjects will initially receive lenalidomide 2.5 mg, and may escalate up to 2.5 mg/wk up to 5 mg 3x/wk, depending toxicity and response.
|
2.5 mg/wk up to 5 mg 3x/wk
Otros nombres:
|
¿Qué mide el estudio?
Medidas de resultado primarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
---|---|---|
Red Blood Cell (RBC) Transfusion Independence
Periodo de tiempo: 6 months
|
Red blood cell (RBC) transfusion independence is reported as the number of subjects who achieve a continuous absence of the intravenous infusion of any RBC transfusion during any consecutive "rolling" 56 days during the treatment period.
|
6 months
|
Medidas de resultado secundarias
Medida de resultado |
Medida Descripción |
Periodo de tiempo |
---|---|---|
Red Blood Cell (RBC) Transfusions
Periodo de tiempo: 6 months
|
The effect on red blood cell (RBC) transfusions was assessed as the number of participants that achieved a greater than 50% decrease in RBC transfusion requirements.
|
6 months
|
Hemoglobin Concentration
Periodo de tiempo: 6 months
|
The effect on hemoglobin concentration was assessed as the change from baseline, measured in g/dL.
|
6 months
|
Neutrophil Response
Periodo de tiempo: 6 months
|
The effect on neutrophil levels was assessed as the change in neutrophil count from baseline.
|
6 months
|
Platelet Response
Periodo de tiempo: 6 months
|
The effect on platelet levels as assessed as the change in platelet count from baseline.
|
6 months
|
Duration of Response
Periodo de tiempo: 6 months
|
The response duration was measured from the last of the consecutive 56 days during which the subject was free of red blood cells (RBC) transfusions to the date of the first RBC transfusion after the 56-day RBC-transfusion-free period.
|
6 months
|
Toxicity
Periodo de tiempo: 6 months
|
Toxicity was assessed as the number of adverse events related to lenalidomide.
|
6 months
|
Colaboradores e Investigadores
Patrocinador
Colaboradores
Investigadores
- Investigador principal: Jason Robert Gotlib, Stanford University
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
Términos MeSH relevantes adicionales
- Neoplasias por tipo histológico
- Neoplasias
- Enfermedades de la médula ósea
- Enfermedades hematológicas
- Enfermedades Genéticas Congénitas
- Condiciones precancerosas
- Anemia Hipoplásica Congénita
- Anemia Aplásica
- Síndromes de insuficiencia congénita de la médula ósea
- Trastornos de insuficiencia de la médula ósea
- Leucemia Mieloide
- Aplasia de glóbulos rojos, pura
- Síndromes mielodisplásicos
- Leucemia
- Leucemia Mieloide Aguda
- Preleucemia
- Anemia
- Anemia, Diamond-Blackfan
- Efectos fisiológicos de las drogas
- Agentes antineoplásicos
- Factores inmunológicos
- Inhibidores de la angiogénesis
- Agentes moduladores de la angiogénesis
- Sustancias de crecimiento
- Inhibidores del crecimiento
- Lenalidomida
Otros números de identificación del estudio
- IRB-16822
- SU-12082009-4523 (Otro identificador: Stanford University)
- RV-0365 (Otro identificador: Celgene Corporation)
- HEMMDS0022 (Otro identificador: OnCore)
Plan de datos de participantes individuales (IPD)
¿Planea compartir datos de participantes individuales (IPD)?
Información sobre medicamentos y dispositivos, documentos del estudio
Estudia un producto farmacéutico regulado por la FDA de EE. UU.
Estudia un producto de dispositivo regulado por la FDA de EE. UU.
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. .
Ensayos clínicos sobre Lenalidomide
-
University Hopsital Schleswig Holstein Campus LübeckReclutamientoMieloma múltiple recién diagnosticadoAlemania