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- Sperimentazione clinica 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
Panoramica dello studio
Stato
Intervento / Trattamento
Descrizione dettagliata
Tipo di studio
Iscrizione (Effettivo)
Fase
- Fase 1
Contatti e Sedi
Luoghi di studio
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California
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Stanford, California, Stati Uniti, 94305
- Stanford University School of Medicine
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Criteri di partecipazione
Criteri di ammissibilità
Età idonea allo studio
Accetta volontari sani
Sessi ammissibili allo studio
Descrizione
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
Piano di studio
Come è strutturato lo studio?
Dettagli di progettazione
- Scopo principale: Trattamento
- Assegnazione: N / A
- Modello interventistico: Assegnazione di gruppo singolo
- Mascheramento: Nessuno (etichetta aperta)
Armi e interventi
Gruppo di partecipanti / Arm |
Intervento / Trattamento |
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Sperimentale: 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.
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2.5 mg/wk up to 5 mg 3x/wk
Altri nomi:
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Cosa sta misurando lo studio?
Misure di risultato primarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
---|---|---|
Red Blood Cell (RBC) Transfusion Independence
Lasso di tempo: 6 months
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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.
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6 months
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Misure di risultato secondarie
Misura del risultato |
Misura Descrizione |
Lasso di tempo |
---|---|---|
Red Blood Cell (RBC) Transfusions
Lasso di tempo: 6 months
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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.
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6 months
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Hemoglobin Concentration
Lasso di tempo: 6 months
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The effect on hemoglobin concentration was assessed as the change from baseline, measured in g/dL.
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6 months
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Neutrophil Response
Lasso di tempo: 6 months
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The effect on neutrophil levels was assessed as the change in neutrophil count from baseline.
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6 months
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Platelet Response
Lasso di tempo: 6 months
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The effect on platelet levels as assessed as the change in platelet count from baseline.
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6 months
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Duration of Response
Lasso di tempo: 6 months
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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.
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6 months
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Toxicity
Lasso di tempo: 6 months
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Toxicity was assessed as the number of adverse events related to lenalidomide.
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6 months
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Collaboratori e investigatori
Sponsor
Collaboratori
Investigatori
- Investigatore principale: Jason Robert Gotlib, Stanford University
Studiare le date dei record
Studia le date principali
Inizio studio
Completamento primario (Effettivo)
Completamento dello studio (Effettivo)
Date di iscrizione allo studio
Primo inviato
Primo inviato che soddisfa i criteri di controllo qualità
Primo Inserito (Stima)
Aggiornamenti dei record di studio
Ultimo aggiornamento pubblicato (Effettivo)
Ultimo aggiornamento inviato che soddisfa i criteri QC
Ultimo verificato
Maggiori informazioni
Termini relativi a questo studio
Termini MeSH pertinenti aggiuntivi
- Neoplasie per tipo istologico
- Neoplasie
- Malattie del midollo osseo
- Malattie ematologiche
- Malattie genetiche, congenite
- Condizioni precancerose
- Anemia, ipoplastica, congenita
- Anemia, aplastica
- Sindromi da insufficienza congenita del midollo osseo
- Disturbi da insufficienza del midollo osseo
- Leucemia, mieloide
- Aplasia eritrocitaria, pura
- Sindromi mielodisplastiche
- Leucemia
- Leucemia, mieloide, acuta
- Preleucemia
- Anemia
- Anemia, Diamante-Blackfan
- Effetti fisiologici delle droghe
- Agenti antineoplastici
- Fattori immunologici
- Inibitori dell'angiogenesi
- Agenti di modulazione dell'angiogenesi
- Sostanze per la crescita
- Inibitori della crescita
- Lenalidomide
Altri numeri di identificazione dello studio
- IRB-16822
- SU-12082009-4523 (Altro identificatore: Stanford University)
- RV-0365 (Altro identificatore: Celgene Corporation)
- HEMMDS0022 (Altro identificatore: OnCore)
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Prove cliniche su Lenalidomide
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