- ICH GCP
- US Clinical Trials Registry
- Klinisk forsøg NCT00976248
Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia
Phase II Study of Everolimus (RAD001) in Primary Therapy of Waldenstrom's Macroglobulinemia
Studieoversigt
Status
Betingelser
Intervention / Behandling
Detaljeret beskrivelse
- Participants will take RAD001 orally once a day in the morning. Each treatment cycle lasts for four weeks. Participants will receive up to 72 cycles of treatment.
- During each cycle, participants will be asked to visit the clinic for scheduled tests and exams. They will visit the clinic on the first day of each of the first three cycles, and then once every 3 cycles. During the visits, participants will have a physical exam and blood tests.. Participants may also have CT scans of the chest, abdomen and pelvis as well as a bone marrow aspirate and biopsy.
Undersøgelsestype
Tilmelding (Faktiske)
Fase
- Fase 2
Kontakter og lokationer
Studiesteder
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Massachusetts
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Boston, Massachusetts, Forenede Stater, 02115
- Dana-Farber Cancer Institute
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Deltagelseskriterier
Berettigelseskriterier
Aldre berettiget til at studere
Tager imod sunde frivillige
Køn, der er berettiget til at studere
Beskrivelse
Inclusion Criteria:
- 18 years of age or older
- Adequate liver and renal function as outlined in the protocol
- Fasting serum cholesterol 300mg/dl or less OR 7.75mmol/L or less AND fasting triglycerides 2.5 x institutional ULN or less.
- Clinicopathological diagnosis of Waldenstrom's macroglobulinemia as defined by consensus panel of the Second International Workshop on Waldenstrom's macroglobulinemia
- No previous therapy for WM
- Measurable disease, defined as presence of immunoglobulin M (IgM) paraprotein with a minimum IgM level of 2 times the upper limit of each institution's normal value or greater is required
- ECOG Performance status of 0-2
- Patients must have a life expectancy of at least 3 months
- Baseline platelet and absolute neutrophil as outlined in the protocol
- INR and PTT 1.5 x normalized ratio or less
- A male subject agrees to use an acceptable method for contraception for the duration of study and for 8 weeks after the last dose of the study drug
- Female subject either post-menopausal or surgically sterilized or willing to use acceptable methods of birth control for the duration of the study and for 8 weeks after the last dose of study drug
Exclusion Criteria:
- Patients experiencing symptomatic hyperviscosity and requiring plasmapheresis. This includes any patient who, in the judgement of the investigator requires urgent response and will not be eligible. These patients have hyperviscosity which includes serum IgM levels of 5000 mg/dL or greater. Symptoms may include nosebleeds, visual complications, fatigue, headaches, confusion, etc.
- Patients, who have had a major surgery or significant traumatic injury within 4 weeks of start of study drug, patients who have not recovered from the side effects of any major surgery or patients that may require major surgery during the course of the study.
- Patients receiving chronic, systemic treatment with corticosteroids or another immunosuppressive agent. Topical or inhaled corticosteroids are allowed.
- Patients should not receive any immunization with attenuated live vaccines within one week of study entry or during study period.
- Patients who have had any severe and/or uncontrolled medical conditions or other conditions that would affect their participation in the study.
- Impairment of gastrointestinal function or gastrointestinal disease that may significantly alter the absorption of RAD001.
- Female patients that are pregnant or breast feeding, or adults of reproductive potential who are not using effective birth control methods.
- Patients with known hypersensitivity to RAD001 or other rapamycins or to its excipients
- Patients with other malignancies within the past 3 years except for adequately treated carcinoma of the cervix or basal or squamous cell of the skin
- Patients with known history of HIV seropositivity
- History of noncompliance to medical regimens
- Patients unwilling to or unable to comply with the protocol
Studieplan
Hvordan er undersøgelsen tilrettelagt?
Design detaljer
- Primært formål: Behandling
- Tildeling: N/A
- Interventionel model: Enkelt gruppeopgave
- Maskning: Ingen (Åben etiket)
Våben og indgreb
Deltagergruppe / Arm |
Intervention / Behandling |
---|---|
Eksperimentel: RAD001
RAD001, oral, 10 mg, daily
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Taken orally once a day
Andre navne:
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Hvad måler undersøgelsen?
Primære resultatmål
Resultatmål |
Foranstaltningsbeskrivelse |
Tidsramme |
---|---|---|
Overall Response Rate of RAD001 in Patients With Previously Untreated WM
Tidsramme: End of Treatment, an average of 16 months
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Overall Response = Complete Response + Near Complete Response + Very Good Partial Response + Partial Response + Minor Response Complete Response: resolution of all symptoms, normalization of serum IgM levels with complete disappearance of IgM paraprotein by immunofixation, and resolution of any adenopathy or splenomegaly. A near CR (nCR) is defined as fulfilling all CR criteria in the presence of positive immunofixation test for an IgM paraprotein. Very Good Partial Response: > 90% reduction in serum IgM levels. Partial Response: > 50% reduction in serum IgM levels. Minor Response: 25-49% reduction in serum IgM levels Progressive Disease: greater than 25% increase in serum IgM level occurs from the lowest attained response value or progression of clinically significant disease related symptom(s). Stable Disease: < 25% change in serum IgM levels, in the absence of new or increasing adenopathy or splenomegaly and/or other progressive signs or symptoms of WM |
End of Treatment, an average of 16 months
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Time to Progression With Single Agent RAD001 Therapy in Previously Untreated WM.
Tidsramme: End of Treatment, an average of 16 months
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Progression is defined as a 25% increase in serum IgM from the lowest attained response value or progression of clinically significant disease related symptoms.
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End of Treatment, an average of 16 months
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Time to Next Therapy With Single Agent RAD001 Therapy in Previously Untreated WM
Tidsramme: End of follow-up, an average of 18 months
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End of follow-up, an average of 18 months
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Samarbejdspartnere og efterforskere
Sponsor
Efterforskere
- Ledende efterforsker: Steven Treon, MD, Dana-Farber Cancer Institute
Publikationer og nyttige links
Generelle publikationer
- Treon SP, Tripsas CK, Ioakimidis L, Warren D, Patterson C, Heffner L, Eradat H, Gregory SA, Thomas S, Advani R, Baz R, Badros, Ashraf Z, Matous J, Anderson KC, Ghobrial IM Prospective, Multicenter Study of the MTOR Inhibitor Everolimus (RAD001) As Primary Therapy in Waldenstrom's Macroglobulinemia ASH Annual Meeting Abstracts 2011 118: 2951
- Treon SP, Tripsas CK, Meid K, Patterson CJ, Heffner H, Gregory SA, Thomas SK, Advani RH, Baz R, Badros AZ, Matous J, Murphy TJ, Ghobrial IM. Prospective, Multicenter Study Of The MTOR Inhibitor Everolimus (RAD001) As Primary Therapy In Waldenstrom's Macroglobulinemia Blood 2013 122:1822.
- Treon SP, Meid K, Tripsas C, Heffner LT, Eradat H, Badros AZ, Xu L, Hunter ZR, Yang G, Patterson CJ, Gustine J, Castillo JJ, Matous J, Ghobrial IM. Prospective, Multicenter Clinical Trial of Everolimus as Primary Therapy in Waldenstrom Macroglobulinemia (WMCTG 09-214). Clin Cancer Res. 2017 May 15;23(10):2400-2404. doi: 10.1158/1078-0432.CCR-16-1918. Epub 2016 Nov 11.
Datoer for undersøgelser
Studer store datoer
Studiestart
Primær færdiggørelse (Faktiske)
Studieafslutning (Faktiske)
Datoer for studieregistrering
Først indsendt
Først indsendt, der opfyldte QC-kriterier
Først opslået (Skøn)
Opdateringer af undersøgelsesjournaler
Sidste opdatering sendt (Faktiske)
Sidste opdatering indsendt, der opfyldte kvalitetskontrolkriterier
Sidst verificeret
Mere information
Begreber relateret til denne undersøgelse
Nøgleord
Yderligere relevante MeSH-vilkår
- Hjerte-kar-sygdomme
- Karsygdomme
- Sygdomme i immunsystemet
- Neoplasmer efter histologisk type
- Neoplasmer
- Lymfoproliferative lidelser
- Lymfesygdomme
- Immunproliferative lidelser
- Hæmatologiske sygdomme
- Hæmoragiske lidelser
- Hæmostatiske lidelser
- Paraproteinæmier
- Blodproteinforstyrrelser
- Neoplasmer, Plasmacelle
- Waldenstrom Makroglobulinæmi
- Lægemidlers fysiologiske virkninger
- Antineoplastiske midler
- Immunsuppressive midler
- Immunologiske faktorer
- Everolimus
Andre undersøgelses-id-numre
- 09-214
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Kliniske forsøg med Waldenstroms makroglobulinæmi
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Acerta Pharma BVAktiv, ikke rekrutterendeWaldenström Macroglobulinemia (WM)Spanien, Forenede Stater, Det Forenede Kongerige, Frankrig, Italien, Grækenland, Holland
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BeiGeneRekrutteringWaldenstrom Makroglobulinæmi | Waldenstroms tilbagevendende makroglobulinæmi | Waldenstroms Macroglobulinemia RefractoryForenede Stater, Australien, Frankrig, Kina, Spanien, Det Forenede Kongerige
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Beijing InnoCare Pharma Tech Co., Ltd.AfsluttetWaldenstroms tilbagevendende makroglobulinæmi | Waldenstroms Macroglobulinemia RefractoryKina
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Dana-Farber Cancer InstituteNational Cancer Institute (NCI)RekrutteringMyelomatose | Vægttab | Faste | MGUS | Kræftforebyggelse | Ulmende Waldenstrom Macroglobulinemia (WM)Forenede Stater
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Weill Medical College of Cornell UniversityMayo Clinic; Janssen Scientific Affairs, LLCAfsluttetWaldenstrom Makroglobulinæmi | Waldenstroms tilbagevendende makroglobulinæmi | Waldenstroms Macroglobulinemia Refractory | Waldenstrøms sygdom | Waldenström; Hypergammaglobulinæmi | Waldenstroms makroglobulinæmi af lymfeknuder | Waldenstroms makroglobulinæmi, uden omtale af remissionForenede Stater
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Mustang BioNational Cancer Institute (NCI)RekrutteringMantelcellelymfom tilbagevendende | Mantelcellelymfom refraktært | Lille lymfatisk lymfom, tilbagefald | Waldenstroms tilbagevendende makroglobulinæmi | Follikulært B-celle non-Hodgkins lymfom | B-celle lymfom refraktær | Waldenstroms Macroglobulinemia Refractory | Kronisk lymfoid leukæmi i tilbagefaldForenede Stater
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Mustang BioTilmelding efter invitationDiffust storcellet B-celle lymfom | Blastisk Plasmacytoid Dendritisk Cell Neoplasma (BPDCN) | Hårcelleleukæmi | Mantelcellelymfom tilbagevendende | Mantelcellelymfom refraktært | Kronisk lymfatisk leukæmi i tilbagefald | Lille lymfatisk lymfom, tilbagefald | Waldenstroms tilbagevendende makroglobulinæmi og andre forholdForenede Stater
Kliniske forsøg med RAD001
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Children's Hospital Medical Center, CincinnatiNovartisAfsluttetTuberøs sklerose | AngiolipomForenede Stater
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Novartis PharmaceuticalsAfsluttetMetastatisk nyrecellekarcinom (mRCC)Tyskland
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University of Alabama at BirminghamNational Cancer Institute (NCI); Children's Hospital of Philadelphia; Washington... og andre samarbejdspartnereAfsluttet
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Novartis PharmaceuticalsAfsluttet
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Novartis PharmaceuticalsAfsluttetLymfangioleiomyomatose (LAM) | Tuberøs sklerosekompleks (TSC)Forenede Stater, Det Forenede Kongerige, Tyskland, Italien, Den Russiske Føderation, Holland, Japan, Canada, Polen, Frankrig, Spanien
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Radiation Therapy Oncology GroupNational Cancer Institute (NCI); NRG OncologyAfsluttetTumorer i hjernen og centralnervesystemetForenede Stater, Israel, Canada
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Guangdong Provincial People's HospitalNovartisUkendtNeuroendokrine tumorer | Carcinoid tumorKina
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Fox Chase Cancer CenterRekrutteringMetastatisk bugspytkirtelkræftForenede Stater
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Novartis PharmaceuticalsAfsluttetHepatocellulært karcinomForenede Stater, Spanien, Korea, Republikken, Holland, Taiwan
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Novartis PharmaceuticalsAfsluttetNeuroendokrint karcinom i lunge og thymusItalien, Det Forenede Kongerige, Holland, Spanien, Tyskland, Frankrig, Grækenland, Danmark, Sverige