Design and rationale of FOCUS (PX-171-011): A randomized,
open-label, phase 3 study of carfilzomib versus best supportive
care regimen in patients with relapsed and refractory multiple
myeloma (R/R MM)

J 2012

Design and rationale of FOCUS (PX-171-011): A randomized, open-label, phase 3 study of carfilzomib versus best supportive care regimen in patients with relapsed and refractory multiple myeloma (R/R MM)

HÁJEK, Roman; Richard BRYCE; Sunhee RO; Barbara KLENCKE; Heinz LUDWIG et al.

Základní údaje

Originální název

Design and rationale of FOCUS (PX-171-011): A randomized, open-label, phase 3 study of carfilzomib versus best supportive care regimen in patients with relapsed and refractory multiple myeloma (R/R MM)

Autoři

HÁJEK, Roman; Richard BRYCE; Sunhee RO; Barbara KLENCKE a Heinz LUDWIG

Vydání

BMC Cancer, England, London : BioMed Central, 2012, 1471-2407

Další údaje

Jazyk

angličtina

Typ výsledku

Článek v odborném periodiku

Obor

30200 3.2 Clinical medicine

Stát vydavatele

Spojené státy

Utajení

není předmětem státního či obchodního tajemství

Impakt faktor

Impact factor: 3.333

Označené pro přenos do RIV

Organizační jednotka

Lékařská fakulta

DOI

https://doi.org/10.1186/1471-2407-12-415

UT WoS

000311048500001

Klíčová slova anglicky

Multiple myeloma; Proteasome inhibitor; Phase 3 trial; Relapsed; Refractory; Overall survival

Příznaky

Mezinárodní význam, Recenzováno

Změněno: 23. 4. 2014 15:15, Ing. Mgr. Věra Pospíšilíková

Anotace

V originále

Carfilzomib is a next-generation proteasome inhibitor with single-agent activity in patients with relapsed and refractory multiple myeloma (R/R MM). In PX-171-003-A1, a single-arm phase 2 study of carfilzomib monotherapy in heavily pretreated patients, the overall response rate was 23.7%, 37% of patients achieved >= minimal response and median overall survival (OS) was 15.6 months. Based on this study, carfilzomib was recently approved by the US Food and Drug Administration for the treatment of R/R MM. Herein we describe the trial design and rationale for a phase 3 randomized study, FOCUS (CarFilzOmib for AdvanCed Refractory MUltiple Myeloma European Study), being conducted to compare OS after treatment with single-agent carfilzomib to best supportive care (BSC) regimen in R/R MM. Conclusions: This phase 3 trial will provide more rigorous data for carfilzomib, as this is the first carfilzomib study with OS as the primary endpoint and will not be confounded by crossover and will provide more robust secondary response and safety results that will add to the data set from prior phase 2 studies. FOCUS will facilitate regulatory approvals around the world and expand treatment options for patients with R/R MM.

Přiložené soubory

Hajek_2012_-_BMC_Cancer.pdf

Požádat o autorskou verzi souboru

Citovat

HÁJEK, Roman; Richard BRYCE; Sunhee RO; Barbara KLENCKE a Heinz LUDWIG. Design and rationale of FOCUS (PX-171-011): A randomized, open-label, phase 3 study of carfilzomib versus best supportive care regimen in patients with relapsed and refractory multiple myeloma (R/R MM). BMC Cancer. England: London : BioMed Central, 2012, roč. 12, Sep 19, s. 415-421. ISSN 1471-2407. Dostupné z: https://doi.org/10.1186/1471-2407-12-415.

@article{1069223,
   author = {Hájek, Roman and Bryce, Richard and Ro, Sunhee and Klencke, Barbara and Ludwig, Heinz},
   article_location = {England},
   article_number = {Sep 19},
   doi = {https://doi.org/10.1186/1471-2407-12-415},
   keywords = {Multiple myeloma; Proteasome inhibitor; Phase 3 trial; Relapsed; Refractory; Overall survival},
   language = {eng},
   issn = {1471-2407},
   journal = {BMC Cancer},
   title = {Design and rationale of FOCUS (PX-171-011): A randomized, open-label, phase 3 study of carfilzomib versus best supportive care regimen in patients with relapsed and refractory multiple myeloma (R/R MM)},
   volume = {12},
   year = {2012}
}

TY  - JOUR
ID  - 1069223
AU  - Hájek, Roman - Bryce, Richard - Ro, Sunhee - Klencke, Barbara - Ludwig, Heinz
PY  - 2012
TI  - Design and rationale of FOCUS (PX-171-011): A randomized, open-label, phase 3 study of carfilzomib versus best supportive care regimen in patients with relapsed and refractory multiple myeloma (R/R MM)
JF  - BMC Cancer
VL  - 12
IS  - Sep 19
SP  - 415-421
EP  - 415-421
PB  - London : BioMed Central
SN  - 14712407
KW  - Multiple myeloma
KW  - Proteasome inhibitor
KW  - Phase 3 trial
KW  - Relapsed
KW  - Refractory
KW  - Overall survival
N2  - Carfilzomib is a next-generation proteasome inhibitor with single-agent activity in patients with relapsed and refractory multiple myeloma (R/R MM). In PX-171-003-A1, a single-arm phase 2 study of carfilzomib monotherapy in heavily pretreated patients, the overall response rate was 23.7%, 37% of patients achieved >= minimal response and median overall survival (OS) was 15.6 months. Based on this study, carfilzomib was recently approved by the US Food and Drug Administration for the treatment of R/R MM. Herein we describe the trial design and rationale for a phase 3 randomized study, FOCUS (CarFilzOmib for AdvanCed Refractory MUltiple Myeloma European Study), being conducted to compare OS after treatment with single-agent carfilzomib to best supportive care (BSC) regimen in R/R MM. Conclusions: This phase 3 trial will provide more rigorous data for carfilzomib, as this is the first carfilzomib study with OS as the primary endpoint and will not be confounded by crossover and will provide more robust secondary response and safety results that will add to the data set from prior phase 2 studies. FOCUS will facilitate regulatory approvals around the world and expand treatment options for patients with R/R MM.
ER  -

HÁJEK, Roman; Richard BRYCE; Sunhee RO; Barbara KLENCKE a Heinz LUDWIG. Design and rationale of FOCUS (PX-171-011): A randomized, open-label, phase 3 study of carfilzomib versus best supportive care regimen in patients with relapsed and refractory multiple myeloma (R/R MM). \textit{BMC Cancer}. England: London : BioMed Central, 2012, roč.~12, Sep 19, s.~415-421. ISSN~1471-2407. Dostupné z: https://doi.org/10.1186/1471-2407-12-415.

Přehled o publikaci