RISE UP

Sise afihan RISE UP, iwadii isegun TUNTUN kan lati ṣe agbeyẹwo isoro irora ati arunmọleegun ninu aarun sẹjẹdọlẹ

RISE UP (2021‑001674‑34) jẹ idanwo isegun ti ifarahan 2/3, iruniloju, aileto, ti placebo ndari, ọlọpọ ibi¹

Iwadii isegun yii nse igbelewọn ilagbara ati aabo mitapivat ninu itẹju aarun sẹjẹdọlẹ fun awọn olukopa ọmọ ọdun 16 ati jubẹẹlọ.¹

WO AGBEKALẸ IWADII ISEGUN

WO AWỌN KOKO AMUYẸ IFISI/KOKO AMUYẸ AYỌKURO

Iwadii fihan wipe ju idaji awọn alaisan lọ ni yoo kopa ninu idanwo isegun kan bi olupese itọju ilera wọn ba jiroro rẹ pẹlu wọn.²

AGBEKALE IWADII ISEGUN RISE UP¹

OJU ORO AKOSO 3 (Ti nforukọsilẹ bayii)

BID=ẹmeji lojumọ; Hb=hemoglobin.

AWỌN KOKO AMUYẸ IKÀKÚN¹

Ọjọ ori 16 tabi jubẹẹlọ, pẹlu akọsilẹ iwadii arun sọjọdọlẹ (HbSS, HbSC, HbS/β0 thalassemia, HbS/β+ thalassemia, tabi awọn orisii other sickle cell syndrome variants sẹjẹdọlẹ yooku)
O keere tan isoro irora sejẹdọlẹ 2 ti kosi ju 10 lọ ninu awọn osu 12 to kọja saaju ki wọn to gba
- Ti o tumọsi asiko irora to gboro, aya riro to gboro, okó lile pipẹ, tabi ẹjẹ didi ninu ẹdọ tabi ọlọ inu
Bi lilo hydroxyurea, oogun hydroxyurea gbọdọ wa nilẹ fun o keere tan ọjọ 90 saaju aileto

AWỌN KOKO AMUYẸ IYỌKURO¹

Oyun tabi ifọmọlọyan
Gbigba eto ẹjẹ loorekoore
Aisedeede Hepatobiliary, aarun ẹdo lile, aarun oronro, tabi aarun kidirin lile
Saaju ifarahan si itọju ilera jiini tabi saaju mudunmudun eegun tabi ipaarọ sẹẹli
To ngba voxelotor, crizanlizumab, tabi L-glutamine lọwọlọwọ
To ngba itọju pẹlu awọn ohun ti ngbe hematopoietic larugẹ
Lilo CYP3A4/5 to lagbara ti ngbogunti tabi awọn imugberu CYP3A4 lagbara

PK ACTIVATION NGBARUKUTI ILERA SẸẸLI ẸJẸ PUPA

Mitapivat — iwadii oogun ninu RISE UP — jẹ ohun ti a fi nsewadii, lilo, ohun imusisẹ ẹnsaimu PK^1,3

Imusisẹ ẹnsaimu PK le se imugberu ilera, agbara, ati igba awọn sẹlli ẹjẹ pupa (RBC) fun awọn alaisan toni aiṣedede sẹlli ẹjẹ pupa¹

Pipọsi imuwa ATP, lati se iranlọwọ ipese awọn agbara ti RBC nilo
Dindin 2,3-DPG ku eyiti o npada mu atẹgun gberu sii fun hemoglobin, ti o si ndin aisan ku
Sise amojuto awọn ohun to ndin ijamba sẹlli ku

KỌ BI MITAPIVAT TI LE SISẸ NINU SẸJẸDỌLẸ

ATP=adenosine triphosphate; MOA=mechanism of action; PK=pyruvate kinase; 2,3-DPG=2,3-diphosphoglycerate.

KANSI AGIOS MEDICAL AFFAIRS

Fun awọn idahun pato ibeere nipa RISE UP, fi atẹjise imeeli ransẹ si medinfo@agios.com.

FI ATEJISE IMEELI RANSE SI MED AFFAIRS NISISINYI

MURASILẸ FUN IFARAHAN RISE UP TO KAN

Kọ sii nipa RISE UP, pẹlu alayẹ ẹkunrẹrẹ ibiwẹ, gbogbo awọn afojusun iwadii, ati koko alaye ifisi ati iyọkuro. Lẹyin naa ba awọn alaisan rẹ to yege sọrọ nipa iwadii naa.

Bi o ba ni awọn ibeere, fi imeeli ránṣẹ́ sí:
medinfo@agios.com (US)
ex-usmedinfo@agios.com (ita US)

I'm ReadySABẸWO SII CLINICALTRIALS.GOV

Isanpada wa fun awọn jagunjagun o si le ma tii kopa ninu RISE UP.

A fun awọn Akọni ní gbà-má-bínú wọ́n si le ma tii kopa ninu RISE UP. Akọni Àìsàn Fòní Kú Fọ̀la Nde jẹ ọrọ kan ti a sẹda ti awujọ àìsàn fòní kú fọ̀la nde si nlo.

O ntọkasi àwọn ọlọ́dọnni ti ngbe pẹlu àìsàn fòní kú fọ̀la nde.

A ko fọwọsi mitapivat fun itọju aarun sẹjẹdọlẹ.

A ko fọwọsi mitapivat fun itọju àìsàn fòní kú fọ̀la nde.

Awọn Itọkasi:

1. Data lori faili. Agios Pharmaceuticals, Inc.

2. Iwadii!America. Akojọ ero awujọ apapọ. Osu keje, ọdun 2017. Ti a yẹwo ni Osu Kẹwa, ọjọ 18, ọdun 2023. https://www.researchamerica.org/wp- content/uploads/2022/07/July2017ClinTrialMi norityOversamplesPressReleaseSlidesFINAL_ 0-1.pdf

3. Howard J, Kuo KHM, Oluyadi A, et al. Ifarahan 2/3, iwadii aileto, iruniloju, ti placebo ndari ti mitapivat ninu awọn alaisan toni aaru sẹjẹdọlẹ/ Ejẹ. 2021;138(suppl 1):3109.