A supervised 12-week cycling-based aerobic exercise intervention performed twice per week.

Exercise

근육 긴장 장애인 1형과 2형 환자를 대상으로 멕실레틴의 효과와 안전성을 알아보는 26주간의 약물 시험입니다.

0 mg (matching sachet volumes to low, medium and high dose active drug)

Placebo

Mexiletine PR 167 mg (mexiletine HCl 200 mg) Mexiletine PR 333 mg (mexiletine HCl 400 mg) OR Mexiletine PR 500 mg (mexiletine HCl 600 mg)

Mexiletine granules for prolonged-release oral suspension

0mg(낮음, 중간, 높음 용량의 활성 약물에 맞는 파우치 용량)

위약

멕실레틴 PR 167mg(멕실레틴 HCl 200mg) 멕실레틴 PR 333mg(멕실레틴 HCl 400mg) 또는 멕실레틴 PR 500mg(멕실레틴 HCl 600mg)

장기 방출 경구 현탁액용 멕실레틴 과립

1형 및 2형 근긴장성 이영양증 환자에서 1일 1회 멕실레틴 PR의 효능 및 안전성

Orkambi® 치료 환자에게서 코 세포 모델을 통해 호흡 개선을 확인하는 연구입니다.

Orkambi®의 임상 효능을 예측하기 위한 호흡기 상피 기능 평가의 검증.

망막 질환을 가진 환자에게 주사한 치료제의 장기 안전성과 효과를 비교하는 시험입니다.

Participants previously treated with sub-retinal injection of BIIB111 in antecedent studies 273CH301 (NCT03496012) and 273CH203 (NCT03507686) will be enrolled. Participants previously treated with this same sub-retinal injection (rAAV2-REP1) in antecedent studies 20150371 (NCT02553135), Pro00028599 (NCT02077361), THOR-TUE-01 (NCT02671539), CHM09/01 (NCT01461213) and REGEN2015 (NCT02407678) will also be invited for enrollment. This is a follow-up study, investigational product was administered in the previous study.

Administered as specified in the treatment arm.

BIIB111

Participants previously treated with sub-retinal injection of BIIB112 in the antecedent study 274RP101 (NCT03116113) will be enrolled. This is a follow-up study, investigational product was administered in the previous study.

BIIB112

Untreated participants who served as controls in the antecedent study 273CH301 (NCT03496012), investigating treatment with BIIB111, will be enrolled. This is a follow-up study, participants will not be administered study medication nor receive a sham surgery.

선행 연구 273CH301(NCT03496012) 및 273CH203(NCT03507686)에서 BIIB111의 망막하 주사 치료를 받은 참가자가 등록될 예정입니다. 선행 연구 20150371(NCT02553135), Pro00028599(NCT02077361), THOR-TUE-01(NCT02671539), CHM09/01(NCT01461213) 및 REGEN2015(NCT02407678)에서 동일한 망막하 주사(rAAV2-REP1) 치료를 받은 참가자도 등록될 예정입니다. 본 연구는 후속 연구이며, 연구용 의약품은 이전 연구에서 투여되었습니다.

선행 연구 274RP101(NCT03116113)에서 BIIB112의 망막하 주사 치료를 받은 참가자가 등록될 예정입니다. 본 연구는 후속 연구이며, 연구용 의약품은 이전 연구에서 투여되었습니다.

BIIB111 치료를 조사하는 선행 연구 273CH301(NCT03496012)에서 대조군으로 참여했던 미치료 참여자가 등록될 예정입니다. 본 연구는 추적 연구이며, 참여자에게는 연구 약물이 투여되지 않으며, 가짜 수술도 시행되지 않습니다.

맥락막변성증 치료를 위한 BIIB111과 X연관성 색소성 망막염 치료를 위한 BIIB112의 장기 안전성 및 효능 추적 조사

During the Primary Treatment Period, all participants will receive open-label ravulizumab via intravenous (IV) infusion starting on Day 1. The end of the Primary Treatment Period will be triggered when the last enrolled participant completes between 26 and 50 weeks in the study (depending on the number of adjudicated On-Trial Relapse observed).

After completion of the Primary Treatment Period, all participants will have the opportunity to continue receiving ravulizumab in the Long-Term Extension Period of the study. For each participant, the Long-Term Extension Period continues for up to 3 years, or until ravulizumab is approved and/or available (in accordance with country-specific regulations), whichever occurs first.

Participants will receive a weight-based loading dose of ravulizumab via IV infusion on Day 1, followed by weight-based maintenance doses on Day 15, then once every 8 weeks until end of the Long-Term Extension Period.

Ravulizumab

Metformin, started at 500 mg per day per os and titrated up to 2000 mg during 2 years (increase of 500 mg every two weeks)

Metformin

Placebo (coated tablet similar to metformin tablet titrated following the same schedule as in the experimental arm), started at 500 mg per day per os and titrated up to 2000 mg during 2 years (increase of 500 mg every two weeks)

Control group : Placebo (coated tablet similar to metformin tablet titrated following the same schedule as in the experimental arm) started at 500 mg per day per os and titrated up to 2000 mg during 2 years (increase of 500 mg every two weeks)

anaesthetic and steroid injection into the SIJ through US guidance

sacroiliac injection with local anaesthetic and corticosteroids

sacroiliac injection

treatment arm (fruquintinib+paclitaxel) : Fruquintinib once daily, 3 weeks on/1week off combined with Paclitaxel 80mg/㎡ day1, 8, 15 of 4 weeks cycle.

treatment arm(fruquintinib +paclitaxel)- subjects will receive Fruquintinib orally, once daily for 3 wks on/ 1 wk off combined with paclitaxel 80mg/㎡ at day 1,8,15 of 4-week cycle. Patients will receive a cycles of 4 weeks of study treatment or until the occurrence of progressive disease (PD), death, unacceptable toxicity, withdrawal of consent or other conditions that meet the end of treatment criteria.

fruquintinib +paclitaxel

control arm (placebo+paclitaxel): Fruquintinib placebo once daily, 3 weeks on/1week off combined with Paclitaxel 80mg/㎡ day1, 8, 15 of 4 weeks cycle.

control arm(fruquintinib placebo + paclitaxel)- subjects will receive Fruquintinib placebo orally, once daily for 3 wks on/ 1 wk off combined with paclitaxel 80mg/㎡ at day 1,8,15 of 4-week cycle. Patients will receive a cycles of 4 weeks of study treatment or until the occurrence of progressive disease (PD), death, unacceptable toxicity, withdrawal of consent or other conditions that meet the end of treatment criteria.

fruquintinib placebo + paclitaxel

Participants with normal hepatic function (Serum bilirubin and Aspartate aminotransferase \[AST\] less than or equal to \[\<=\] Upper limit of normal \[ULN\]) will be administered with Belantamab mafodotin

Belantamab mafodotin will be administered

Belantamab mafodotin

Participants with moderate hepatic impairment (Serum bilirubin greater than \>1.5 - 3 times ULN and any AST) will be administered with Belantamab mafodotin

Participants with severe hepatic impairment (Serum bilirubin \>3 times ULN and any AST) will be administered with Belantamab mafodotin

Participants randomised to the intervention group receive Metformin XR plus standard of care for 104 weeks.

Dosage will depend on individual participant's level of tolerance to Metformin XR as well as their estimated glomerular filtration rate (eGFR). The dosage will be between 500-2000mg/day.

Metformin XR

Participants randomised to the control group receive placebo plus standard of care for 104 weeks.

Placebo is inactive tablets that is identical to the intervention Metformin tablets.

Control

Meg Hockley

Randall Faull, Prof

Terry Jennings

Scott Crawford, Dr

Rudi Koks, Study Coordinator

Adam Mullan, Dr

Ling Yap, Study Coordinator

Rob Walker, Professor

Lynette Knuth

Mahmoud Amer, Dr

Rhian Clissold, Dr

Matt Hall, Dr

Stewart Lambie, Dr

Aimun Ahmed, Dr

Aine DeBhalis, Dr

Osasuyi Iyasere, Dr

Osasuyi, Iyasere, Dr

Ayesha Irtiza-Ali, Dr

Chris Goldsmith, Dr

Matt Todd, Dr

Stephanie Bolton, Dr

Alastair Woodman, Dr

Frank McCarroll, Dr

Neal Morgan, Dr

Tom Connor, Dr

Mohsen El Kossi, Dr

Ragada El-Damanawi, Dr

Dominic De Takats, Dr

John Sayer, Dr

Yuki Heath, Dr

Mohd Furqan, Dr

Conor Byrne, Dr

Daniel Gale, Dr

Adam Rumjon, Dr

Participants received ELX 200 milligram (mg) once daily (qd)/TEZ 100 mg qd/IVA 150 mg every 12 hours (q12h) in the treatment period approximately 13 weeks.

Fixed dose combination (FDC) tablet for oral administration.

ELX/TEZ/IVA

Escalating doses of NXP900 are planned with a starting dose level of 20 mg once per day.

NXP900 is an orally administered SRC/YES1 kinase inhibitor

NXP900

Participants will receive the selected dose of NXP900

Jordi Rodon Ahnert, MD, PhD

Japanese and Chinese participants will initiate treatment with DRF 231 milligrams (mg), oral capsule, twice daily on Day 1 through Day 7, followed by DRF 462 mg, oral capsules, twice daily from Day 8 up to Week 48.

Administered as specified in the treatment arm

Diroximel fumarate

* Up to 24 weeks in open-label period
* Up to 4 weeks in randomized double-blind period

AXS-12 (reboxetine)

Up to 4 weeks in randomized double-blind period

(closed 11/10/10) Patients receive doxorubicin IV and cyclophosphamide IV on day 1 and pegfilgrastim subcutaneously (SC) on day 2 or filgrastim (G-CSF) SC on days 3-10. Treatment repeats every 14 days for 6 courses. Beginning 2 weeks after completion of doxorubicin and cyclophosphamide, patients receive paclitaxel IV over 3 hours on day 1 and pegfilgrastim SC on day 2. Treatment repeats every 14 days for 6 courses.

pegfilgrastim

AC regimen

cyclophosphamide

doxorubicin hydrochloride

paclitaxel

(closed 11/10/10) Patients receive doxorubicin IV on day 1, oral cyclophosphamide on days 1-7, and G-CSF SC on days 2-7. Treatment repeats every 7 days for 15 courses. Beginning 2 weeks after completion of cyclophosphamide, patients receive paclitaxel and pegfilgrastim as in arm I.

(closed 11/10/10) Patients receive doxorubicin, cyclophosphamide, and pegfilgrastim or G-CSF as in arm I. Beginning 2 weeks after completion of doxorubicin and cyclophosphamide, patients receive paclitaxel IV over 1 hour on day 1. Treatment repeats every 7 days for 12 courses.

(closed 11/10/10) Patients receive doxorubicin, cyclophosphamide, and G-CSF as in arm II. Beginning 2 weeks after completion of cyclophosphamide, patients receive paclitaxel as in arm III.

Patients receive doxorubicin IV and cyclophosphamide IV on day 1 and pegfilgrastim SC on day 2. Treatment repeats every 14 days for 4 courses. Patients receive doxorubicin IV and cyclophosphamide IV on day 1 and pegfilgrastim SC on day 2. Treatment repeats every 14 days for 6 courses. Beginning 2 weeks after completion of doxorubicin and cyclophosphamide, patients receive paclitaxel IV over 3 hours on day 1 and pegfilgrastim SC on day 2. Treatment repeats every 14 days for 6 courses.

Patients receive doxorubicin, cyclophosphamide, and pegfilgrastim as in arm V. Beginning 2 weeks after completion of doxorubicin and cyclophosphamide, patients receive paclitaxel IV over 1 hour on day 1. Treatment repeats every 7 days for 12 courses.

A4250(오데빅시밧)의 장기 안전성과 효과 지속성을 PFIC 아동 환자에서 평가하는 연장 연구입니다.

Capsules for oral administration (40 or 120 µg/kg) once daily for 72 weeks, or 40 µg/kg/day for the first 12 weeks followed by 120 µg/kg/day for the remaining 60 weeks"

A4250 is a small molecule and selective inhibitor of IBAT

A4250 (odevixibat)

Dexamethasone 40 mg daily x 4 days only in the first cycle and subcutaneous romiplostim weekly for up to 12 months

Romiplostim:

1. The starting dose should be 3 mcg/kg/week. It could be start during de 4 days of dexamethasone.
2. Patients will weekly receive dose increases of romiplostim in increments of 1 mcg/kg up to a maximum dose of 10 mcg/kg in an attempt to reach a target platelet count higher than 50x109/L.
3. Otherwise, if platelets are lower than 50x109/L treatment with romiplostim will go on until Day 365 since randomization.

Patients will be reviewed weekly for 8 weeks (56 days). After Week 8, patients will be reviewed every 2 weeks (14 days) for 8 additional weeks and then monthly until Week 52 (365 days) from randomization.

romiplostim plus dexamethasone

Dexamethasone 40 mg daily x 4 days for up to 3 cycles every 14 to 28 days

Patients will be reviewed weekly until the completion of dexamethasone cycles and for a minimum of 8 weeks (56 days). After that, every 2 weeks (14 days) for 8 additional weeks and then monthly until Week 52 (365 days) from randomization.

Dexamethasone

Fundación Pública Andaluza para la gestión de la Investigación en Sevilla

HER2-low, unresectable, and/or metastatic breast cancer participants previously treated with chemotherapy randomized to DS8201a

DS-8201a is a lyophilized powder reconstituted into a sterile aqueous solution (100 mg/5 mL) to be administered intravenously

Trastuzumab deruxtecan (DS-8201a)

HER2-low, unresectable, and/or metastatic breast cancer participants previously treated with chemotherapy randomized to Physician's choice from the following options:

* Capecitabine
* Eribulin
* Gemcitabine
* Paclitaxel
* Nab-paclitaxel

Administered according to label, as one option for Physician's Choice (determined before randomization)

Capecitabine

Eribulin

Gemcitabine

Paclitaxel

Nab-paclitaxel

Rituximab (375mg/m2) was given once (day1) and a Daratumumab (16mg/kg) was given four times (day8,15,22,29).

All subjects were randomly assigned to group A (active comparator) and group B (experimental). For subjects in group B (experimental), rituximab (375mg/m2) was given once (day1) and Daratumumab (16mg/kg) was given four times (day8,15,22,29).

Daratumumab（Anti-CD38 Monoclonal Antibody）

All subjects were randomly assigned to group A (active comparator) and group B (experimental). For subjects in group A (active comparator) , rituximab (375mg/m2) was given once. For subjects in group B (experimental), rituximab (375mg/m2) was given once (day1) and anti-CD38 monoclonal antibody (16mg/kg) was given four times (day8,15,22,29).

Rituximab

Institute of Hematology & Blood Diseases Hospital, China

Lei Zhang, MD

Yunfei Chen, MD

FOLFIRINOX: Oxaliplatin, Leucovorin and 5-Fluorouracl, Irinotecan,

Alternating FOLFOX and FOLFIRI administered via intravenous infusion. FOLFOX: Oxaliplatin 85 mg/m2 on Day 1; Leucovorin 400 mg/m2 on day 1; 5-FU: 400 mg/m2 IV push on Day 1 followed by 2400 mg/m2 continuous infusion over 46-48 hours. FOLFIRI: Irinotecan 180 mg/m2 on day 1, leucovorin 400 mg/m2, 5-FU: 400 mg/m2 IV push on Day 1 followed by 2400 mg/m2 continuous infusion over 46-48 hours

운동

1형 및 2형 근긴장성 이영양증 환자에서 1일 1회 멕실레틴 PR의 효능 및 안전성

Orkambi®의 임상 효능을 예측하기 위한 호흡기 상피 기능 평가의 검증.

맥락막변성증 치료를 위한 BIIB111과 X연관성 색소성 망막염 치료를 위한 BIIB112의 장기 안전성 및 효능 추적 조사

라불리주맙

메트포르민, 위약

천골장골 주사

프루퀸티닙 + 파클리탁셀, 프루퀸티닙 위약 + 파클리탁셀

벨란타맙 마포도틴

메트포르민 XR, 대조군

ELX/TEZ/IVA, 부가가치세

NXP900

디록시멜 푸마르산염

AXS-12(레복세틴), 위약

페그필그라스팀, AC 요법, 사이클로포스파마이드, 독소루비신 염산염, 파클리탁셀

A4250(오데빅시밧)

로미플로스팀 플러스 덱사메타손, 덱사메타손

트라스투주맙 데룩스테칸(DS-8201a), 카페시타빈, 에리불린, 젬시타빈, 파클리탁셀, 나브-파클리탁셀

다라투무맙(항-CD38 단일클론 항체), 리툭시맙

폴피리녹스