Participants initially received secukinumab 75mg subcutaneously once every four weeks in the extension study. If the signs and symptoms of the participants were not adequately controlled with the 75mg dose, as determined by the investigator, the dose could be escalated to 150mg subcutaneously. For patients weighing 50kg and over, the dose could further be escalated to 300mg subcutaneously every four weeks. The dose escalation from secukinumab 75mg subcutaneously to 300mg subcutaneously was to be implemented in two steps, with the first step being an increase to 150mg subcutaneously, followed by another escalation to 300mg subcutaneously, based on the judgement of the investigator.

Secukinumab solution for subcutaneous injections was provided in PFS. Initially, participants continued to receive secukinumab at either 75 mg (in 0.5mL) or 150 mg (in 1mL) every 4 weeks, consistent with their dosage at the Week 100 visit of the core study. The dose could be escalated from 75 mg to 150 mg for patients whose signs and symptoms were not fully controlled, as judged by the investigator, with the current 75 mg dose. Furthermore, the dose could also be escalated to 300 mg every 4 weeks for patients weighing 50kg and over who were currently on the 150 mg dose and whose signs and symptoms were not well-controlled, as judged by the investigator. The dose escalation from secukinumab 75 mg to 300 mg was to be implemented in two steps (first 150 mg and then 300 mg based on the investigator's judgment). At each study treatment time point, one or two subcutaneous injections in the form of PFS were administered.

Secukinumab

Participants initially received secukinumab 150mg subcutaneously once every four weeks in the extension study. If the signs and symptoms of the participants were not adequately controlled with the 150mg dose, as determined by the investigator, the dose could be escalated to 300mg subcutaneously.

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

Inclisiran sodium 300 mg subcutaneous (sc) injection (given at Days 1, 90 and 270)

Inclisiran sodium 300 mg (equivalent to 284 mg inclisiran) in 1.5 mL solution for subcutaneous injection

Inclisiran

Placebo sc injection (given at Day 1, 90 and 270)

Sterile normal saline (0.9% sodium chloride in water for injection) for subcutaneous injection

Placebo

Inclisiran sodium 300 mg sc injection (given at Days 450 and 630). In addition, participants assigned to placebo in Part 1 received inclisiran sodium 300 mg sc injection on Day 360, while participants assigned to inclisiran in Part 1 received placebo sc injection on Day 360.

Pharmaceutically manufactured. Cannabidiol is formulated as a clear gel for transdermal delivery.

Pharmaceutically manufactured cannabidiol formulated as a clear gel that can be applied to the skin (transdermal delivery)

ZYN002 - transdermal gel

Placebo is formulated as a clear gel for transdermal delivery.

Placebo formulated as a clear gel that can be applied to the skin (transdermal delivery)

Other Names:

Placebo Comparator Matching Placebo

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

6 subjects each will receive RVP-001 at a dose of 2 mg Mn/kg

RVP-001

6 subjects each will receive RVP-001 at a dose of 7 mg Mn/kg

6 subjects each will receive RVP-001 at a dose of 12 mg Mn/kg

각 피험자 6명에게 2mg Mn/kg의 용량으로 RVP-001을 투여합니다.

각 피험자 6명에게 RVP-001을 7mg Mn/kg의 용량으로 투여합니다.

각 피험자 6명에게 12mg Mn/kg의 용량으로 RVP-001을 투여합니다.

Kristin DeFrancesco

Amit Mahajan, MD

Sophia D'Alessandro

John Chen, MD, PhD

Nick Cloney

Matthew DeSalvo, MD

Jenn Cornelius-Green

Edvin Isufi, MD

Helen Zuleta

Shruti Raja, M.D.

망간 기반 MRI 조영제 RVP-001의 2상 용량 증가 안전성 및 효능 연구

어린이 중증 근무력증 환자에게 라불리주맙 주사 치료의 몸 반응과 약물 동작을 살펴보는 연구입니다.

All participants will receive a weight-based loading dose of ravulizumab IV on Day 1, followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for participants weighing ≥ 20 kg, or once every 4 weeks (q4w) for participants weighing \< 20 kg, for a total of 122 weeks of treatment.

Ravulizumab will be administered by intravenous (IV) infusion.

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening.

Fixed-dose combination granules for oral administration.

Participants will receive ELX/TEZ/IVA in the morning and IVA in the evening with the dose(s) to be based on the outcome of Part A.

7.5mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water

CNM-Au8 is a dark red/purple-colored liquid formulation consisting of a stable suspension of faceted clean surfaced elemental gold nanocrystals in buffered deionized water with a concentration of up to 0.5 mg/mL of gold. The formulation is buffered by sodium bicarbonate present at a concentration of 0.546 mg/mL. There are no other excipients. The drug product is formulated to be taken orally and will be provided in single dose HDPE containers. The study doses vary by the concentration of gold nanocrystals per milliliter in a volume of 60 mL.

gold nanocrystals

15mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water

30mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water

60mg suspension of clean-surfaced, faceted, gold nanocrystals in 120ml of sodium bicarbonate buffered water

성인 발작성 야간 혈색소뇨증 환자를 대상으로 라불리주맙의 효과를 평가하는 시험입니다.

During the Primary Treatment Period, participants will receive a weight-based loading dose of ravulizumab on Day 1 followed by weight-based maintenance dose of ravulizumab on Day 15 and once every 8 weeks (q8w) thereafter for a total of 26 weeks. On Day 183, all participants will enter a 32-week Extension Treatment Period and receive ravulizumab. Beginning on Day 183, participants will receive a maintenance dose of ravulizumab q8w for an additional 32 weeks.

Participants will be randomized to receive ARO-ANG3 SC on Day 1 and Day 84 during the initial 36 Weeks of the study and on Day1 and Months 3, 6, 9, 12, 15, 18, and 21 of the extension period

ARO-ANG 3 Injection

Obese pts with hypertension, starting treatment with eplerenone 25mg twice daily (BD). ABPM wil be scheduled at wks 8, 16 and 24.

At wk 8 if mean ABPM \< 130/80mm Hg will continue to monotherapy with eplerenone. If not controlled (mean ABPM \> 130/80mm Hg) at wk8, amlodipine 5mg OD will be added.

At wk 16 if mean ABPM \< 130/80mm Hg will continue to monotherapy with eplerenone, or dual therapy with eplerenone and amlodipine. If not controlled (mean ABPM \> 130/80mm Hg) at wk16, indapamide 1,5mg OD will be added. All patients will be followed up for 24 weeks.

At randomization, pts meeting inclusion/exclusion criteria will be randomized (1:1) to either eplerenone (E) 25mg bd or valsartan (V) 160mg od for 8 wks. At 8, 16 and 24 wks, pts at both arms will be evaluated with ABPM primary, as well as home and office BP measurements. At wk 8, pts with controlled hypertension (mean ABPM \<130/80mmHg), will continue in monotherapy with eplerenone or valsartan and pts with uncontrolled hypertension (mean ABPM ≥130/80mmHg) will continue with the addition of amlodipine (C) 10mg od. At wk 16, pts achieving BP control will continue in either monotherapy (E), (V) or dual therapy (E+C), (V+C). However, in pts not achieving ABPM target, a third drug, will be added \[indapamide (D) 1.25 mg od\]. All groups at both arms will be evaluated at 24 wks by ABPM.

Eplerenone (-based therapy) arm

Obese pts with hypertension, starting treatment with irbesartan 150mg once daily (OD). ABPM wil be scheduled at wks 8, 16 and 24.

At wk 8 if mean ABPM \< 130/80mm Hg will continue to monotherapy with irbesartan. If not controlled (mean ABPM \> 130/80mm Hg) at wk8, amlodipine 5mg OD will be added.

At wk 16 if mean ABPM \< 130/80mm Hg will continue to monotherapy with irbesartan, or dual therapy with irbesartan and amlodipine. If not controlled (mean ABPM \> 130/80mm Hg) at wk16, indapamide 1,5mg OD will be added. All patients will be followed up for 24 weeks.

Valsartan (-based therapy) arm

Open label, single arm treatment with bbT369

bbT369 is a genetically modified autologous T cell immunotherapy product consisting of T cells that are transduced with a single lentiviral vector (LVV) to express anti-CD79a and anti-CD20 chimeric antigen receptors (CARs) and transfected with an mRNA encoding the CBLB-targeting megaTAL enzyme to edit the CBLB gene, suspended in a cryopreservative solution.

bbT369

All participants will receive a weight-based loading dose of ravulizumab on Day 1, followed by weight-based maintenance treatment with ravulizumab on Day 15 and either once every 8 weeks (q8w) thereafter or once every 4 weeks (q4w) depending on weight.

Participants will receive Ravulizumab via intravenous (IV) infusion.

study eye treated with 100 μg (94.3 μg free acid) of OLX10212

Clear colorless solution dissolved in 1X PBS and injected intravitreally

OLX10212 is a cell penetrating asymmetric small interference RNA (cp-asiRNA)

study eye treated with 250 μg (235.8 μg free acid) of OLX10212

study eye treated with 500 μg (471.5 μg free acid) of OLX10212

study eye treated with 750 μg (707.3 μg free acid) of OLX10212

study eye treated with 950 μg (895.9 μg free acid) of OLX10212

study eye treated with a total of 3 intravitreal injections of 750 μg (707.3 μg free acid) of OLX10212 each 28 days apart

study eye treated with a total of 3 intravitreal injections of 950 μg (895.9 μg free acid) of OLX10212 each 28 days apart

DZD6008

Shum

The monotherapy group will be treated with the following treatments:

A) 1 to 2 weeks of Amlodipine 5mg followed by 6 to 7 weeks of Amlodipine 10mg B) 1 to 2 weeks of Lisinopril 10mg followed by 6 to 7 weeks of Lisinopril 20mg C) Approximately 8 weeks of 25mg Chlortalidone

Participants will be randomly allocated to one of six possible sequences of treatments of the three-treatment-three period Williams design: ABC, ACB, BAC, BCA, CAB, and CBA.

Amlodipine 5mg and Amlodipine 10mg will be one of the treatments in which patients will receive on the monotherapy arm and on the dual therapy arm.

Amlodipine

Lisinopril 10mg and Lisinopril 20mg will be one of the treatments in which patients will receive on the monotherapy arm.

Lisinopril 20mg will be one of the treatments in which patients will receive on the dual therapy arm.

Lisinopril

Chlortalidone 25mg will be one of the treatments in which patients will receive on the dual therapy arm and monotherapy arm.

Chlortalidone

The dual-therapy group will be treated with the following treatments:

A) Approximately 8 weeks of Amlodipine 5mg and Lisinopril 20mg B) Approximately 8 weeks of Amlodipine 5mg and Chlortalidone 25mg C) Approximately 8 weeks of Lisinopril 20mg and Chlortalidone 25mg D) Approximately 8 weeks of Amiloride 10mg and Chlortalidone 25mg

Participants will be randomly allocated to one of four possible sequences of treatments of the four-treatment four-period Williams design: ABDC, BCAD, CDBA, and DACB.

Amiloride 10mg will be one of the treatments in which patients will receive on the dual therapy arm.

Amiloride

Cambridge University Hospitals NHS Foundation Trust

This study will employ the traditional "3+3" dose escalation design. Ultimately, two combination dosing cohorts will be selected to enter Part 2, the dose expansion phase, based on a comprehensive evaluation of the safety and pharmacokinetic (PK) data obtained by the investigators and the sponsor.

ZG006 will be administered as an intravenous (IV) infusion.

ZG006

ZG005 will be administered as an intravenous (IV) infusion.

ZG005

The safety and pharmacokinetic (PK) data obtained in Part 1 will be used to determine two expansion phase doses of the ZG006 and ZG005 combination. The dose expansion study will further evaluate the efficacy of the two selected combination dosing cohorts in patients with advanced small cell lung cancer and neuroendocrine carcinoma.

Participants will receive the RP2D of ZG006 in combination with ZG005 identified in Phase Ib of the study.

All eligible participants will be enrolled to receive \[18F\]CTT1057 imaging agent on Day 1 and have PET/CT scan

[18F]CTT1057

스테로이드 치료에 실패한 성인 ITP 환자에게 이아날루맙과 엘트롬보파그를 추가 투여해 치료 실패 시간을 늘리는 연구입니다.

Participants will receive eltrombopag and ianalumab lower dose

Concentrate for solution for infusion for intravenous use

Ianalumab

Eltrombopag

Participants will receive eltrombopag and ianalumab higher dose

Participants will receive eltrombopag and placebo

Concentrate for solution for infusion for intravenous use.

세쿠키누맙

라불리주맙

인클리시란, 위약

ZYN002 - 경피 젤, 위약

ELX/TEZ/IVA, 부가가치세

망간 기반 MRI 조영제 RVP-001의 2상 용량 증가 안전성 및 효능 연구

라불리주맙

ELX/TEZ/IVA, 부가가치세

금 나노결정

라불리주맙

아로앙 3 주사

에플레레논(-기반 치료)군, 발사르탄(-기반 치료)군

bbT369

라불리주맙

OLX10212는 세포 투과성 비대칭 소형 간섭 RNA(cp-asiRNA)입니다.

DZD6008

암로디핀, 리시노프릴, 아밀로라이드, 클로르탈리돈

ZG006, ZG005

[18F]CTT1057

이아날루맙, 엘트롬보파그, 위약