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NCT ID: NCT04153149 Active, not recruiting - Clinical trials for Transthyretin Amyloidosis (ATTR) With Cardiomyopathy

HELIOS-B: A Study to Evaluate Vutrisiran in Patients With Transthyretin Amyloidosis With Cardiomyopathy

Start date: November 26, 2019
Phase: Phase 3
Study type: Interventional

This study will evaluate the efficacy and safety of vutrisiran 25 mg administered subcutaneously (SC) once every 3 months (q3M) compared to placebo in patients with ATTR amyloidosis with cardiomyopathy.

NCT ID: NCT04152200 Active, not recruiting - Clinical trials for Primary Hyperoxaluria

A Study to Evaluate Lumasiran in Patients With Advanced Primary Hyperoxaluria Type 1

ILLUMINATE-C
Start date: January 21, 2020
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the efficacy, safety, pharmacokinetics (PK), and pharmacodynamics (PD) of lumasiran in patients with Advanced Primary Hyperoxaluria Type 1 (PH1).

NCT ID: NCT04147182 Active, not recruiting - TCC Clinical Trials

Perioperative Intravesical Instilation of Hypertonic Saline Following Bladder Tumor Resection

Start date: December 2, 2018
Phase: Phase 1
Study type: Interventional

Hypertonic saline at the concentration of 3% (hypersel) has been injected for years into echinococcal cysts to kill live scolices, and is also known to kill suspended cells by means of a steep osmotic gradient and the investigators postulate that perioperative intravesical instillation of hypersel for patients with low grade bladder cancer is safe and may be active against shedded and suspended tumor cells and thus may decrease recurrence risk.

NCT ID: NCT04136171 Active, not recruiting - Clinical trials for Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

CARDIO-TTRansform: A Study to Evaluate the Efficacy and Safety of Eplontersen (Formerly Known as ION-682884, IONIS-TTR-LRx and AKCEA-TTR-LRx) in Participants With Transthyretin-Mediated Amyloid Cardiomyopathy (ATTR CM)

Start date: March 13, 2020
Phase: Phase 3
Study type: Interventional

To evaluate the efficacy of eplontersen compared to placebo in participants with ATTR-CM receiving available standard of care (SoC). For more information, please visit https://www.cardio-ttransform.com.

NCT ID: NCT04133909 Active, not recruiting - Clinical trials for Pulmonary Disease, Chronic Obstructive

Mepolizumab as Add-on Treatment IN Participants With COPD Characterized by Frequent Exacerbations and Eosinophil Level

MATINEE
Start date: October 30, 2019
Phase: Phase 3
Study type: Interventional

This is a multi-center, randomized, placebo-controlled, double-blind, parallel group study designed to confirm the benefits of mepolizumab treatment on moderate or severe exacerbations in chronic obstructive pulmonary disease (COPD) participants given as an add on to their optimized maintenance COPD therapy. The maximum duration of participant participation is approximately 109 weeks, consisting of 2 screening visits (up to 3 weeks), a run-in period (up to 2 weeks), and an intervention period of at least 52 weeks and up to 104 weeks. 800 participants will be randomized in a 1:1 ratio to receive mepolizumab 100 milligrams (mg) or placebo every 4 weeks for at least 13 doses (52 weeks treatment period) up to a maximum of 26 doses (104 weeks treatment period). The number of randomized participants may increase up to approximately 1400.

NCT ID: NCT04133636 Active, not recruiting - Multiple Myeloma Clinical Trials

A Study of JNJ-68284528, a Chimeric Antigen Receptor T Cell (CAR-T) Therapy Directed Against B-cell Maturation Antigen (BCMA) in Participants With Multiple Myeloma

CARTITUDE-2
Start date: November 7, 2019
Phase: Phase 2
Study type: Interventional

The purpose of this study is to evaluate the overall minimal residual disease (MRD) negative rate of participants who receive JNJ-68284528.

NCT ID: NCT04130191 Active, not recruiting - Clinical trials for Hereditary Angioedema (HAE)

A Study of Lanadelumab in Persons With Hereditary Angioedema (HAE) Type I or II

ENABLE
Start date: December 11, 2019
Phase:
Study type: Observational

The main aim of this study is to compare the number of HAE attacks occuring in persons using lanadelumab with the number of HAE attacks before lanadelumab treatment was started. Data from participants who start the study after 1 March 2021, will be collected for 24 months; data from all other participants (who started the study before 1 March 2021) will be collected for 36 months. Participants will report information in a smartphone application at study start and for the next 3 months and then every 6 months until the study ends; data will also be collected by the study doctor during routine clinic visits

NCT ID: NCT04129502 Active, not recruiting - Clinical trials for Advanced/Metastatic Non-Small Cell Lung Cancer (NSCLC)

TAK-788 as First-Line Treatment Versus Platinum-Based Chemotherapy for Non-Small Cell Lung Cancer (NSCLC) With EGFR Exon 20 Insertion Mutations

Start date: January 10, 2020
Phase: Phase 3
Study type: Interventional

The purpose of this study is to compare the effectiveness of TAK-788 as first-line treatment with that of platinum-based chemotherapy in participants with locally advanced or metastatic non-small cell lung cancer (NSCLC) whose tumors has epidermal growth factor receptor (EGFR) exon 20 insertion mutations. Participants will be randomly assigned to one of the two treatment groups- TAK-788 group or Platinum-based chemotherapy group. Participants will receive TAK-788 orally and pemetrexed/cisplatin or pemetrexed/carboplatin via vein until the participants experience worsening disease (PD) as assessed by blinded independent review committee (IRC), intolerable harmful effects or another discontinuation criteria.

NCT ID: NCT04127006 Active, not recruiting - Clinical trials for Retinitis Pigmentosa

Rate of Progression in EYS Related Retinal Degeneration

Pro-EYS
Start date: February 25, 2020
Phase:
Study type: Observational

The overall goal of this project funded by the Foundation Fighting Blindness is to characterize the natural history of disease progression in patients with EYS mutations in order to accelerate the development of outcome measures for clinical trials.

NCT ID: NCT04123418 Active, not recruiting - Clinical trials for Multiple Myeloma (MM)

A Study of WVT078 in Patients With Multiple Myeloma (MM)

Start date: December 5, 2019
Phase: Phase 1
Study type: Interventional

The design of a phase I, open-label, dose finding study was chosen in order to establish a safe and tolerated dose of single agent WVT078 alone and in combination with WHG626 in patients relapses and/or refractory Multiple Myeloma (MM)