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NCT ID: NCT04562389 Recruiting - Myelofibrosis Clinical Trials

Study of Selinexor in Combination With Ruxolitinib in Myelofibrosis

SENTRY
Start date: March 11, 2021
Phase: Phase 3
Study type: Interventional

This is a global, multicenter Phase 1/3 study to evaluate the efficacy and safety of selinexor plus ruxolitinib in JAK inhibitor (JAKi) treatment-naïve myelofibrosis (MF) participants. The study will be conducted in two phases: Phase 1 (open-label) and Phase 3 (double-blind). Phase 1 (enrollment completed) was an open-label evaluation of the safety and recommended dose (RD) of selinexor in combination with ruxolitinib and included a dose escalation using a standard 3+3 design (Phase 1a) and a dose expansion part (Phase 1b). In Phase 3, JAKi treatment-naïve MF participants are enrolled in 2:1 ratio to receive the combination therapy of selinexor + ruxolitinib or the combination of placebo + ruxolitinib.

NCT ID: NCT04561804 Completed - Weight Loss Clinical Trials

NAFLD and Liver Fibrosis in Obese Adolescents

NAFLD
Start date: December 1, 2018
Phase:
Study type: Observational [Patient Registry]

Nonalcoholic fatty liver disease (NAFLD) is a chronic liver disease resulting from excessive fat accumulation in the liver. Due to its close association with obesity, it has become the most common liver disease in children in the United States. NAFLD can result in progressive fibrosis and lead to end-stage liver disease. Best practices in management of pediatric NAFLD are not clearly defined. Our aim is to clarify the natural history of NAFLD in obese children after weight loss surgery compare to lifestyle intervention. Our secondary aim is to investigate the added value of elastography for the screening and diagnosis of NASH with fibrosis.

NCT ID: NCT04561518 Recruiting - ATTR Amyloidosis Clinical Trials

ConTTRibute: A Global Observational Study of Patients With Transthyretin (TTR)-Mediated Amyloidosis (ATTR Amyloidosis)

ConTTRibute
Start date: November 23, 2020
Phase:
Study type: Observational

The purpose of this study is to: - Describe epidemiological and clinical characteristics, natural history and real-world clinical management of ATTR amyloidosis patients - Characterize the safety and effectiveness of patisiran and vutrisiran as part of routine clinical practice in the real-world clinical setting - Describe disease emergence/progression in pre-symptomatic carriers of a known disease-causing transthyretin (TTR) variant

NCT ID: NCT04558268 Not yet recruiting - Clinical trials for Recurrent Miscarriage

Low Dose Prednisone Therapy in Women With Recurrent Pregnancy Loss

Start date: December 2020
Phase: Phase 2/Phase 3
Study type: Interventional

For many years there is a lack of large randomized controlled trials that study the effect of low dose prednisone in women with RPL and thus the evidence of a probable efficacy of prednisone in RPL women remains limited and unclear. As the ESHRE recommended in 2018 (2) we aim to assess the effect of such treatment in a large trial that includes unexplained and abnormal autoimmune profile RPL patients. we also aim to assess the side effects of the treatment in RPL pregnant women.

NCT ID: NCT04557735 Recruiting - Clinical trials for Thrombotic Microangiopathy

Study of Ravulizumab in Pediatric Participants With HSCT-TMA

Start date: December 7, 2020
Phase: Phase 3
Study type: Interventional

This study will evaluate the safety, efficacy, pharmacokinetics, and pharmacodynamics of ravulizumab administered by intravenous infusion to pediatric participants, from 1 month to < 18 years of age, with HSCT-TMA. The treatment period is 26 weeks, followed by a 26-week off-treatment follow-up period.

NCT ID: NCT04557462 Recruiting - Clinical trials for Primary IgA Nephropathy

A Rollover Extension Program (REP) to Evaluate the Long-term Safety and Tolerability of Open Label Iptacopan/LNP023 in Participants With Primary IgA Nephropathy

Start date: September 20, 2021
Phase: Phase 3
Study type: Interventional

The purpose of this study is to evaluate the long-term safety and tolerability, of open label iptacopan in primary IgA nephropathy participants who have completed either the CLNP023X2203 or CLNP023A2301 clinical trials. The open-label design of the current study is appropriate to provide study participants the opportunity to receive treatment with iptacopan until marketing authorizations are received and the drug product becomes commercially available while enabling collection of long-term safety and tolerability data for the investigational drug. Furthermore efficacy assessments conducted every 6 months will afford the opportunity to evaluate the clinical effects of iptacopan on long-term disease progression.

NCT ID: NCT04555421 Not yet recruiting - Clinical trials for Overweight and Obesity

Using the Lumen Device for Prediabetes Prevention

Start date: October 2020
Phase: N/A
Study type: Interventional

To examine the effect of using Lumen on metabolic parameters and anthropometric variables. This will be done from baseline to the end of a 12 weeks intervention in adults with prediabetes..

NCT ID: NCT04552899 Terminated - Clinical trials for Idiopathic Pulmonary Fibrosis

A Study to Evaluate the Efficacy and Safety of Recombinant Human Pentraxin-2 (rhPTX-2; PRM-151) in Participants With Idiopathic Pulmonary Fibrosis

STARSCAPE
Start date: March 19, 2021
Phase: Phase 3
Study type: Interventional

This phase III study will evaluate the efficacy, safety and pharmacokinetics (PK) of recombinant human pentraxin-2 (rhPTX-2; PRM-151) zinpentraxin alfa, compared with placebo in participants with idiopathic pulmonary fibrosis (IPF).

NCT ID: NCT04551066 Active, not recruiting - Myelofibrosis Clinical Trials

To Evaluate the Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis (LIMBER-313)

Start date: May 27, 2021
Phase: Phase 3
Study type: Interventional

The purpose of the study is to compare the efficacy of parsaclisib when combined with ruxolitinb versus placebo combined with ruxolitinib in participants with myelofibrosis.

NCT ID: NCT04551053 Active, not recruiting - Myelofibrosis Clinical Trials

To Evaluate Efficacy and Safety of Parsaclisib and Ruxolitinib in Participants With Myelofibrosis Who Have Suboptimal Response to Ruxolitinib (LIMBER-304)

Start date: May 26, 2021
Phase: Phase 3
Study type: Interventional

The purpose of the study is to compare the efficacy and safety of parsaclisib when combined with ruxolitinb versus placebo combined with ruxolitinib in participants with myelofibrosis who have suboptimal response while receiving ruxolitinib monotherapy.