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NCT ID: NCT05321524 Terminated - Biliary Atresia Clinical Trials

Obeticholic Acid in Pediatric Subjects With Biliary Atresia

CARE
Start date: July 1, 2015
Phase: Phase 2
Study type: Interventional

This is a Phase 2, multicenter, open-label, single dose and multi-dose, dose-finding study with an optional open-label extension (OLE) to assess the safety, tolerability, and pharmacokinetics of obeticholic acid (OCA) in pediatric subjects with biliary atresia with successful hepatoportoenterostomy (HPE, also known as a Kasai portoenterosomy). The OLE will continue to evaluate safety, tolerability, pharmacodynamics, and efficacy of OCA. In addition, a change in vitamin A and D levels, and where possible the degree of change in liver stiffness, will be assessed during the OLE.

NCT ID: NCT05307679 Terminated - Dup15q Syndrome Clinical Trials

A Study to Evaluate the Safety and Efficacy of Basmisanil Treatment in Children Aged 2-14 Years With Dup15q Syndrome

Start date: December 16, 2022
Phase: Phase 2
Study type: Interventional

This study consists of two parts. Part 1 will evaluate the safety, efficacy, and pharmacodynamics of 52-weeks of basmisanil treatment in children and adolescents (aged 2-14 years) with Dup15q syndrome. Part 1 will test the hypothesis that negative allosteric modulation of a GABAA receptor subtype can address excessive receptor function and positively impact core neurodevelopmental disease feature in individuals with Dup15q syndrome. Part 2 is an optional 2-year open-label extension to evaluate long-term safety, tolerability, and to provide supportive evidence of benefit of continued treatment with basmisanil in selected efficacy outcomes.

NCT ID: NCT05305040 Terminated - Clinical trials for Cytomegalovirus Infections

Study of Posoleucel (ALVR105,Viralym-M) for Multi-Virus Prevention in Patients Post-Allogeneic Hematopoietic Cell Transplant

Prevent
Start date: March 24, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

This is a Phase 3 study to evaluate posoleucel (ALVR105, Viralym-M); an allogeneic, off-the-shelf multi-virus specific T cell therapy that targets six viral pathogens: BK virus, cytomegalovirus, adenovirus, Epstein-Barr virus, human herpesvirus 6 and JC virus.

NCT ID: NCT05293236 Terminated - COVID-19 Clinical Trials

ApTOLL for the Treatment of COVID-19

Start date: January 14, 2022
Phase: Phase 1
Study type: Interventional

There is a clear and urgent medical need of developing new medicinal products for COVID-19 since there are poor pharmacological tools to block the progression of patients to cytokine storm syndrome (CSS). To this aim, this Phase Ib clinical study (APTACOVID) pretends to determine whether ApTOLL, in combination with the standard of care, is safe and shows any biological effect) in those patients infected with SARS-CoV-2 who are not developed CSS yet.

NCT ID: NCT05289492 Terminated - Multiple Myeloma Clinical Trials

Study of Novel Regimens in Relapsed or Refractory Multiple Myeloma

Start date: May 1, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

This is a multicenter, open-label, phase I/II clinical study, to assess the safety, tolerability, antitumor activity, pharmacokinetics and pharmacodynamics of EOS884448 (also known as EOS-448 or GSK4428859A), alone or in combination with iberdomide with and without dexamethasone in participants with relapsed/refractory multiple myeloma (RRMM).

NCT ID: NCT05282121 Terminated - Liver Diseases Clinical Trials

A Study to Test Whether BI 685509 Alone or in Combination With Empagliflozin Helps People With Liver Cirrhosis Caused by Viral Hepatitis or Non-alcoholic Steatohepatitis (NASH) Who Have High Blood Pressure in the Portal Vein (Main Vessel Going to the Liver)

Start date: May 26, 2022
Phase: Phase 2
Study type: Interventional

This study is open to adults with liver cirrhosis caused by hepatitis B, hepatitis C or nonalcoholic steatohepatitis (NASH). People can join this study if they have high blood pressure in the portal vein (main vessel going to the liver). The purpose of this study is to find out whether a medicine called Avenciguat (BI 685509) taken alone or in combination with a medicine called empagliflozin helps people with this condition. Participants take Avenciguat (BI 685509) as tablets twice a day for 8 weeks. Half of the participants with NASH who also have type 2 diabetes take empagliflozin as tablets once a day in addition to Avenciguat (BI 685509). Participants are in the study for about 3 months. During this time, they visit the study site about 10 times. At 2 of the visits, the doctors check the pressure in a liver vein to see whether the treatment works. This is done with a catheter (a long thin tube) and gives information about the pressure in the portal vein. The doctors also regularly check participants' health and take note of any unwanted effects.

NCT ID: NCT05267574 Terminated - Clinical trials for Primary Mitochondrial Myopathy

An Open Label, Long Term Safety Study of REN001 in Primary Mitochondrial Myopathy Patients (Stride Ahead)

Start date: February 1, 2022
Phase: Phase 2/Phase 3
Study type: Interventional

This study is designed to evaluate the long-term safety and tolerability of REN001 administered once daily to subjects with PMM due to mitochondrial DNA mutations (mtDNA-PMM) or nuclear DNA mutations (nDNA-PMM). Subjects with mtDNA mutations will have previously completed Study REN001-201 or participated in Study REN001-101. Subjects with nDNA mutations who enroll in this study will be REN001- naïve.

NCT ID: NCT05256134 Terminated - Alzheimers Disease Clinical Trials

A Study to Evaluate the Efficacy and Safety of Gantenerumab in Participants at Risk for or at the Earliest Stages of Alzheimer's Disease (AD)

SKYLINE
Start date: April 19, 2022
Phase: Phase 3
Study type: Interventional

A study to evaluate the efficacy and safety of gantenerumab in amyloid-positive, cognitively unimpaired participants at risk for or at the earliest stages of AD. The planned number of participants for this study is approximately 1200 participants randomized in a 1:1 ratio to receive either gantenerumab or placebo (600 participants randomized to gantenerumab and 600 participants randomized to placebo).

NCT ID: NCT05247307 Terminated - COVID-19 Clinical Trials

Efficacy of the Infusion of Donor Plasma in COVID-19 Infection

Start date: March 31, 2020
Phase: Phase 3
Study type: Interventional

Objectives Primary objectives To assess the superiority or not of the infusion of anti-COVID immune plasma infusion versus a placebo (non-immune plasma) to patients over 65 years of age recently infected with the SARS-CoV-2 coronavirus, to prevent progression to severe forms of the disease with hospital admission. Secondary objectives To assess if the intervention improves the clinical evolution of the target patient. - Improvement of the disease. - Disease deterioration Methodology Design Prospective, randomized (1:1; experimental group: control group), double-blind study, in which the doctors who prescribe the treatments and those who assess the results do not know the treatment received by the patient. Methods. Patients 65 years of age or older will be included, diagnosed with SARS-CoV-2 coronavirus infection, in which no more than 7 days have elapsed since the onset of symptoms or diagnosis to plasma infusion.All patients included in the study will receive the same standard treatment that is deemed appropriate at any time, understanding as standard treatment that established at any time by the guidelines established.Infusion of 300 cc of convalescent donor plasma from COVID 19, administered within more than 168 hours from the onset of symptoms. For the study to be blind, the infusion of non-convalescent donor plasma, obtained before the start of the epidemic, is required to guarantee the absence of anti-COVID antibodies in the plasma of the control group. Analysis of data: Initially, the homogenization of the data referring to the two groups of studies will be evaluated. Classical statistical tests will be applied, such as Wilcoxon's non-parametric tests for the comparison of means of continuous variables, as well as the Chi-square test (or Fisher's exact test) to evaluate the association between categorical variables. The result variables are: 1. Hospital admission 2. Clinical course 3. Mortality 4. Complications and/or adverse effects of plasma.

NCT ID: NCT05244070 Terminated - Lymphoma Clinical Trials

A Study to Evaluate the Safety and Tolerability of BMS-986403 in Participants With Relapsed and/or Refractory Chronic Lymphocytic Leukemia or Small Lymphocytic Lymphoma

Start date: September 14, 2022
Phase: Phase 1
Study type: Interventional

The purpose of this study is to evaluate the safety and preliminary efficacy of BMS-986403 in participants with relapsed and/or refractory chronic lymphocytic leukemia (R/R CLL) or small lymphocytic lymphoma (SLL).