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NCT ID: NCT06289998 Recruiting - Clinical trials for Autosomal Dominant Polycystic Kidney Disease (ADPKD)

Study of Tamibarotene in Patients With ADPKD

Start date: December 22, 2023
Phase: Phase 2
Study type: Interventional

Clinical trial of tamibarotene in patients with Autosomal Dominant Polycystic Kidney Disease

NCT ID: NCT06285097 Recruiting - Melanoma Clinical Trials

A Study of PF-07820435 as a Single Agent and in Combination in Participants With Advanced Solid Tumors

Start date: February 8, 2024
Phase: Phase 1
Study type: Interventional

This study aims to evaluate the safety, and early signals of anti-tumor activity of PF-07820435 when administered alone (Part 1A) or in combination with sasanlimab (Part 1B; Part 2) in patients with selected advanced or metastatic solid tumors. Part 1 will be dose-finding and Part 2 of the study will further evaluate PF-07820435 at the recommended dose for combination expansion in patients with selected advanced solid tumors.

NCT ID: NCT06283966 Recruiting - Clinical trials for COPD (Chronic Obstructive Pulmonary Disease)

A Study Evaluating the Efficacy of Budesonide, Glycopyrronium and Formoterol Fumarate Metered Dosed Inhaler on Cardiopulmonary Outcomes in Chronic Obstructive Pulmonary Disease

THARROS
Start date: February 21, 2024
Phase: Phase 3
Study type: Interventional

This study will evaluate the effect of triple ICS/LAMA/LABA therapy with BGF MDI 320/14.4/9.6 μg on cardiopulmonary outcomes relative to LAMA/LABA therapy with GFF MDI 14.4/9.6 μg in a population with COPD and elevated cardiopulmonary risk.

NCT ID: NCT06280391 Recruiting - Bronchiectasis Clinical Trials

A Proof-of-Concept Study to Assess the Efficacy, Safety and Tolerability of Itepekimab (Anti-IL-33 mAb) in Participants With Non-cystic Fibrosis Bronchiectasis

Start date: February 20, 2024
Phase: Phase 2
Study type: Interventional

ACT18018 is a multinational, randomized, double-blind, placebo-controlled, parallel-group, Phase 2 study with 3 treatment groups. The purpose of this study is to evaluate efficacy, safety and tolerability with 2 dosing regimens of itepekimab compared with placebo in male and/or female participants with NCFB aged 18 years of age up to 85 years of age (inclusive). Study details include: - The study duration (screening, 24-52-week treatment, 20-week safety follow-up) will be up to 47-77 weeks. - The treatment duration will be up to 24-52 weeks. - The follow-up duration will be 20 weeks. - Site/phone visits are at a monthly interval.

NCT ID: NCT06279221 Recruiting - Alopecia Areata Clinical Trials

Litfulo Capsules Special Investigation

Start date: March 1, 2024
Phase:
Study type: Observational

The objective of this Study is to confirm the safety during the long-term use of this drug and the effectiveness during the use of this drug under the actual use in the patients treated with this drug.

NCT ID: NCT06278337 Recruiting - Autoimmune Diseases Clinical Trials

X-linked Moesin Associated Immunodeficiency

X-MAIDReg
Start date: August 12, 2021
Phase:
Study type: Observational

Moesin deficiency was initially described in 7 male participants aged 4 to 69 years and is characterized by lymphopenia of the 3 lineages and moderate neutropenia. Genetically, 6 out of 7 participants had the same missense mutation in the moesin gene located on the X chromosome. The 7th patient has a mutation leading to the premature introduction of a STOP codon into the protein.Clinically the 7 participants with X-linked moesin-associated immunodeficiency all presented with recurrent bacterial infections of the respiratory, gastrointestinal or urinary tracts, and some had severe varicella.Therapeutically, in the absence of a molecular diagnosis and due to his SCID-like phenotype, one patient was treated with geno-identical hematopoietic stem cell transplantation . The remaining are untreated or treated with immunoglobulin substitution and/or prophylactic antibiotics. Since this study, the moesin gene has been integrated into DNA chips used for the molecular diagnosis of immune deficiencies in several countries. Physicians in Canada, the United States, Japan, South Africa and Europe have contacted us with a total of 16 known participants to date. Because of their very low severe, uncontrolled CMV infection and the absence of treatment recommendations, two 2 American participants were treated with allogeneic transplantation with severe post-transplant complications (1), and one of the participants died as a result of the transplant. Management of XMAID participants therefore varies widely from country to country, depending on age at diagnosis and clinical picture. It ranges from no treatment treatment (associated with recurrent infections and skin manifestations), IgIv substitution and/or antibiotic prophylaxis antibiotic prophylaxis, with low toxicity and apparent efficacy, and allogeneic transplantation, with all the risks risks involved (graft-related toxicity, graft versus host, disease, rejection, risk of infection). The Investigators therefore feel it is important to review the diagnosis, clinical presentation and management of X-MAID participants. The study the investigator propose will enable to understand the presentation of X-MAID participants, establish guidelines and provide the best treatment for each patient according to his or her clinical picture

NCT ID: NCT06278207 Recruiting - Clinical trials for Type 2 Diabetes Mellitus

An Observational Study Called FINEROD to Learn More About the Use of the Treatment Finerenone Including How Safe it is and How Well it Works Under Real-world Conditions

FINEROD
Start date: May 15, 2024
Phase:
Study type: Observational

This is an observational study, in which data from people in Japan with chronic kidney disease (CKD) together with type 2 diabetes (T2D) are studied. The participants in this study are already receiving the study treatment finerenone as part of their regular care from their doctors. In observational studies, only observations are made without specified advice or interventions. CKD is a long-term progressive decrease in the kidneys' ability to work properly. In people with T2D, the body does not make enough of a hormone called insulin, or does not use insulin well enough. The resulting high blood sugar levels can cause damage to the kidneys. CKD often occurs together with T2D or as a consequence of T2D. Finerenone works by blocking certain proteins, called mineralocorticoid receptors. By doing this, it may reduce damage to kidneys, heart and blood vessels. Finerenone was recently approved in the US and is now available for doctors to prescribe to people with CKD together with T2D. Consequently, there is a need to collect more information about how finerenone is used, its safety and how well it works under real-world conditions. The main purpose of this study is to collect and describe the characteristics of people with CKD and T2D who are receiving initiate finerenone treatment as prescribed by their doctors. To do this, the researchers will collect general information of the participants such as age or gender and data on kidney function and possible heart problems. The researchers will also collect data on any other disease or medical condition in the participants and on other medications used while taking finerenone. The data will come from a network of commercial electronic health records (EHRs) and national claims data in Japan. They cover the period from July 1st, 2021 until September 2023. Only already available data is collected and studied. There are no required visits or tests in this study.

NCT ID: NCT06278051 Recruiting - Clinical trials for Venous Thromboembolism

An Observational Study to Learn More About How Safe Treatment With Rivaroxaban is in Children in Japan With Venous Thromboembolism

Start date: February 19, 2024
Phase:
Study type: Observational

This is an observational study in which only data will be collected from children with venous thromboembolism who are prescribed rivaroxaban or warfarin by their doctors. Venous thromboembolism (VTE) is a condition in which people have problems due to the formation of blood clots in their veins. The study drug rivaroxaban is an approved treatment for VTE in children and adults in Japan. It is a blood thinner that prevents the blood from clotting by blocking a protein responsible for blood clotting. Warfarin is another blood thinner that is available for VTE. In this study warfarin is the reference drug. A previous study was carried out to learn about how well rivaroxaban works and how safe it is in children with VTE. However, to better understand the safety of this drug in children, more knowledge is needed about the use of rivaroxaban in the real world. The main purpose of this study is to learn more about the occurrence of major bleeding in children taking rivaroxaban. Major bleeding can be bleeding within the skull, bleeding inside the eye, bleeding from an organ in the digestive system, or bleeding which requires being given blood from a donor. In addition, this study will help learn more about the following in children with VTE: - The occurrence of major bleeding during treatment with rivaroxaban and during treatment with warfarin - The occurrence of bleeding of importance in children being treated with rivaroxaban and in children being treated with warfarin. Bleeding of importance in children can be: bleeding from the lung, blood in the kidney, heavy menstrual bleeding - The occurrence of major bleeding and bleeding of importance in children who are taking drugs called anti platelet agents and NSAIDs to prevent blood clots at the same time as rivaroxaban, who are taking a drug that blocks the action of a protein called 'CYP3A4' at the same time as rivaroxaban, who have reduced kidney function, who have taken rivaroxaban for a long time, or who have taken other drugs by mouth to prevent blood clots before starting rivaroxaban - The occurrence of repeated VTE on treatment with rivaroxaban and on treatment with warfarin The children with VTE in this study are already receiving rivaroxaban or warfarin treatment as part of their regular care from their doctors. The data will come from an electronic health records database created by a company called Medical Data Vision. The data will be collected between January 2021 and June 2024. Researchers will look at the health information from children less than 18 years of age with VTE in Japan who are prescribed treatment with rivaroxaban or warfarin during the study period. In this study, only available data from routine care are collected. No visits or tests are required as part of this study

NCT ID: NCT06275724 Recruiting - Clinical trials for Hypercholesterolaemia

Specified Drug-use Survey of Leqvio for s.c. Injection.

Start date: March 4, 2024
Phase:
Study type: Observational

The objective of this study is to evaluate the long-term safety of Leqvio in patients with familial hypercholesterolaemia or hypercholesterolaemia in post-marketing clinical practice

NCT ID: NCT06269484 Recruiting - Liver Cirrhosis Clinical Trials

A Phase IIb Study to Evaluate the Safety of Zibotentan/Dapagliflozin in Participants With Cirrhosis-ZEAL-UNLOCK

ZEAL UNLOCK
Start date: February 15, 2024
Phase: Phase 2
Study type: Interventional

This is a Phase IIb multicentre, randomised, double-blind, parallel-group, placebo-controlled study to evaluate the safety of zibotentan/dapagliflozin in combination as compared to zibotentan monotherapy as well as zibotentan/dapagliflozin and zibotentan monotherapy as compared to placebo in patients with cirrhosis.