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NCT ID: NCT04789070 Recruiting - Clinical trials for Inclusion Body Myositis

Phase III Trial of Sirolimus in IBM

Start date: July 1, 2022
Phase: Phase 3
Study type: Interventional

The hypothesis is that Sirolimus, (Rapamycin (R)) which is currently used in organ transplantation and works by blocking the activity of T effector cells but preserving T regulatory cells, as well as by inducing autophagy (protein degradation), will be effective in IBM to slow or stabilize disease progression, helping to maintain patient function and independence. This phase III trial will confirm pilot data showing statistically significant clinical outcomes.

NCT ID: NCT04788888 Recruiting - Clinical trials for Symptomatic Severe Aortic Stenosis

Evaluation of TAVR Using the NAVITOR Valve in a Global Investigation

VANTAGE
Start date: June 13, 2021
Phase: N/A
Study type: Interventional

Evaluation of TAVR using the NAVITOR valve in a Global Investigation.

NCT ID: NCT04779320 Recruiting - Clinical trials for Crohn's Disease (CD)

A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Crohn's Disease (CD)

Start date: April 30, 2022
Phase: Phase 3
Study type: Interventional

Vedolizumab is a medicine that helps to reduce inflammation and pain in the digestive system. In this study, children and teenagers with moderate to severe Crohn's disease will be treated with vedolizumab. The main aim of the study is to check if participants achieve remission after treatment with the vedolizumab. Remission means symptoms improve or disappear and an endoscopy shows no signs of inflammation. Participants will receive 3 infusions of vedolizumab over 6 weeks. Then, those who have a clinical response will receive either a high dose or low dose of vedolizumab once every 8 weeks. They will receive the same dose every time.

NCT ID: NCT04779307 Recruiting - Colitis, Ulcerative Clinical Trials

A Study of Vedolizumab in Children and Teenagers With Moderate to Severe Ulcerative Colitis (UC)

Start date: April 18, 2022
Phase: Phase 3
Study type: Interventional

Vedolizumab is a medicine that helps to reduce inflammation and pain in the digestive system. In this study, children and teenagers with moderate to severe ulcerative colitis will be treated with vedolizumab. The main aim of the study is to check if participants achieve remission after treatment with vedolizumab. Remission means symptoms improve or disappear and an endoscopy shows no or limited signs of disease. The study is also evaluating side effects of vedolizumab in the children and teenager with moderately to severely active ulcerative colitis. Participants will receive 3 infusions of vedolizumab over 6 weeks. Then, those who have a clinical response will receive 1 of 3 doses of vedolizumab once every 8 weeks. They will receive the same dose every time.

NCT ID: NCT04775485 Recruiting - Clinical trials for Advanced Solid Tumor

A Study to Evaluate DAY101 in Pediatric and Young Adult Patients With Relapsed or Progressive Low-Grade Glioma and Advance Solid Tumors

FIREFLY-1
Start date: April 22, 2021
Phase: Phase 2
Study type: Interventional

FIREFLY-1 is an ongoing, Phase 2, multi center, open-label study to evaluate the safety and efficacy of oral pan-RAF inhibitor DAY101 in pediatric, adolescent, and young adult patients with recurrent or progressive low-grade glioma or an advanced solid tumor harboring a known RAF alteration.

NCT ID: NCT04774718 Recruiting - Clinical trials for ALK Fusion-positive Solid or CNS Tumors

A Study Evaluating the Safety, Pharmacokinetics, and Efficacy of Alectinib in Pediatric Participants With ALK Fusion-Positive Solid or CNS Tumors

Start date: September 14, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

This study will evaluate the safety, pharmacokinetics, and efficacy of alectinib in children and adolescents with ALK fusion-positive solid or CNS tumors for whom prior treatment has proven to be ineffective or for whom there is no satisfactory standard treatment available.

NCT ID: NCT04773782 Recruiting - CNS Tumor Clinical Trials

A Study of Avapritinib in Pediatric Patients With Solid Tumors Dependent on KIT or PDGFRA Signaling

Start date: February 24, 2022
Phase: Phase 1/Phase 2
Study type: Interventional

This is a Phase 1/2, multicenter, open-label trial of avapritinib in participants 2 to < 18 years of age with advanced relapsed/refractory (R/R) solid tumors, including central nervous system (CNS) tumors, that harbor a PDGFRA and/or KIT mutation (including non-synonymous point mutations, insertions, and deletions) or amplification, or DMG-H3K27a who have no available curative treatment options. This is a single-arm trial in which all participants will receive avapritinib. The study consists of 2 parts: dose confirmation, safety, and PK (Part 1) and initial efficacy, safety, and PK at the Part 2 recommended dose (Part 2).

NCT ID: NCT04772079 Recruiting - Plaque Psoriasis Clinical Trials

A Study to Evaluate the Drug Levels, Efficacy and Safety of Deucravacitinib in Pediatric Participants With Moderate to Severe Plaque Psoriasis

Start date: March 23, 2021
Phase: Phase 3
Study type: Interventional

The purpose of this pediatric study is to evaluate the drug levels, efficacy and safety of Deucravacitinib in pediatric participants aged 4 to <18 years with moderate to severe plaque psoriasis. This study includes two cohorts; Cohort 1 (age 12 to <18 years) and Cohort 2 (age 4 to <12 years), with two parts; for each cohort. Part A will evaluate the drug levels of BMS-986165 to enable selection of 2 dose levels to be studied in Part B. Part B will assess the efficacy and safety of two dose levels in pediatric participants with moderate to severe plaque psoriasis. The 5-year long-term extension (LTE) period will observe the long-term safety and tolerability of deucravacitinib in pediatric participants with psoriasis who have completed Parts A or B of the study.

NCT ID: NCT04771130 Recruiting - Clinical trials for Acute Myeloid Leukemia

A Study of BGB-11417 in Participants With Myeloid Malignancies

Start date: May 24, 2021
Phase: Phase 1/Phase 2
Study type: Interventional

The study will determine the safety, tolerability, recommended Phase 2 dose (RP2D) and preliminary efficacy of BGB-11417 as monotherapy and in combination with azacitidine in participants with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)or MDS/myeloproliferative neoplasm (MPN) .

NCT ID: NCT04769817 Recruiting - Prostate Cancer Clinical Trials

ProsTIC Registry of Men Treated With PSMA Theranostics

Start date: May 1, 2021
Phase:
Study type: Observational [Patient Registry]

This is a descriptive, observational, prospective, open-ended, registry utilising electronic data capture to collect information on the outcomes of men treated with prostate specific-membrane antigen (PSMA) theranostics.