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Remote Evaluation and Surveillance of Patients With Interstitial Lung Disease: Transforming ILD Care Delivery With Remote Monitoring
The purpose of this interventional study is to identify which combination of remote monitoring devices (e.g. home spirometry, pulse oximetry, scale, ePROs) is …
A Phase 3, Randomized, Multicenter, Double-Blind, Placebo-Controlled Study of Acoramidis for Transthyretin Amyloidosis Prevention in the Young (ACT-EARLY Trial)
Transthyretin amyloidosis (ATTR) is a disease where the normally occurring transthyretin (TTR) protein falls apart and forms amyloid, a sticky plaque-like subs…
Phase-2 Study of Vedolizumab Plus Post-Transplant Cyclophosphamide and Short Course Tacrolimus for Graft-versus-Host Disease Prevention After Reduced Intensity Conditioning Peripheral Blood Stem Cell Allogeneic Hematopoietic Cell Transplantation
This phase II trial studies how well vedolizumab plus post-transplant cyclophosphamide (PTCy) and short course tacrolimus work for the prevention of graft vers…
Uric Acid, Klotho and Salt Sensitivity in Young Adults Born Preterm
The purpose of this research is to learn about how salt in the diet influences blood pressure in young adults who were born prematurely.
A Phase III, Randomised, Double-blind, Placebo-controlled, Parallel-group, Pivotal Trial to Assess the Efficacy and Safety of Sonlicromanol in Adult Subjects With a Genetically Confirmed Mitochondrial DNA tRNALeu(UUR) m.3243A>G Variant
The KHENERFIN study aims to determine whether the study medicine, sonlicromanol, is able to reduce symptoms of fatigue and the impact of fatigue on daily life,…
Phase I Trial of Ruxolitinib in Combination With a Pediatric Based-regimen for Adolescents and Young Adults (AYAs) With Ph-like Acute Lymphoblastic Leukemia (ALL)
This study will test if adding ruxolitinib to standard multi-drug chemotherapy regimen will be safe and tolerated in adolescents and young adults with newly di…
Evaluation of Efficacy and Safety of D-galactose Supplementation in SLC35A2-CDG, a Disorder of Hypogalactosylation
This is a multicenter, randomized, double-blind, placebo-controlled, cross-over study to evaluate the efficacy and safety of AVTX-801 in subjects with SLC35A2-…
A Multicenter Prospective Study to Develop a Blood-based Biomarker Test for Aiding the Diagnosis/Prognosis of Traumatic Brain Injury in Adult Subjects (CLIN12.1) and for Monitoring the Development of Secondary Events in Patients Diagnosed With Traumatic Brain Injury (CLIN12.2)
The study is intended to cover two purposes: first, to develop a blood-based biomarker test for aiding the diagnosis of traumatic brain injury (TBI) in adult p…
Open-label Single-arm Phase 2 Trial of Trastuzumab Deruxtecan in Previously Treated HER2-Immunohistochemistry (IHC) 0 Advanced Breast Cancer
The purpose of this study is to test the good and bad effects of a drug called trastuzumab deruxtecan (T-DXd) in adult patients with metastatic HER2-negative b…
PNOC031: Protocol for Embryonal Tumor With Multilayered Rosettes (ETMR)
This is an open-label, comprehensive, iterative investigation of evaluating the use of induction chemotherapy, high-dose chemotherapy, and focal radiation ther…
Examining the Feasibility and Acceptability of a Journaling Intervention in Alleviating Temporomandibular Pain
The goal of this clinical trial is to learn whether a 2-week gratitude journaling activity is feasible and acceptable for individuals with Temporomandibular Di…
A PHASE 1 STUDY TO EVALUATE SAFETY, TOLERABILITY, PHARMACOKINETICS, PHARMACODYNAMICS, RELATIVE BIOAVAILABILITY, FOOD EFFECT, METABOLISM & EXCRETION, AND DRUG-DRUG INTERACTION POTENTIAL OF PF-08103402 IN HEALTHY ADULTS AND/OR ADULTS WITH MILD TO MODERATE ASTHMA
The purpose of this study is to learn about the safety of a new study medicine called PF-08103402 in healthy adults (do not have disease) and or in adults with…