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A Single Arm, Multicenter, Open-label Extension (OLE) Trial to Evaluate Long-term Safety and Tolerability of Pelacarsen (TQJ230) in Participants Who Completed the Parent Lp(a)HORIZON Trial
This open-label extension study will provide post-trial access to pelacarsen (TQJ230) to participants who have successfully completed the double-blind parent s…
Phase II Study of the Combination of Blinatumomab and Ponatinib in Patients With Philadelphia Chromosome (Ph)-Positive and/or BCR-ABL Positive Acute Lymphoblastic Leukemia (ALL)
This phase II trial studies how well blinatumomab, methotrexate, cytarabine, and ponatinib work in treating patients with Philadelphia chromosome (Ph)-positive…
A Phase 1-2, Double-Blind, Sham-Controlled Multiple Ascending Dose Study to Evaluate Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Intrathecally-Administered ION440 in Patients With MECP2 Duplication Syndrome
The primary purpose of this study is to evaluate the safety and tolerability of ION440.
A National Registry For Pulmonary Alveolar Proteinosis
The major goal of Part A of this study is to establish a National PAP Registry to help make reliable new research tests available to doctors to improve the dia…
A Phase I/II, Dose-escalation and Dose-optimization Study to Evaluate Safety, Tolerability, Pharmacokinetics, Pharmacodynamics, and Efficacy of MT-4561 in Patients With Various Advanced Solid Tumors and to Evaluate Effect of MT-4561 on Pharmacokinetics of Oral Midazolam
This is a First In Human (FIH), multicenter, open-label, Phase I/II study to evaluate safety, tolerability, Pharmacokinetics (PK), pharmacodynamics, and effica…
A Two-part Multicenter Study: a Randomized, Double-blind, Placebo-controlled Dose-escalation Safety Phase (Part 1) Followed by Double-blind, Placebo-controlled, Adaptive Phase (Part 2) Study to Evaluate the Safety and Efficacy of AB-1003 in Adult Subjects With LGMD2I/R9 Mutations in the Gene Encoding Fukutin Related Protein (FKRP)
The purpose of this study is to evaluate the safety and tolerability of a single intravenous infusion of AB-1003 in adults diagnosed with limb girdle muscular …
An Open-Label Phase 2 Study to Evaluate the Safety, Tolerability, and Effect on Albuminuria of MZE829 in Adults With Proteinuric Chronic Kidney Disease and the APOL1 High Risk Genotype
This is purpose of this study is to evaluate the safety, tolerability, and effect on Albuminuria of MZE829 in Adults with APOL1 Kidney Disease
Retrospective and Longitudinal Prospective Natural History Study of GEMIN5-Related Neurodevelopmental Disorder
This study will include a comprehensive retrospective chart review and a longitudinal prospective observational natural history study to characterize the pheno…
An 18-month, Open-label, Single-arm Safety Extension Study of an age-and Bodyweight-adjusted Oral Finerenone Regimen, in Addition to an ACEI or ARB, for the Treatment of Children and Young Adults From 1 to 18 Years of Age With Chronic Kidney Disease and Proteinuria
Researchers are looking for a better way to treat children who have chronic kidney disease (CKD), which is long-term kidney disease, and proteinuria, a conditi…
Evaluation of Recipients With and Without Proteinuria, Pre and Post Kidney Transplantation
Patients with focal segmental glomerulosclerosis (FSGS) who progress rapidly to end stage renal disease (ESRD) are at highest risk for development of recurrenc…
Brain Metabolism Observed at 7 Tesla in Health and Metabolic Disease.
The goal is to develop methodology to monitor flux in the citric acid cycle in brain via 13C nuclear magnetic resonance (NMR) spectroscopy at 7 Tesla.
A Phase I/II, Open-label, Multicenter Study to Evaluate the Safety, Pharmacokinetics, Pharmacodynamics, and Preliminary Efficacy of Anti-GPRC5D CAR-T Cell Product (OriCAR-017) in Subjects With Relapsed/Refractory Multiple Myeloma.
The is a first clinical study for Oricell Therapeutics Inc. in the United States to evaluate the safety, PK, PD and preliminary efficacy of our anti-GPRC5D cel…