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A Phase 1/2, Open-Label, Dose-Escalation, Safety, Pharmacokinetic, and Pharmacodynamic Study of Oral Nuvisertib (TP-3654) in Patients With Intermediate or High-Risk Primary or Secondary Myelofibrosis
This study is a Phase 1/2, multicenter, dose-escalation, open-label trial to assess safety, tolerability, pharmacokinetics and pharmacodynamics of nuvisertib (…
A Comprehensive View of Patient Experiences of Individuals Participating in Pulmonary Fibrosis Clinical Study
Taking part in clinical trials usually favors a particular demographic group. But there is limited research available to explain what research attributes affec…
Standardizing Treatments for Pulmonary Exacerbations: A Platform for Evaluating Treatment Decisions to Improve Outcomes (STOP360) Aminoglycoside Study (AG Study)
The purpose of this study is to look at pulmonary exacerbations in people with cystic fibrosis (CF) that need to be treated with antibiotics given through a tu…
Placebo-Controlled and Randomized Phase 2 Trial of CMV-MVA Triplex Vaccination in HLA-Matched Related Stem Cell Donors to Enhance CMV-Specific Immunity and Prevent CMV Viremia in Recipients After Hematopoietic Stem Cell Transplant
This phase II clinical trial tests how well the cytomegalovirus-modified vaccinica Ankara (CMV-MVA) Triplex vaccine given to human leukocyte antigens (HLA) mat…
Cord Blood Transplantation in Children and Young Adults With Hematologic Malignancies and Non-malignant Disorders
This is a single-arm study to investigate 1-year treatment related mortality (TRM) in patients with life threatening non-malignant and malignant hematologic di…
Utilization of Explanted Lungs for Isolation of Tissue Samples and Primary Cell Lines to Study Pulmonary Fibrosis
The goal of this study is to use the tissues from the explanted lungs in order to better study the cause of pulmonary fibrosis at a cellular level.
KB407-02 A Phase 1 Study of Inhaled KB407, a Replication-Defective, Non-Integrating Vector Expressing Human Cystic Fibrosis Transmembrane Conductance Regulator, for the Treatment of Cystic Fibrosis
This study will evaluate safety and tolerability of ascending doses of nebulized KB407 in adults with cystic fibrosis.
Atrial Late Gadolinium Enhancement in Patients with Repaired Congenital Heart Disease
In this research study the investigators want to learn more about how well the investigators can visualize scar tissue in the heart by MRI. In patients with co…
A Research Study to Advance the CF Therapeutics Pipeline for People Without Modulators
The REACH study is for people with CF who do not take cystic fibrosis transmembrane conductance regulator (CFTR) modulators. The goal of the REACH study is to …
TIDES 2.0: Prevalence and Longitudinal Course of Depression, Anxiety, and Behavior Problems in Children With Cystic Fibrosis Under 12 Years of Age
This is a longitudinal, observational epidemiological study designed to estimate the prevalence of depression, anxiety, and behavior problems in children ages …
A Phase 1b Study of Safety, Tolerability of Ropeginterferon Alfa-2b Add-on to Ongoing Ruxolitinib Therapy in Myelofibrosis (RopeRux in Myelofibrosis)
The purpose of this clinical trial is to learn if the study drug ropeginterferon alfa- 2b added to, standard of care, ruxolitinib is safe and effective in trea…
CF Wellness Program: ORBIT Phase 2 Pilot RCT
This study is a pilot randomized control trial (RCT; N=80) comparing the Cystic Fibrosis Wellness Program (CFWP) to usual care (UC) to evaluate (1) Interventio…