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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.
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 …
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…
Study to Assess Potential Impairments in Estradiol Augmentation of Gonadotropin Secretion in Polycystic Ovary Syndrome
The purpose of this study is to determine if estradiol augmentation of luteinizing hormone (LH) secretion secretion (primary endpoint) and follicle-stimulating…
Autosomal Dominant Polycystic Kidney Disease Data Repository
Autosomal dominant polycystic kidney disease (PKD) is the most common inherited kidney disease, affecting more than 400,000 people in the U.S. and 5 million pe…
BEGIN Novel ImagiNG Biomarkers
To determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary a…
Quantitation and Spatial Registration of Airways Dysfunction With Dynamic 19F MRI in Cystic Fibrosis
The purpose of this study is to look at lung ventilation in people with cystic fibrosis over time (1 year) using magnetic resonance imaging (MRI) with an inhal…
Evaluation of the Clinical and Psychological Impact of Vitamin D Replacement in Adolescent Females at Risk for Polycystic Ovarian Syndrome (PCOS)
This study is a double blind, placebo controlled, randomized trial of study subjects with PCOS and low vitamin D to 2 groups- placebo and vitamin D replacement…
A Phase 1/2, Multicenter Study Evaluating the Safety, Tolerability, and Biodistribution of RCT2100 With Single-Ascending Doses in Healthy Participants and Multiple-Ascending Doses and Proof-of-Concept in Participants With Cystic Fibrosis
This is the first-in-human study with RCT2100 and is designed to provide safety and tolerability data for future clinical studies.
A Phase 1a/1b, First-in-human, Multicenter Study to Assess the Efficacy and Safety of RGT-61159 for Treatment of Patients With Relapsed/Refractory Adenoid Cystic Carcinoma (ACC) or Colorectal Carcinoma (CRC)
Phase 1 study to evaluate safety, tolerability and anti-tumor activity of RGT-61159 in patients with ACC or CRC
OnTrackCF: Engagement, Feasibility, and Acceptability Study
This is a multi-site, nonrandomized study using mixed methods approach to evaluate the feasibility, acceptability, and user engagement of OnTrackCF for adults …