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A Phase 3b Multicenter Open-label Trial of the Safety, Tolerability, and Efficacy of Tolvaptan in Infants and Children 28 Days to Less Than 18 Years of Age With Autosomal Recessive Polycystic Kidney Disease (ARPKD)
To evaluate the safety of tolvaptan in pediatric subjects with ARPKD
Personalized Theratyping Trial
The purpose of this study is to explore the use of off-label CFTR modulators that may affect CFTR function in patients with CFTR mutations that are not current…
Autosomal Dominant Polycystic Kidney Disease Patient Registry
The purpose of the ADPKD Registry is to create an online patient network that includes at least 5,000 people with Autosomal Dominant Polycystic Kidney Disease …
Symptoms of Polycystic Ovarian Syndrome Ameliorated by Keto-Adaptation Pilot
The SPARK- Pilot study (Symptoms of PCOS Ameliorated by Responses to Keto-adaptation) is a 12-week clinical trial at The Ohio State University designed to expl…
Advancing Telemedicine in Pulmonology: Acoustic-waveform Respiratory Evaluation (AWARE) Via Sensing and Machine Learning on Smartphones
The study will evaluate the feasibility of using smartphone speakers and microphones to evaluate the caliber of the airways, detect airway obstruction, aid in …
A Phase 2 Trial of Ivonescimab for Patients With Advanced, Metastatic Salivary Gland Cancers (I-MAC)
The goal of this study is to evaluate the efficacy and safety of Ivonescimab in participants with advanced, metastatic salivary gland cancers. The name of the…
The Measurement and Analysis of Maximal Expiratory Flow Volume Loops at Low Lung Volumes in Children With Cystic Fibrosis and Normal Routine Lung Function.
Goal is to physiologically detect unsuspected small airways obstruction in children and adults with treated heterozygous and homozygous cystic fibrosis. Unsusp…
Study to Evaluate if Androgen-receptor Blockade (Spironolactone) Improves Progesterone-suppression of Wake Luteinizing Hormone Pulse Frequency in Pubertal Girls With Hyperandrogenism
The purpose of this study is to determine if, in mid- to late pubertal girls with hyperandrogenism, androgen-receptor blockade (spironolactone) improves the ab…
Comparative Meal Response to Plant vs. Animal Protein in Women With PCOS
Polycystic ovary syndrome (PCOS) is a common endocrine disorder characterized by insulin resistance, hyperandrogenism, and reproductive dysfunction. Dietary st…
Phase II Trial of Puxitatug Samrotecan (AZD8205) in Advanced, Recurrent or Metastatic (R/M) Aggressive Adenoid Cystic Carcinoma Subtype I (ACC-I)
Phase II open label study designed to evaluate the efficacy and safety of P-Sam in patients with aggressive, solid, NOTCH mutant or p63 low (B7-H4 high) R/M AC…
An Open-label, Single Center Study Assessing Tenapanor as a Non-CFTR-mediated Treatment of CF-related Constipation in People With CF.
Tenapanor is the newest FDA-approved drug for IBS with constipation (IBS-C). This study seeks to understand tenapanor as a treatment for cystic fibrosis-relate…
FLOWER: Following Longitudinal Outcomes With Epidemiology for Rare Diseases
FLOWER is a completely virtual, nationwide, real-world observational study to collect, annotate, standardize, and report clinical data for rare diseases. Patie…