Clinical Trials

Has your child received a Fontan palliation operation? If so, they may be able to join a clinical trial that is testing whether exercise can help patients who have had this procedure. Your child must be between 8 and 19 years old, had a Fontan palliation, and can exercise. A study team member will observe the participants doing aerobic and resistance exercises live on video. The study is taking place in Palo Alto, California.

Do you want to help find new treatments for heart failure with preserved ejection fraction? This study is testing whether a medicine called spironolactone can help treat heart failure with preserved ejection fraction when paired with the standard treatment. Spironolactone helps to remove excess water and sodium from the body. To participate in this study, you must be between 50 and 99 years old and diagnosed with heart failure with preserved ejection fraction.

Do you have heart failure with preserved ejection fraction (HFPEF)? This study is testing the efficacy of a diuretic medicine called spironolactone that stops your body from absorbing too much salt and keeps your potassium levels from dropping too low. Participants of this study must be between 50 and 99 years old and already taking diuretic medicine. This study takes place in multiple locations across the United States and Sweden.

Do you or a loved one have pneumonia? This study will test whether combining two medicines to reduce inflammation of the airway and to keep it open may help prevent respiratory failure in people who have pneumonia. To participate in this study, you must be at least 18 years old and have severe pneumonia with low blood oxygen levels.

Do you have symptoms of a lung disease (such as long-term wet cough, bronchiectasis, or recurrent pneumonia) with no genetic diagnosis? This study aims to develop new ways to diagnose primary ciliary dyskinesia (PCD) or primary immune deficiency (PID), two conditions that can have similar symptoms. Researchers will combine information from genetic testing, lung imaging, and lung function tests to diagnose and tell the difference between these two conditions. Participants in this study must be 5 to 45 years old and must have symptoms of a serious lung disease but no diagnosis. This study is taking place at multiple locations in the United States and in Canada, including the NIH Clinical Center in Bethesda, Maryland.

The investigators propose to study the relationship between right ventricle (RV) steatosis and RV function, exercise capacity, and outcomes in humans with pulmonary arterial hypertension (PAH) and to identify potential drivers of lipid accumulation.

The Investigators propose a randomized trial of smart texts versus usual care for 6 months. The Investigators will randomize 100 PAH patients to the mHealth intervention or usual care. The Investigators will test the effect of a text-based mHealth intervention on HRQOL in PAH using the PAH-specific emPHasis-10 questionnaire. The Investigators will also test the effect of an mHealth intervention on exercise capacity, measured by a supervised home-based 6MWD test. Finally, the Investigators will examine the effect of the intervention on time to clinical worsening (composite of PAH therapy escalation, PAH hospitalization, and death) one year after randomization.

The investigators propose to study the relationship between right ventricle (RV) steatosis and RV function, exercise capacity, and outcomes in humans with pulmonary arterial hypertension (PAH) and to identify potential drivers of lipid accumulation.

Patients with pulmonary arterial hypertension (PAH) have reduced health related quality of life (HRQOL) and impaired exercise capacity. Despite fourteen approved therapies, most patients die within ten years. Increasing physical activity is highly efficacious in PAH, resulting in six-minute walk distance (6MWD) and HRQOL improvement that often exceeds the effect of medications. Prior activity studies required inpatient rehabilitation, which is impractical, hard to sustain, and poorly scalable to a rare disease.

The Investigators propose a randomized trial of smart texts versus usual care for 6 months. The Investigators will randomize 100 PAH patients to the mHealth intervention or usual care. The Investigators will test the effect of a text-based mHealth intervention on HRQOL in PAH using the PAH-specific emPHasis-10 questionnaire. The Investigators will also test the effect of an mHealth intervention on exercise capacity, measured by a supervised home-based 6MWD test. Finally, the Investigators will examine the effect of the intervention on time to clinical worsening (composite of PAH therapy escalation, PAH hospitalization, and death) one year after randomization.

The investigators' central hypothesis is that early combination therapy with two PAH-specific oral therapies that have been shown to be well tolerated in the pediatric population, sildenafil and bosentan, will result in better World Health Organization (WHO) functional class at 12 months after initiation of PAH treatment than therapy with sildenafil alone.