Clinical Trials

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.

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.

Are you an adult with pulmonary arterial hypertension (PAH) and currently taking vasodilators? This study is testing the efficacy of a new medicine called famotidine for people with PAH. Participants in this study must be between 18 and 80 years old and willing to take famotidine or a placebo for 6 months. This study takes place in Seattle, Washington.

Are you an adult diagnosed with cystic fibrosis who would like to help test web-based educational resources designed to inform and prepare patients to make decisions about lung transplantation? This study will determine if it is feasible for study participants to complete use of an assigned web-based educational program, and evaluate whether providing certain educational resources leaves participants more prepared to make future treatment decisions involving lung transplantation with their health care provider(s). To participate in this study, you must be at least 18 years old. This study takes place in Seattle, Washington.

Are you a woman 18 years old or older who has been diagnosed with LAM? This study is examining whether long-term treatment with medicine called sirolimus can stop lung damage in women who have LAM. This study takes place at multiple locations in the United States.

Do you or your child have symptoms of a serious lung disease but no diagnosis? This study aims to develop new ways to diagnose primary ciliary dyskinesia (PCD) and 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 ant tell the difference between these two conditions. Participants in this study must be 5 to 45 years old. This study is taking place at multiple locations in the United States and in Canada.

The primary purpose of this study is to investigate whether a voice-based platform is better for delivering an electronic health intervention to older adults than a text/typing-based platform. We have an evidence-based electronic health intervention (Elder Tree, ET) that has been shown to improve quality of life, physical and socio-emotional health outcomes for older adults with multiple chronic conditions when delivered via a text/typing-based system. The current project would test whether such patients would benefit even more if ET were delivered via a voice-based system (vs. the text-based system) because they would use it more consistently. ET is an existing intervention providing tools, motivation, and support on a computer platform to help older adults manage their health.

The purpose of the research is to understand structural plaque abnormalities that make a carotid plaque unstable and brake off (embolize) which would help to predict and treat individuals who are likely to suffer not only classic episodic major strokes but also cognitive impairment.

The main goal of this study is to determine whether the treatment will lead to a complete absence of severe vaso-occlusive events (VOEs) in patients with severe SCD.

This study will pilot test a just-in-time (JIT) adaptive intervention to reduce severe respiratory illness, for children with severe cerebral palsy (CP). The intervention program, called RE-PACT, delivers timely, customized action planning and health coaching when mobile text messaging with families predicts hospitalization risk is elevated. A total of n=90 caregivers of children with severe CP will be enrolled from the University of Wisconsin-Madison (UW) and the University of California, Los Angeles (UCLA) and can expect to be on study for up to 6 months.