Clinical Trials

This is a multicenter, randomized, placebo-controlled, sequential dose-escalating, double-masked, safety study of sildenafil in premature infants with severe bronchopulmonary dysplasia,

The purpose of this study is to identify the optimal deimplementation strategies for an overused practice: continuous pulse oximetry monitoring of children hospitalized with bronchiolitis who are not receiving supplemental oxygen.

This is a multicenter, randomized, placebo-controlled, sequential dose-escalating, double-masked, safety study of sildenafil in premature infants with severe bronchopulmonary dysplasia (BPD).

Do you have COPD? This study will be looking for genes that may play a role in the development of COPD by looking at the blood from patients who have COPD, as well as the blood from those patients’ family members. To participate in this study, you must be 52 years old or younger and must not be pregnant or have other serious lung diseases such as lung cancer. This study will be conducted in Boston, Massachusetts.

The MIDAS study aims to follow LAM patients who are currently taking, have previously failed or been intolerant of, or may (at some time in the future) take mTOR inhibitors (sirolimus or everolimus) as part of their clinical care. Adult female TSC patients may also enroll, with or without lung cysts.

Are you an adult woman who would like to help develop methods to prevent the worsening of LAM? This study aims to discover whether earlier and longer treatment with a lower dose of sirolimus can help prevent LAM from getting worse over time. To participate in this study, you must be female, be at least 18 years old, and have been diagnosed with LAM. This study is located in Atlanta, Georgia; Boston, Massachusetts; Chicago, Illinois; Cincinnati, Ohio; Denver, Colorado; Nashville, Tennessee; Palo Alto, California; Philadelphia, Pennsylvania; and Seattle, Washington.

Does your infant have a low platelet count? This study is investigating the safety and efficacy of platelet transfusion as a treatment for infants with thrombocytopenia. Researchers are looking at the positive effects of platelet transfusion against the negative effects, such as the release of inflammatory molecules and the formation of blood clots. To participate in this study, your child must be younger than 6 months old and have a low platelet count (less than 100 × 109/L). This study takes place in Boston, Massachusetts.

Does your infant have a low platelet count? This study investigates immature platelet counts as a marker for bleeding risk in newborns with thrombocytopenia, compared with platelet counts alone. Immature platelets are the most recently produced platelets and may be a better marker of platelet production. To participate in this study, you must have a newborn who is less than 32 weeks’ gestational age, has a birth weight greater than 500 grams, and has a platelet count less than 100 × 109/L. This study takes place in multiple locations in the United States, the Netherlands, and Sweden.