Clinical Research

For 150 years, Boston Children’s has embodied a culture of scientific investigation that has shaped pediatric medicine and changed children’s lives. This legacy continues today as we lead the world in pediatric research, empowering clinicians and scientists to challenge the status quo and seek better answers for our patients.

Our research enterprise is the world's largest and most highly-funded pediatric hospital. In FY2018, we received more funding from the National Institutes of Health than any other children’s hospital in the nation. We perform research in a vast range of specialties, revolutionizing treatments for children with common conditions, such as asthma, diabetes and obesity, to children with rare and complex disorders.

Recent Breakthroughs

Erica Esrick, MD, presents the results of a novel approach to gene therapy for sickle cell disease

Gene therapy to boost fetal hemoglobin continues to do well in sickle cell trial

Three adult patients with sickle cell disease (SCD) are doing well after receiving an infusion of their own stem cells that had been genetically engineered to induce them to stop producing harmful “sickle” hemoglobin and start producing a healthy form of hemoglobin.

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patient receiving stem cell transplant

A big step toward curbing graft-vs.-host disease after bone marrow transplant

A drug used for rheumatoid arthritis has moved a step closer to FDA approval for a desperately needed new use. The drug, abatacept, has gained FDA Breakthrough Therapy Designation for preventing acute, severe graft-versus-host disease (GvHD) in patients receiving bone marrow transplants. 

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A potential Diamond-Blackfan anemia treatment swims into view

Zebrafish, besides being popular in aquariums, make good stand-ins for studying human diseases. One such test came up with a new potential treatment for a rare blood disorder, Diamond-Blackfan anemia (DBA): a drug called trifluoperazine, normally used to treat psychotic disorders. It’s now in a clinical trial for DBA in adults.
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Would your child benefit from a clinical trial?

Clinical research takes scientific discoveries and transforms them into new treatments and approaches to medical care that improve the health. Learn about the importance of clinical research, its benefits and considerations.

 

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Find a clinical research study

Boston Children's leads or participates in hundreds of clinical trials. Use this database to find out which trials are recruiting, who can enroll and more.

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Resources for the research community

A central hub of resources to support the Boston Children’s research community through guidance on planning, designing, implementing and reporting.

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Building on our extraordinary leadership in pediatric science at Boston Children’s, we translate our findings — and those of others — to fundamentally change how disease is treated and cured.

David A. Williams, MD
Senior Vice President and Chief Scientific Officer