| Study Protocol Number | Study Title and Description |
|---|---|
| 25-0689 |
Testing the Effects of CNP-103 in People with Recently Diagnosed Type 1 Diabetes
The main goal of this study is to learn whether CNP-103 is safe and well tolerated in teenagers and young adults with recently diagnosed type 1 diabetes. We will look at how people react to CNP-103 compared to a placebo which looks like the study drug but has no active medicine. |
| 23-1690 |
Understanding Physical Activity and Bone Health in Female Youth
We want to understand how physical activity and bone health are related in female youth. For one year, we will look at physical activity and bone health in active teenagers without an eating disorder and teenagers who are getting care for a restrictive eating disorder. |
| 26-0382 |
A Phase 3 Multicenter, Double-Masked, Randomized, Placebo-Controlled Study to Investigate the Safety, Tolerability, and Efficacy of Gildeuretinol Acetate (ALK-001) on the Progression of Stargardt Disease (STGD) - NORTHSTAR
To evaluate the efficacy of ALK-001 compared with placebo in slowing the progression of retinal atrophic lesion growth in participants with STGD |
| 26-0145 |
New Treatment for Children and Young Adults with Newly Diagnosed Type 1 Diabetes (BARICADE-PRESERVE)
This study is testing a new drug called baricitinib in children and young adults who have recently been diagnosed with type 1 diabetes (T1D). We want to learn whether it can help preserve beta cells and slow the progression of T1D. |
| 25-2333 |
A Phase 3, Multicenter, Prospective, Randomized, Open-label Efficacy and Safety Study of Intravenous Brincidofovir versus Intravenous Cidofovir for Treatment of Adenovirus Infection in Pediatric and Adult Subjects After Allogeneic Hematopoietic Cell Transplantation (allo-HCT)
Primary Objective To assess efficacy of intravenous (IV) brincidofovir (BCV), compared with IV cidofovir (CDV), in subjects after allo-HCT with adenovirus (AdV) viremia. Secondary Objectives To assess the safety of IV BCV, compared with IV CDV, in subjects after allo-HCT with AdV viremia. To assess IV BCV pharmacokinetics (PK). |
| 25-2429 |
How Physical Activity Affects Bone Health in Teens With and Without Cerebral Palsy
This study wants to find out how physical activity affects bone strength in teens with and without cerebral palsy (CP). |
| 25-1257 |
Learning How Infections During Pregnancy Affect Babies
In this study we want to learn how babies are affected when their mothers have a viral infection during pregnancy. To do this, we will collect blood samples from pregnant women who have viral infections (like COVID-19) and those who do not. |
| 25-1102 |
How Safe is Hepatitis C Treatment for Pregnant Women and their Babies?
This study wants to learn how treating Hepatitis C during pregnancy affects the health of pregnant women and their babies. |
| 25-0693 |
New Drug Treatment to Protect Insulin-Making Cells in Kids and Adolescents with Type 1 Diabetes
In this study we are testing a new drug called teplizumab to see if it can help protect the cells that make insulin in people with type 1 diabetes. We want to know if it can improve blood sugar levels, reduce the need for extra insulin, and lower the risk of low blood sugar. |
| 25-0323 |
Evaluating the Safety and Antibody Protection of a Second RSV Vaccine in Pregnancy
This study will look at whether getting a second dose of RSVpreF (Abrysvo®) vaccine during another pregnancy is safe and can help your body make antibodies that may protect your baby from Respiratory Syncytial Virus (RSV). We will also check to see if you still have antibodies from the dose you got during your previous pregnancy. |
| 25-0039 |
A Phase 1/2, Open-Label Study to Evaluate the Safety and Efficacy of Autologous CD19-specific Chimeric Antigen Receptor T cells (CABA-201) in Subjects with Active Idiopathic Inflammatory Myopathy or Active Juvenile Idiopathic Inflammatory Myopathy
This is a Phase 1/2, open-label study designed to evaluate the safety and efficacy of CABA-201 in patients with active IIM or JIIM and to identify an appropriate dose for future studies. |
| 24-1345 |
Registry Study for Pregnant Women with Multiple Sclerosis
This registry wants to learn whether taking Vumerity (Diroximel Fumarate) during pregnancy impacts the health of pregnant women with MS or their babies. We will compare the health of women who took Vumerity during pregnancy with the health of women who took other MS medicines or no MS medicine during pregnancy. |
| 24-0432 |
Modulation of the Immune System in Down Syndrome for Improved Outcomes and Neurodevelopment - 1 (MISSION-1)
In this study, we want to learn more about inflammation and the immune system in people with Down syndrome. We are testing whether a study drug called tofacitinib can help lower inflammation and improve health and development in children and young adults with Down syndrome. |
| 24-0908 | |
| 23-2327 |
Pregnant Women Needed for a Clinical Trial of Choline Supplements
This study looks at how choline supplements and exposure to cannabis-based products affect pregnancy and child health. |
| 24-0027 |
The Family Matters Study
In this study, we want to learn if eating meals together as a family can help improve your child’s health, and the health of other family members too! |
| 23-1622 |
Learning How Environment Impacts Child Health and Development
In this study, we want to better understand how the environment and things that happen early in children’s lives—even before they are born—affect their development, health, and well-being. |
| 22-1840 |
PRenatal and Obstetric Maternal Exposures and ISlet Autoantibodies in Early Life - The PROMISE Study
This study wants to learn more about how exposures during pregnancy like infections, diet and growth, can affect a baby’s chances of developing type 1 diabetes later in life. |
| 22-0853 |
Child Bipolar Disorder Network (CBN) Study: Treating Youth with or at Risk for Bipolar
We want to find better ways to treat bipolar disorders. We are looking for children, teens, and young adults, aged 9 to 21 years old, to join our study. |
| 20-0482 |
A Patient Registry to Learn More about People with X and Y Syndromes
This study will collect data from health records about people that have variations in their X & Y chromosomes to learn about their health. We also ask if people are willing to join future studies about X & Y variations. |
| 21-3533 |
Study Exploring Genetic Causes for Recurrent Pregnancy Loss
We are looking for couples that have had two or more pregnancy losses with no known cause. We want to find out if genetics might be a reason for recurrent pregnancy loss (RPL). |
| 23-2361 |
The COVID-19 and Diabetes Assessment (CODA) Study
This study wants to learn more about how COVID-19 and diabetes are connected. |
| 19-3027 |
Learning More About Turner Syndrome (INSIGHTS Registry)
This study wants to collect medical information from people with Turner Syndrome at clinics across the country to learn more about the condition. We are gathering this data in a national registry to help improve the health and care for people with Turner Syndrome in the future. |
| 20-0706 |
Understanding the Role of Mother’s Triglycerides in Fetal Growth
This study wants to learn how a type of fat in the blood, called triglycerides, affects how the fetus grows. |
| 18-2740 |
Study on the Safety and Effectiveness of a New Oral Drug Combination Therapy to Treat Bone Cancer in Children and Adults
This study wants to find a new combination of drugs that is safe and effective to treat people who have bone cancer that has come back or not responded to other treatments. |
| 23-1018 |
Physical Health and Wellness in Teens
In this study, we want to learn about the effects of physical activity on the health of teenagers. We will compare the physical, mental, and emotional health of teens who take part in physical activities with those who do not during a fall semester (10–14 weeks). |
| 15-2170 |
Human Trisome Project
This study wants to better understand Down syndrome and related health problems by comparing blood and saliva samples from people with and without Down syndrome. Right now, we are mainly looking for people who do not have Down syndrome but do have chronic constipation to join our study. |
| 13-2102 | |
| 25-1531 |
REST: Improving Sleep Health and Timing to Support Health in Teens
The purpose of this study is to learn if improving sleep habits and sleep timing can help improve blood sugar and heart health in teens who do not get enough sleep. |
| 23-2499 |
Preventing Mood, Anxiety, and Trauma Symptoms After Cesarean Delivery
This study will look at whether a one-time session can help you prepare for delivery, reduce concerns about giving birth, and improve perinatal mood, anxiety, and trauma symptoms (PMATS). |
| 25-2690 |
A Study of Montelukast for Kidney and Heart Health in Young Adults with Type 2 Diabetes
The goal of this study is to learn more about inflammation and how well the kidneys and blood vessels work in young adults with type 2 diabetes (T2D). |
| 24-1783 |
Emotion Regulation and Executive Functioning Intervention in treatment of child SITBs
This intervention development and proof-of-concept study addresses the need for a developmentally tailored, flexible intervention that targets risk factors and features of children with SITBs. We will systematically and iteratively adapt and combine existing EBIs for children targeting emotion dysregulation and executive functioning for SITBs and evaluate feasibility and safety of SURE-Kids, as we... |
| 23-2367 |
Comparing Medications to Treat Eosinophilic Esophagitis in Children and Young Adults
In this study we want to compare how well two different drugs work to treat kids and young adults that have a narrow or stiff esophagus because of a health problem called eosinophilic esophagitis. |
| 18-0461 |
Safety and Effectiveness of Surgical Treatment for Left and Right Congenital Diaphragmatic Hernia
In this study we want to learn about how safe and how well a treatment called Fetoscopic Endoluminal Tracheal Occlusion works to treat babies that have a condition called right and left Congenital Diaphragmatic Hernia. |
| 25-1919 |
Study of Targeted Drug Treatment for Kids and Young Adults with Newly Diagnosed Type 1 Diabetes
In this study we are testing how two drugs, Thymoglobulin ® and verapamil, work together to treat children and young adults with newly diagnosed type 1 diabetes. |
| 23-0755 |
Improving Treatment for Pulmonary Arteriovenous Malformations
In this study we want to collect baseline data on how well treatment works for kids and adults with pulmonary arteriovenous malformations. |
| 24-0061 |
New Device to Manage Insulin Delivery for Kids and Adults with Cystic Fibrosis Related Diabetes
In this study we want to learn how a device that automates insulin delivery (called the iLet Bionic Pancreas) helps people with Cystic Fibrosis Related Diabetes manage their insulin levels. We want to learn how well this device works compared to a normal insulin delivery method. |
| 24-1750 |
Oral Venetoclax Combined with Chemotherapy to Treat Children with Acute Myeloid Leukemia
In this study, we want to learn whether adding a new oral drug called Venetoclax to standard chemotherapy affects how long children and young adults with acute myeloid leukemia (AML) stay in remission and what side effects they experience. |
| 25-1807 |
A Study Testing if SAB-142 Can Slow the Progression of Type 1 Diabetes in People Recently Diagnosed
This study wants to see if a new drug called SAB-142 in addition to regular insulin therapy can help slow down Type 1 Diabetes (T1D) in people who were diagnosed in the last 100 days. |
| 25-0997 |
Creating a Digital Tool to Support Teen Mental Health
In this study, we want to learn how teens handle stress, deal with feelings of depression, and manage habits like eating, exercise, and sleep. We will use what we learn to make a Digital App that helps teens struggling with these challenges. |
| 24-2267 |
Understanding Health Problems Connected to Cystic Fibrosis Related Diabetes (SPeCTRuM)
In this study we want to learn more about how common health problems like high blood pressure, vision problems, and kidney disease are in people with cystic fibrosis-related diabetes (CFRD). |
| 24-1490 |
Impact of Sleep Health in Adolescents with Type 1 Diabetes
In this study we want to see if improving the amount and quality of sleep will help improve the overall health of adolescents that have type 1 diabetes (TID). |
| 24-2571 |
Methods for Improving Sleep for Concussion Recovery
In this study we want to determine the best intervention for improving sleep quality in people recovering from a concussion. To do this we are comparing two sleep interventions and seeing how the impact the recovery time for a concussion. |
| 22-0998 |
CAR T-Cell Treatment for Children with Refractory or Relapsed B-Cell Acute Lymphoblastic Leukemia
In this study we want to learn more about a new treatment called CD19x22 CAR T cells. We want to see how well it works as a treatment for B-cell Acute Lymphoblastic Leukemia (B-ALL) when the cancer has come back after treatment or hasn’t responded to previous treatments. |
| 24-0387 |
Learning More About Achondroplasia (Biobank Study)
In this study we want to learn more about achondroplasia and the genetic factors and health outcomes linked to it. To do this, we are collecting DNA (from saliva samples) from people who have achondroplasia and their family members. |
| 23-0690 |
Effect of Protein Supplement on Liver Fat in Adolescents
This study wants to learn if taking a protein supplement drink twice a day for 8 weeks can help lower the amount of fat that is stored in the liver. |
| 23-1536 |
Improving Kidney Transplant Outcomes in Children and Adolescents
In this study, we want to see if two anti-rejection drugs (belatacept and sirolimus) can better protect your new kidney after kidney transplant, compared to the currently used drugs (tacrolimus and MMF). |
| 23-0839 |
Safety and Effectiveness of a New Drug Treatment for IC-MPGN
In this study, we want to learn more about a new drug called iptacopan to see if it can help treat a rare kidney disease called IC-MPGN (immune complex mediated membranoproliferative glomerulonephritis). |
| 21-4941 |
Semaglutide as a Treatment for Adolescents and Adults with Polycystic Ovary Syndrome (PCOS)
In this study we want to learn if a medicine approved for diabetes and weight loss called Ozempic® and Wegovy® (semaglutide) can help improve the frequency of periods and the way the body uses sugar in women with polycystic ovary syndrome (PCOS). |
| 22-0180 |
Cognitive-Behavioral Therapy and Exercise Training in Teenage Girls At-Risk for Type 2 Diabetes
Our team is here to find out if taking part in a cognitive-behavioral therapy group, exercise training group, or combination of therapy and exercise training lowers stress, improves mood, raises physical activity and fitness, and lowers diabetes risk among teen girls at risk for diabetes. |