To discover the efficacy of a multidimensional sleep health intervention on concussion symptom resolution time and sleep health assessments following adolescent concussion, compared to SOC sleep health guidance. To discover the efficacy of a multidimensional sleep health intervention on concussion symptom resolution time and sleep health assessments following adolescent concussion, compared to SOC...
We are studying how a type of chemotherapy called doxorubicin affects the heart and arteries. We are looking for lymphoma survivors and healthy adults to join this study.
This study plans to learn more about psychological interventions that can be used to address anxiety for youth with at high risk for type 1 diabetes and their caregivers. Anxiety is a common reaction to learning you or a loved one are at risk for type 1 diabetes.
This study looks at how choline supplements and exposure to cannabis-based products affect pregnancy and child health.
This study plans to learn more about how providing interventions in the home during family meals affects the health of children. We want to learn how we can best educate families about things that affect the health of children.
To determine the safety of targeted immunotherapy with daratumumab (DARA) IV after total body irradiation (TBI)-based myeloablative conditioning and allogeneic hematopoietic cell transplantation (HCT) for children, adolescents, and young adults (CAYA) with high-risk T-cell acute lymphoblastic leukemia (T-ALL) or T-cell lymphoblastic lymphoma (T-LLy).
The objective of this study is to evaluate disease progression, using multiple modalities, in subjects with genetically-confirmed STGD1.
To understand variations in the health of people with achondroplasia
This is a study wants to find out if the birth control pill Slynd affects bone health in adolescent girls ages 14 to 17.
Can ADAP assay accurately detect GADA, IA2A, IAA and ZnT8A in self-collected capillary dried blood spot specimens.
This study will evaluate the effectiveness and safety of risdiplam (Evrysdi®) administered to patients under 2 years of age genetically diagnosed with spinal muscular atrophy (SMA) and 2 SMN2 copies who have previously received onasemnogene abeparvovec.
To compare whether de novo use of belatacept and sirolimus versus tacrolimus and MMF maintenance therapy will prevent the development of DSA or maintain optimal graft function (eGFR).
Primary objective: To assess the persistence rate of patients undergoing pulmonary arteriovenous malformation (PAVM) embolization at multiple hereditary hemorrhagic telangiectasias (HHT) centers of excellence in the United States using standardized reports. Second objective: To assess the impact of technical factors like the type of embolic device and embolization technique (feeding artery vs. ve...
ECHO is a national research initiative sponsored by the National Institutes of Health (NIH) to determine what factors give children the highest likelihood of achieving the best health outcomes over their lifetimes.
To demonstrate the efficacy of different doses of frexalimab in comparison with placebo and in addition to standard of care (SOC) on endogenous insulin secretion in participants (12-21 y.o.) with newly diagnosed T1D over a 52-week period
This study is testing whether taking an essential amino acid (EAA) supplement for 8 weeks will lower fat in the liver compared to a placebo.
To evaluate the effect of three doses of Diamyd compared to placebo in terms of (1) beta cell function; and (2) glycemic control in adolescents and adults recently diagnosed with type 1 diabetes (T1D), who carry the human leukocyte antigen (HLA) DR3-DQ2 haplotype and have antibodies against GAD65.
To demonstrate the superiority of iptacopan compared to placebo in reducing proteinuria at 6 months of treatment
• Examine the clinical efficacy of 1000 mg/m2 /day of oral DFMO to stabilize or improve loss of β cell function in persons with recent onset T1D. Our primary endpoint defining clinical efficacy will be based on mixed-meal stimulated C-peptide AUC in the treatment group compared to placebo after 6 months of DFMO treatment. • Examine the safety and tolerability of 1000 mg/m2 /day of oral DFMO i...
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.
To evaluate the safety and effectiveness of the Spinal Kinetics M6-C™ artificial cervical disc in patients with contiguous twolevel symptomatic cervical radiculopathy with or without cord compression.
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.
The primary objective of this Phase I open-label study is to assess the safety and tolerability of CD19x22 CAR T cells in pediatric patients with R/R B-ALL.
This study seeks to better understand the effects of cannabis use during adolescence on decision making and reward responsivity and may identify biomarkers that help distinguish youth who experience impairments in decision making and are at higher risk of the negative effects of cannabis use.
This study is designed as a real-world evaluation of two integrated treatments for substance use and trauma, Risk Reduction Through Family Therapy (RRFT) or Encompass Treatment for adolescents with co-occurring Substance Use Disorder (SUD) and symptoms of Post-Traumatic Stress Disorder (PTSD).
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.
The proposed project presents an opportunity to expand use cases for a social engagement virtual reality (VR) program. The purpose is to assess the feasibility, usability, and acceptability of VR for anxiety symptoms associated with social interactions in public places, using a technology developed by Rey (previously Oxford VR or OVR). This project is a first step toward understanding new populati...
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).
This study wants to learn more about hearing loss using samples from a child, both of their biological parents, and/or other family members. We will use the samples to study heritable risk factors for hearing loss and changes in gene expression that lead to hearing loss and/or ear abnormalities.
This study wants to collect medical information from individuals 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.
This study wants to learn how a type of fat in the blood, called triglycerides, affects how the fetus grows.
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).
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.
This study wants to learn about how total joint replacements affect the lives and physical abilities of children and young adults as they grow older.
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.
The purpose of this study is to investigate high frequency ultrasound as a future modality for the diagnosis of chronic exertional compartment syndrome (CECS).
Our goal is to determine if 12 sessions of Parent Management Training can reduce anger outbursts and irritable moods in children aged 10-14.
To compare the physical, mental, and emotional effects of high school girls' flag football participation to those of high school girls who do not participate in organized sport over a 9 month period.
The primary objective of this study is to evaluate the efficacy of subtypeselective Janus kinase (JAK) inhibitors in the preservation of β cell function in participants with recent onset Stage 3 T1D (diagnosed within 100 days)
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.
The purpose of this study is to determine how the body's immune system interacts with nuts and other foods to cause allergic reaction. This study also plans to validate a test that is being developed to help predict reactions to peanuts.