Research Studies Finder

The purpose of this study is to find out more about mRNA-3705, a study drug that is being tested and is not approved by the U.S. Food and Drug Administration (FDA). In this study you will receive mRNA-3705, which is being developed to treat people with isolated MMA with MUT deficiency mRNA-3705. mRNA-3705 can only be used in a study like this one. We want to see what dose of the study drug is safe, how the study drug affects the function of cells, and how much study drug is in the blood after patients receive it.

This study will provide early access to an investigational drug which does not yet have marketing authorization for human use, Arimoclomol, for a group of patients suffering from Niemann-Pick Disease Type C that are not eligible for or able to participate in other clinical trials.

CReWS (the Collaborative Registry for Williams Syndrome) has been developed for, and is owned by the Williams Syndrome Association. The Registry collects data about the lives of people diagnosed with Williams syndrome (WS) in order to advance our understanding of the condition and its underlying causes, and develop better treatments for persons with Williams syndrome.CReWS collects and organizes information & medical records on people with Williams syndrome and connects families to doctors and scientists currently researching the condition. All participants will be invited to answer surveys on medical, developmental, and social concerns beginning at the time of registration and continuing periodically going forward. For those who attend WS Clinical Consortium sites, the medical providers will add clinical data to their research records. We expect the registry will:

• Foster & facilitate research into medical, developmental, and social concerns for those with Williams syndrome.
• Collect reliable and complementary data from both clinicians and families.
• Establish best practices for care of children and adults with WS.
• Gather longitudinal data to better understand long-term outcomes.
• Establish a large study cohort for a rare condition.
• Support research in WS by more easily connecting those who conduct research, with individuals who are interested in participating in research.

HIV infection can cause harm to the brain and negatively affect memory, learning, and decision making in daily activities. Smoking may affect the ability of medications to reduce HIV in the bloodstream (viral load), which may affect long-term survival and brain health. This study will look for changes in the brain that might have occurred due to HIV infection or smoking.

Our team has partnered with Moderna on a clinical trial for patients with glycogen storage disease type 1a (GSD1a). We would like to invite you to take part in a clinical trial for an mRNA investigational drug that could potentially correct the cause of GSD1a, by teaching your body to break down glycogen, correct low glucose, and avoid starch intake. The purpose of this trial is to see if a new investigational mRNA study drug, called mRNA-3745, is safe and if it works in the way researchers expect. mRNA technology uses messenger ribonucleic acid (mRNA), an instructional molecule that naturally occurs in the body and carries information to cells. In this case, we are studying if mRNA-3745 given through an IV infusion can instruct the body to make the protein that is missing in people with GSD1a.

mRNA-3725 is not approved by the FDA; the FDA has allowed us to provide this investigational medicine to patients enrolled in this phase 1/2 trial. Phase 1/2 clinical trials like this one aim to test the safety, side effects, and best dose of an investigational medication. In the phase 2 part, participants usually receive the highest dose of treatment discovered in the phase 1 part. This is the first study in which humans are administered this study drug.

If successful, mRNA-3745 would enable certain organs in the body to effectively break down glycogen and avoid low glucose levels and starch intake. If mRNA-3745 could correct the cause of GSD1a, it would help keep your glucose levels where they need to be all the time.

Participation in this clinical trial could last up to approximately two years, depending on which stage you enroll in and whether or not you decide to participate in the long-term follow-up period of the study. Some of the visits in this trial will require you to stay overnight at the hospital, and some of the visits will require you to visit the hospital or doctors office. Most of the visits in this clinical trial can be done at your home, where a home health nurse will perform the assessments needed.

All study-related procedures, travel, and compensation are provided.

The first few years of life are a time of rapid growth and brain development. The Healthy Brain and Child Development (HBCD) Study will help us better understand how a child may be shaped by experiences and conditions in pregnancy and early life. These include social and environmental experiences in early life and exposure to substances (such as opioids, alcohol, tobacco, and cannabis) during pregnancy. The HBCD Study will enroll a large cohort of participating families from across the U.S. and follow them and their children through early childhood. The researchers want to better understand how the brain develops and is affected by exposure to substances and other environmental conditions during pregnancy and after birth.

The Children's Hospital of Philadelphia is recruiting teen drivers to participate in a study that is testing different types of driver training program for reducing new driver crash risk. This study is open to teens between the ages of 16-17 years old who already have an active Pennsylvania learner permit.

Researchers at CHOP are trying to improve outcomes in the treatment of adolescents with anorexia nervosa. Eligible families will receive Family Based Treatment for anorexia nervosa. Some adolescents with anorexia nervosa will also receive Cognitive Remediation Therapy (a treatment that increases flexible thinking). Both parents are required to participate and any siblings living at home are asked to come to the first four treatment sessions. Families will receive treatment for approximately 6 months and will be asked to complete number of assessments during treatment and at two time points after treatment ends.

The Childrens Hospital of Philadelphia is seekinghealthy adolescents between the ages of 12-13, who are currently in the 7^thgrade,to participate in the Sleep and Growth Study 2. The purpose of this research is to learn more about the changes in sleep and body size in adolescents as they transition from middle to high school. Participants will be asked to visit CHOP so that we can measure their bone density. Following this visit, participants will wear a sleep tracker over a 2-week period and speak to a team member by phone on three occasions about the types of food eaten in the previous 24 hours. If you and your child agree to participate, the study will involve 3 visits over approximately 2 years. All participants are compensated for their time and effort.For more information and study details, please call 215-590-2383, ext. 1, or emails_grow@chop.edu.

PEDSnet and DBPNet seek youth (13-24), parents, and clinicians/scientists for the Intellectual and Developmental Disabilities (IDD) Research Prioritization Study. The goal is to develop a prioritized list of research questions that will immediately impact patient health outcomes, focusing on children with IDD.
Participants will answer open-ended questions on Viva Engage, a Microsoft platform, to share their thoughts and opinions. The study is entirely online, and parents and youth will be compensated for their time. To participate or learn more, complete our eligibility screener.