Clinical Research Study Finder

Children's Hospital of Philadelphia leads or participates in hundreds of clinical research studies and clinical trials. Many of the studies that are currently enrolling patients can be found in this database. Use this finder to learn more about the purpose of these studies and clinical trials, find out who can participate, and tell us you're interested in enrolling. You can also look for studies that offer remote participation from home!

Find a Clinical Research Study

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Designate - Siplizumab in T1DM

The goal of this study is to identify a safe dosing regimen for siplizumab in participants with type 1 diabetes. Siplizumab is a drug used to help treat recent onset type 1 diabetes mellitus (T1DM). This study is interested in participants aged 18-45 years with T1DM, who are within 18 months of receiving their diagnosis. Participants will be randomized (selected at random by the study team) to one of four possible treatment groups. All groups will receive weekly siplizumab doses administered subcutaneously (injected into the body through the skin) for a total of 12 weeks.

RM-493-035: Setmelanotide in Patients with Gene Defects in the MC4R Pathway

The purpose of this study is to find out if an investigational drug called setmelanotide (RM-493) can help control body weight in people with certain genetic modifications in their genes that play a key role in the regulation of body weight. Males and females, age 6 to 65, with obesity and certain genetic variants (MC4R pathway) may be eligible to participate. If you are confirmed eligible for the study, then participation will last up to 64 Weeks. Participants will complete Screening (Up to 8 Weeks), Double-Blind Treatment (Up to 52 Weeks), and Follow-Up Period (4 Weeks). Participants will complete research procedures including physical exams, blood draws, ECGs, and questionnaires. Participant will also need to self-inject the study drug once daily for the duration of their participation.

ThecaFlex Investigational Device for the Treatment of SMA

Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by the degeneration of spinal cord motor neurons, resulting in progressive muscle weakness, paralysis, and atrophy. One treatment option available to patients is called nusinersen. This medication is administered by lumbar puncture (LP; a needle inserted between the bones of your spine) every 4 months after the initial 4 loading doses.
Lumbar puncture, however, can be challenging for some patients due to the presence of spine deformity caused by the disease. Additionally, children usually require anesthesia for this procedure. This study will be utilizing a new investigational research device, called the ThecaFlex DRx (Alcyone Therapeutics) system as an alternative to repeated lumbar puncture.
The ThecaFlex DRx system is a port and catheter that is surgically implanted to provide access to the space between the bones of your spine to avoid the need for repeated lumbar puncture and repeated anesthesia. After the device is inserted, the prescribed nusinersen is administered via the port located just beneath the skin near your abdomen. The catheter guides the medication to the intrathecal space, where it needs to be delivered.
You/your child may qualify for this study if you/they are:
  • diagnosed with SMA and are prescribed nusinersen (Spinraza) treatment
  • older than 3 years of age
  • resistant to lumbar puncture
It is important to note that this study is the first in human for this device and it has not yet been approved by the FDA.

PETITE-T1D

The purpose of this study is to evaluate the safety and pharmacokinetics (drug levels in the blood) of teplizumab in children less than 8 years old. The study will also assess if your child stays in Stage 2 T1D or progresses to Stage 3 T1D. Your child may join the study only if they are in Stage 2 T1D.

Previous studies in adults and children older than 8 years of age suggest that taking teplizumab, a drug that affects your child’s immune system, may stop, delay, or decrease the attack on the cells that make insulin. This slowing or blocking of the immune attack may reduce the risk of short-term and long-term consequences of T1D.

To join this study, your child must have been diagnosed with Stage 2 T1D based on two positive T1D-related antibodies and confirmed dysglycemia (abnormal blood sugar levels) based on an oral glucose tolerance test, fasting plasma glucose test, or an increase in a blood test called the hemoglobin A1c. 

The study is “open-label.” That means that if your child qualifies for the study, your child will receive teplizumab. There is no placebo in this study.

Selpercatinib in Thyroid Cancer (Collaboration with the International Thyroid Oncology Group)

This study involves taking a study drug called selpercatinib. The overall goal of this study is to find out how effective the study drug is in combination with radioactive iodine (I-131) in treating people with thyroid cancer. The study drug will be given in combination with treatments you will receive as part of your regular cancer care (I-131 and thyrotropin alfa). 

Parenting Intervention Study

CHOP PriCARE is a FREE, VIRTUAL workshop for caregivers of children 18 months - 6 years of age. Caregivers attend the workshop for 80 minutes once a week for 6 weeks. Workshops are offered mornings and evenings throughout the year. The goal of the workshop is to teach ways to interact with young children that improve their behavior and build a strong bond between caregiver and child. The PriCARE workshop is a research project with two groups of caregivers. One group attends the workshop, and one group does not attend. Caregivers in both groups are very important for this project. All caregivers will be interviewed and get paid for their participation. To learn how you can participate, call 267-318-1707 or email pricare@chop.edu.


Larotrectinib RAI Thyroid

This study enrolls patients with newly diagnosed differentiated thyroid cancer (papillary thyroid cancer or follicular thyroid cancer) that has spread to the lungs and has a genetic mutation called and NTRK fusion. The study involves taking a study drug called larotrectinib for approximately 6 months prior to receiving radioiodine therapy (RAI). The overall goal of this study is to find out if taking the study drug before RAI works better than receiving RAI alone (the usual standard of care treatment). 

Breath Biomarkers of Mitochondrial Dysfunction

This prospective cohort study will compare breath biomarkers from subjects with mitochondrial disease to healthy controls. The age range for the study is 4-80 years and all subjects will be recruited at CHOP. Participation would consist of a one time visit, up to 2 hours maximum, to collect identifiable information and breath sample. The goal is to identify VOC biomarkers that characterize mitochondrial disease compared to healthy controls, which could lead to a non-invasive breath test for mitochondrial function. Subjects will be compensated as a form of appreciation for participating in the study. 

Down Syndrome and Sleep Apnea Study (DOSA)

This research study is being conducted to look at oxygen as a treatment of obstructive sleep apnea (OSA) in children with Down syndrome. The main reason for the study is to show how 6 months of oxygen treatment while sleeping affects the participants sleep, quality of life, breathing, heart and if it makes the participant feel better.

ABA3 GVHD ProRx post Allo HACT

This study involves taking the study drug, abatacept, by intravenous (IV) infusion. Along with the study drug, participants will take a either cyclosporine or tacrolimus, and methotrexate, which are standard medications for transplant patients. Participants, 6 years of age and older, will be randomized (placed into a group by chance) by the study team. After being placed into a group, you will receive additional doses of either abatacept (the study drug) or a placebo (an inactive substance). 


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