The Pediatric Clinical Trials Unit offers research for all ages not just children and adolescents. We specialize in rare and genetic diseases. Before clinical trials are performed with children/adolescents they are first tested with adults to determine the safety, efficacy, and dosage of the treatment. UConn Health has partnered with Connecticut Children’s to be able to provide the best research and safety facility for our subjects.
Dr. Rebecca Riba-Wolman, M.D., Pediatric Endocrinologist, Connecticut Children’s
Dr. Emily Germain-Lee, M.D., Chief, Division of Pediatric Endocrinology, Connecticut Children’s Medical Center
Shaylee King, M.S., Study Coordinator, UConn Health
Julieta Bonvin-Sallago, M.S., Study Coordinator, Connecticut Children’s
Julie Vigil, M.S, MPH, CHC, CHRC, Administrative Director, UConn Health
Malaya Mount, Registered Dietician, Connecticut Children’s
Amber Barry RN, BSN
Shila Bhandari RN
Iris Ferrecchia RN
Love Joy Melendez RN, BSN
Stephanie Ruggiero, RN, BSN
A Phase 3, Randomized, Double-blind, Placebo-controlled Study of Adeno-associated Virus Serotype 8-mediated Gene Transfer of Glucose-6-phosphatase in Patients With Glycogen Storage Disease Type Ia
*Recruiting Soon*-An Open-Label Dose-Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of mRNA-3745 in Participants with Glycogen Storage Disease Type 1a (GSD1a)
Long-Term Follow-up to Evaluate the Safety and Efficacy of Adeno Associated Virus (AAV) Serotype 8 (AAV8)-Mediated Gene Transfer of Glucose-6-Phosphatase (G6Pase) in Adults with Glycogen Storage Disease Type Ia (GSDIa)
ClinicalTrials.gov Identifier: NCT03970278
(Active - no longer recruiting)
Safety and Dose-Finding Study of DTX401 (AAV8G6PC) in Adults with Glycogen Storage Disease Type Ia (GSDIa)
ClinicalTrials.gov Identifier: NCT03517085
(No longer recruiting)
The Pediatric Clinical Trials Unit is always adding and looking to add more studies. If you are potential subject or a sponsor looking for a site to run your clinical trial please contact PediatricClinicalTrials@uchc.edu for more information.
What Are Clinical Trials?
Clinical trials are highly regulated research studies that help to determine the safety, efficacy and tolerance of an investigational product such as drug or device. These studies are performed by using volunteer patients who agree to participate and are followed by a highly trained team of doctors and other study staff. If you have more questions about Clinical Trials please visit www.ClinicalTrials.gov for more information.
In the News
First Patient Receives Novel Gene Therapy for GSD
UConn Health Center was the first site to infuse a subject across the globe with a novel gene therapy. This trial was the first gene therapy for the treatment of Glycogen Storage Disorder Type Ia.
If you would like to learn more and see if you or a loved one may qualify for one of our clinical trials please email us at PediatricClinicalTrials@uchc.edu or contact Shaylee King at 860-679-6584, firstname.lastname@example.org.
Answers to Common Questions on Clinical Trials
What are the phases of clinical trials?
There are several phases of clinical trials:
Preclinical/Phase 0 - testing the Investigation Medicinal Product (IMP) (new drug or device) in non-human subjects, such as animals. This helps to determine preliminary safety and efficacy data. This is also when scientists are able to determine how long the drug or device can stay in the body at a therapeutic level.
Phase I/II - can sometimes be done in conjunction with one another. These phases help to determine the dose levels for maximum therapeutic effect. Additionally, this is when side effects are monitored and documented for severity and safety.
Phase III - these trials are done with similar set ups of that of phase II trials but these are conducted with more participants to continue to monitor efficacy and safety of the IMP.
Phase IV - the FDA (or other governmental regulatory authority, depends on the country) has approved and released the IMP to the public and it is continued to be monitored in larger numbers of patients. You would be able to get this product from your own Doctor.
What protections are there for people who participate in clinical trials?
All clinical trials are performed under the surveillance of an Institutional Review Board (IRB). The purpose of an IRB is to ensure that all research is done in an ethical manner and that all information provided to subjects is presented in a clear and concise manner. During the time of consent, you will be given the information on who to contact at the UConn Health IRB, if you were to have questions on the specific study you have enrolled in.
What is Informed Consent?
Informed Consent is a form and a process to which all research done with humans includes. This means that when you come to a research site, the first thing you will do is go over the informed consent forms with the study team. The forms will outline exactly why the study is occurring, and what will be asked of you throughout the duration of the trial. You are free to ask as many questions as you would like and also, if after everything is explained, you decide the trial is not something you would like to do, you do not have to sign it. There will be no consequences to not signing. Moreover, throughout the duration of the trial, after consent is signed, you can withdraw from the study at any point, without any fear of consequences.
Who can participate?
All clinical trials have to follow a protocol (a list of instructions for all study sites performing that trial). Within the protocol, there is a list of inclusion and exclusion criteria that study staff must follow. If you meet all of the inclusion criteria and do not match any of the exclusion criteria, then with your consent, you will be eligible to participate in the study. This is determined through a prescreening phone call and a screening visit with the study team. You will notified if you do not meet the criteria to be able to participate in the study.
Who will I be meeting with?
You will be meeting with the study doctor, and their staff. The staff includes the Study Coordinators and Study Nurses, who are well versed in the study procedures and will be able to answer any questions you may have throughout the trial. The Study Coordinators will be your main contact throughout the duration of the trial, they will be able to help you for any study related matters.
What happens if I want to participate in a clinical trial?
If you find a clinical trial that you may qualify for. Your first step should be to reach out to the contact person for the study. With your consent they will ask you some qualifying questions over the phone to see if you meet the basic criteria for enrollment. If you do, you will be brought to the site for what is known as a screening visit. During this visit you and the study team will review the informed consent forms. If consent is given, some assessments will be performed. This can include a physical exam, blood/urine labs, an EKG to review your heart, a review of your medical history, and you may be asked to answer some study specific surveys. The study team will walk you through any assessments or questions you may have. Do not hesitate to ask questions!
What is a placebo?
A placebo is an inactive substance such like a pill or a liquid (it will match the form of the IMP). A placebo offers no medical value. It is important to use placebos in research because it helps to compare the data from the placebo group to the experimental group to be able to fully assess the effectiveness of the treatment.
What does Blinded/Open Label mean?
Blinded means that the participant will not know what experimental group they have been assigned too. Double-Blinded means that the participant and the study team are both unaware of what treatment the subject receives. There are some trials that are called Open-Label studies, in these studies, the subject and the study team are aware of the treatment that the participant has been assigned to. Your study team will inform you which type your study falls into.
What are adverse reactions?
An adverse reaction would be anything that would result in an unfavorable change to that participant’s health, this could be due to the IMP or not. Your study team will be documenting any changes to your health through the study (if there are any). Any known adverse reactions or side effects will be told to you during the informed consent process.
What are the benefits and risks associated with clinical trials?
Some of the benefits of participating in a clinical trial is that you may receive a treatment for your condition that may not be widely available to others (at the current moment) and your health will be closely monitored by a study team. A potential risk may be that you receive no treatment for your condition and are assigned to the placebo group. The data collected will still help the overall collective research process and may be able to help others with the same condition. You will still be closely monitored by the study team. You may also experience some side effects or adverse reactions to the IMP. All known side effects or reactions will be outlined in the informed consent forms, and if any new side effects are reported throughout the trial you will be informed. Additionally, during the trial, if you do experience any side effects or adverse reactions, your study doctor will be able to help you. It is critical that you tell your study team how you are feeling at each visit.
What should I know before I join a clinical trial?
Medical advancements through clinical trials cannot happen without the participation of human subjects. By participating in clinical trials you will be a part of something much bigger than yourself, you may have the ability to help yourself and many others. You should understand that your participation is voluntary and is greatly appreciated by everyone, and that the study team is here to help you throughout the trial. Being a part of research is much different than being treated by your normal doctor, so please ask questions when you have them. Lastly, your personal and direct contribution to scientific discovery is greatly appreciated.
Can I leave a trial after it has started?
At any point in the trial, you are able to leave, with no consequences. If you do leave, please inform the study team as to why you are leaving the trial.
Will I be paid for participating?
Most trials will compensate their participants in a few different ways. One of those ways could be with money, or they could cover your transportation, meals, accommodations cost etc. You can ask your study team how/if you will be compensated for a particular study.