Current Clinical Trials

Currently Recruiting Trials

A Randomized Study of BPN14770 in Male Adolescents (Aged 12 to < 18 Years) With Fragile X Syndrome

Description: This is a 2-part study, with each part having a unique set of objectives for male adolescents aged 12 to < 18 years with fragile X syndrome (FXS). Part 1 is an open-label, single-dose, pharmacokinetics (PK) assessment of BPN14770 25 mg and 50 mg, while Part 2 is double-blind (DB) and randomized between two treatment groups (Study Drug and Placebo).

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Assessment of Potential for Chronic Liver Injury in Participants Treated With Epidiolex (Cannabidiol) Oral Solution

Description:  This study will monitor for potential chronic liver injury and liver fibrosis, in participants treated with cannabidiol oral solution.

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The Balance Study

Description:  Bipolar depression can change the way a child or teen interacts with the world around them. If you or your child have bipolar depression, we are inviting you to take part in the Balance Study for children and teens, aged 10 to 17 years of age, who have been struggling with bipolar depression. The purpose of this study is to evaluate an investigational drug for bipolar depression in children and teens. The Balance Study will enroll about 380 children and teens with bipolar depression at participating study centers throughout North America and Europe.

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Does Your Child Have Epilepsy

Description: This is a clinical research study of a medication that may treat both anxiety and epilepsy. The study will be for 16 weeks with 11 study visits.

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Explore our Fragile X Syndrome Clinical Research Trial

Description: The behavioral issues associated with a Fragile X diagnosis can create daily challenges for your child.  Learn more about a clinical trial evaluating investigational topical cannabidiol (CBD) gel to see if it helps improve Fragile X-related behavioral issues.

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Forward-March Fragile X Study

Description: FORWARD-MARCH is the next step following a highly successful research study called FORWARD (Fragile X Online Registry With Accessible Research Database) that was started in 2012. The FORWARD study created the largest database of information on fragile X syndrome (FXS) in the United States. Data from the FORWARD study is being used by researchers to learn about the lives of people with FXS, including medication use, behaviors, and development over time, and share findings with other researchers and clinicians.

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LIFE-DSR

Description:  The Longitudinal Investigation for the Enhancement of Down Syndrome Research (LIFE-DSR) is a multi-year, coordinated research study by medical and academic professionals to track and analyze the medical and physical data of 270 adults with Down syndrome. Because LIFE-DSR is an “observational and natural history” study, it isn’t testing the impact or effectiveness of a certain medication or therapy. Rather, LIFE-DSR “observes” people as they are by recording medical, physical, and behavioral data points, like: height, weight, blood/ plasma samples, key behaviors, sleeping patterns, and blood pressure. Researchers will use the information collected from all study participants, to better understand the clinical profile of people with Down syndrome.

Learning more about the physiology of people with Down syndrome gives researchers better insight into the connection between Down syndrome and Alzheimer’s disease. LIFE-DSR aims to address the questions of why the rate of Down syndrome-related Alzheimer’s disease is so high, and what therapies and treatments can be developed to prevent it.

The people with Down syndrome who take part in LIFE-DSR are known as “participants,” and their caregivers/family members are called “study partners.” All study participants are
attached to a specialized clinical “site” located in different cities across the U.S., and develop an ongoing relationship with the clinical team and coordinators at their site through visits
every 16 months. Individuals 25 years and older can consider joining the study.

We welcome new LIFE-DSR participants, and we hope you will consider signing up! LIFE-DSR depends on volunteers from the community who want to help researchers learn more about Down syndrome.

Scientists want to learn more about how adults with Down syndrome (DS) change as they age. They particularly want to develop tools to measure the changes that are associated with Alzheimer’s disease for people with Down syndrome. Scientists experienced in research are conducting the study, along with participating doctors, and the LuMind IDSC Foundation.

People who choose to participate in the LIFE-DSR study can expect:

1. Three or more visits to your clinic site over two years. At the site, you will meet with your team and the study doctor. You will have a physical exam, including a blood draw.
2. You will be asked to have a conversation called a “cognitive assessment,” and it will help the researchers measure things that can’t be measured with a scale or a tape measure, like memory, language, and the ability to recognize objects.
3. Depending on which site you are connected to, you may be asked to participate in additional tests, such as a PET scan of your brain, a collection of spinal fluid, or a discussion of how you can set goals to work on skills or overcome challenges that are important to you.
4. You and your study partner will learn about measurements and tests available at the site you are connected to. You will have the opportunity to ask questions before you agree to participate. You and your study partner will “give consent” before you join the study.

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Epidiolex® For the Treatment Of Anxiety Comorbidity In Refractory Pediatric Epilepsy (EAPE)

Description:  This research study titled, Epidiolex® For the Treatment Of Anxiety Comorbidity In Refractory Pediatric Epilepsy (EAPE), is being conducted by Dr. Jay Salpekar to evaluate if the drug FDA approved drug, Epidiolex®, helps control anxiety and mood symptoms associated with recurrent seizures in addition to seizure control. The length of the study is 4 months and consists of 11 visits; some are remote and some are onsite.

If you or a family member have any questions or are interested in learning more about the study please contact the study staff by replying to this notice or by contacting the staff directly with the information below.

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Perinatal Arterial Stroke: A Multi-site RCT of Intensive Infant Rehabilitation (I-ACQUIRE)

Description:  Bipolar depression can change the way a child or teen interacts with the world around them. If you or your child have bipolar depression, we are inviting you to take part in the Balance Study for children and teens, aged 10 to 17 years of age, who have been struggling with bipolar depression. The purpose of this study is to evaluate an investigational drug for bipolar depression in children and teens. The Balance Study will enroll about 380 children and teens with bipolar depression at participating study centers throughout North America and Europe.

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