The Jazz Piccolo Study

Is your child suffering from seizures and less than 2 years of age?
If so, your child may qualify for the Jazz Piccolo Study!

The Jazz Piccolo Study (Piccolo means “little one” in Italian) is investigating the safety and effectiveness of cannabidiol (or CBD oral solution) in infants (2 years of age and younger) with Tuberous Sclerosis Complex (TSC), Lennox-Gastaut Syndrome (LGS) or Dravet Syndrome (DS) who experience inadequately controlled seizures.

TSC, LGS and DS are rare, early-onset epilepsies with life-long impact. Current treatment options for seizures associated with these conditions have substantial limitations, including poor seizure control and numerous side effects. Studies have shown that poor seizure control leads to additional health issues and a lower quality of life for those affected. Therefore, early treatment and control of seizures may be critical in improving the outcomes for children with these conditions.

To find out if your child may be eligible contact a study site location near you.

The drug as used in this study is considered investigational. It is a cannabidiol (or CBD oral solution) that is taken twice daily and has been approved by the U.S. Food and Drug Administration for the treatment of seizures associated with LGS, DS, or TSC in patients 1 year of age and older. It’s also approved in the European Union (EU) & Great Britain for adjunctive use to treat seizures associated with LGS and DS, in conjunction with clobazam, in patients 2 years of age and older, and for adjunctive use to treat seizures associated with TSC in patients 2 years of age and older. The cannabidiol in the investigational drug (known as GWP42003-P) is extracted from cannabis plants with a minimal amount of the component which causes a ‘high’ (known as tetrahydrocannabinol or THC).

What is Lennox-Gastaut Syndrome?

What is Tuberous Sclerosis Complex?

What is Dravet Syndrome?

What is Lennox-Gastaut Syndrome?
Lennox-Gastaut Syndrome (LGS) is a severe epilepsy syndrome that develops in young children and often leads to lifelong disability. Nobody is born with LGS. It develops over time. LGS is a rare disease (approximately one person in every 2,000). About 50,000 people in the United States and 1 million people worldwide have LGS. For more information on LGS, please visit www.lgsfoundation.org.

What is Tuberous Sclerosis Complex?
Tuberous Sclerosis Complex (TSC) is a genetic disorder that causes tumors to form in many different organs, primarily in the brain, eyes, heart, kidney, skin and lungs. The aspects of TSC that most strongly impact quality of life are generally associated with the brain: seizures, developmental delay, intellectual disability and autism. At least two children born each day will have TSC. Current estimates place tuberous sclerosis complex-affected births at one in 6,000. Nearly 1 million people worldwide are estimated to have TSC, with approximately 50,000 in the United States. For more information on TSC, please visit www.tscalliance.org.

What is Dravet Syndrome?
Dravet syndrome is a rare type of epilepsy that starts in the first year of a baby’s life. Its first occurrence is usually a long-lasting seizure (more than five minutes) that’s triggered by a high fever. Children with Dravet syndrome have a wide range of seizure types and severity. They also have many other symptoms, including developmental setbacks, speech and language problems, and balance and walking issues. Researchers estimate that between one in 15,700 and one in 40,000 infants born in the U.S. have Dravet syndrome. For more information, please visit www.dravetfoundation.org.

What do I need to know about the Study?

The Jazz Piccolo Study will involve up to 10 visits to a study clinic and 12 telephone calls and will include a:

4 week screening/baseline period
52-week treatment period
10-day taper following completion of the 52-week treatment regiment
4-week safety/follow-up period

Children who are eligible and participate in the Jazz Piccolo Study will receive the investigational drug, as well as study-related visits, tests and assessments, at no cost. Participants can stop taking part in the clinical trial at any time without giving a reason. Caregivers of participants may also be reimbursed for some study-related expenses, such as costs associated with travel and hotels.

The research team will be able to explain more about what the Jazz Piccolo Study will involve, and it is up to you to decide if you want your child to take part. Participation in this study is voluntary. Your decision to participate or not participate will have no effect on the medical care your child receives now or in the future.

Is my Child Eligible to Participate in the Jazz Piccolo Study?

Here is a list of key Inclusion/Exclusion Criteria to participate in the study:

KEY INCLUSION CRITERIA

Children with confirmed:

– Tuberous Sclerosis Complex: 1 month to < 2 years of age; or
– Dravet Syndrome: 1 year to < 2 years of age, or
– Lennox-Gastaut Syndrome: 1 year to < 2 years of age

Has seizures not adequately controlled through their current antiseizure medications
Currently receiving 1 or more antiseizure medications
A suitable Video EEG or VEEG for confirmation of diagnosis; VEEG is short for a video EEG (electroencephalograph) which records a patient’s experience on video while an EEG test records brainwave function

KEY EXCLUSION CRITERIA

Clinically significant unstable medical condition other than epilepsy
Clinically significant symptoms or illness which could affect the seizure frequency
Has undergone surgery for epilepsy within 6 months
Significantly impaired hepatic (liver) function
Currently using recreational or medicinal cannabis, cannabinoid-based medications or CBD

Frequently Asked Questions

What is a study?

A study (also called a clinical trial) is a medical investigation that helps to answer important questions about an investigational medication, including how well the medication works for a certain condition and how safe and tolerable the medication is. All medications must be tested in clinical research studies before they can be approved by regulatory authorities and prescribed to patients.

Why are studies important?

A study is the process by which new and innovative medications, interventions, and treatments are approved and brought to the general public, so people can hopefully live happier and healthier lives. Even over-the-counter medications, such as aspirin that you may have used for a headache, have gone through the clinical research study process before they were available at your local pharmacy. By enrolling in a study you have the opportunity to help both your child and other children who have the same medical condition.

What is the purpose of the Piccolo study?

The purpose of this study is to find out how well GWP42003-P (the study medicine) works, and if it is safe to use in children less than 2 years of age with uncontrolled seizures due to Tuberous sclerosis complex (TSC), Lennox-Gastaut syndrome (LGS) or Dravet syndrome (DS).

What will the Piccolo study involve?

This clinical study involves 3 stages, (1) screening, (2) treatment, and (3) end of treatment.
The first visit will be a visit to the study center. For the second visit, you will receive a telephone call from the study staff. During these visits, the study staff will ask you about your child’s medical history, epilepsy history, demographic data, any medications your child may be taking, and any anti-seizure medication, rescue medication, or vaccination he or she has received.

Some tests will be performed to check that it is appropriate for your child to take part. If the tests show that it is appropriate (known as ‘eligible’), you and your child will be asked to attend up to 9 additional visits at the study site and have up to 11 additional telephone appointments.

Your child may also have a video EEG, which is a test to record the electrical activity of the brain. In certain cases, the video EEG may be collected from your child’s medical record, if it is available.

You will also be asked to complete an electronic daily diary throughout the study to record information on your child’s quality of life, their seizures, any changes in their health, and any other medications he or she may be taking. You will also discuss the seizure types your child is experiencing with the study physician.

If your child is eligible at the beginning of the treatment period, they will be enrolled in the study and be given GWP42003 P (the study medicine). At each visit, the study physician will perform a physical examination, to ensure your child is in good health. Safety tests will be performed, which include measuring your child’s vital signs and collecting blood and urine samples for laboratory safety tests. Your child will have electrocardiograms, which take a tracing of the electrical impulses of their heartbeat.

Extra blood samples at some visits will be taken to test how the study medicine has been absorbed into your child’s body. You will also need to record details of your child’s meals (timing and content) in the electronic diary for 24 hours before each of the visits where certain blood tests known as PK or pharmacokinetics samples are collected.

Your child will also have another video EEG at the End-of-Treatment Visit which will last up to 24 hours depending on how long the investigator feels is necessary, and although unlikely, possibly may require an overnight stay. You will also be asked to use the electronic diary to complete questionnaires on your child’s quality of life.

Will compensation for time and travel be provided?

Caregivers of children who qualify to take part in the study may receive reimbursement for time and travel. Please discuss this with the study team.

Is there a cost to participate?

There is no cost to participate in the Piccolo study. If your child is eligible and you decide to take part:

Your child will receive study-related care from a team of experienced doctors and nurses throughout the study
All study-related visits, tests, assessments, and investigational medication will be provided at no cost to you

What else do I need to consider?

The research team will be able to explain more about what the Piccolo study will involve, and it is up to you to decide if you want your child to take part. Participation in this study is voluntary. Your decision to participate or not participate will have no effect on the medical care your child receives now or in the future. If your child is eligible and you choose to have them participate in the Piccolo study, your child may leave the study at any time without providing any reason.

Where are the study sites located?

The Piccolo study is being conducted across several study sites in the United States. Find a study site location near you.

Study Site Locations >