Honeycomb: Evaluation of Radiprodil in Children with GRIN-related Disorder
The Honeycomb study is looking at a new medicine called Radiprodil for children (aged 6 months to 12 years) with a rare genetic condition known as GRIN-related disorder. This condition can cause serious symptoms like seizures or behavioural issues. The study's main goals are to check if Radiprodil is safe for these children and to learn how their bodies react to the medicine. Researchers also want to see if it might help ease their symptoms. All children taking part will receive Radiprodil, and the doctors will carefully adjust the dose for each child. The study has two main parts: an initial period of up to six months and a longer-term period for those who wish to continue. This is a chance to explore a potential new treatment for children living with GRIN-related disorder.
At a glance
What is this study about?
The Honeycomb study is focused on helping children with a rare genetic condition called GRIN-related disorder. This condition is caused by a change in a specific gene, which can lead to problems like uncontrolled seizures or difficulties with behaviour. Currently, there aren't many treatments specifically for GRIN-related disorder, so finding new options is very important.
This study is testing a new medicine called Radiprodil. Researchers want to understand if Radiprodil can safely help children with this condition. They will watch closely for any side effects and learn how the body processes the medicine. They also hope to see if Radiprodil might improve the children's symptoms, whether it's helping with seizures or behavioural challenges.
Because this is a very early stage study (called Phase 1B), all the children involved will receive the new medicine, Radiprodil. This helps the medical team learn as much as possible about it. The study is designed to be very careful, with doctors adjusting the dose of the medicine specifically for each child to find what works best and safely.
Key takeaways
- Targets children aged 6 months to 12 years with GRIN-related disorder.
- Tests a new medicine called Radiprodil for safety and potential help with symptoms.
- All participating children will receive the study medicine.
- Study involves careful dose adjustment and close monitoring.
- Initial participation lasts up to six months, with an option for longer-term treatment.
- Participation is voluntary and you can withdraw at any time.
Who may be eligible?
To be considered for this study, children need to be between 6 months and 12 years old and have a GRIN-related disorder caused by a specific type of genetic change. This change means their bodies have too much activity in a certain part of the brain.
Children in the study will fall into one of two groups: either they experience at least one seizure every week that hasn't been well controlled by at least two other seizure medicines, or they have significant behavioural problems or movement issues that their caregiver has noticed. It's important that any other treatments your child is already receiving for their seizures or other conditions are stable and won't interfere with the new medicine.
Children cannot join if they have other serious medical, brain, or mental health conditions not related to GRIN-related disorder, or if they have severe liver problems. They also can't take part if they've had brain surgery for epilepsy or are currently taking certain other medicines that might react badly with Radiprodil, such as particular types of seizure medicines or hormone treatments like steroids.
Could this study suit you?
Answer these quick questions to see if you may be eligible. This is a guide only — the research team makes the final call.
- Is your child between 6 months and 12 years old?
- Does your child have a GRIN-related disorder with a specific genetic change (GoF)?
- Does your child have at least one observable seizure per week (not controlled by at least two other medicines) OR significant behavioural/movement problems?
- Is your child's current seizure or other medical treatment stable?
- Has your child had brain surgery for epilepsy?
- Is your child currently taking medicines like felbamate, memantine, perampanel, or hormone therapies like prednisolone?
What does participation involve?
If your child takes part in the study, their journey will usually last up to six months initially, with the option to continue for longer. It starts with a "screening" period (about 35 days) where doctors check if your child is suitable and observe their seizures or behaviour for four weeks.
Next is the "titration period" (about 51 days), where your child will stay overnight at the hospital. They'll start taking Radiprodil twice a day, and the dose will be slowly increased until the doctors find a safe and potentially effective amount. During this time, they’ll regularly check your child’s blood to see how much medicine is in their system and watch for any side effects.
Once the right dose is found, your child will enter the "maintenance period" (up to 53 days), continuing on that dose. This part also includes another overnight hospital stay at the end. After this, you'll have the choice for your child to join Part B, a longer-term treatment plan. If you choose not to continue, there's a 15-day period where the medicine is gradually reduced, followed by a final safety check-up 14 days after the last dose.
Potential risks and benefits
Locations (15)
- Mid-Atlantic Epilepsy and Sleep CenterVerified postcodeBethesda, United States
- Columbia University Irving Medical Center, Dept of NeurologyVerified postcodeNew York, United States
- Queensland Children's HospitalVerified postcodeSouth Brisbane, Australia
- The Hospital for Sick Children (Sick Kids)Verified postcodeToronto, Canada
- BC Children's HospitalVerified postcodeVancouver, Canada
- Abteilung für Neuropädiatrie, Klinik und Poliklinik für Kinder - und Jugendmedizin, Universitätsklinikum LeipzigVerified postcodeLeipzig, Germany
- KBO-Kinderzentrum München gemeinnützige GmbHVerified postcodeMünchen, Germany
- Istituto di Ricovero e Cura a Carattere Scientifico (IRCCS) - Ospedale Pediatrico Bambino GesuVerified postcodeRome, Italy
- Azienda Ospedaliero Universitaria Careggi (AOUC) Firenze - Azienda Ospedaliera Universitaria MeyerVerified postcodeFlorence, Italy
- ERASMUS Medisch Centrum, Developmental & Genetic pediatricsVerified postcodeRotterdam, Netherlands
- UMC Utrecht - Wilhelmina Kinderziekenhuis, Polikliniek KinderneurologieVerified postcodeUtrecht, Netherlands
- Universitat de Barcelona - Hospital Sant Joan de Deu Barcelona (HSJDB)Verified postcodeBarcelona, Spain
Common questions
What is GRIN-related disorder?
It's a rare genetic condition caused by a change in a specific gene, leading to symptoms like seizures or behavioural difficulties in children.
What is Radiprodil?
Radiprodil is a new medicine being tested in this study to see if it can help children with GRIN-related disorder.
Will my child definitely get the new medicine?
Yes, in this study, all children who participate will receive Radiprodil, and the dose will be carefully adjusted for them.
How long will the study last for my child?
The first main part of the study is expected to last up to six months, with an option to continue in a longer-term treatment period.
Can I take my child out of the study if we change our mind?
Yes, you can withdraw your child from the study at any time, and it will not affect their regular medical care.
How to find out more
Always speak to your GP or specialist before deciding to take part in a study.
Discussion
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