marigold cdkl5 study

Is your child suffering from early onset intractable seizures?

Have they been genetically tested?

If your child tested positive for the CDKL5 gene mutation and is suffering from seizures and behavioral disorders, there may be a clinical trial for your child.

It is hypothesized that decreasing electrical activity in the brain may improve seizure control and other behavioral abnormalities in children with the CDKL5 mutation.

Currently, there are no approved therapies for CDKL5 Deficiency Disorder (CDD). Any therapy that reduces the frequency, duration or severity of seizures may positively impact quality of life for the child and family.

The Marigold study is exploring whether ganaxolone, an investigational medication can reduce seizures, increase seizure free days and improve behavior in children with CDD.

Find out if your child prequalifies

infant with cdkl5 disorder

About CDD

CDKL5 Deficiency Disorder (CDD) is a serious and rare genetic disorder that is caused by a mutation of the cyclin-dependent kinase-like 5 (CDKL5) gene. The CDKL5 gene produces a protein that is important for normal brain development and function.

CDD predominantly affects girls and is characterized by early onset intractable seizures, severe developmental delay and intellectual disability, disturbed sleep, and movement complications. Typically, the seizures are resistant to existing antiepileptic medication. Treatment-resistant epilepsy can be fatal and patients are often treated with high doses of multiple antiepileptic drugs (AEDs) that worsen the cognitive, behavioral and physical consequences of the underlying disorder. Together, this has a negative impact on quality of life in these children and young adults.

Currently, there are no approved therapies for CDD. Any therapy that reduces the frequency, duration or severity of seizures may positively impact quality of life for the child and family.

Find out if your child prequalifies

What is the Marigold Study?

It is hypothesized that treatment with ganaxolone, an investigational medication, will increase inhibitory (quieting) signaling within the brain thus reducing overall electrical activity. It is believed that this could improve not only seizure control, but also other behavioral abnormalities in children with the CDKL5 mutation.

Ganaxolone is very similar to allopregnanolone which is a naturally occurring neurosteroid in our bodies. Like allopregnanolone, ganaxolone binds to GABAA receptors in the brain and enhances inhibitory signaling. Ganaxolone binds to these receptors at a site distinct from benzodiazepines which are a group of existing antiepileptic drugs. The purpose of the Marigold study is to test whether ganaxolone can be used to treat CDD.

The Marigold Study is a global, double-blind, randomized, placebo-controlled trial of ganaxolone treatment in children and young adults with CDD. The trial consists of a 6-week prospective baseline period to collect seizure data, followed by a 17-week double-blind treatment phase, which is then followed by a long-term open-label phase.

If your child qualifies and agrees to participate, your child may be in the study for up to 23 weeks. At the screening visit to determine eligibility per inclusion/exclusion criteria, you will need to provide a 2-month daily historical seizure calendar which must include seizure type, frequency, and denote seizure free days. After meeting the eligibility criteria, patients will be randomly assigned to receive either the test medication or placebo for 17 weeks. Neither you nor the study staff will know which medication you are receiving. Your child may remain on the standard anti-seizure medication he/she is currently taking.

Find out if your child prequalifies

Does My Child Qualify?

Your child must meet the following criteria to be eligible for the study:

  • CDKL5 mutation confirmed by a genetic laboratory
  • Between the ages of 2-21 years
  • Suffer from approximately 16 or more major motor or drop seizures per month

 
The study doctor will require a pre-screening for your child and ask you questions about your child’s medical history and seizures to see if your child can participate in the study. You will be asked to keep track of your child’s seizures (including the type and number of seizures your child has every day, and the days when your child doesn’t have any seizures) for 8 weeks.

If you require financial assistance to get your child to a required study visit, the sponsor will reimburse for reasonable expenses (parking and travel).

does my child qualify

Participation in the Marigold Study is completely voluntary. Ask your doctor if being in this study is right for your child.

Find out if your child prequalifies

field of marigolds

Clinical Research

Clinical research studies evaluate investigational medications to determine if they are safe and effective for patients before they are made available to the public. Studies are carefully designed and monitored by health professionals, with patient safety as a priority.

Before enrolling in clinical research studies, participants review and sign an informed consent document, stating that the study details have been explained to them and their questions have been answered. This document is not a contract; participation in a clinical research study is completely voluntary and study participants can leave the study for any reason at any time.

Frequently Asked Questions

Can my child take CBD and participate in this trial?

Patients on cannabidiol (CBD) can participate in the study if they discontinue use of CBD and undergo a six-week washout period before being randomized into the 17-week double-blind treatment period of the study. Once the double-blind period is complete, if the patient enters the open-label portion of the study, they can take CBD.

Is there help for transportation costs?

If you require financial assistance to get your child to a required study visit, the sponsor will reimburse for reasonable expenses (parking and travel).

Can my child be seen by my regular physician?

Your child will be seen by the physician that is currently participating in the study. You can also continue to see your child’s regular physician, who will be apprised of your child’s treatment and condition throughout the study.

Am I guaranteed to get ganaxolone in this trial?

Following the 17-week double-blind treatment period, all participants will be able to receive ganaxolone during the open-label phase of the trial. The selection of who receives ganaxolone or placebo in the treatment period is completely random and remains secret during the course of the trial. There is no way of knowing which your child will receive.

Who can join this study?

Individuals between the ages of 2 and 21 with early-onset, difficult-to-control seizures, neurodevelopmental impairment, and a confirmed pathogenic CDKL5 gene variant may be eligible for the study.

See clinicaltrials.gov for details, or ask your doctor if being in this study is right for your child.

What do I have to do to be considered for the study?

Your child will need to go through a pre-screening process where the study doctor will ask you questions about your child’s medical history and seizure history to see if your child is a good candidate for the study.

Daily seizure history including seizure type, frequency, and seizure-free days in the past 2 months will help determine how your child’s seizures fluctuate. If you do not have this information, you will be asked to sign an informed consent and prospectively chart seizures for 8 weeks before coming in for a screening visit.

What treatment options are provided?

Patients will be randomized into the clinical trial to receive either ganaxolone or a placebo (non-active substance), in addition to their standard anti-seizure treatment. The drug will be administered as either a drinkable liquid or as a capsule, and it must be taken with food. Patients must maintain stable background medications while in the clinical trial.

Are there side effects?

Ganaxolone has been shown to be generally safe and well-tolerated in clinical studies to date. Over 1,600 people of all ages have taken ganaxolone, some for as long as four years. Common side effects include sleepiness, dizziness, and fatigue.

Will this help my child?

Previous studies have shown that ganaxolone reduced seizures in CDD. This trial intends to find out if ganaxolone is more effective in reducing seizures compared to placebo and whether there are other benefits associated with ganaxolone treatment.

Because of the nature of the trial, even if ganaxolone is effective, your child might be in the control group that receives the placebo. Ask your doctor if being in this study is right for your child.

What is a clinical trial?

Clinical trials are research studies used to find better ways to treat diseases such as genetic epilepsies. Researchers use clinical trials to learn whether a potential drug is safe and effective. Research has shown that patients tend to show benefits while participating in clinical trials.

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