There are lots of ways you can get involved in research, from completing a survey to taking part in a clinical trial.
CDKL5 Clinical Trials
Be in a study
What is a clinical trial?
Clinical trials are types of research studies, that involves patients, which are aimed at evaluating a new way to manage a condition. The aim of a clinical trial is to find out if a new treatment, like a new drug, diet or medical device is safe and effective in people. Commonly clinical trials are used to establish if a new treatment is more effective and/or has a less harmful side effect than the current treatment.
As with all research there is no guarantee of success – clinical trials are experimental by nature and there is a chance that the new way will be no better, or not as good, as the current treatment. Unexpected side-effects are also a possibility, or you may be put into the ‘control group’, which receives a placebo (dummy drug) or the standard existing treatment, rather than the new treatment.
Clinical trial take place globally and there are a number of different databases available to help you find out more.
Global Clinical trials database that includes both private and public funded studies around the world, including the UK.
The information on this database is provided by NHS National Institute for Health Research, and includes only UK trials.
The table below shows the current clinical trials and research studies for CDKL5 deficiency.
Company
Drug
Indication
Pre-Clinical Phase 1 Phase 2 Phase 3
Marinus Therapeutics
PTC – Therapeutics
Ovid- Therapeutics
Zogenix
Ganaxolone
Ataluren
OV935 – (TAK -935)
Fenfluramine
Seizure
Seizure
Seizure
Seizure
Company – Marinus Therapeutics
Drug Ganaxolone – Indication Seizure
Pre-Clinical
Phase 1 Phase 2 Phase 3
Company – Marinus Therapeutics
Drug Ganaxolone – Indication Seizure
Pre-Clinical
Phase 1 Phase 2 Phase 3
Company – Marinus Therapeutics
Drug Ganaxolone – Indication Seizure
Pre-Clinical
Phase 1 Phase 2 Phase 3
Company – Marinus Therapeutics
Drug Ganaxolone – Indication Seizure
Pre-Clinical
Phase 1 Phase 2 Phase 3
How can I take part in a clinical trial?
If you would like your child to take a part in a clinical trial in the first instance we recommend you speak to your/your child’s health team about trials that may be suitable.
MARIGOLD STUDY – CDKL5 DEFICIENCY DISORDER
Marinus is committed to developing ganaxolone to improve the lives of children suffering from CDKL5 deficiency disorder (CDD). Marinus’ pivotal Phase 3 Trial, #TheMarigoldStudyis now enrolling. If your child is between the ages of 2-21 w/ a confirmed #CDKL5 diagnosis, they might be a fit.
Visit: www.themarigoldstudy.com.
Trials currently recruiting patients
THE ARCADE STUDY ADDS TWO RARE DISORDERS, CDKL5 DEFICIENCY DISORDER AND DUPLICATION 15Q SYNDROME, TO TAK-935/OV935 CLINICAL DEVELOPMENT.
Takeda Pharmaceutical Company Limited[TSE: 4502] (“Takeda”) and Ovid Therapeutics Inc. (NASDAQ: OVID) today provided an overview of their TAK-935/OV935 broad clinical development program.
The companies plan to initiate three clinical trials: in pediatric patients with Dravet syndrome and Lennox-Gastaut syndrome, in pediatric patients with CDKL5 deficiency disorder (CDD) and Duplication 15q (Dup15q) syndrome, and an extension trial for patients with developmental and epileptic encephalopathies (DEEs) who participated in a previous TAK-935/OV935 clinical study.
Click here to read the full announcement and view the fact sheet.
Visit http://arcadestudy.com for more information.