ClinicalTrials.gov record NCT05126914

Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents

Record copied from ClinicalTrials.gov: Oct 10, 2026AI summaries generated: Aug 22, 2026

Multicentre Real-life Follow-up Study of Rare Epileptic Syndromes in Children and Adolescents

StatusRecruitingThe study is currently enrolling participants. Only the study team can say whether a particular person can join.
Study typeObservationalA study in which researchers do not assign participants to a treatment or other intervention. They observe or measure what happens, which may include study visits, tests, questionnaires or a review of medical records.
AgeUp to 15 years old
SexAll
LocationFrance
AI summaryWhat this study is about, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

This prospective, multicentre observational study follows children and adolescents (up to 15 years) diagnosed with rare epilepsies. It collects real-life information on treatment responses, seizure frequency (using parent-kept seizure calendars), and the evolution of cognitive and psychiatric comorbidities. The study will compare different management approaches over time; the primary outcome is rate of decrease in seizures measured over 5 years.

What it is studying. The study is testing how children and adolescents with rare epilepsies respond to treatments in routine care, how cognitive and psychiatric comorbidities change over time, and how different real-life management methods relate to seizure control and medium-term outcomes. It will use seizure calendars kept by parents as part of current care to measure changes in seizure frequency, with a main measurement over five years.

What the researchers hope to learn. To evaluate response to treatments in real-life care; to measure the rate of decrease in epileptic seizures over time; to describe the evolution of cognitive and psychiatric comorbidities; and to compare different management approaches to inform future knowledge about prognosis and endpoints in rare pediatric epilepsies.

What this study is about

The study's own summary, as published on ClinicalTrials.gov. Underlined terms have a short explanation.

Rare epilepsies as a whole account for 20-30% of epilepsies, but knowledge about prognostic factors is currently limited. This means that it is difficult to provide adequate information to families at diagnosis and during follow-up. Prognostic factors are also important for management as they can have an impact on the patient's outcome (time to intervention, choice of one molecule over another, etc.). Finally, few treatments are currently available for these epilepsies. One of the limitations to the development of treatments is the lack of real life data as it is difficult to create reliable primary endpoints such as the rate of patients becoming seizure free naturally compared to a therapeutic intervention. The aim of this real-life study is to evaluate the response to treatment as well as to see the evolution of cognitive and psychiatric comorbidities. As explained above, there are very few randomised trials except for 3 rare epilepsies (infantile spasm syndrome, Dravet syndrome, Lennox-Gastaut syndrome). This has led to the virtual absence of management recommendations, including for the three syndromes mentioned above, where attempts at treatment algorithms have been proposed, although these have not been able to be considered as evidence-based recommendations. As a result, there is some diversity in the management of rare epilepsies from one centre to another. However, this diversity in management can be an asset in a real-life study. This will make it possible to compare different management methods, both in terms of seizure control and medium-term outcome.

Eligibility

The criteria exactly as listed on ClinicalTrials.gov. Only the study team can say whether they apply to you.

Inclusion criteria

  • Diagnosis for rare epilepsy (based on ORPHA codes)
  • holders of parental authority not opposed
  • Be followed in one of the declared centers of the study

Exclusion criteria

  • opposition from the holders of parental authority or the patient

Study description and what participation involves

The registry record has no detailed description beyond the summary above.

AI summaryWhat participation may involve, in plain languageShow ▾Hide ▴

AI-written from the registry record, not the study's own wording. May contain errors or omissions. Check the registry text on this page and the original record ↗ before acting on it.

Participants are followed prospectively during their usual clinical care. Parents are asked to keep a seizure calendar (used as part of current care) and the study will collect information on treatments used and seizure frequency. The study will also record the course of cognitive and psychiatric comorbidities as assessed in routine visits. The registry does not specify additional invasive procedures beyond standard care.

How the study is structured. A prospective observational cohort study. Participants are followed during their usual care at participating centres; the study does not assign treatments. Real-life data are collected prospectively, including parent-kept seizure calendars, with outcomes tracked up to five years.

Questions you could ask

These questions are suggestions for a conversation with the study team or your own clinician, not advice.

  • This is an observational study, so the study itself gives no treatment. What would taking part involve for me, and would it change my usual care?
  • The listed sites are in France. Is any of them reachable for me, and does the study help with travel?
  • Which of the eligibility criteria would need to be checked with tests before I could join?

More on CDKL5 deficiency disorder

This trial is also listed on this site under Dravet syndrome.

Clinical trials22 trials listed for CDKL5 deficiency disorderEvery trial the registry lists for CDKL5 deficiency disorder, with recruitment status, phase and sites, updated from ClinicalTrials.gov.See all trials →Research updatesWhat changed in CDKL5 deficiency disorder researchLatest monthly update: September 2026. Includes: EEG background changes. New research summarized each month in plain language, with links to every source paper.Read the latest update →

Important dates

Study start
Dec 11, 2025
Primary completion
December 2028 (estimated)
Study completion
December 2028 (estimated)
Target enrollment
1000 participants (estimated)
Last updated on registry
Feb 24, 2026
Data verified
Oct 10, 2026

Sponsor and research team

The lead sponsor funds and oversees the trial. A pharmaceutical or biotech company sponsor typically means the study is testing that company's drug or device; a hospital, university, or government agency usually indicates investigator-initiated or publicly funded research.

Lead sponsor
Assistance Publique - Hôpitaux de Paris
Collaborators
  • Orphelia Pharma

Central study contact

If you're interested in participating, the central contact can answer general questions about the study — including eligibility requirements and how to find a recruiting site near you. Actual eligibility is determined by the site's medical team.

Participating sites (11)

Terms used on this page · explanations by MedicalUpdates.ai

follow-up
The period after treatment or enrolment during which participants are observed or contacted, for example to record side effects or outcomes.
endpoints
Something the study measures to judge its results, such as a symptom score, a blood test or survival. Outcomes are grouped as primary, secondary and sometimes exploratory.
randomised
Participants are assigned to the study groups by chance, like a coin toss, rather than chosen by the doctor or the participant. This is done so the groups are comparable.
Inclusion Criteria
The conditions a person must meet to be considered for the study, for example a confirmed diagnosis, an age range or a test result.
Exclusion Criteria
The conditions that rule a person out even if they meet the inclusion criteria, for example other illnesses, certain medicines or pregnancy.

The study record on this page is reproduced from ClinicalTrials.gov, a database of the U.S. National Library of Medicine, where it was submitted by the study sponsor. MedicalUpdates.ai does not own this content, is not affiliated with or endorsed by ClinicalTrials.gov, NIH, or the U.S. Government, and does not verify the record. Plain-language notes, the glossary and the questions above are added by this site; text in the boxes marked "AI summary" was written by an AI model and may contain errors. Registry records change over time. Check the original record for the current version and discuss eligibility or participation with a qualified healthcare professional.

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