Clinical trial NCT06447675: Efficacy and Safety Evaluation of Hyperthermic Baths in the Treatment of Seizures in Children With CDKL5 Deficiency
Official title: Efficacy and Safety Evaluation of Hyperthermic Baths in the Treatment of Seizures in Children With CDKL5 Deficiency: A Single-center, Self Control Trial
Study type: INTERVENTIONAL
Overall status: RECRUITING
Phase: NA
Conditions: CDKL5 Deficiency Disorder
Keywords: Hyperthermic Baths
Drug Resistant Epilepsy
CDKL5
Lead sponsor: Xuanwu Hospital, Beijing
Sponsor class: OTHER
First posted: 2024-06-07
Enrollment: 8
Sex: ALL
Minimum age (years): 6
Maximum age (years): 6
Standard ages: CHILD
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

Brief summary: The primary objective of this research is to study the efficacy and safety of hyperthermic baths as adjunctive therapy for reducing the frequency of seizures in CDKL5 deficiency.

Detailed description: This project aims to include 8 participants, and evaluate the effectiveness and safety of hyperthermic baths in patients with CDKL5 deficiency through A single-center, preliminary feasibility clinical trial. It is expected to provide new therapeutic options for patients with CDKL5 deficiency with alternative treatment options.

Eligibility criteria: Inclusion Criteria:

* A diagnosis of CDKL5 deficiency, including molecular confirmation of a pathogenic or likely pathogenic CDKL5 variant and refractory seizures
* Male or female participants aged 6 months to less than 6 years
* Parent(s) or LAR willing to give written informed consent, after being properly informed of the nature and risks of the study and prior to engaging in any study-related procedures
* Failure to control seizures despite appropriate trial of 1 or more anti-seizure medications at therapeutic doses
* Have a history of at least 16 countable seizures per 4 weeks during the 8 weeks prior to screening. Countable seizures will be defined by the following:Seizures with or without impairment of consciousness with a clear motor component, including generalized tonic-clonic, focal to bilateral tonic-clonic, atonic, bilateral clonic, bilateral tonic, focal motor seizures with or without impaired awareness, or infantile spasms. Clusters of infantile spasms/tonic seizures will be counted as a single seizure.
* Participants should be on a stable regimen of anti-seizure medications for ≥ 4 weeks prior to the screening visit, without a foreseeable change in dosing for the duration of the DB phase.
* Vagus nerve stimulator (VNS) are permitted, but should be implanted for 1 year prior to screening and settings should be unchanged throughout the study.
* Parent/caregiver is able and willing to maintain an accurate and complete daily seizure eDiary for the duration of the study.

Exclusion Criteria:

* Any history of previous brain disease (trauma, etc.) that likely to precipitate seizures.
* Tissue damage (eczema, etc.) in the area where hyperthermic baths will be applied on the skin.
* Seizures associated with hyperthermic baths.
* Presence of any clinical condition that in the opinion of the principal investigator makes the patient not suitable to participate in the trial.

Primary outcomes:
measure: Seizure frequency (SF28)
timeFrame: Up to 3 month after hyperthermic baths
Secondary outcomes:
measure: Seizure Responder Rate
timeFrame: Up to 3 month after hyperthermic baths
measure: Seizure-free Days
timeFrame: Up to 3 month after hyperthermic baths
Other outcomes:
Locations:
city: Beijing
country: China
facility: Xuanwu Hospital,Capital Medical University
state: Beijing
Study officials:
affiliation: Xuanwu Hospital, Beijing
name: Liankun Ren, MD
role: PRINCIPAL_INVESTIGATOR
References: