Efficacy and Safety Evaluation of Hyperthermic Baths in the Treatment of Seizures in Children With CDKL5 Deficiency

Not Applicable

Recruiting

• Conditions: CDKL5 Deficiency Disorder
• Interventions: Other: Hyperthermic Baths

• Registration Number: NCT06447675
• Lead Sponsor: Xuanwu Hospital, Beijing

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.

Study Timeline

• Study First Submitted: 2024-06-03
• Study First Submitted QC: 2024-06-03
• Study First Posted: 2024-06-07
• Study Start: 2024-09-14
• Status Verified: 2025-02
• Last Update Submitted: 2025-02-27
• Last Update Posted: 2025-03-04
• Primary Completion: 2025-07-31
• Study Completion: 2025-12-31

Recruitment & Eligibility

• Status: RECRUITING
• Sex: All
• Target Recruitment: 8

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
Hyperthermic Baths	Hyperthermic Baths	Patients with CDKL5 deficiency assigned to hyperthermic baths will have their body immersed in a hot water (40\~42°C) bath for 20 min per session.

Primary Outcome Measures

Name	Time	Method
Seizure frequency (SF28)	Up to 3 month after hyperthermic baths	Seizure frequency (SF28) is defined as seizure count per month (28-day) period. The SF28 is calculated as follows, where D=total number of days for which seizure information is collected for the specific 28-day interval: SF28=(Total number of seizures in D days/D)\*28. In addition, the baseline seizure frequency is defined as mean of 3-month SF28 in the baseline period. The seizure frequency in double-blind phase is defined as SF28 per month during the double-blind period. Percent change in seizure frequency=100\*(double-blind SF28-baseline SF28)/baseline SF28.

Secondary Outcome Measures

Name	Time	Method
Seizure-free Days	Up to 3 month after hyperthermic baths	Change in percentage of seizure-free days over the entire blinded phase as compared to the entire baseline phase. The number of seizure-free days was normalized to 84-day baseline and blinded phases for each subject.
Seizure Responder Rate	Up to 3 month after hyperthermic baths	The proportion of patients with a ≥ 50% reduction from Baseline in seizure frequency.

Trial Locations

Locations (1)

Xuanwu Hospital,Capital Medical University; 🇨🇳 Beijing, Beijing, China