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

Efficacy and Safety Evaluation of Hyperthermic Baths in the Treatment of Seizures in CDKL5 Deficiency Disorder: A Single-center, Self Control Trial

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.

Study Overview

• Status: Completed
• Conditions: CDKL5 Deficiency Disorder
• Intervention / Treatment: Other: Hyperthermic Baths

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 Type: Interventional
• Enrollment (Actual): 8
• Phase: Not Applicable

Contacts and Locations

Study Locations

• China
  • Beijing Municipality
    • Beijing, Beijing Municipality, China, 100053
      • Xuanwu Hospital,Capital Medical University

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: Child
• Accepts Healthy Volunteers: No

Description

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 2 or more anti-seizure medications at therapeutic doses
• Have a history of at least 8 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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: 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.	Other: Hyperthermic Baths; The intervention will consist of 12 weeks of repeated, daily 20-min body immersions.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Seizure frequency (SF28)	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 SF28 in the baseline period. The seizure frequency in the treatment phase is defined as SF28 per month during the treatment period. Percent change in seizure frequency=100*(treatment SF28-baseline SF28)/baseline SF28.	Up to 12 week after hyperthermic baths

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Seizure Responder Rate	The proportion of patients with a ≥ 50% reduction from Baseline in seizure frequency.	Up to 12 week after hyperthermic baths
Seizure-free Days	Seizure-free days over the entire treatment phase	Up to 12 week after hyperthermic baths

Collaborators and Investigators

• Sponsor: Xuanwu Hospital, Beijing
• Investigators: Principal Investigator: Liankun Ren, MD, Xuanwu Hospital, Beijing

Study record dates

Study Major Dates

• Study Start (Actual): September 20, 2024
• Primary Completion (Actual): June 25, 2025
• Study Completion (Actual): September 30, 2025

Study Registration Dates

• First Submitted: June 3, 2024
• First Submitted That Met QC Criteria: June 3, 2024
• First Posted (Actual): June 7, 2024

Study Record Updates

• Last Update Posted (Actual): April 15, 2026
• Last Update Submitted That Met QC Criteria: April 12, 2026
• Last Verified: April 1, 2026

More Information

Terms related to this study

• Keywords: CDKL5; Drug Resistant Epilepsy; Hyperthermic Baths
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Epilepsy; Drug Resistant Epilepsy; CDKL5 deficiency disorder
• Other Study ID Numbers: 2024-111-002

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: NO

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: No
• Studies a U.S. FDA-regulated device product: No