Efficacy and Safety of Daily Home-based Hyperthermic Baths for Reducing Epileptic Seizures in CDKL5 Deficiency Disorder: A PROBE Clinical Trial

Efficacy and Safety of Daily Home-based Hyperthermic Baths for Reducing Epileptic Seizures in CDKL5 Deficiency Disorder: A Prospective, Randomized, Parallel-Controlled, Open-Label, Blinded Endpoint (PROBE) Clinical Trial

The primary objective of this research is to study the efficacy and safety of daily, home-based hyperthermic baths for reducing epileptic seizures in CDKL5 Deficiency Disorder.

Study Overview

• Status: Not yet recruiting
• Conditions: CDKL5 Deficiency Disorder
• Intervention / Treatment: Behavioral: Home-based hyperthermic Baths

Detailed Description

This is a prospective, randomized, open-label, blinded-endpoint study designed to evaluate the efficacy and safety of daily home-based hyperthermic bathing in participants with CDKL5 deficiency disorder. Eligible participants will be randomized in a 1:1 ratio to receive either hyperthermic baths in addition to usual antiseizure care or usual antiseizure care alone without hyperthermic baths. Efficacy will be assessed primarily by changes in epileptic seizure burden, and safety will be evaluated throughout the study period.

• Study Type: Interventional
• Enrollment (Estimated): 34
• Phase: Not Applicable

Contacts and Locations

• Study Contact: Name: Liankun Ren, MD, PhD; Phone Number: +86 13681576621; Email: renlk2022@outlook.com

Participation Criteria

Eligibility Criteria

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

Description

Inclusion Criteria:

1. A diagnosis of CDKL5 deficiency disorder, including molecular confirmation of a pathogenic CDKL5 variant and exhibiting epileptic seizures.
2. Male or female participants aged 6 months to 14 years, inclusive.
3. Patients presenting with drug-resistant epilepsy that seizures were uncontrolled despite the appropriate trial of at least two antiseizure medications (ASMs).
4. At least 8 motor seizures during each 4 weeks in the 8-week historical seizure records preceding screening.
5. Treatment regimens of 0-4 ASMs were required to be stable for at least 4 weeks before screening and throughout the intervention. Ongoing ketogenic diets were allowed if remain unchanged during screening and treatment.

6. Subjects who have undergone surgical implantation of vagus nerve stimulation (VNS) will be eligible for inclusion in the study, provided they meet all of the following criteria:

   1. The VNS device has been in place for at least one year prior to the screening visit;
   2. The stimulation parameters remain unchanged throughout the duration of the trial.
7. Parents or caregivers must be able and willing to maintain an accurate and comprehensive daily electronic seizure diary throughout the duration of the study.
8. Participants' legal guardians willing to provide written informed consent.

Exclusion Criteria:

1. Patients who had any history of previous brain disease (including trauma, encephalitis, etc.) that were likely to precipitate seizures; defined by MRI outcomes.
2. Participants with skin ulceration or dermatologic conditions that make bathing unsuitable.
3. Parents or guardians who, based on past experiences, determine that the child has an intolerance to thermal baths.
4. A history of seizures induced by thermal baths, or an increase in seizure frequency after thermal baths.
5. Patients who presented any clinical condition that in the opinion of the principal investigator made the patient not suitable to participate in the survey.

Study Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: Randomized
• Interventional Model: Parallel Assignment
• Masking: Single

Number of Arms

2

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: Home-based hyperthermic baths group; Patients with CDKL5 deficiency disorder assigned to the daily home-based hyperthermic bathing group will undergo immersion in a 40-42°C water bath for 20 minutes per session under continuous parental supervision.	Behavioral: Home-based hyperthermic Baths; A 12-week daily home-based hyperthermic bathing intervention consisting of immersion in a 40-42°C water bath for 20 minutes per session under continuous parental supervision. The intervention will be administered according to the study protocol.
No Intervention: Control Group; Participants assigned to the control group will not receive hyperthermic baths during the study period and will continue their usual care, including their existing antiseizure treatment regimen. Seizure frequency and safety outcomes will be assessed according to the study protocol.

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Seizure Responder Rate	The proportion of participants achieving at least a 50% reduction in seizure frequency from baseline during the 12-week home-based hyperthermic baths period.	Baseline to week 12 at home-based hyperthermic baths phase

Secondary Outcome Measures

Outcome Measure	Measure Description	Time Frame
Change in Seizure Frequency	Change in the frequency of epileptic seizures from baseline to the 12-week home-based hyperthermic baths period.	Baseline to week 12 at home-based hyperthermic baths phase
Seizure-free Days	Number of days without epileptic seizures during the 12-week treatment period.	Baseline to week 12 at home-based hyperthermic baths phase

Collaborators and Investigators

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

Study record dates

Study Major Dates

• Study Start (Estimated): August 1, 2026
• Primary Completion (Estimated): December 31, 2027
• Study Completion (Estimated): December 31, 2027

Study Registration Dates

• First Submitted: May 15, 2026
• First Submitted That Met QC Criteria: May 15, 2026
• First Posted (Actual): May 22, 2026

Study Record Updates

• Last Update Posted (Actual): May 22, 2026
• Last Update Submitted That Met QC Criteria: May 15, 2026
• Last Verified: May 1, 2026

More Information

Terms related to this study

• Keywords: epilepsy; fever; CDKL5 Deficiency Disorder; home-based hyperthermic baths; genotype-phenotype
• Additional Relevant MeSH Terms: Brain Diseases; Central Nervous System Diseases; Nervous System Diseases; Body Temperature Changes; Pathological Conditions, Signs and Symptoms; Signs and Symptoms; Epilepsy; Fever; CDKL5 deficiency disorder
• Other Study ID Numbers: 2026-161-001

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