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

CDKL5 Deficiency Disorder Clinical Trial

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

Clinical Trial Details — Status: Not yet recruiting

Administrative data

• NCT number: NCT06447675
• Other study ID #: 2024-111-002
• Status: Not yet recruiting
• Phase: N/A
• Start date: June 1, 2024
• Est. completion date: December 31, 2025

Study information

• Verified date: May 2024
• Source: Xuanwu Hospital, Beijing
• Contact: Liankun Ren, MD
• Phone: +86 13681576621
• Email: renlk2022[@]outlook.com
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial 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.

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.

Recruitment information / eligibility

• Status: Not yet recruiting
• Enrollment: 8
• Est. completion date: December 31, 2025
• Est. primary completion date: December 31, 2024
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Months to 6 Years

Eligibility

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.

Related Conditions & MeSH terms

• CDKL5 Deficiency Disorder
• Fever
• Hyperthermia
• Seizures

Intervention

Other:
• Hyperthermic Baths
The intervention will consist of 12 weeks of repeated (2x/day) 20-min body immersions.

Locations

Country	Name	City	State
China	Xuanwu Hospital,Capital Medical University	Beijing	Beijing

Sponsors (1)

Lead Sponsor	Collaborator
Xuanwu Hospital, Beijing

Country where clinical trial is conducted

China, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	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 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.	Up to 3 month after hyperthermic baths
Secondary	Seizure Responder Rate	The proportion of patients with a = 50% reduction from Baseline in seizure frequency.	Up to 3 month after hyperthermic baths
Secondary	Seizure-free Days	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.	Up to 3 month after hyperthermic baths