Targeting the Gut to Improve Seizure Control in CDKL5 Deficiency Disorder (CDD)
ConditionCDKL5
Trial statusRecruiting
Trial phaseNot applicable
Trial typeInterventional
Biological sexAll
Age3-50
SponsorUniversity of Milan
About this trial
Standard anti-seizure medications have limited efficacy in seizure control in cyclin-dependent kinase-like 5 deficiency disorder (CDD).
The study will investigate whether targeting the gut-microbiota-brain axis in CDD patients can alleviate seizures and ameliorate other comorbidities.
Eligibility criteria
This trial does not accept healthy volunteersQualifiers
None
Disqualifiers
None
Trial design
Design model
Sequential
Treatments tested in this trial
alpha-lactalbumin, fructooligosaccharides, inulin
Dietary supplementThe first round supplementation will be administered for 3-month period (i.e. 12 weeks). One dose/day (2g sachet) is intended to be administered orally once a day after dissolving in water. At the scheduled visits/phone contacts \[i.e., baseline, 12 weeks, and 16 weeks (post washout)\], seizure frequency and entity of the critical episodes will be recorded. Gut microbiome characterization, clinical scales and dietary intake will be assessed.
alpha-lactalbumin, sodium butyrate, fructooligosaccharides, inulin
Dietary supplementThe second round supplementation will be administered for 3-month period (i.e. 12 weeks). For participants weighing \<30 kg, a 4 g dose (i.e., one 4 g sachet) is intended to be administered orally once a day after dissolving in water. For participants weighing ≥30 kg, a 4 g dose (i.e., 4 g sachets) is intended to be administered orally twice a day (12h interval) after dissolving in water. At the scheduled visits/phone contacts \[i.e., 28 weeks and 32 weeks (post washout)\], seizure frequency and entity of the critical episodes will be recorded. Gut microbiome characterization, clinical scales and dietary intake will be assessed.
Treatment groups
20 Participants
are divided into 1 treatment groupGroup A: CDD armExperimental treatment 2 interventions
Trial outcomes
Primary outcomes
1
Epilepsy
to evaluate the number of CDD patients considered treatment responders (seizure reduction ≥50%, ≥75% or ≥100% from baseline in monthly seizure counts) during the 12- week treatment period in 1st and 2nd round of supplementation
Time frame
Change at 3 months from baseline of each round of supplementationSecondary outcomes
1
Gut microbiota
to evaluate indicator species that could help as biomarkers for guiding clinicians to choose the intervention with the most likelihood of improve patient quality of life.
Time frame
Change at 3 months from baseline of each round of supplementation2
Sleep disturbances
Decreased Sleep SDSC scale (max score=125) by at least 5%
Time frame
Change at 3 months from baseline of each round of supplementation3
gastrointestinal discomfort
Decrease GISI scale (max score = 17), by at least 2 points
Time frame
Change at 3 months from baseline of each round of supplementationOther outcomes
1
Clinical features
Clinical Global Impression of Change (CGIC), ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change\] decrease of at least 1 point
Time frame
Change at 3 months from baseline of each round of supplementation2
Parental stress
Caregiver burden by Parenting Stress Index (PSI-SF); clinically significance \> 85%, decrease by at least 5%
Time frame
Change at 3 months from baseline of each round of supplementationSponsors and contacts
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Source ClinicalTrials.gov