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Clinical Trials in Germany / NCT05373719
Active, not recruiting Observational

Observational Study in Patients With Cyclin-dependent Kinase-like 5 Deficiency Disorder

NCT05373719 · tracked via the Priya Life Science Germany tracker
Sponsor
Loulou Foundation
Phase
Observational
Started
2022-09-30
Last updated
2025-03-10

Condition(s) studied

Cyclin-Dependent Kinase-Like 5 Deficiency Disorder

Investigational drug(s) / intervention(s)

No intervention; observational

No intervention; observational: No intervention; observational

Study summary

Observational, noninterventional, 3-year study to examine the presence of detectable differences in neurocognitive, developmental, motor, neurophysiologic, and quality of life measures over time in patients with cyclin-dependent kinase-like 5 deficiency disorder.

Eligibility

Sex
ALL
Min age
—
Max age
55 Years
Healthy volunteers
No
Inclusion Criteria: 1. Documented diagnosis of Cyclin-dependent Kinase-like 5 (CDKL5) deficiency disorder with pathogenic or likely pathogenic of CDKL5 variants 2. Ages newborn to 55 years old 3. Parent/Caregiver who is willing and capable of providing written informed consent 4. Parent/caregiver lives with or has daily contact with study participant and able to provide consistent information across the study Exclusion Criteria: 1. Any clinically significant neurocognitive deficit not attributable to Cyclin-dependent Kinase-like 5 (CDKL5) deficiency disorder or a secondary cause that may, in the opinion of the investigator, confound interpretation of study results (e.g., extreme prematurity, other genetic variants, unclassified CDKL5 variant, abnormal brain imaging, imaging and/or injury not otherwise attributable to CDD). 2. Any condition that, in the opinion of the investigator, would put the patient at undue risk or make it unsafe for the patient to participate 3. Any condition which would make the individual or the caregiver, in the opinion of the investigator, unsuitable for the study

Primary outcome measure(s)

  • Bayley Scale of Infant and Toddler Development, Fourth Edition (BSID-4) — Baseline
    Suitability of this cognition and global development scale in CDD will be assessed based on the number of tests completed, ceiling/floor effect and variance estimate.
  • Vineland 3 — Baseline
    Suitability of this adaptive behavior scale in CDD will be assessed based on the number of tests completed, ceiling/floor effect and variance estimate.
  • Gross Motor Function Measure (GMFM) — Baseline
    Suitability of this motor function scale in CDD will be assessed based on the number of tests completed, ceiling/floor effect and variance estimate.
  • Sleep Disturbance Scale for Children (SDSC) — Baseline
    Suitability of this sleep quality scale in CDD will be assessed based on the number of tests completed, ceiling/floor effect and variance estimate.

Trial sites (23)

FacilityCityRegionStatus
UCLA Mattel Children's Hospital Los Angeles California
Children's Hospital Colorado Aurora Colorado
Center for Rare Neurological Diseases Norcross Georgia
Boston Children's Hospital Boston Massachusetts
New York University New York New York
Cincinnati Children's Hospital Medical Center Cincinnati Ohio
The Cleveland Clinic Cleveland Ohio
Childrens Hospital of Philadlephia Division of Neurology Philadelphia Pennsylvania
Baylor College of Medicine Houston Texas
Alberta Children's Hospital, Pediatric Epilepsy and Child Neurology Calgary Canada
The Hospital of Sick Children Toronto Canada
Hopital Necker- Enfants Malades Paris France
DRK Berlin - Epilepsiezentrum und Neuropädiatrie Berlin Germany
Schön Klinik Vogtareuth Vogtareuth Germany
Azienda Ospedaliero-Universitaria Meyer Florence Italy
Fondazione Policlinico Universitario A Gemelli, IRCCS Roma Italy
Ospedale Pediatrico Bambino Gesù Rome Italy
Azienda Ospedaliera Universitaria Integrata di Verona Verona Italy
Hospital Infantil Universitario Niño Jesús Madrid Spain
Ruber Internacional Hospital Madrid Spain
Hospital UniversitarioVithas Madrid Madrid Spain
Ruber Internacional Hospital Madrid Spain
Mediclinic Dubai United Arab Emirates
Official registry record

This page summarises publicly available registry data for informational purposes — not medical advice. Eligibility is determined by each study team; patients should discuss participation with their clinician.

View NCT05373719 on ClinicalTrials.gov ↗ ← All trials in Germany