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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT05558371




Registration number
NCT05558371
Ethics application status
Date submitted
28/07/2022
Date registered
28/09/2022

Titles & IDs
Public title
International CDKL5 Clinical Research Network
Scientific title
Multi-Site Validation of Biomarkers and Core Clinical Outcome Measures for Clinical Trials Readiness in CDKL5 Deficiency Disorder
Secondary ID [1] 0 0
5U01NS114312
Secondary ID [2] 0 0
19-2756
Universal Trial Number (UTN)
Trial acronym
ICCRN
Linked study record

Health condition
Health condition(s) or problem(s) studied:
CDKL5 0 0
CDKL5 Deficiency Disorder 0 0
CDD 0 0
Condition category
Condition code

Intervention/exposure
Study type
Observational
Patient registry
Target follow-up duration
Target follow-up type
Description of intervention(s) / exposure
Other interventions - No intervention.

Other interventions: No intervention.
No intervention administered as part of this study; observational only.
Intervention code [1] 0 0
Other interventions
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
CDKL5 Deficiency Disorder (CDD) Clinical Severity Assessment - Clinician (CCSA-Clinician)
Timepoint [1] 0 0
5 years
Primary outcome [2] 0 0
CDKL5 Deficiency Disorder (CDD) Clinical Severity Assessment - Caregiver (CCSA-Caregiver)
Timepoint [2] 0 0
5 years
Primary outcome [3] 0 0
CDKL5 Deficiency Disorder (CDD) Development Questionnaire - Caregiver (CDQ-Caregiver)
Timepoint [3] 0 0
5 years
Primary outcome [4] 0 0
Communication and Symbolic Behavior Scales - Developmental Profile Infant Toddler Checklist (CSBS-DP ITC)
Timepoint [4] 0 0
5 years
Primary outcome [5] 0 0
Sleep Disorder Scale for Children (SDSC)
Timepoint [5] 0 0
5 years
Primary outcome [6] 0 0
Quality of Life Inventory - Disability (QI-Disability)
Timepoint [6] 0 0
5 years
Primary outcome [7] 0 0
CDKL5 Deficiency Disorder (CDD) Gross Motor (CDD-Motor)
Timepoint [7] 0 0
5 years
Primary outcome [8] 0 0
CDKL5 Deficiency Disorder (CDD) Fine Motor (CDD-Hand)
Timepoint [8] 0 0
5 years
Primary outcome [9] 0 0
Electroencephalogram/Evoked Potentials (EEG/EP) characteristics in CDKL5 Deficiency Disorder.
Timepoint [9] 0 0
5 years
Secondary outcome [1] 0 0
Frequency of different mutations in the CDKL5 Deficiency Disorder population
Timepoint [1] 0 0
5 years
Secondary outcome [2] 0 0
Frequency of medications and their indications in the CDKL5 Deficiency Disorder population
Timepoint [2] 0 0
5 years
Secondary outcome [3] 0 0
Social Determinants of Health (SDOH) in CDKL5 Deficiency Disorder population
Timepoint [3] 0 0
5 years

Eligibility
Key inclusion criteria
* All children diagnosed with CDD age 1-month to 100 years of age that are receiving care at one of the study institutions or are registered with the International CDKL5 Disorder Database will be considered for the study population.
Minimum age
1 Month
Maximum age
100 Years
Sex
Both males and females
Can healthy volunteers participate?
No
Key exclusion criteria
* Individuals who do not meet study inclusion criteria.

Study design
Purpose
Duration
Selection
Timing
Prospective
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
15/02/2021
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
15/02/2027
Actual
Sample size
Target
1000
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
Nedlands WA
Recruitment hospital [1] 0 0
Telethon Kids Institute/Perth Children's Hospital - Perth
Recruitment postcode(s) [1] 0 0
6009 - Perth
Recruitment outside Australia
Country [1] 0 0
United States of America
State/province [1] 0 0
California
Country [2] 0 0
United States of America
State/province [2] 0 0
Colorado
Country [3] 0 0
United States of America
State/province [3] 0 0
Massachusetts
Country [4] 0 0
United States of America
State/province [4] 0 0
Missouri
Country [5] 0 0
United States of America
State/province [5] 0 0
New York
Country [6] 0 0
United States of America
State/province [6] 0 0
Ohio
Country [7] 0 0
United States of America
State/province [7] 0 0
Pennsylvania
Country [8] 0 0
United States of America
State/province [8] 0 0
Texas

Funding & Sponsors
Primary sponsor type
Other
Name
University of Colorado, Denver
Address
Country
Other collaborator category [1] 0 0
Government body
Name [1] 0 0
National Institute of Neurological Disorders and Stroke (NINDS)
Address [1] 0 0
Country [1] 0 0
Other collaborator category [2] 0 0
Government body
Name [2] 0 0
National Institutes of Health (NIH)
Address [2] 0 0
Country [2] 0 0
Other collaborator category [3] 0 0
Other
Name [3] 0 0
International Foundation for CDKL5 Research
Address [3] 0 0
Country [3] 0 0

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Timothy A Benke, MD PhD
Address 0 0
University of Colorado, Denver
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Sharon R Pincus, MA
Address 0 0
Country 0 0
Phone 0 0
303-949-7116
Fax 0 0
Email 0 0
sharon.pincus@cuanschutz.edu
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
Yes
What data in particular will be shared?
The ICCRN will be utilizing the National Institute of Mental Health (NIMH) Data Archive (NDA), previously known as the National Database for Autism Research (NDAR), as our final deidentified repository of our data. We will utilize Global Unique Identifiers to ensure we have deconflicted patient data across all institutions. Each institution will be directly uploading patient data to our central REDCap database to allow for cleaning of data prior to upload to NDA. Our data elements will be harmonized with other NDA elements where appropriate at the start of the study. The one exception is patient videos which will be reviewed at Telethon kids secure network for analysis, but the scored results of validated motor scales will be uploaded to NDAR in a deidentified fashion. All scales, outcome measures, and protocols (both for scoring and implementation of COMs) will also be shared through publications.

Supporting document/s available: Study protocol, Statistical analysis plan (SAP), Informed consent form (ICF)
When will data be available (start and end dates)?
Available data will be released to the repository and will become available to the scientific community one year after publication of planned analyses, or after a period of 5 years from the date when the data were collected, whichever comes first.
Available to whom?
NIMH NDA policy will govern access criteria.
Available for what types of analyses?
How or where can data be obtained?
IPD available at link: https://nda.nih.gov/


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT05558371