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International CDKL5 Registry

NCT04486768 · Status: SUSPENDED · Type: OBSERVATIONAL · Enrollment: 500

Last updated 2026-04-21

No results posted yet for this study

Summary

Owing to the recent classification of CDKL5 Deficiency Disorder (CDD) as a unique disorder, there is a limited understanding of overall disease natural history and meaningful outcome measures. An international patient registry aimed at collecting both patient/caregiver and clinician-entered demographic, patient-reported outcome (PRO) and treatment data would benefit both the scientific and patient communities. This CDD registry will follow up to 500 patients diagnosed with CDD over several years through both the patients/caregivers and their clinicians. Initial data will be collected upon enrollment in the registry, followed by the collection of additional CDD-specific data on a bi-annual/ annual basis. No procedures will be performed as part of this registry. Clinician-entered data will be collected following standard of care visits conducted as part of patients' ongoing clinical care. Ultimately, the goal is to create a contact registry to allow patients/families to be alerted about relevant clinical trials and to collect valuable information that is accessible to the patient and scientific communities, thereby aiding and encouraging research in CDD.

Conditions

  • CDKL5 Deficiency Disorder (CDD)

Interventions

OTHER

Survey Instrument

This registry contains several surveys that will be released for patient/caregiver completion at enrollment and at time points following enrollment. These surveys can be completed on any computer that is connected to the internet.

Sponsors & Collaborators

  • Loulou Foundation

    collaborator OTHER

  • International Foundation for CDKL5 Research

    collaborator UNKNOWN

  • CDKL5 Alliance

    collaborator UNKNOWN

  • University of Pennsylvania

    lead OTHER

Principal Investigators

  • Dan Lavery, PhD · Director, CDKL5 Program of Excellence, Orphan Disease Center

Eligibility

Sex
ALL
Healthy Volunteers
No

Timeline & Regulatory

Start
2018-12-05
Primary Completion
2028-12-31
Completion
2028-12-31

Countries

  • United States

Study Locations

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Entities

Companies

Read the full study record

This page highlights key information. For complete eligibility criteria, study locations, investigator contacts, and the full protocol, visit the original record on ClinicalTrials.gov.

View NCT04486768 on ClinicalTrials.gov