RECRUITING

International CDKL5 Registry

Study Overview

This clinical trial focuses on testing the efficacy of different digital interventions to promote re-engagement in cancer-related long-term follow-up care for adolescent and young adult (AYA) survivors of childhood cancer.

Description

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.

Official Title

Orphan Disease Center CDKL5 Deficiency Disorder International Patient Registry

Quick Facts

Study Start:2018-12-05
Study Completion:2028-12-31
Study Type:Not specified
Phase:Not Applicable
Enrollment:Not specified
Status:RECRUITING

Study ID

NCT04486768

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Ages Eligible for Study:Not specified
Sexes Eligible for Study:ALL
Accepts Healthy Volunteers:Not specified
Standard Ages:CHILD, ADULT, OLDER_ADULT
Inclusion CriteriaExclusion Criteria
  1. * Person of any age, living or deceased;
  2. * Be a patient or the legal guardian (parent or caregiver) of a patient with a diagnosis of CDD (Diagnoses must be confirmed by a clinician or genetic test);
  3. * Have the ability to understand and complete an informed consent process where applicable per local regulations or have a legal guardian to provide consent on the patient's behalf if the patient is under the legal age, per local regulations, or otherwise unable to provide consent.
  1. * Patient with a diagnosis of CDD who is under the legal age, per local regulations, enrolling without a legal guardian;
  2. * Legal guardian of a patient who is 1) over the legal age, per local regulations, and 2) is able to read and provide consent and enter data. (We require that patients over the legal age who are capable of reading and understanding and informed consent provide data directly.)

Contacts and Locations

Study Contact

Theresa E Berger, MBE
CONTACT
445-236-4543
ODCRegistry@pennmedicine.upenn.edu

Principal Investigator

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

Study Locations (Sites)

University of Pennsylvania Orphan Disease Center
Philadelphia, Pennsylvania, 19104
United States

Collaborators and Investigators

Sponsor: University of Pennsylvania

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

Study Record Dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Registration Dates

Study Start Date2018-12-05
Study Completion Date2028-12-31

Study Record Updates

Study Start Date2018-12-05
Study Completion Date2028-12-31

Terms related to this study

Keywords Provided by Researchers

  • CDKL5
  • CDD
  • CDKL5 Disorder
  • CDKL5 Deficiency
  • pediatric epilepsy
  • epilepsy
  • CDKL5 Registry
  • CDKL5 Patient Registry
  • CDD Registry
  • CDD Patient Registry

Additional Relevant MeSH Terms

  • CDKL5 Deficiency Disorder (CDD)