Observational Study to Assess Natural History in Cockayne Syndrome Patients (NCT00985413) | Clinical Trial Compass
TerminatedNot Applicable
Observational Study to Assess Natural History in Cockayne Syndrome Patients
Stopped: The company DNage is in receivership and no longer functional
United States, France, United Kingdom40 participantsStarted 2009-09
Plain-language summary
This is an Observational Study of children under the age of 11 diagnosed with Cockayne Syndrome to assess the natural progression of Cockayne Syndrome disease, with special attention to hearing and physical changes in length or height, weight, head circumference, and arm span during standard treatment.
The primary analytical objective is to determine the rate of linear growth over a 6-month period in children \< 2 years of age and over a 12-month period in children ≥ 2 years of age.
Who can participate
Age range
1 Year – 11 Years
Sex
ALL
See this in plain English?
AI-rewrites the medical criteria so a patient or caregiver can understand them. Always confirm with the trial site.
Inclusion Criteria:
* Pediatric patients with a documented diagnosis of CS, as suggested by clinical features and possible confirmation by genetic consultation and analysis
* Age of participation:
* At least 12 months of age at the time of signing Informed Consent/Assent
* Female patient's age will not be greater than 10 years of age at the time of signing Informed Consent/Assent
* Male patient's age will not be greater than 11 years of age at the time of signing Informed Consent/Assent
Exclusion Criteria:
* Severe contractures or physical deformities that in the opinion of the investigator would prevent accurate measurement of height, length and ulna length
* Patients that have taken growth hormone or growth hormone related medications within 12 months prior to the date of Informed Consent/Assent
* Known history of inborn error of hyperprolinemia (Type I or Type II)
* Clinical features present at the time of initial screening that are associated with the terminal phases of the natural progression of CS suggesting safe travel and completion of the study and its assessments to be unlikely as judged by the Investigator, including any of the following:
* Continuous or intermittent dependence on supplemental oxygen at home during the prior six months
* Two or more hospitalizations for pneumonia during the prior 12 months;
* A documented net weight loss of at least 10%, which has not been recovered and which includes a significant net weight loss (beyond the estim…
Questions worth asking your doctor
Bring these to your next appointment. They're a starting point for a shared conversation — not a sign you qualify or a recommendation to enrol.
1Based on my diagnosis and history, is this trial worth exploring for me — or is there a standard treatment we should try first?
2What does this trial's phase tell us about how much is already known about its safety and benefit?
3What would taking part actually involve for me — visits, tests, time, and travel?
4What are the known and possible risks or side effects I should weigh, and how would they be monitored?
5If this trial isn't the right fit, what other options or trials would you suggest I look into?
Generated to help you prepare — always confirm anything about your own eligibility and care with the study team and your doctor.
Questions for the trial coordinator
The trial coordinator is the person who runs the study day to day. These cover the practical side — logistics, costs, and what taking part would actually mean for your life. The study team confirms whether you meet the criteria; these are questions to ask, not a sign you qualify.
1What does taking part actually involve week to week — how many visits, where, and how long does each one take?
2What costs are covered by the study, and what might I have to pay for myself, including travel, parking, or time off work?
3What happens during screening, and what happens if the study team confirms I don't meet the criteria after those tests?
4Who pays for the scans, blood work, and other tests the trial requires — the study, my insurance, or me?
5How will being in the trial affect my regular care, and will my own doctor stay informed and involved?
6Can I leave the trial at any point if I change my mind, and what would happen to my care if I do?
A starting point for the conversation — always confirm anything about your own eligibility, costs, and care with the study team and your doctor.
What they're measuring
1
The primary objective is to determine the rate of linear growth over a 6-month period in children < 2 years of age and over a 12-month period in children ≥ 2 years of age.