Cockayne Syndrome, Type I and IICurves-CSNCT05090917
Growth Curves in Cockayne Syndrome Type 1 and Type 2
An observational study of Cockayne Syndrome, Type I and II, run by University Hospital, Strasbourg, France at 1 site in France.
Trial summary
Despite the natural progression of Cockayne's syndrome, affected patients also present with variable neurological and gastrointestinal damage (gastroesophageal reflux, recurrent vomiting, swallowing disorders, etc.) with varying repercussions on their growth. Read more about this study
- Who is it for?
- Ages 1 year to 17 years
- Conditions the study is for
- Cockayne Syndrome, Type I and II
- How many people
- 85 participants
- Where
- 1 siteFrance
- Study dates
- 1 Nov 2020to 15 May 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 1 year to 17 years
- All sexes
- No healthy volunteers
- Child
Study population: Minor patient with cockayne syndrome with molecular confirmation, treated between 01/01/1985 and 01/03/2020
Who may be able to join
- Minor patient
- Cockayne syndrome with molecular confirmation, treated between 01/01/1985 and 01/03/2020
- Collection of at least one growth data (height, weight, head circumference) for a given age
- Clinic compatible with type 1 or 2 Cockayne syndrome
- Holder of parental authority not opposing, after information, the reuse of their child's medical data for the purposes of this scientific research.
Who cannot join
- Refusal to participate in the study.
- Lack of molecular confirmation of Cockayne syndrome
- Lack of growth data for a given age.
- Clinic not compatible with type 1 or 2 Cockayne syndrome. Exclusion of type 3 Cockayne syndromes, trichothiodystrophy and xeroderma-pigmentosum.
Trial locations and contacts
1 site in 1 country.
Saïd CHAYER, PhD, HDR contact
France1 site, 1 recruiting
- Recruiting
Service de Pédiatrie1 - Hôpitaux Universitaires de Strasbourg
Strasbourg, 67091
Vincent LAUGEL, MD, PhD, 33 3 88 12 77 43, vincent.laugel@chru-strasbourg.fr
Saïd CHAYER, PhD, HDR, said.chayer@chru-strasbourg.fr
About this study
Official title: Development of Growth Curves in Cockayne Syndrome Type 1 and Type 2
Despite the natural progression of Cockayne's syndrome, affected patients also present with variable neurological and gastrointestinal damage (gastroesophageal reflux, recurrent vomiting, swallowing disorders, etc.) with varying repercussions on their growth. Acute intercurrent events such as seizures, constipation, infections can also interact with their metabolism, food intake and influence their growth. The nutritional deficit potentially involved in this growth retardation can be responsible for many manifestations such as anemia, bone fractures, fatigue, coagulation disorders responsible in total for the reduction in quality and life expectancy.
Faced with growth retardation in patients with Cockayne syndrome, medical management is difficult to establish. Is this delay part of the natural course of the pathology?
- Conditions
- Cockayne Syndrome, Type I and II
- Keywords
- Cockayne Syndrome, Cockayne Syndrome, Type I and II, Growth curves
- Medical subject headings
- Cockayne Syndrome
What is being measured
The primary outcome is the main question the study is designed to answer.
Development of growth curves in Cockayne syndrome type 1 and type 2 from existing medical data
Measured: Files analysed retrospectively from January 01, 1985 to March 01, 2020 will be examined]
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted25 Oct 2021
- Study start1 Nov 2020
- Primary completion15 May 2022 (estimated)
- Study completion15 May 2022 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cockayne Syndrome, Type I and IIStudies of the same condition, open and under way.
- More trials run by University Hospital, Strasbourg, FranceThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case-control
- Time perspective
- Retrospective
- Enrolment
- 85 participants (estimated)
Sponsor and investigators
- Lead sponsor
- University Hospital, Strasbourg, France
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
- Investigators
- Vincent LAUGEL, MD, PhD, Principal investigator, Service de Pédiatrie1 - Hôpitaux Universitaires de Strasbourg
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05090917
- Sponsor's protocol ID
- 7886
- Acronym
- Curves-CS
- Status last verified
- Oct 2021
- First submitted
- 11 Oct 2021
- Last updated
- 1 Dec 2021
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 1 Dec 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Development of Growth Curves in Cockayne Syndrome Type 1 and Type 2
- ClinicalTrials.gov ID
- NCT05090917
- Acronym / sponsor ID
- Curves-CS / 7886
- Conditions
- Cockayne Syndrome, Type I and II
- Keywords
- Cockayne Syndrome, Cockayne Syndrome, Type I and II, Growth curves
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 85 participants (estimated)
- Lead sponsor
- University Hospital, Strasbourg, FranceUniversity, hospital or other
- Study start
- 1 Nov 2020 (actual)
- Primary completion
- 15 May 2022 (estimated)
- Study completion
- 15 May 2022 (estimated)
- First posted
- 25 Oct 2021 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
Despite the natural progression of Cockayne's syndrome, affected patients also present with variable neurological and gastrointestinal damage (gastroesophageal reflux, recurrent vomiting, swallowing disorders, etc.) with varying repercussions on their growth. Acute intercurrent events such as seizures, constipation, infections can also interact with their metabolism, food intake and influence their growth. The nutritional deficit potentially involved in this growth retardation can be responsible for many manifestations such as anemia, bone fractures, fatigue, coagulation disorders responsible in total for the reduction in quality and life expectancy.
Faced with growth retardation in patients with Cockayne syndrome, medical management is difficult to establish. Is this delay part of the natural course of the pathology?
Study design
- Observational model
- Case-control
- Time perspective
- Retrospective
Who may qualify
- Ages
- 1 year to 17 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child
- Study population
- Minor patient with cockayne syndrome with molecular confirmation, treated between 01/01/1985 and 01/03/2020
Key inclusion criteria (5 listed)
- Minor patient
- Cockayne syndrome with molecular confirmation, treated between 01/01/1985 and 01/03/2020
- Collection of at least one growth data (height, weight, head circumference) for a given age
- Clinic compatible with type 1 or 2 Cockayne syndrome
- Holder of parental authority not opposing, after information, the reuse of their child's medical data for the purposes of this scientific research.
Key exclusion criteria (4 listed)
- Refusal to participate in the study.
- Lack of molecular confirmation of Cockayne syndrome
- Lack of growth data for a given age.
- Clinic not compatible with type 1 or 2 Cockayne syndrome. Exclusion of type 3 Cockayne syndromes, trichothiodystrophy and xeroderma-pigmentosum.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Development of growth curves in Cockayne syndrome type 1 and type 2 from existing medical data
Time frame: Files analysed retrospectively from January 01, 1985 to March 01, 2020 will be examined]
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 1 Dec 2021.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Saïd CHAYER, PhD, HDR contact
Vincent LAUGEL, MD, PhD principal investigator
Service de Pédiatrie1 - Hôpitaux Universitaires de Strasbourg
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cockayne Syndrome, Type I and II, run by University Hospital, Strasbourg, France at 1 site in France.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 4 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 1 Dec 2021. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
Contact Vincent LAUGEL, MD, PhD (33 3 88 12 77 43, vincent.laugel@chru-strasbourg.fr), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Development of growth curves in Cockayne syndrome type 1 and type 2 from existing medical data (time frame: Files analysed retrospectively from January 01, 1985 to March 01, 2020 will be examined]). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 1 Dec 2021.