- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT04223908
InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia (InFocus)
Study Overview
Status
Intervention / Treatment
Detailed Description
cross-sectional study involving a sample of FCS and MCS patients in 1:1 ratio. The study was run in seven academic reference centers in France. Adult patients with documented history of fasting TG > 10 mmol/L (885 mg/dL) were recruited. Patients had to be genetically characterized or MCS patients had a MCS score ≤ 9 [11]. Group sample sizes of 19 FCS and 19 MCS have been calculated to achieve an at least 80% power to detect a 40% difference in the frequency of abdominal pain assuming that 90% of FCS patients would complain about abdominal pain.
The study protocol was approved by a nationally appointed ethics committee (Comité de Protection des Personnes - Ile de France 5) under number 18040. The study protocol complied with the ethical guidelines of the Declaration of Helsinki and the French Bioethics Law Jarde. The data management complied with the CNIL requirement. Each patient was informed about the aims and constraints of the study and signed a non-opposition form prior to any study procedure.
Health Burden assessment The French IN-FOCUS study was designed to capture current and retrospective data in FCS or MCS. Patients were called by the participating centers and were proposed to answer a paper questionnaire or to connect to a web-based questionnaire The questionnaire was divided into sections specifically assessing various domains of their experience: demographic data, signs, symptoms and complications of the disease, healthcare circuit, management, relationship with healthcare providers, and burden of the disease on daily life. (Details are available as supplementary material S1). The questionnaire focused on symptoms because the interest of quality of life assessment tools is debated in rare diseases with episodic manifestations. Indeed, most of these tools refer to a limited recall period.
Data analysis The statistical analysis was performed by Soladis (Lyon, France) using the Statistical Analysis System (SAS) software 9.4 (SAS Institute, Cary, NC, USA). Continuous variables were summarized by the number of observed data, mean, standard deviation (SD), median, first and third quartiles. Categorical variables were described as numbers and percentages calculated on the number of observed data. FCS and MCS subjects were compared using Student's t test or U Mann-Whitney's test. Categorical variables were compared by the chi squared test or the Fisher's exact test. Tests were two-sided and the significance threshold was set at 5%. No adjustment of the Type 1 error risk was made for multiplicity. Impact of history of AP was evaluated in a multivariable model (logistic regression for binary variables, ordinal logistic regression for ordinal variables) including the type of disease (FCS or MCS), history of AP and the interaction between these two predictors.
Study Type
Enrollment (Actual)
Contacts and Locations
Study Locations
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Saint-Maur-des-Fossés, France, 94 100
- new French society of atherosclerosis
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Participation Criteria
Eligibility Criteria
Ages Eligible for Study
Accepts Healthy Volunteers
Genders Eligible for Study
Sampling Method
Study Population
Description
Inclusion Criteria:
- any adult FCS patient genetically documented any adult MCS patient genetically or phenotypically documented
Exclusion Criteria:
- legal restrictions
Study Plan
How is the study designed?
Design Details
- Observational Models: Case-Control
- Time Perspectives: Retrospective
Cohorts and Interventions
Group / Cohort |
Intervention / Treatment |
---|---|
FCS
patient with genetically documented familial chylomicronemia syndrome
|
The French IN-FOCUS study was designed to capture current and retrospective data in FCS or MCS.
Patients were called by the participating centers and were proposed to answer a paper questionnaire or to connect to a web-based questionnaire The questionnaire was divided into sections specifically assessing various domains of their experience: demographic data, signs, symptoms and complications of the disease, healthcare circuit, management, relationship with healthcare providers, and burden of the disease on daily life.
(Details are available as supplementary material S1).
The questionnaire focused on symptoms because the interest of quality of life assessment tools is debated in rare diseases with episodic manifestations.
Indeed, most of these tools refer to a limited recall period.
|
MCS
patient with genetically or phenotypically documented multifactorial chylomicronemia syndrome
|
The French IN-FOCUS study was designed to capture current and retrospective data in FCS or MCS.
Patients were called by the participating centers and were proposed to answer a paper questionnaire or to connect to a web-based questionnaire The questionnaire was divided into sections specifically assessing various domains of their experience: demographic data, signs, symptoms and complications of the disease, healthcare circuit, management, relationship with healthcare providers, and burden of the disease on daily life.
(Details are available as supplementary material S1).
The questionnaire focused on symptoms because the interest of quality of life assessment tools is debated in rare diseases with episodic manifestations.
Indeed, most of these tools refer to a limited recall period.
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
abdominal pain
Time Frame: 12 previous months record
|
Symptoms were assessed as present/absent within the 12 months prior to the study; if present, the patient was asked to rate their frequency (yearly, every 3 months, monthly, weekly, daily using a 5-point verbal numeric scale) and severity using a 7-point verbal numeric scale with 1=very mild and 7=very important.
|
12 previous months record
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
dietary burden
Time Frame: 12 previous months records
|
Consuming more fats than allowed to avoid attracting attention Longing for rich-fat food Meals lack variety Carefully reading the food labelling Feeling frustrated by his/her diet Controlling fat consumption is a hard task Avoids alcohol consumption to prevent symptoms Fasting voluntarily Preparing a special meal different from the rest of the family Symptoms were assessed as present/absent within the 12 months prior to the study; if present, the patient was asked to rate their frequency (yearly, every 3 months, monthly, weekly, daily using a 5-point verbal numeric scale) and severity using a 7-point verbal numeric scale with 1=very mild and 7=very important. |
12 previous months records
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psychological burden
Time Frame: 12 previous months record
|
Feeling anxious when eating out Feeling anxious if not compliant with his/her diet Trouble concentrating due to insufficient satiety Feeling guilty when drinking small amounts of alcohol Symptoms were assessed as present/absent within the 12 months prior to the study; if present, the patient was asked to rate their frequency (yearly, every 3 months, monthly, weekly, daily using a 5-point verbal numeric scale) and severity using a 7-point verbal numeric scale with 1=very mild and 7=very important.
|
12 previous months record
|
Collaborators and Investigators
Collaborators
Investigators
- Study Chair: Philippe Moulin, New French Atherosclerosis Siciety
Study record dates
Study Major Dates
Study Start (Actual)
Primary Completion (Actual)
Study Completion (Actual)
Study Registration Dates
First Submitted
First Submitted That Met QC Criteria
First Posted (Actual)
Study Record Updates
Last Update Posted (Actual)
Last Update Submitted That Met QC Criteria
Last Verified
More Information
Terms related to this study
Keywords
Additional Relevant MeSH Terms
Other Study ID Numbers
- 2018-A01735-50
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
IPD Plan Description
IPD Sharing Supporting Information Type
- Study Protocol
- Statistical Analysis Plan (SAP)
- Informed Consent Form (ICF)
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Studies a U.S. FDA-regulated device product
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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