InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia

Familial Chylomicronemia Syndrome Clinical Trial

— InFocus  

Official title:

InFocus France Epidemiological Study of Health Burden in Major Hypertriglyceridemia

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT04223908
• Other study ID #: 2018-A01735-50
• Status: Completed
• Start date: September 19, 2018
• Est. completion date: September 1, 2019

Study information

• Verified date: January 2020
• Source: New French Society of Atherosclerosis
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial Summary

FCS and MCS patients recruited from 7 academic reference centers were invited to answer a paper or a web questionnaire. Questions encompassed demographics, physical, cognitive and mental symptoms, health care circuit, past and current disease management, satisfaction regarding healthcare providers and impact on daily life.

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 52
• Est. completion date: September 1, 2019
• Est. primary completion date: September 1, 2019
• Gender: All
• Age group: 18 Years to 100 Years

Eligibility

Inclusion Criteria:

• any adult FCS patient genetically documented any adult MCS patient genetically or phenotypically documented

Exclusion Criteria:

• legal restrictions

Related Conditions & MeSH terms

• Familial Chylomicronemia Syndrome
• Hyperlipoproteinemia Type I
• Hypertriglyceridemia
• Multifactorial Chylomicronemia Syndrome
• Syndrome

Intervention

Other:
• observational : questionnaries for assessment of health burden
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.

Locations

Country	Name	City	State
France	new French society of atherosclerosis	Saint-Maur-des-Fossés

Sponsors (2)

Lead Sponsor	Collaborator
New French Society of Atherosclerosis	Akcea Therapeutics

Country where clinical trial is conducted

France, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	abdominal pain	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	dietary burden	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
Secondary	psychological burden	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