Thank you for your interest in participating in the study entitled "Exploring the Burden of Disease and Quality of Life in people with Metachromatic Leukodystrophy (MLD) in the United States and Europe: A Descriptive Study". This study is an international research programme that is being offered to the United States and some countries in Europe, including France. It is offered to parents of children with MLD or relatives of sick children to better understand the impact of the disease on patients and their families beyond the direct medical implications that it causes.
This international study is being conducted by the biotechnology company Orchard Therapeutics in partnership with ELA [European Leukodystrophy Association], the association supporting the families of MLD patients in France. This study is in no way a promotional study of a pharmaceutical product. It is a life-course research study with an MLD patient to better understand the experiences of parents and patients with MLD and to measure the burden of the disease. This study is organised in the form of questionnaires and your answers will enable healthcare professionals, patient organisations (including ELA) and the biotechnology company Orchard to better understand the disease and better respond to your needs.
The survey questionnaire was carried out with experts in the disease. Argo Santé is an independent research firm specialising in human and social sciences and the expertise of sick or disabled people in order to improve care, health and life paths for those living with an illness or loss of independence. An investigator from ARGO Santé will assist you in filling out the survey questionnaires that will be proposed to you. The biotechnology company ORCHARD Therapeutics will not be involved at any time in the collection of information or the analysis of results.
Your participation in this research is entirely free and voluntary. Your decision will not prejudice the care of your child's illness or that of the child you take care of. If you do not wish to participate, simply do not volunteer on the LEUCONNECT platform or, if you volunteer but no longer wish to participate, tell the interviewer at the first contact.
The expected results of this survey will provide a better understanding of the journey faced by patients and parents. This study may also lead to reflection on the implementation of actions to facilitate certain stages in the lives of people with MLD. We are very interested in your testimony and your valuable experience on this subject and we thank you in advance for your participation.
Who is this questionnaire for?
- For parents or relatives of minor children with MLD who have not received gene therapy treatment
- If their child is deceased, for the parents of children who have been dead for less than three years.
- For people with access to computer equipment and an internet connection and residing in France.
If you volunteer to participate in this survey, these criteria will be verified by the investigator via the Leuconnect secure platform prior to your inclusion.
How is this survey conducted?
If you are eligible and once you agree to participate, this survey will be conducted in two stages.
You will be asked before starting the survey to confirm verbally to the interviewer that you agree to participate in this study and that you do not object to the use and processing of your data collected during this interview for the purposes of the study.
Initially, an online questionnaire on living with the disease and its repercussions on all aspects of your daily life and your child's quality of life will be offered to you, which the interviewer, to facilitate the completion of the questionnaire, will fill out for you in accordance with your answers. You will see your answers displayed on your computer screen or smartphone by connecting to the application whose link will be sent to you via the secure messaging system of the LEUCONNECT platform managed by the ELA association. The application used (GoToMeeting) secures your personal data; the interviewer who will assist you in completing the questionnaires will not have access to your photo but simply to your voice in order to complete the questionnaire.
This first stage of the survey will take between 45 and 60 minutes.
The second stage, which is a more "relaxed" telephone interview will be offered to you if you wish. It will be scheduled at a later date via the messaging system of the secure LEUCONNECT platform. This interview will allow us not only to hear your personal testimony about your experience of your child's illness (or of the sick child you take care of if you are a close relative) but also to gather information about your quality of life through a standard measure such as the one we used for your child in the first stage of the survey.
The objective of these interviews is to provide avenues for understanding the burden of the disease and to identify ways to improve the course of your and your child’s life, by in no way disrupting those who have volunteered to participate.
If one of our questions or themes to be explored makes you feel uncomfortable or disturbed, or if you simply do not wish to answer them, you can let us know and adapt the questioning accordingly to preserve your well-being.
The interview will last between 30 minutes - one hour, depending on how much time you can devote to us.
This study is anonymous and confidential
We will not collect any personal data allowing us to identify you during the study (surname, first name, initials, department, care settings, etc.) or elements of your medical file. All the answers you provide will be treated in the strictest confidence and all analyses will be carried out in an anonymous and aggregated manner. Your anonymity will thus be totally preserved, in accordance with French regulations on the protection of personal data and patient health data. None of the information you entrust to us will be transmitted to other people.
The information collected will remain between you and the ARGO Santé investigators and the analyses resulting from it will only be used in the context of the study. On the other hand, if during one of these two stages (questionnaires then interview), we feel a form of uneasiness or distress on your part, we will take the liberty of alerting the association so that it can come back to you and offer you support if you wish.
It is important to note that ARGO Health investigators are not doctors or caregivers and will not be able to answer medical questions about you or the effectiveness of treatments and clinical trials. For all these questions, we invite the respondents to the survey to contact their attending physician or the ELA association, which will be able to provide them with information and appropriate care.
The individual interviews in the second stage of the study will be recorded in "audio" mode for the purposes of the study. Once the recordings have been transcribed, they will be destroyed. The transcripts of the interviews (completely anonymous) as well as the answers to the questionnaires will be kept for the entire duration of the study, at least until the study report or the scientific publication of the study results, or even later if developments in the study are envisaged for scientific purposes. They will then be archived in accordance with regulations (in general, for at least 15 years after the end of the study or its early termination).
You retain all your rights relating to the Data Protection Act specified below.
In application of the provisions of the amended law of 6 January 1978 relating to information technology, data files and liberties, as well as the European Regulation 2016/679 relating to the protection of individuals with regard to the processing of personal data and the free circulation of such data (GDPR), you have at any time the right to access, rectify and delete data collected about you, as well as the right to limit and oppose the transmission of data covered by professional secrecy that may be used in the context of this study and processed. These rights shall be exercised with the investigator (researcher). However, once your interview is coded and mixed with the other interviews as part of the collective analysis and overview of the results, it will no longer be possible to remove your data. You have the right to file a complaint with the Commission Nationale de l'Informatique et des Libertés - [CNIL: National Commission on Informatics and Liberty] (https://www.cnil.fr/fr/plaintes).
For more information about the study and the use of your information, contact (firstname.lastname@example.org).