An interventional study of Follow-up diary and questionnaire in Spinal Muscular Atrophy 1, sponsored by Assistance Publique - Hôpitaux de Paris. Completed at 1 site in France. Open to participants aged 1 Day to 1 Year. Per ClinicalTrials.gov, last updated 2025-09-11.
Sponsored by Assistance Publique - Hôpitaux de Paris · Not applicable, Interventional, and Supportive care
The purpose of this study is to evaluate the quality of supportive and palliative care for SMA type 1 patients.
Spinal Muscular Atrophy Type I (SMA I) is the most severe form of SMA. It presents in infancy and death occurs by 2 years. There is actually no curative treatment for this pathology. Support and help must be provided from the time of presentation till death and usually this period is quite short, about a couple of months. Variations in medical practice have be seen, depending on the medical experience and sometimes coupled with differences in family resources and values. The aim of the study is to evaluate the needs of the patients and their families, the medical practices, and to describe a cohort of SMA type 1 patients with the natural history of this disease. For this, a follow-up diary will be done, and this diary will be completed by the families and the different practitioners working with the patient. Will be noted in it : physical signs, all therapeutic choices and actions, evaluation of the pain and treatments. A special part of this follow-up diary will be completed by the medical doctors, after the death of the patient, with all the medication used at time of death and the conditions of the death. One year after the death of the patient, a questionnaire will be proposed to the parents of the child by a psychologist. This questionnaire will estimate the benefice of the follow-up diary, and the improvements to give in the diagnostic strategies, recommendations for assessment and monitoring, and therapeutic interventions in SMA type 1.
50 studies on the registry are indexed under Spinal Muscular Atrophies of Childhood; 17 are open to participants now.
This study's enrollment of 39 is above the median of 21 across 40 interventional studies indexed under Spinal Muscular Atrophies of Childhood.
Browse Spinal Muscular Atrophies of Childhood studies →Assistance Publique - Hôpitaux de Paris is the lead sponsor of 3,505 studies on the registry; 1,006 are open to participants now.
Counted across the registry records on this site, refreshed daily.
Exclusion Criteria:
A follow-up diary will be completed by the families and the different practitioners working with the patient. One year after the death of the patient, a questionnaire will be proposed to the parents of the child by a psychologist.
Other: Follow-up diary and questionnaire
A follow-up diary will be completed by the families and the different practitioners working with the patient. One year after the death of the patient, a questionnaire will be proposed to the parents of the child by a psychologist.
Also known as: Supportive and Palliative care
%O2
Quantitative evaluation of care : oxygen therapy and Invasive ventilation
Time frame: until 2 years
Qualitative evaluation of the practices of care
Analysis of the semi-structured questionnaire completed remotely by the methods of analysis semi-qualitative. Identification of the principal challenges faced by parents and satisfaction criteria or non-face to the proposed solutions.
Time frame: until 2 years
Evaluation of nutritional status
Quantitative evaluation of care : nutritional status and enteral nutrition
Time frame: until 2 years
Evaluation of orthopedic facilities
Quantitative evaluation of care : kinesitherapy and orthopedic facility
Time frame: until 2 years
Evaluation of comfort
quantitative evaluation of care : criterion for pain analgesics and sedatives care
Time frame: until 2 years
Plan to share: No
This study is completed, as verified in Sep 2025. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.
Spinal Muscular Atrophies of Childhood→
Assistance Publique - Hôpitaux de Paris