NCT01862042

Palliative Care in Spinal Muscular Atrophy (SMA) 1

Evaluation of Palliative and Supportive Care for Spinal Muscular Atrophy (SMA) Type 1 Patients

• Status: Completed
• Phase: N/A
• Study type: Interventional
• Enrollment: 39 (actual)
• Sponsor: Assistance Publique - Hôpitaux de Paris · Academic / Other
• Sex: All
• Age: 1 Day – 1 Year
• Healthy volunteers: Not accepted

Summary

The purpose of this study is to evaluate the quality of supportive and palliative care for SMA type 1 patients.

Detailed description

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.

Conditions

• Spinal Muscular Atrophy 1

Interventions

Type	Name	Description
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.

Timeline

• Start date: 2012-06-01
• Primary completion: 2018-06-11
• Completion: 2018-06-11
• First posted: 2013-05-24
• Last updated: 2025-09-11

Locations

1 site across 1 country: France