Overview

This trial is active, not recruiting.

Condition spinal muscular atrophy 1
Treatment follow-up diary and questionnaire
Sponsor Assistance Publique - Hôpitaux de Paris
Start date June 2012
End date December 2018
Trial size 39 participants
Trial identifier NCT01862042, AOM 11183

Summary

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

United States No locations recruiting
Other Countries No locations recruiting

Study Design

Intervention model single group assignment
Masking open label
Primary purpose supportive care
Arm
(Other)
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.
follow-up diary and questionnaire Supportive and Palliative care
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.

Primary Outcomes

Measure
%O2
time frame: until 2 years

Secondary Outcomes

Measure
Qualitative evaluation of the practices of care
time frame: until 2 years
Evaluation of nutritional status
time frame: until 2 years
Evaluation of orthopedic facilities
time frame: until 2 years
Evaluation of comfort
time frame: until 2 years

Eligibility Criteria

Male or female participants up to 1 year old.

Inclusion Criteria: - SMA type 1 under 1 an - Genetic confirmation Exclusion Criteria: - No genetic confirmation - SMA type 1 over 1 year

Additional Information

Official title Evaluation of Palliative and Supportive Care for Spinal Muscular Atrophy (SMA) Type 1 Patients
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.
Trial information was received from ClinicalTrials.gov and was last updated in June 2016.
Information provided to ClinicalTrials.gov by Assistance Publique - Hôpitaux de Paris.