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Study examines how to better support families of infants with fatal muscle disease

NCT ID NCT01862042

Knowledge-focused Sponsor: Assistance Publique - Hôpitaux de Paris Source: ClinicalTrials.gov ↗

What the study statuses mean

This study's is highlighted.

Recruitment status, easiest to join first

Recruiting now
This trial is taking on new participants right now.
Not yet recruiting
Registered, but not yet taking participants.
By invitation only
Not open to general applications. Only people the study team invites can take part.
Paused
Paused for now. It may or may not start again.
Ongoing
Running, but no longer taking on new participants.
Completed This study
The trial has finished. Results may not be published yet.
Stopped early
Stopped early, before it reached the end. That can be for many reasons, including safety.
Cancelled
Cancelled before anyone took part.

Expanded access (not trials)

Expanded access
Not a trial. This treatment can be requested outside a study, case by case, for people who qualify.
Expanded access (paused)
Not a trial. The treatment can normally be requested outside a study, but is unavailable right now.
Expanded access (ended)
Not a trial. The treatment could once be requested outside a study, but no longer can.
Approved
The treatment has been approved, so it is available normally rather than through this programme.

When the status isn't known

Details not published
The full record has not been published yet, so there is little to show here.
Status unknown
This status has not been confirmed recently, so it may be out of date.

First seen Jun 26, 2026 · Last updated Jun 26, 2026 · Updated 1 time

Summary

This study looked at the quality of supportive and palliative care for infants under one year old with spinal muscular atrophy (SMA) type 1, a severe genetic muscle disease. Researchers followed 39 infants and asked families to keep a diary about care. One year after the child's death, parents completed a questionnaire with a psychologist to share their experiences and challenges. The goal was to understand what works well and what could be improved in end-of-life care for these infants.

What this could mean

Our plain-language read of the trial. This is informational only, not medical advice or a prediction.

What this could lead to
If successful, this study could help improve how doctors and families manage care for infants with SMA type 1, making the experience more comfortable and supportive.
What could go wrong
This is a small, observational study with only 39 participants and no new treatment being tested. Results may not apply to all patients or change standard care.

This is an AI summary of the original study and may miss details. Read our disclaimer.

Study facts

What this study's own registry entry says, in plain language.

Phase

Not a phased trial

Phase numbers describe drug development. The registry uses this when they do not apply, as it does for trials of devices, procedures or behaviour changes, and for observational studies.

Participants

39 people

The number who actually took part.

Started

Jun 2012

Finished

Jun 2018

Lead sponsor

Other sponsor

The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.

Who can take part

This study's own entry requirements. Only the study team can say for certain whether you qualify.

Ages

1 day to 1 year

Sex

Anyone

Healthy volunteers

Not accepted

This study is not open to healthy volunteers. The entry requirements below say who it is open to.

Show the full entry requirements

Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.

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

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Conditions

The condition(s) this trial relates to.

Spinal Muscular Atrophies of Childhood spinal muscular atrophy spinal muscular atrophy, type 1

As listed by the trial registrant

The condition terms exactly as the trial's registrant entered them.

Spinal muscular atrophy 1

Contacts and locations

Locations

  • Necker Hospital

    Paris, 75015, France

More trials for these conditions

Other studies related to the condition(s) this trial covers.