NCT05291962

Brief Summary

Background and Aim: Spinal muscular atrophy (SMA) is a neuromuscular disease characterized by progressive symmetrical weakness and atrophy of proximal muscles causing from degeneration of anterior horn cells of spinal cord. Nusinersen must be administered intrathecally and this treatment is specially for spinal muscular atrophy. Procedural sedation is commonly enough for intrathecal treatment in children. In this retrospective study, the investigators aimed to present our experience in procedural sedation for the intrathecal treatment of patients with SMA 1,2 and 3 in our hospital.

Trial Health

87
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
14

participants targeted

Target at below P25 for all trials

Timeline
Completed

Started Jan 2022

Shorter than P25 for all trials

Geographic Reach
1 country

1 active site

Status
completed

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Start

First participant enrolled

January 1, 2022

Completed
1 month until next milestone

Primary Completion

Last participant's last visit for primary outcome

January 31, 2022

Completed
Same day until next milestone

Study Completion

Last participant's last visit for all outcomes

January 31, 2022

Completed
8 days until next milestone

First Submitted

Initial submission to the registry

February 8, 2022

Completed
1 month until next milestone

First Posted

Study publicly available on registry

March 23, 2022

Completed
Last Updated

March 23, 2022

Status Verified

March 1, 2022

Enrollment Period

1 month

First QC Date

February 8, 2022

Last Update Submit

March 18, 2022

Conditions

Muscular Atrophy, SpinalChild, OnlyAnesthesia

Keywords

Spinal muscular atrophyProcedural sedationChildrenIntratechal treatment

Outcome Measures

Primary Outcomes (7)

  • Procedural sedation

    The number of pediatric patients with SMA I,II and III who underwent intrathecal treatment required procedural sedation

    4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

  • Anesthetics use

    The anesthetic agents and their doses that used during procedure

    4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

  • Age

    Age in year

    4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

  • Gender

    Gender as female/male

    4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

  • Weight

    Weight in kilograms

    4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

  • American Society of Anesthesiologists (ASA) physical status

    ASA score

    4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

  • Type of SMA

    SMA I, SMA II and SMA III

    4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

Secondary Outcomes (4)

  • Presence of scoliosis

    4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)(

  • How many times intrathecal treatment was performed for each patient

    4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

  • By whom the procedure was performed

    4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

  • Length of time spent in recovery room

    4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)

Interventions

Retrospective observational studyOTHER

Data from anesthesia charts and electronic medical records of 14 patients with SMA type I, II and III who underwent procedural sedation,such as; the demographics data of the patients (age, gender, weight, American Society of Anesthesiologist (ASA) Physical Status), which anesthetic agent was used and their doeses, during of anesthesia, presence of scoliosis, by whom the procedure was performed etc. was reviewed.

Eligibility Criteria

AgeUp to 18 Years
Sexall
Healthy VolunteersYes
Age GroupsChild (0-17), Adult (18-64)
Sampling MethodProbability Sample
Study Population

Pediatric patients younger than 18 years old with SMA 1,2 and 3 who were to undergo intrathecal treatment under procedural sedation in our clinic

You may qualify if:

  • Pediatric patients younger than 18 years old with SMA 1,2 and 3 who were to undergo intrathecal Nusinersen treatment were included in this study.

You may not qualify if:

  • Patients whose procedures were performed under fluoroscopic guidance in the radiology unit were excluded from the study because their procedures were performed in another hospital.
  • Patients without a diagnosis of SMA

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (1)

Feyza Sever

Ankara, Çankaya, 06800, Turkey (Türkiye)

Location

MeSH Terms

Conditions

Muscular Atrophy, Spinal

Condition Hierarchy (Ancestors)

Spinal Cord DiseasesCentral Nervous System DiseasesNervous System DiseasesMotor Neuron DiseaseNeurodegenerative DiseasesNeuromuscular Diseases

Study Officials

  • Feyza Sever

    Ankara City Hospital Bilkent

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
OTHER
Time Perspective
RETROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Principal investigator

Study Record Dates

First Submitted

February 8, 2022

First Posted

March 23, 2022

Study Start

January 1, 2022

Primary Completion

January 31, 2022

Study Completion

January 31, 2022

Last Updated

March 23, 2022

Record last verified: 2022-03

Locations

TU(1)