Muscular Atrophy, SpinalNCT05291962
Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy
An observational study of Muscular Atrophy, Spinal and Child, Only, run by Ankara City Hospital Bilkent at 1 site in Turkey (Türkiye).
Trial 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. Read more about this study
- Who is it for?
- Ages Up to 18 yearsHealthy volunteers can take part
- Conditions the study is for
- Muscular Atrophy, Spinal, Child, Only, Anesthesia
- What is being tested?
- Retrospective observational study
- How many people
- 14 participants
- Where
- 1 siteTurkey (Türkiye)
- Study dates
- 1 Jan 2022to 31 Jan 2022
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages Up to 18 years
- All sexes
- Healthy volunteers accepted
- Child, Adult
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
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
1 site in 1 country.
About this study
Official title: Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy Undergoing Intrathecal Treatment
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.
Read the detailed description
Design: This research is a retrospective obsevational study. Methods: After approval Institutional Review Board, 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. All our patients with SMA type II and III who underwent intrathecal treatment are given sedation with different combination of anesthetic agent. Due to peripheral intravenous line was placed previously, intravenous induction was performed in all of our patients For procedural sedation, anesthesiologists used different combination of midazolam, ketamine, propofol, fentanyl or remifentanyl, depending on the patient's requirement. They were oxygenated with a face mask or nasal cannula while their spontaneous breathing continues. All patients were recovered in the recovery room.
Statistical analysis: The data were analyzed by using SPSS Statistics Software (SPSS 24, Chicago, IL, USA) Descriptive statistics were used in this study. Mean and standart deviation were calculated for Age (year), Weight (kilogram), Midazolam Dose (mg/kg), Fentanyl Dose (mcg/kg), Remifentanyl Dose (mcg/kg), Propofol Dose (mg/kg), Ketamine Dose (mg/kg) and Length of Stay in Recovery Room (minute) and frequency analysis were made for the number of intervention.
- Conditions
- Muscular Atrophy, Spinal, Child, Only, Anesthesia
- Being tested
- Retrospective observational study
- Keywords
- Spinal muscular atrophy, Procedural sedation, Children, Intratechal treatment
- Medical subject headings
- Muscular Atrophy, Spinal
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
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.
What is being measured
The primary outcome is the main question the study is designed to answer.
Procedural sedation
The number of pediatric patients with SMA I,II and III who underwent intrathecal treatment required procedural sedation
Measured: 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
Measured: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Age
Age in year
Measured: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Gender
Gender as female/male
Measured: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Weight
Weight in kilograms
Measured: 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
Measured: 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
Measured: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Secondary outcomes (4)
Presence of scoliosis
Yes or no
Measured: 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
The number of intrathecal treatments was administered for each patient over 4 years was recorded.
Measured: 4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
By whom the procedure was performed
It was recorded who performed the lumbar puncture procedure from a neurologist, neurosurgeon, or anesthesiologist.
Measured: 4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Length of time spent in recovery room
Recovery time in minutes
Measured: 4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Mar 2022
- Study start1 Jan 2022
- Primary completion31 Jan 2022
- Study completion31 Jan 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Muscular Atrophy, SpinalStudies of the same condition, open and under way.
- More trials run by Ankara City Hospital BilkentThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Other
- Time perspective
- Retrospective
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Ankara City Hospital Bilkent
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Feyza Sever, Ankara City Hospital Bilkent
- Investigators
- Feyza Sever, Principal investigator, Ankara City Hospital Bilkent
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT05291962
- Sponsor's protocol ID
- 2019-159
- Status last verified
- Mar 2022
- First submitted
- 8 Feb 2022
- Last updated
- 23 Mar 2022
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 23 Mar 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Procedural Sedation for Pediatric Patients With Spinal Muscular Atrophy Undergoing Intrathecal Treatment
- ClinicalTrials.gov ID
- NCT05291962
- Acronym / sponsor ID
- 2019-159
- Conditions
- Muscular Atrophy, Spinal, Child, Only, Anesthesia
- Keywords
- Spinal muscular atrophy, Procedural sedation, Children, Intratechal treatment
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Ankara City Hospital BilkentUniversity, hospital or other
- Study start
- 1 Jan 2022 (actual)
- Primary completion
- 31 Jan 2022 (actual)
- Study completion
- 31 Jan 2022 (actual)
- First posted
- 23 Mar 2022 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Detailed description
Design: This research is a retrospective obsevational study. Methods: After approval Institutional Review Board, 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. All our patients with SMA type II and III who underwent intrathecal treatment are given sedation with different combination of anesthetic agent. Due to peripheral intravenous line was placed previously, intravenous induction was performed in all of our patients For procedural sedation, anesthesiologists used different combination of midazolam, ketamine, propofol, fentanyl or remifentanyl, depending on the patient's requirement. They were oxygenated with a face mask or nasal cannula while their spontaneous breathing continues. All patients were recovered in the recovery room.
Statistical analysis: The data were analyzed by using SPSS Statistics Software (SPSS 24, Chicago, IL, USA) Descriptive statistics were used in this study. Mean and standart deviation were calculated for Age (year), Weight (kilogram), Midazolam Dose (mg/kg), Fentanyl Dose (mcg/kg), Remifentanyl Dose (mcg/kg), Propofol Dose (mg/kg), Ketamine Dose (mg/kg) and Length of Stay in Recovery Room (minute) and frequency analysis were made for the number of intervention.
Study design
- Observational model
- Other
- Time perspective
- Retrospective
Who may qualify
- Ages
- Up to 18 years
- Sex
- All
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- 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
Key inclusion criteria (1 listed)
- 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.
Key exclusion criteria (2 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Retrospective observational studyData 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. | Other | – |
Primary outcome
Procedural sedation
Time frame: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
The number of pediatric patients with SMA I,II and III who underwent intrathecal treatment required procedural sedation
Anesthetics use
Time frame: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
The anesthetic agents and their doses that used during procedure
Age
Time frame: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Age in year
Gender
Time frame: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Gender as female/male
Weight
Time frame: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Weight in kilograms
American Society of Anesthesiologists (ASA) physical status
Time frame: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
ASA score
Type of SMA
Time frame: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
SMA I, SMA II and SMA III
4 secondary outcomes
Presence of scoliosis
Time frame: 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
Time frame: 4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
By whom the procedure was performed
Time frame: 4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Length of time spent in recovery room
Time frame: 4 years The data of the patients were retrospectively analyzed between July 2017 and December 2021.)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 23 Mar 2022.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Feyza Sever principal investigator
Ankara City Hospital Bilkent
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Muscular Atrophy, Spinal and Child, Only, run by Ankara City Hospital Bilkent at 1 site in Turkey (Türkiye).
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 1 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 23 Mar 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in Turkey (Türkiye). Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Procedural sedation (time frame: 4 years (The data of the patients were retrospectively analyzed between July 2017 and December 2021.)). The study lists 7 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 23 Mar 2022.