Spinal Muscular AtrophyNCT03808233
Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy
An observational study of Spinal Muscular Atrophy, run by University of Texas Southwestern Medical Center.
Trial summary
The purpose of this study is to investigate the measurement properties of the Ability Captured Through Interactive Video Evaluation-mini (ACTIVE-mini) for quantifying movement in infants with Spinal Muscular Atrophy (SMA). Read more about this study
- Who is it for?
- Any age
- Conditions the study is for
- Spinal Muscular Atrophy
- What is being tested?
- ACTIVE mini
- How many people
- 25 participants
- Where
- No sites listed yet
- Study dates
- 5 Nov 2016to 31 Aug 2018
- 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 Any age
- All sexes
- Child, Adult, Older adult
Study population: SMA
Who may be able to join
- -
- Participants will be included in this study if they:
- are non-rolling at time of enrollment
- age birth to 25 months
- have no concomitant system pathology that would limit clinical evaluation.
- These inclusion criteria are set for both affected babies and healthy controls.
Who cannot join
- Participants will be excluded from this study if they:
- demonstrate evidence of renal dysfunction, central nervous system damage, neuro-degenerative or neuromuscular disease other than SMA type I or II;
- require mechanical ventilation of any type > 16 hours per day.
About this study
The purpose of this study is to investigate the measurement properties of the Ability Captured Through Interactive Video Evaluation-mini (ACTIVE-mini) for quantifying movement in infants with Spinal Muscular Atrophy (SMA). Specifically, I will investigate within-day and between-day test-retest reliability and calculate the minimal detectable change of the ACTIVE-mini. Additionally, I will determine the concurrent validity of the ACTIVE-mini with The Children's Hospital of Philadelphia Infant Test for Neuromuscular Disease (CHOP INTEND) and the construct validity of the ACTIVE-mini in infants with SMA using a known group methodology.
Read the detailed description
This study was a cross-sectional, repeated measure design, investigating the test-retest within- and between-day reliability and the convergent construct validity and the known-groups method construct validity of the ACTIVE-mini. The group variable included two levels (non-rolling infants with SMA and function matched non-rolling typically developing infants). The dependent variables included a predicted CHOP INTEND score determined by data captured by the ACTIVE-mini and the CHOP INTEND extremity score. The dependent variables were collected at two time points over two days at a minimum of 24 hours and no more than 30 days between collections. Variables were collected in a standard order of assessment. All participants underwent all assessments as set out in the procedures. Based on results from pilot data, the conclusion was made that the ACTIVE-mini could quantify infant movement parameters well enough to warrant further investigation. These data were important in establishing the feasibility of the specific aims of this research, because they provide initial validation of the ability of ACTIVE-mini to quantify basic infant movement parameters
- Conditions
- Spinal Muscular Atrophy
- Being tested
- ACTIVE mini
- 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.
ACTIVE miniOther
the ACTIVE-mini, is a non-invasive method of recording movement in an infant using a KINECT camera system. This camera is positioned over a resting infant and measures natural movement over a two minute time period.
| Study group | Role | What they receive |
|---|---|---|
| Spinal Muscular Atrophy Type 1 | Non rolling infants or children with SMA Receives: Other: ACTIVE mini | |
| Non Rolling Function matched control | Non rolling typically developing infant Receives: Other: ACTIVE mini |
What is being measured
The primary outcome is the main question the study is designed to answer.
ACTIVE mini
the ACTIVE-mini, is a physiological parameter non-invasive method of recording movement in an infant using a KINECT camera system. This camera is positioned over a resting infant and measures natural movement over a two minute time period.
Measured: 2 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Jan 2019
- Study start5 Nov 2016
- Primary completion1 Jun 2018
- Study completion31 Aug 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Spinal Muscular AtrophyStudies of the same condition, open and under way.
- More trials run by University of Texas Southwestern Medical CenterThe 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
- Cohort
- Time perspective
- Prospective
- Enrolment
- 25 participants (actual)
Sponsor and investigators
- Lead sponsor
- University of Texas Southwestern Medical Center
- Sponsor type
- University, hospital or other
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03808233
- Sponsor's protocol ID
- STU 052016-109
- Status last verified
- Jan 2019
- First submitted
- 29 Jun 2018
- Last updated
- 17 Jan 2019
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 17 Jan 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Reliability and Validity of the ACTIVE-mini for Quantifying Movement in Infants With Spinal Muscular Atrophy
- ClinicalTrials.gov ID
- NCT03808233
- Acronym / sponsor ID
- STU 052016-109
- Conditions
- Spinal Muscular Atrophy
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 25 participants (actual)
- Lead sponsor
- University of Texas Southwestern Medical CenterUniversity, hospital or other
- Study start
- 5 Nov 2016 (actual)
- Primary completion
- 1 Jun 2018 (actual)
- Study completion
- 31 Aug 2018 (actual)
- First posted
- 17 Jan 2019 (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.
The purpose of this study is to investigate the measurement properties of the Ability Captured Through Interactive Video Evaluation-mini (ACTIVE-mini) for quantifying movement in infants with Spinal Muscular Atrophy (SMA). Specifically, I will investigate within-day and between-day test-retest reliability and calculate the minimal detectable change of the ACTIVE-mini. Additionally, I will determine the concurrent validity of the ACTIVE-mini with The Children's Hospital of Philadelphia Infant Test for Neuromuscular Disease (CHOP INTEND) and the construct validity of the ACTIVE-mini in infants with SMA using a known group methodology.
Detailed description
This study was a cross-sectional, repeated measure design, investigating the test-retest within- and between-day reliability and the convergent construct validity and the known-groups method construct validity of the ACTIVE-mini. The group variable included two levels (non-rolling infants with SMA and function matched non-rolling typically developing infants). The dependent variables included a predicted CHOP INTEND score determined by data captured by the ACTIVE-mini and the CHOP INTEND extremity score. The dependent variables were collected at two time points over two days at a minimum of 24 hours and no more than 30 days between collections. Variables were collected in a standard order of assessment. All participants underwent all assessments as set out in the procedures. Based on results from pilot data, the conclusion was made that the ACTIVE-mini could quantify infant movement parameters well enough to warrant further investigation. These data were important in establishing the feasibility of the specific aims of this research, because they provide initial validation of the ability of ACTIVE-mini to quantify basic infant movement parameters
Study design
- Observational model
- Cohort
- Time perspective
- Prospective
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Spinal Muscular Atrophy Type 1 | – | Other: ACTIVE miniNon rolling infants or children with SMA |
| Non Rolling Function matched control | – | Other: ACTIVE miniNon rolling typically developing infant |
Who may qualify
- Sex
- All
- Age groups
- Child, Adult, Older adult
- Study population
- SMA
Key inclusion criteria (6 listed)
- -
- are non-rolling at time of enrollment
- age birth to 25 months
- have no concomitant system pathology that would limit clinical evaluation.
- These inclusion criteria are set for both affected babies and healthy controls.
Key exclusion criteria (3 listed)
- demonstrate evidence of renal dysfunction, central nervous system damage, neuro-degenerative or neuromuscular disease other than SMA type I or II;
- require mechanical ventilation of any type > 16 hours per day.
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 |
|---|---|---|
| ACTIVE minithe ACTIVE-mini, is a non-invasive method of recording movement in an infant using a KINECT camera system. This camera is positioned over a resting infant and measures natural movement over a two minute time period. | Other | Non Rolling Function matched control, Spinal Muscular Atrophy Type 1 |
Primary outcome
ACTIVE mini
Time frame: 2 minutes
the ACTIVE-mini, is a physiological parameter non-invasive method of recording movement in an infant using a KINECT camera system. This camera is positioned over a resting infant and measures natural movement over a two minute time period.
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 17 Jan 2019.
- Individual participant dataSharing planned: No
Related trials for Spinal Muscular Atrophy
RecruitingObservational study
An International Federated Model for Wearable-derived Remote Longitudinal Motor Monitoring in Young Children With Spinal Muscular Atrophy Compared With Healthy Controls: Active-NBS Study (UK)
Spinal Muscular Atrophy (SMA)
Enrolling by invitationPhase 1/2
Gene Therapy for IGHMBP2-Related Diseases
SMARD1, CMT2S
RecruitingPhase 4
A Study Evaluating the Effectiveness and Safety of Risdiplam Administered as an Early Intervention in Pediatric Participants With Spinal Muscular Atrophy After Gene Therapy
Muscular Atrophy, Spinal
RecruitingPhase 4
ASsessing The REAl-world Safety & Effectiveness of Spinal Muscular Atrophy Participants Treated With Intrathecal Onasemnogene Abeparvovec-brve (OAV101B) (ITVISMA®): A U.S. Pragmatic Multicenter Study (STREAM)
Spinal Muscular Atrophy
Not yet recruitingEarly Phase 1
SPX-01 Gene Therapy in Pediatric Patients With Spinal Muscular Atrophy Type 1 (RISE)
Spinal Muscular Atrophy (SMA)
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.
The record lists no contact. Use the official record to reach the sponsor.
Questions about this trial
What is this clinical trial studying?
An observational study of Spinal Muscular Atrophy, run by University of Texas Southwestern Medical Center.
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 6 inclusion and 3 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 17 Jan 2019. Status can change between updates, so confirm with the study contact before making plans.
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?
ACTIVE mini (time frame: 2 minutes). 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 17 Jan 2019.