Rett SyndromeNCT03848442
'Uptime' Participation Intervention in Girls and Women With Rett Syndrome
A study testing 'Uptime' participation in people with Rett Syndrome, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
Trial summary
The aim of the study is to evaluate the feasibility and health-related effects of an individualized 12wk 'uptime' participation intervention in girls and women with Rett syndrome. Girls and women above 5 years of age with Rett syndrome and a confirmed MECP2 mutation will be included. Read more about this study
- Who is it for?
- Ages 5 years and older, women only
- Conditions the study is for
- Rett Syndrome
- What is being tested?
- 'Uptime' participation
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 14 participants
- Where
- 1 siteDenmark
- Study dates
- 7 Feb 2017to 15 Dec 2017
- Phase
- Interventional
- 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 5 years and older
- Women only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- Girls and women above 5 years of age with Rett syndrome and a confirmed MECP2 mutation
- Hoffer ambulation scale levels I-IV
Who cannot join
- Girls and women who have undergone orthopedic surgery in the lower extremities within the last 6 months
- Girls and women who have undergone spinal fusion within the last 12 months
Trial locations and contacts
1 site in 1 country.
Denmark1 site
Center for Rett syndrome, Department of Paediatrics and Adolescents Medicine
Copenhagen, København Ø, 2100
About this study
Official title: Feasibility and Effectiveness of an Individualized 12-week 'Uptime' Participation Intervention (U-PART) in Girls and Women With Rett Syndrome
The aim of the study is to evaluate the feasibility and health-related effects of an individualized 12wk 'uptime' participation intervention in girls and women with Rett syndrome. Girls and women above 5 years of age with Rett syndrome and a confirmed MECP2 mutation will be included. Each individual program focuses on participation in enjoyable activities to promote 'uptime' in home, school/day center and community settings. Primary outcomes are sedentary time and daily steps. Secondary outcomes are gross motor skills, walking capacity, quality of life and participation-level goals. Outcomes are evaluated on four occasions: at baseline and after a 6-week interval, immediately following the 12-week intervention program and 12 weeks after the intervention program.
- Conditions
- Rett Syndrome
- Being tested
- 'Uptime' participation
- Medical subject headings
- Rett Syndrome
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
'Uptime' participationOther
The intervention are designed for each individual and are consistent with the model for participation-based interventions. The interventions are goal-oriented (meaningful and family/client-selected goals), family/client-centered (promoted self-determination and decision making), collaborative (partnership between caregivers), strength-based (built on participant and caregiver strengths) and ecological (natural environment and real-life experiences). Design of the intervention comprised three steps: 1) Preparation period (6 weeks); 2) Intervention period (12 weeks); 3) Follow-up period (12 weeks)
| Study group | Role | What they receive |
|---|---|---|
| UPART intervention group | Experimental | A 1-group pretest-posttest design was conducted. Outcomes were evaluated on four occasions; twice during baseline separated by six weeks and immediately following a 12-week 'uptime' participation intervention and after a further 12 weeks (follow-up). Receives: Other: 'Uptime' participation |
What is being measured
The primary outcome is the main question the study is designed to answer.
ActivPAL
Change in sedentary time
Measured: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
Stepwatch Activity Monitor
Change in daily step count
Measured: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
Secondary outcomes (4)
Rett Syndrome Gross Motor Scale
Change in gross motor skills
Measured: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
2 minute walk test
Change in walking capacity
Measured: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
Quality of Life Inventory - Disability, questionnaire
Change in quality of life. The Quality of Life Inventory-Disability was used to assess quality of life. This 32 item-questionnaire was developed for children 5-18 years of age with ID. Responses are used to calculate an overall score and six subscale scores (social interaction, positive emotions, physical health, negative emotions, leisure/outdoors and independence), each scored on a 0-100 scale with higher scores representing better quality of life.
Measured: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
Goal attainment scaling
Change in participation level goals. Goal attainment scaling is a validated method for evaluating achievement of individual goals. Individual goals were assessed on a five-point rating scale ranging from -2 to +2. Baseline was set at -2, the expected level after the intervention at zero and the most favorable outcome at +2. The goals were graded in relation to frequency or duration of an activity.
Measured: Assessed twice; at baseline and after the 12 week intervention
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted20 Feb 2019
- Study start7 Feb 2017
- Primary completion15 Dec 2017
- Study completion15 Dec 2017
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Rett SyndromeStudies of the same condition, open and under way.
- More trials run by Copenhagen University Hospital, RigshospitaletThe 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
- Interventional
- Phase
- Phase N/A
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 14 participants (actual)
Sponsor and investigators
- Lead sponsor
- Copenhagen University Hospital, Rigshospitalet
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Michelle Stahlhut, Copenhagen University Hospital, Rigshospitalet
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03848442
- Sponsor's protocol ID
- Center for RTT- 2017
- Status last verified
- Feb 2019
- First submitted
- 8 Feb 2019
- Last updated
- 21 Feb 2019
- Results posted
- No
- Data monitoring committee
- 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 21 Feb 2019. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Feasibility and Effectiveness of an Individualized 12-week 'Uptime' Participation Intervention (U-PART) in Girls and Women With Rett Syndrome
- ClinicalTrials.gov ID
- NCT03848442
- Acronym / sponsor ID
- Center for RTT- 2017
- Conditions
- Rett Syndrome
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 14 participants (actual)
- Lead sponsor
- Copenhagen University Hospital, RigshospitaletUniversity, hospital or other
- Study start
- 7 Feb 2017 (actual)
- Primary completion
- 15 Dec 2017 (actual)
- Study completion
- 15 Dec 2017 (actual)
- First posted
- 20 Feb 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 aim of the study is to evaluate the feasibility and health-related effects of an individualized 12wk 'uptime' participation intervention in girls and women with Rett syndrome. Girls and women above 5 years of age with Rett syndrome and a confirmed MECP2 mutation will be included. Each individual program focuses on participation in enjoyable activities to promote 'uptime' in home, school/day center and community settings. Primary outcomes are sedentary time and daily steps. Secondary outcomes are gross motor skills, walking capacity, quality of life and participation-level goals. Outcomes are evaluated on four occasions: at baseline and after a 6-week interval, immediately following the 12-week intervention program and 12 weeks after the intervention program.
Study design
- Primary purpose
- Other
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| UPART intervention group | Experimental | Other: 'Uptime' participationA 1-group pretest-posttest design was conducted. Outcomes were evaluated on four occasions; twice during baseline separated by six weeks and immediately following a 12-week 'uptime' participation intervention and after a further 12 weeks (follow-up). |
Who may qualify
- Ages
- 5 years and older
- Sex
- Female only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (2 listed)
- Girls and women above 5 years of age with Rett syndrome and a confirmed MECP2 mutation
- Hoffer ambulation scale levels I-IV
Key exclusion criteria (2 listed)
- Girls and women who have undergone orthopedic surgery in the lower extremities within the last 6 months
- Girls and women who have undergone spinal fusion within the last 12 months
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 |
|---|---|---|
| 'Uptime' participationThe intervention are designed for each individual and are consistent with the model for participation-based interventions. The interventions are goal-oriented (meaningful and family/client-selected goals), family/client-centered (promoted self-determination and decision making), collaborative (partnership between caregivers), strength-based (built on participant and caregiver strengths) and ecological (natural environment and real-life experiences). Design of the intervention comprised three steps: 1) Preparation period (6 weeks); 2) Intervention period (12 weeks); 3) Follow-up period (12 weeks) | Other | UPART intervention group |
Primary outcome
ActivPAL
Time frame: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
Change in sedentary time
Stepwatch Activity Monitor
Time frame: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
Change in daily step count
4 secondary outcomes
Rett Syndrome Gross Motor Scale
Time frame: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
2 minute walk test
Time frame: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
Quality of Life Inventory - Disability, questionnaire
Time frame: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up
Goal attainment scaling
Time frame: Assessed twice; at baseline and after the 12 week intervention
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 21 Feb 2019.
- Individual participant dataSharing planned: No
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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.
The record lists no contact. Use the official record to reach the sponsor.
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A study testing 'Uptime' participation in people with Rett Syndrome, run by Copenhagen University Hospital, Rigshospitalet at 1 site in Denmark.
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 2 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 21 Feb 2019. 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 Denmark. 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?
ActivPAL (time frame: Assessed four times; twice at baseline; once after the 12 week intervention and once after a further 12 week follow-up). The study lists 2 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 21 Feb 2019.