Sickle Cell DiseaseNCT03585543

Self-Management for Youth and Families Living With SCD - SMYLS

A study testing Voice Crisis Alert V2 in people with Sickle Cell Disease, run by Medical University of South Carolina at 1 site in United States.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record

Search all trials

Trial summary

Who is it for?: Ages 8 to 17 years
Conditions the study is for: Sickle Cell Disease
What is being tested?: Voice Crisis Alert V2
Type of treatment: Lifestyle or therapy programme
Could I receive a placebo?: No, everyone receives an active treatment
How many people: 30 participants
Where: 1 siteUnited States
Study dates: 16 Mar 2018to 31 Jan 2019
Phase: Interventional
Are there results?: Yes, posted in the registrySummary in the doctors' view

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 8 to 17 years
All sexes
No healthy volunteers
Child

Who may be able to join

children ages 8 - 17 years and parent or primary caregiver 18 years or older
child with sickle cell disease, as reported by clinician at MUSC Pediatric Sickle Cell Clinic

Who cannot join

Parent/caregiver or child with cognitive disability or delay that precludes ability to participate
Lack of wi-fi access

Trial locations and contacts

1 site in 1 country.

United States1 site

Medical University of South Carolina
Charleston, South Carolina, 29425

Other trials in United States

About this study

Official title: Self-Management for Youth and Families Living With Sickle Cell Disease - SMYLS

The purpose of this proposal is to integrate family-centered self-management strategies with mobile health (mHealth) technology to improve reach, self-management behaviors, and child and caregiver physical and psychosocial symptoms and quality of life. Specifically, the investigators propose to conduct feasibility testing of SMYLS, which has been adapted based on user feedback in the first phase of this study. First the investigators will work with the Medical University of South Carolina (MUSC) Pediatric Sickle Cell Clinic to identify and recruit families with children with sickle cell disease (SCD) in the community, statewide. Next, the investigators will test the feasibility of the intervention with 30 dyads of children ages 8 - 17 with sickle cell disease and their parent or primary caregiver, (N=60)

Conditions: Sickle Cell Disease
Being tested: Voice Crisis Alert V2
Medical subject headings: Anemia, Sickle Cell

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

Voice Crisis Alert V2Behavioural
Intervention consists of a mHealth app delivered via smartphone

Study groups in NCT03585543
Study group	Role	What they receive
Single group intervention arm	Experimental	Not described Receives: Behavioral: Voice Crisis Alert V2

What is being measured

The primary outcome is the main question the study is designed to answer.

Rates of Recruitment
Number of weeks required to recruit 30 participants.
Measured: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.
Participant Adherence to Intervention
Number of participants who used the intervention (mHealth application) from baseline to mid-intervention, from mid-intervention to end-of-intervention, and from end-of-intervention to follow-up, assessed by number of participants who logged into and used the app, stored in the app's back end database.
Measured: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
Acceptability of Intervention
Number of participants reporting problems with the intervention (mHealth app) per week.
Measured: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.
Participant Adherence to Intervention
number of participants who accessed the educational component of intervention, assessed using back end app use database
Measured: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
Participant Adherence to Intervention
Number of participants who accessed the symptom monitoring component of the intervention, assessed using the app back end database
Measured: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
Participant Adherence to Intervention
number of participants who sent messages to nurse practitioner
Measured: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months

Secondary outcomes (10)

Difference in Mean Pain Score Rating From Baseline to End-of-intervention
Patient Reported Outcome Measurement Information System (PROMIS) Pain Interference Pediatric Short Form 8a: Change in score at 12 weeks minus baseline. Raw scores range from 0 - 32, with 0 being the lowest pain rating and 32 the highest pain rating. Any decrease in mean difference between scores = improved
Measured: baseline, 12 weeks
Difference in Mean Caregiver Self-efficacy Score From Baseline to End-of-intervention
Self-efficacy for Managing Chronic Disease 6-item scale. Scores for each item range from 1 - 10 with 1 indicating lower confidence/self-efficacy and 10 indicating higher confidence/self-efficacy. Scores for each item are averaged for the mean total score. Change in score at 12 weeks minus baseline. Any increase in difference in mean scores = improved
Measured: baseline, 12 weeks
Differences in Mean Scores for Quality of Life From Baseline to End-of-intervention
Pediatric Quality of Life Inventory (Peds QL): Scores are transformed on a scale from 0 - 100, with 0 indicating the highest possible problems with quality of life and 100 indicating the lowest possible problems with quality of life. Change in score at 12 weeks minus baseline. Any increase in difference between mean scores = improved
Measured: baseline, 12 weeks
Difference in Mean Scores for Fatigue From Baseline to End-of-intervention
Patient Reported Outcomes Measurement Information System (PROMIS) Fatigue Pediatric Short Form 10a: Raw Scores range from 0 - 40 with 0 being the least amount of fatigue and 40 the most fatigue. Difference in mean scores at 12 weeks minus baseline. Any decrease in rating = improved
Measured: baseline, 12 weeks
Difference in Mean Scores for Anxiety From Baseline to End-of-intervention
Patient Reported Outcomes Measurement System (PROMIS) Pediatric Short Form Anxiety 8a: Raw Scores range from 0 - 32, with 0 being the lowest anxiety rating and 32 the highest anxiety rating. Change in score at 12 weeks minus baseline. Any decrease difference in mean scores = improved
Measured: baseline, 12 weeks
Differences in Mean Scores on Depressive Symptoms From Baseline to End-of-intervention
Patient Reported Outcomes Measurement System (PROMIS) Pediatric short form depression 8a: Raw Scores range from 0 - 32, with 0 being the lowest depression rating and 32 the highest depression rating. Change in score at 12 weeks minus baseline. Any decrease in differences in mean scores = improved
Measured: baseline,12 weeks
Differences in Mean Scores for Pain Intensity From Baseline to End-of-intervention
Patient Reported Outcome Measurement Information System (PROMIS) Pain Intensity Pediatric 3a. Raw scores range from 3 - 15, with 3 being the lowest pain intensity rating and 15 being the highest pain intensity rating. Change in score at 12 weeks minus baseline. Any decrease in mean difference between scores = improved
Measured: baseline, 12 weeks
Differences in Mean Scores for Disease-related Quality of Life From Baseline to End-of-intervention
Pediatric Quality of Life Inventory (Peds QL) with SCD module: Scores are transformed on a scale from 0 - 100, with 0 indicating the highest possible problems with quality of life and 100 indicating the lowest possible problems with quality of life. Change in score at 12 weeks minus baseline. Any increase in difference between mean scores = improved
Measured: baseline, 12 weeks
Clinic Appointment Attendance
Number of missed clinic appointments at baseline compared to 12 weeks
Measured: 12 weeks
Home Medication Administration
Adherence to home medications, measured by number of medications marked as taken in the app database.
Measured: 12 weeks

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

16 Mar 201831 Jan 2019

First posted13 Jul 2018
Study start16 Mar 2018
Primary completion31 Jan 2019
Study completion31 Jan 2019

Resources

Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
Other trials for Sickle Cell DiseaseStudies of the same condition, open and under way.
More trials run by Medical University of South CarolinaThe 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: Supportive care
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Enrolment: 30 participants (actual)

IDs, dates and publications

ClinicalTrials.gov ID: NCT03585543
Sponsor's protocol ID: Pro00062837
Status last verified: Oct 2020
First submitted: 4 May 2018
Last updated: 19 Nov 2020
Results posted: Yes, on ClinicalTrials.gov
Data monitoring committee: Yes
FDA-regulated drug: No
FDA-regulated device: No
Individual data sharing: No

Want to know more?

The sponsor has not listed a contact. The official record may have more details.

Talk to your doctor first and take this page with you. Only the study team can confirm whether you can join.

Questions to ask the study team

What is the study trying to find out?
What would I need to do, and how often would I visit?
Could I receive a placebo instead of the treatment?
What are the possible risks and benefits for me?
Will it cost me anything? Are travel or time paid for?
Can I keep seeing my own doctor during the study?
What happens when the study ends?

See locations

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis

All Sickle Cell Disease trials

Study at a glance

From the registry record, last updated by the sponsor on 19 Nov 2020. Recruitment status is as the sponsor reported it: confirm with the study team.

Official title: Self-Management for Youth and Families Living With Sickle Cell Disease - SMYLS
ClinicalTrials.gov ID: NCT03585543
Acronym / sponsor ID: Pro00062837
Conditions: Sickle Cell Disease
Study type: Interventional
Recruitment status: Completed
Enrolment: 30 participants (actual)
Lead sponsor: Medical University of South CarolinaUniversity, hospital or other
Study start: 16 Mar 2018 (actual)
Primary completion: 31 Jan 2019 (actual)
Study completion: 31 Jan 2019 (actual)
First posted: 13 Jul 2018 (actual)
Results: Posted 16 Apr 2020

Clinical summary

The research question as the sponsor registered it. This describes what the study plans to test, not what it found.

Study design

Primary purpose: Supportive care
Allocation: Not applicable
Intervention model: Single group
Masking: None (open label)
Number of arms: 1

Arm	Type	Receives
Single group intervention arm	Experimental	Behavioral: Voice Crisis Alert V2

Who may qualify

Ages: 8 to 17 years
Sex: All
Healthy volunteers: Not accepted
Age groups: Child

Key inclusion criteria (2 listed)

children ages 8 - 17 years and parent or primary caregiver 18 years or older
child with sickle cell disease, as reported by clinician at MUSC Pediatric Sickle Cell Clinic

Key exclusion criteria (2 listed)

Parent/caregiver or child with cognitive disability or delay that precludes ability to participate
Lack of wi-fi access

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
Voice Crisis Alert V2Intervention consists of a mHealth app delivered via smartphone	Behavioural	Single group intervention arm

Primary outcome

Rates of Recruitment
Time frame: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.
Number of weeks required to recruit 30 participants.
Participant Adherence to Intervention
Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
Number of participants who used the intervention (mHealth application) from baseline to mid-intervention, from mid-intervention to end-of-intervention, and from end-of-intervention to follow-up, assessed by number of participants who logged into and used the app, stored in the app's back end database.
Acceptability of Intervention
Time frame: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.
Number of participants reporting problems with the intervention (mHealth app) per week.
Participant Adherence to Intervention
Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
number of participants who accessed the educational component of intervention, assessed using back end app use database
Participant Adherence to Intervention
Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
Number of participants who accessed the symptom monitoring component of the intervention, assessed using the app back end database
Participant Adherence to Intervention
Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months
number of participants who sent messages to nurse practitioner

10 secondary outcomes

Difference in Mean Pain Score Rating From Baseline to End-of-intervention
Time frame: baseline, 12 weeks
Difference in Mean Caregiver Self-efficacy Score From Baseline to End-of-intervention
Time frame: baseline, 12 weeks
Differences in Mean Scores for Quality of Life From Baseline to End-of-intervention
Time frame: baseline, 12 weeks
Difference in Mean Scores for Fatigue From Baseline to End-of-intervention
Time frame: baseline, 12 weeks
Difference in Mean Scores for Anxiety From Baseline to End-of-intervention
Time frame: baseline, 12 weeks
Differences in Mean Scores on Depressive Symptoms From Baseline to End-of-intervention
Time frame: baseline,12 weeks
Differences in Mean Scores for Pain Intensity From Baseline to End-of-intervention
Time frame: baseline, 12 weeks
Differences in Mean Scores for Disease-related Quality of Life From Baseline to End-of-intervention
Time frame: baseline, 12 weeks
Clinic Appointment Attendance
Time frame: 12 weeks
Home Medication Administration
Time frame: 12 weeks

Results and safety

Results posted to the registry on 16 Apr 2020, as reported by the sponsor. Figures are copied from the record, not re-analysed. Full results on ClinicalTrials.gov.

Participant flow

Group	Started	Completed
Group Receiving mHealth Intervention	30	15

Primary outcome: Rates of Recruitment

Time frame: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.. Number. Unit: weeks

Measure	Intervention Group n = 30
Value	14

Primary outcome: Participant Adherence to Intervention

Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months. Number. Unit: participants

Measure	Intervention Group n = 30
Number who used from baseline to 6 weeks	30
Number who used from 6 weeks to 12 weeks	15
Number who used from 12 weeks to 6 months	10

Primary outcome: Acceptability of Intervention

Time frame: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.. Count of participants. Unit: Participants

Measure	Intervention Group n = 30
Value	4

Primary outcome: Participant Adherence to Intervention

Time frame: baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months. Number. Unit: participants

Measure	Intervention Group n = 30
Number who used from baseline to 6 weeks	28
Number who used from 6 weeks to 12 weeks	7
Number who used from 12 weeks to 6 months	4

10 secondary outcomes are reported in the full results.

Adverse events

Time frame: The duration of study participation; 6 months. Other adverse events reported at 0% frequency or more

Group	Serious adverse events	Other adverse events	Deaths
Intervention Group	2 of 30 (6.7%)	0 of 30 (0%)	0 of 30

Limitations and caveats (from the sponsor): Limitations of this feasibility study included technical difficulties with the app, leading to an inability to accurately assess medication adherence data and measurement issues with the clinic appointment attendance. We were unable to assess these additional outcome measures with accuracy.

Loading the posted results from ClinicalTrials.gov…

Research links

Official record on ClinicalTrials.govLast updated by the sponsor 19 Nov 2020.
Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
Individual participant dataSharing planned: No

Related trials for Sickle Cell Disease

Active, not recruitingPhase 1/2
Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Long-term Mitapivat Dosing in Subjects With Stable Sickle Cell Disease: An Extension of a Phase I Pilot Study of Mitapivat
Sickle Cell Disease, Hemolytic Anemia
RecruitingObservational study
HPC Offered for PRESERVE Expansion
Bone Marrow Transplant (BMT)
RecruitingPhase 1
Study to Evaluate the Safety and Tolerability of Escalating Doses of Fostamatinib in Subjects With Stable Sickle Cell Disease
Sickle Cell Disorders, Sickle Cell Disease
RecruitingPhase 4
Clinical and Biomarker Effects of Depot Medroxyprogesterone Acetate in Females With Sickle Cell Disease
Sickle Cell Disease (SCD), Vaso-Occlusive Pain Episode in Sickle Cell Disease
Not yet recruitingPhase 1
Buprenorphine for the Management of Pain in Children With Sickle Cell Disease
Sickle Cell Disease (SCD), Sickle Cell Disease Vaso-occlusive Crisis

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 Voice Crisis Alert V2 in people with Sickle Cell Disease, run by Medical University of South Carolina at 1 site in United States.

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 19 Nov 2020. 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 United States. 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?

Yes. Results are posted in the registry (first posted 16 Apr 2020). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.

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?

Rates of Recruitment (time frame: Assessed each week over a period of 6 months, cumulative data up to 6 months is reported.). The study lists 6 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT03585543.

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 19 Nov 2020.