NCT03585543

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

Completed NA Results posted Last updated 19 November 2020

What this trial tests

NA trial testing Voice Crisis Alert V2 in Sickle Cell Disease in 30 participants. Completed in 31 January 2019.

Timeline

16 March 2018

Primary endpoint
31 January 2019

31 January 2019

Quick facts

Lead sponsor	Medical University of South Carolina
Phase	NA
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	supportive care
Enrollment	30
Start date	16 March 2018
Primary completion	31 January 2019
Estimated completion	31 January 2019
Sites	1 location across United States

Drugs / interventions tested

• Voice Crisis Alert V2

Conditions studied

• Sickle Cell Disease — all drugs for Sickle Cell Disease →

Sponsor

Medical University of South Carolina

Who can join

Adults 8 to 17, any sex, with Sickle Cell Disease. Patients with the condition only — healthy volunteers not accepted.

Results — posted to ClinicalTrials.gov

Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.

Rates of Recruitment Primary · 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.

Group	Value	95% CI
Intervention Group	14

Participant Adherence to Intervention Primary · 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.

Number who used from baseline to 6 weeks

Group	Value	95% CI
Intervention Group	30

Number who used from 6 weeks to 12 weeks

Group	Value	95% CI
Intervention Group	15

Number who used from 12 weeks to 6 months

Group	Value	95% CI
Intervention Group	10

Acceptability of Intervention Primary · 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.

Group	Value	95% CI
Intervention Group	4

Participant Adherence to Intervention Primary · 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

Number who used from baseline to 6 weeks

Group	Value	95% CI
Intervention Group	28

Number who used from 6 weeks to 12 weeks

Group	Value	95% CI
Intervention Group	7

Number who used from 12 weeks to 6 months

Group	Value	95% CI
Intervention Group	4

Participant Adherence to Intervention Primary · 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

Number who used from baseline to 6 weeks

Group	Value	95% CI
Intervention Group	29

Number who used from 6 weeks to 12 weeks

Group	Value	95% CI
Intervention Group	15

Number who used from 12 weeks to 6 months

Group	Value	95% CI
Intervention Group	10

Participant Adherence to Intervention Primary · baseline to 6 weeks, 6 weeks to 12 weeks, 12 weeks to 6 months

number of participants who sent messages to nurse practitioner

Number who used from baseline to 6 weeks

Group	Value	95% CI
Intervention Group	12

Number who used from 6 weeks to 12 weeks

Group	Value	95% CI
Intervention Group	2

Number who used from 12 weeks to 6 months

Group	Value	95% CI
Intervention Group	1

Difference in Mean Pain Score Rating From Baseline to End-of-intervention Secondary · baseline, 12 weeks

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

Group	Value	95% CI
Group Receiving mHealth Intervention	-9.0	-15.0 – -3.0

Difference in Mean Caregiver Self-efficacy Score From Baseline to End-of-intervention Secondary · baseline, 12 weeks

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

Group	Value	95% CI
Group Receiving mHealth Intervention	0.2	-0.5 – 0.9

Differences in Mean Scores for Quality of Life From Baseline to End-of-intervention Secondary · baseline, 12 weeks

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

Group	Value	95% CI
Group Receiving mHealth Intervention	10.9	4.3 – 17.4

Difference in Mean Scores for Fatigue From Baseline to End-of-intervention Secondary · baseline, 12 weeks

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

Group	Value	95% CI
Group Receiving mHealth Intervention	-7.9	-12.9 – -2.9

Difference in Mean Scores for Anxiety From Baseline to End-of-intervention Secondary · baseline, 12 weeks

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

Group	Value	95% CI
Group Receiving mHealth Intervention	1.6	-3.7 – 6.8

Differences in Mean Scores on Depressive Symptoms From Baseline to End-of-intervention Secondary · baseline,12 weeks

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

Group	Value	95% CI
Group Receiving mHealth Intervention	-3.1	-7.3 – 1.1

Adverse events — posted to ClinicalTrials.gov

Time frame: The duration of study participation; 6 months. Reporting threshold: 0%. Adverse-event reports describe events observed during the trial — not all are caused by the drug.

Intervention Group

Serious: 2/30 (7%)

Deaths: 0/30

Serious adverse events (1 terms)

Reaction	System	Intervention Group
Vaso-occlusive crisis	Blood and lymphatic system disorders	—

Most-reported serious reactions: Vaso-occlusive crisis.

Data from ClinicalTrials.gov NCT03585543 adverse events section.

Sponsor's own description

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)

Publications & conference data

1 peer-reviewed publication reference this trial (live from Europe PMC):

1. Feasibility of an mHealth self-management intervention for children and adolescents with sickle cell disease and their families.
   Phillips S, Kanter J, Mueller M, Gulledge A, et al · · 2021 · cited 8× · PMID 33410488 · DOI 10.1093/tbm/ibaa132