Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

Down Syndrome Clinic to You (DSC2U)

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04227197
Recruitment Status : Completed
First Posted : January 13, 2020
Last Update Posted : January 13, 2020
Sponsor:
Collaborator:
Patient-Centered Outcomes Research Institute
Information provided by (Responsible Party):
Brian G Skotko, Massachusetts General Hospital

Brief Summary:
The investigators conducted a national two-arm, randomized controlled trial of caregivers of individuals with DS to assess the efficacy of DSC2U in assuring adherence to evidence-based guidelines. The research plan was approved by the Partners Human Research Committee.

Condition or disease Intervention/treatment Phase
Down Syndrome Other: Down Syndrome Clinic to You (DSC2U) Not Applicable

Detailed Description:

Background: The overwhelming majority of people with Down syndrome do not have access to specialty clinics, a disparity resulting in delayed or missed diagnoses and significant untreated co-morbidities. To meet this critical gap in needs, the investigators created "Down Syndrome Clinic to You" (DSC2U), a novel, web-based tool created for caregivers of individuals with Down syndrome, which generates personalized recommendations for the caregiver and the patient's primary care provider (PCP).

Methods: In a national, randomized controlled trial of 230 caregivers who had children or dependents with Down syndrome without access to a Down syndrome specialist, 117 were randomized to receive DSC2U and 113 to receive usual-care. The primary outcome was adherence to five health evaluations indicated by national guidelines for Down syndrome: celiac screen, sleep study, thyroid test, audiogram, and ophthalmology evaluation.

Layout table for study information
Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 230 participants
Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: None (Open Label)
Primary Purpose: Health Services Research
Official Title: A Randomized, Controlled Trial of an Online Health Tool About Down Syndrome
Actual Study Start Date : October 3, 2017
Actual Primary Completion Date : May 15, 2019
Actual Study Completion Date : May 15, 2019

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Down Syndrome

Arm Intervention/treatment
Experimental: Intervention Group
The participants randomized to the intervention group completed the DSC2U questionnaire, and received online access to a personalized Caregiver Checklist and PCP plan. Caregivers were encouraged to share and discuss the PCP plan at their next wellness visit with the PCPs.
Other: Down Syndrome Clinic to You (DSC2U)
DSC2U is a web-based tool for families to get up-to-date, personalized health and wellness information, based on national guidelines and expert consensus, for a person with Down syndrome.

No Intervention: Control Group
The participants randomized to the control group, received usual care for 7 months, after their scheduled PCP appointment. They did not receive DSC2U during these 7 months, but did receive the online, personalized health assessment tool (DSC2U) at the end of the 7 months, after the primary and secondary outcomes were measured.



Primary Outcome Measures :
  1. Number of indicated evaluations that were completed or recommended by the PCP [ Time Frame: 7 months after the participant's scheduled PCP visit ]
    Adherence to the five health evaluations indicated by national guidelines for Down syndrome. The five health evaluations included: celiac screen, sleep study, thyroid test, audiogram, and ophthalmology evaluation.


Secondary Outcome Measures :
  1. Caregiver experience with the PCP visit questionnaire [ Time Frame: 2 weeks, and 7 months after their scheduled PCP visit ]
    Measure the caregiver experience with the PCP using a 10-point Likert scale with "10" representing "most helpful" and "0" representing "least helpful."

  2. Quality of life outcomes for person with Down syndrome questionnaire [ Time Frame: 2 weeks, and 7 months after their scheduled PCP visit ]
    Ascertained through the six PedsQL 4.0 parent-proxy and PedsQL 2.0 Family Impact Module summary scores.

  3. PCP experience with the patient visit questionnaire [ Time Frame: 2 weeks after the scheduled PCP visit ]
    These measurements were gathered by self-administered mail survey

  4. PCP experience with DSC2U questionnaire [ Time Frame: 2 weeks after the scheduled PCP visit ]
    These measurements were gathered by self-administered mail survey, for the PCP's randomized to the intervention group.



Information from the National Library of Medicine

Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.


Layout table for eligibility information
Ages Eligible for Study:   1 Year and older   (Child, Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Potential eligible participants were directed to the study website (www.dsc2u.org) for the eligibility screening questionnaire in English and Spanish and online consent. After selecting a language preference (English or Spanish), in addition to questions about the child's or dependent's biological sex, race and ethnicity, the eligibility screening questions included:

  1. Do you have a child or dependent with DS?
  2. Is your child or dependent 1 year or older?
  3. When is your child's next annual well visit ("PCP visit")?
  4. Does your child or dependent currently receive care at a DS specialty clinic? (If the child or dependent was actively followed in a DS specialty clinic, even one out of state. For example, a family from Arizona who travels to Texas each year for their child to be seen in a DS specialty clinic would be ineligible)

Inclusion Criteria:

  • If you have a child or dependent with Down syndrome (DS)
  • If your child or dependent is 1 year or older
  • If your child or dependent is does not receive care at a DS specialty clinic

Exclusion Criteria:

  • If your child or dependent does receive care at a DS specialty clinic
  • If your child or dependent is under 1 year old
  • If your child or dependent has a PCP that is already involved in the study

DS occurs naturally and proportionally in all races and ethnicities, so the study's population estimates were proportional to the racial/ethnic distribution of the U.S. population, as reported in the 2010 U.S. Census. To achieve commensurate representation in the study, the investigators applied a quota system in offering enrollment using the race and ethnicity of the individual with DS (not the caregiver).

The investigators' plan was to enroll participants such that there were: no more than 144 white individuals with DS, no fewer than 25 Hispanic or Latino/Latina individuals with DS, and no fewer than 20 black individuals with DS. The team also planned to enroll no more than 120 individuals with DS of one sex.


Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04227197


Locations
Layout table for location information
United States, Massachusetts
Massachusetts General Hospital
Boston, Massachusetts, United States, 02114
Sponsors and Collaborators
Massachusetts General Hospital
Patient-Centered Outcomes Research Institute
Additional Information:
Publications of Results:

Layout table for additonal information
Responsible Party: Brian G Skotko, Director, Down Syndrome Program, Massachusetts General Hospital
ClinicalTrials.gov Identifier: NCT04227197    
Other Study ID Numbers: 2017P000442.
(PCORI) AD-1507-31567 ( Other Grant/Funding Number: Patient-Centered Outcomes Research Institute )
First Posted: January 13, 2020    Key Record Dates
Last Update Posted: January 13, 2020
Last Verified: January 2020
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: Yes
Plan Description: The investigators will submit their complete data set to PCORI in accordance with their guidelines.
Supporting Materials: Study Protocol
Statistical Analysis Plan (SAP)
Informed Consent Form (ICF)
Clinical Study Report (CSR)
Analytic Code
Time Frame: The investigators will submit to PCORI in accordance with their guidelines, and they will plan to post to their online registry.
Access Criteria: The study's data set will be accessed through PCORI. Please see further information on the process of accessing the data-set here: https://help.pcori.org/hc/en-us/sections/360000257660-Data-Management-and-Data-Sharing-Policy
URL: https://www.pcori.org/about-us/governance/policy-data-management-and-data-sharing

Layout table for additional information
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Brian G Skotko, Massachusetts General Hospital:
Online Health Tool
Health evaluations
Celiac Screen
Sleep Study
Thyroid Test
Audiogram
Ophthalmology evaluation
Healthcare outcomes survey
Experience survey
Additional relevant MeSH terms:
Layout table for MeSH terms
Down Syndrome
Syndrome
Disease
Pathologic Processes
Intellectual Disability
Neurobehavioral Manifestations
Neurologic Manifestations
Nervous System Diseases
Abnormalities, Multiple
Congenital Abnormalities
Chromosome Disorders
Genetic Diseases, Inborn