Rasch-built Outcome Measures to Improve ALS Clinical Trials

Study Purpose:

The purpose of this study is to develop improved outcome scales for tracking ALS progression.

Disease:

Amyotrophic Lateral Sclerosis (ALS),  Familial ALS,  Sporadic ALS, Primary Lateral Sclerosis (PLS)

Study Type:

Observational Study

Study Category:

Study Status:

Enrolling

Phase:

Not Applicable

Study Chair(s)/Principal Investigator(s):

Christina Fournier, MD (Emory University )

Clinicaltrials.gov ID (11 digit #):

Neals Affiliated?

No

Coordinating Center Contact Information

Emory University
Karon Simmons / .(JavaScript must be enabled to view this email address) / 404-712-4182
.(JavaScript must be enabled to view this email address) 101 Woodruff Circle
Suite 6000
Atlanta, Georgia 30322 United States

Full Study Summary:

The objective of this study is to build and refine an ALS outcome measure toolbox containing a an externally validated patient-reported questionnaire to assess overall disability and a novel objective exam-based scale to assess overall motor strength using Rasch methodology.

The recently created and validated Rasch-built Overall ALS Disability Scale (ROADS) will be studied longitudinally to evaluate responsiveness, longitudinal performance, predictive features, and clinically meaningful change.  This study will establish clinical trial readiness for the ROADS.  Based on recent work showing that the ROADS has superior item targeting and psychometric properties compared to the ALSFRS-R coupled with prior studies for other diseases showing that Rasch-scales are more responsive than standard ordinal scales, use of the ROADS as an outcome measure in ALS trials is expected to improve clinical trial efficiency and reliability.

A new objective ALS outcome measure will also be created and validated to capture the neurologic exam data that are already collected during research visits and clinical care in a quantifiable, reliable manner.  This scale will offer the same psychometric advantages of the ROADS through use of Rasch methodology and will capture overall motor strength, a highly relevant and important clinical and research outcome for ALS.  Importantly, this scale will be designed for in-person or telemedicine administration, which will add great value to clinical trials as they adapt to the challenges of COVID-19 and will aid clinicians in standardizing their approach to delivering ALS care through virtual platforms.

The hypothesis driving this study is that creating and refining improved outcome measures for ALS using Rasch methodology can facilitate faster translation and approval of disease-modifying therapies for ALS.  These outcome measures are also expected to serve as useful tools for tracking ALS disease progression in the clinic, particularly in the emerging era of mainstream telemedicine care.

Study Sponsor:

Department of Defense

Participant Duration:

Participants are eligible to participate in the study if they are adults age 18 or older, have a diagnosis of ALS, and are able to provide informed consent. Participants will complete questionnaires about their ALS online every month for 6 months. Participants will also do questionnaires by telephone once and will have 2 Zoom video teleconference visits for a neurologic exam. No travel to the study site is required. Each remote study session is expected to take approximately 30 minutes.

Estimated Enrollment:

200

Estimated Study Start Date:

06/24/2021

Estimated Study Completion Date:

03/31/2024

Posting Last Modified Date:

07/06/2022

Date Study Added to alsconsortium.org:

07/06/2022
  • Eligibility Criteria

    Gender:

    Female, Male

    Minimum Age:

    18

    Maximum Age:

    N/A

    Time since Symptom Onset:

    N/A

    Time since Diagnosis:

    N/A

    Can participants use Riluzole?

    Yes


    Adults age 18 or older, have a diagnosis of ALS, and are able to provide informed consent.

  • Site Contact Information

    Emory University
    Karon Simmons / .(JavaScript must be enabled to view this email address) / 404-712-4182
    101 Woodruff Circle
    Atlanta, Georgia 30322
    United States