DMD Voice: Qualitative Interviews With Patients and Caregivers

NCT ID: NCT06925269

Last Updated: 2025-04-30

Study Results

Results pending

The study team has not published outcome measurements, participant flow, or safety data for this trial yet. Check back later for updates.

Basic Information

Get a concise snapshot of the trial, including recruitment status, study phase, enrollment targets, and key timeline milestones.

Recruitment Status

RECRUITING

Total Enrollment

68 participants

Study Classification

OBSERVATIONAL

Study Start Date

2025-03-31

Study Completion Date

2026-12-31

Brief Summary

Review the sponsor-provided synopsis that highlights what the study is about and why it is being conducted.

The purpose of this study is to understand DMD functional losses or abilities and their association with independence and quality of life from the perspective of individuals with DMD and/or and their caregivers.

This is a qualitative interview study in which individuals with DMD and/or their caregivers will be asked to participate in a semi-structured, approximately 60- minute interview. Interviews will focus on functional abilities and independence. Caregivers and boys with DMD will be interviewed. This study includes no treatment nor intervention; however, some participants are being treated by a drug that is approved in the U.S. and the U.K. and under investigation in other geographies.

Detailed Description

Dive into the extended narrative that explains the scientific background, objectives, and procedures in greater depth.

Conditions

See the medical conditions and disease areas that this research is targeting or investigating.

Duchenne Muscular Dystrophy (DMD)

Study Design

Understand how the trial is structured, including allocation methods, masking strategies, primary purpose, and other design elements.

Observational Model Type

COHORT

Study Time Perspective

RETROSPECTIVE

Study Groups

Review each arm or cohort in the study, along with the interventions and objectives associated with them.

Subsample A - Caregivers

Caregivers of individuals with DMD treated with givinostat for at least 2 years

No interventions assigned to this group

Subsample A - Patients

Patients with DMD treated with givinostat for at least 2 years

No interventions assigned to this group

Subsample B - Caregivers

Caregivers of individuals with DMD never treated with givinostat

No interventions assigned to this group

Eligibility Criteria

Check the participation requirements, including inclusion and exclusion rules, age limits, and whether healthy volunteers are accepted.

Inclusion Criteria

Subsample A - Patients

Eligible participants for Subsample A must:

* Have been treated with Givinostat for at least 2 years
* Be at least 10 years of age with signed consent of a parent or legal guardian
* Be currently taking givinostat
* Willing and able to participate in a video and/or audio recorded interview

Subsample A - Caregivers

Eligible caregivers for Subsample A must:

* Be a parent or legal guardian of an individual with DMD that has been taking givinostat for at least two years
* Willing and able to participate in a video and/or audio recorded interview

Subsample B - Caregivers

Eligible caregivers for Subsample B must:

* Provide care to an individual with DMD who is unable to raise their hands above their head as confirmed by the caregiver
* Reside in the United States or Canada
* Able to read, speak, and understand English
* Willing and able to participate in a video and/or audio recorded interview Have access to a stable internet connection

Exclusion Criteria

\-
Minimum Eligible Age

10 Years

Eligible Sex

MALE

Accepts Healthy Volunteers

No

Sponsors

Meet the organizations funding or collaborating on the study and learn about their roles.

Italfarmaco S.A

INDUSTRY

Sponsor Role collaborator

Red Nucleus Enterprise Solutions, LLC

INDUSTRY

Sponsor Role lead

Responsible Party

Identify the individual or organization who holds primary responsibility for the study information submitted to regulators.

Responsibility Role SPONSOR

Principal Investigators

Learn about the lead researchers overseeing the trial and their institutional affiliations.

Mindy Leffler, M.Ed.

Role: PRINCIPAL_INVESTIGATOR

Red Nucleus Solutions

Locations

Explore where the study is taking place and check the recruitment status at each participating site.

Red Nucleus

Yardley, Pennsylvania, United States

Site Status RECRUITING

Countries

Review the countries where the study has at least one active or historical site.

United States

Central Contacts

Reach out to these primary contacts for questions about participation or study logistics.

Mary Hubbard

Role: CONTACT

904-541-9326

Facility Contacts

Find local site contact details for specific facilities participating in the trial.

Mary Hubbard

Role: primary

904-541-9326

Other Identifiers

Review additional registry numbers or institutional identifiers associated with this trial.

CAS-ITF001-01

Identifier Type: -

Identifier Source: org_study_id

More Related Trials

Additional clinical trials that may be relevant based on similarity analysis.

Stem Cell Therapy in Muscular Dystrophy
NCT02241928 WITHDRAWN PHASE1
Exploratory Study of NS-089/NCNP-02 in DMD
NCT04129294 COMPLETED PHASE1/PHASE2