Duchenne Muscular Dystrophy (DMD)NCT06925269
DMD Voice: Qualitative Interviews With Patients and Caregivers
An observational study of Duchenne Muscular Dystrophy (DMD), run by Red Nucleus Enterprise Solutions, LLC at 1 site in United States.
Trial summary
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. Read more about this study
- Who is it for?
- Ages 10 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy (DMD)
- How many people
- 68 participants
- Where
- 1 siteUnited States
- Study dates
- 31 Mar 2025to Dec 2026
- Phase
- Observational
- Are there results?
- Not yet: the study is under way
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 10 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Study population: Caregivers of individuals with Duchenne Muscular Dystrophy (DMD) and patients with DMD
Who may be able to join
- 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
Show all 16Show fewer
- 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
Who cannot join
- -
Trial locations and contacts
1 site in 1 country. Contact the study team or a site near you to ask about taking part.
United States1 site, 1 recruiting
- Recruiting
Red Nucleus
Yardley, Pennsylvania, 19067
Mary Hubbard, 904-541-9326, mhubbard@rednucleus.com
Mindy Leffler
About this study
Official title: Duchenne Muscular Dystrophy Quality of Life: Qualitative Interviews With Patients and Caregivers
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.
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne, givinostat
- Medical subject headings
- Muscular Dystrophy, Duchenne
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| Subsample A - Caregivers | Caregivers of individuals with DMD treated with givinostat for at least 2 years | |
| Subsample A - Patients | Patients with DMD treated with givinostat for at least 2 years | |
| Subsample B - Caregivers | Caregivers of individuals with DMD never treated with givinostat |
What is being measured
The primary outcome is the main question the study is designed to answer.
Qualitative Interview
Participants will participate in a semi-structured interview
Measured: 60 minutes
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted13 Apr 2025
- Study start31 Mar 2025
- Primary completionDec 2025 (estimated)
- Study completionDec 2026 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Duchenne Muscular Dystrophy (DMD)Studies of the same condition, open and under way.
- 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
- Observational
- Observational model
- Cohort
- Time perspective
- Retrospective
- Enrolment
- 68 participants (estimated)
Sponsor and investigators
- Lead sponsor
- Red Nucleus Enterprise Solutions, LLC
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Italfarmaco S.A
- Responsible party
- Sponsor
- Investigators
- Mindy Leffler, M.Ed., Principal investigator, Red Nucleus Solutions
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT06925269
- Sponsor's protocol ID
- CAS-ITF001-01
- Status last verified
- Apr 2025
- First submitted
- 3 Apr 2025
- Last updated
- 30 Apr 2025
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 30 Apr 2025. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Duchenne Muscular Dystrophy Quality of Life: Qualitative Interviews With Patients and Caregivers
- ClinicalTrials.gov ID
- NCT06925269
- Acronym / sponsor ID
- CAS-ITF001-01
- Conditions
- Duchenne Muscular Dystrophy (DMD)
- Keywords
- Duchenne, givinostat
- Study type
- Observational
- Recruitment status
- Recruiting
- Enrolment
- 68 participants (estimated)
- Lead sponsor
- Red Nucleus Enterprise Solutions, LLCIndustry (pharma, biotech, devices)
- Collaborators
- Italfarmaco S.A
- Study start
- 31 Mar 2025 (actual)
- Primary completion
- Dec 2025 (estimated)
- Study completion
- Dec 2026 (estimated)
- First posted
- 13 Apr 2025 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Cohort
- Time perspective
- Retrospective
- Number of arms
- 3
| Arm | Type | Receives |
|---|---|---|
| Subsample A - Caregivers | – | –Caregivers of individuals with DMD treated with givinostat for at least 2 years |
| Subsample A - Patients | – | –Patients with DMD treated with givinostat for at least 2 years |
| Subsample B - Caregivers | – | –Caregivers of individuals with DMD never treated with givinostat |
Who may qualify
- Ages
- 10 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- Study population
- Caregivers of individuals with Duchenne Muscular Dystrophy (DMD) and patients with DMD
Key inclusion criteria (16 listed)
- Subsample A - Patients
- 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
Key exclusion criteria (1 listed)
- -
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Qualitative Interview
Time frame: 60 minutes
Participants will participate in a semi-structured interview
Results and safety
This study is still under way. Results are not available in the source record yet.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 30 Apr 2025.
- Individual participant dataSharing planned: No
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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.
Mindy Leffler, M.Ed. principal investigator
Red Nucleus Solutions
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Duchenne Muscular Dystrophy (DMD), run by Red Nucleus Enterprise Solutions, LLC 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 16 inclusion and 1 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: looking for participants now, as of the sponsor's last update on 30 Apr 2025. 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?
This study is still under way. Results are not available in the source record yet.
How can a doctor refer a patient or contact the research team?
Contact Mary Hubbard (904-541-9326, mhubbard@rednucleus.com), or a site near the patient. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Qualitative Interview (time frame: 60 minutes). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
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 30 Apr 2025.