Duchenne Muscular DystrophyNCT02340923
A Device for Rapid, Painless, Bedside Muscle Evaluation of Children
An observational study of Duchenne Muscular Dystrophy, run by Skulpt, Inc. at 5 sites in United States.
Trial summary
The purpose of this protocol is to perform Electrical Impedance Myography (EIM) testing on healthy children and children with duchenne muscular dystrophy so as to develop a new, convenient tool for the office based assessment of children with a wide variety of neuromuscular conditions. Read more about this study
- Who is it for?
- Ages Up to 18 years, men onlyHealthy volunteers can take part
- Conditions the study is for
- Duchenne Muscular Dystrophy
- How many people
- 327 participants
- Where
- 5 sitesUnited States
- Study dates
- Jan 2015to Aug 2016
- How long will I take part?
- 12 Months
- Phase
- Observational
- Are there results?
- None posted in the registry
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 Up to 18 years
- Men only
- Healthy volunteers accepted
- Child, Adult
Study population: Duchenne Muscular Dystrophy Subjects
Who may be able to join
- 1. Age 0-18
- 2. Male
- 3. Genetic or histopathologic diagnosis of duchenne muscular dystrophy, or signs and symptoms of DMD and genetic or histopathologic diagnosis in a family member.
Who cannot join
- 1. Age over 18
- 2. Female
- 3. Presence of a superimposed neuromuscular or other medical condition that substantially impacts the individual's health or ability to cooperate.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Children's Hospital Colorado
Aurora, Colorado, 80045
Boston Children's Hospital
Boston, Massachusetts, 02115
Skulpt, Inc.
Boston, Massachusetts, 02210
Cincinnati Children's Hospital
Cincinnati, Ohio, 45229
St. Louis Children's Hospital
St Louis, Missouri, 63310
About this study
The purpose of this protocol is to perform Electrical Impedance Myography (EIM) testing on healthy children and children with duchenne muscular dystrophy so as to develop a new, convenient tool for the office based assessment of children with a wide variety of neuromuscular conditions.
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Duchenne Muscular Dystrophy, EIM
- 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 |
|---|---|---|
| Healthy Subjects | Healthy subjects aged 0-18 years old. Subjects cannot have a presence or past history of a neurological disorder or other disease that would be expected to substantially impact health. | |
| Duchenne Muscular Dystrophy Subjects | Male subjects aged 0-18 years old with genetic or histopathologic diagnosis of duchenne muscular dystrophy, or signs and symptoms of DMD and genetic or histopathologic diagnosis in a family member. Additionally, subjects cannot have the presence of a superimposed neuromuscular or other medical condition that substantially impacts the individual's health or ability to cooperate. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Electrical Impedance Myography Measurements
Up to 11 muscles will be measured using EIM technology. These muscles include: Right Lateral Deltoid, Right Biceps, Right Triceps, Right Wrist Flexors, Right Wrist Extensors, Right Vastus Lateralis, Right Tibialis Anterior, Right Gastrocnemius, Right Gluteus Medius, Right Biceps Femoris, and Right Thoracic Parapinal.
Measured: Baseline (all subjects) & 3 months, 6 months, 1 year for returning subjects only
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted19 Jan 2015 (estimated)
- Study startJan 2015
- Primary completionJun 2016
- Study completionAug 2016
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Publications (1)Papers the sponsor has linked to this study.
- Other trials for Duchenne Muscular DystrophyStudies 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
- Cross-sectional
- Enrolment
- 327 participants (actual)
- Target duration
- 12 Months
Sponsor and investigators
- Lead sponsor
- Skulpt, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02340923
- Sponsor's protocol ID
- PED-003-2014
- Other IDs
- 2R44NS073188
- Status last verified
- Aug 2016
- First submitted
- 3 Dec 2014
- Last updated
- 11 Aug 2016
- Results posted
- No
- Data monitoring committee
- Yes
Publications
Leitner ML, Kapur K, Darras BT, Yang M, Wong B, Dalle Pazze L, Florence J, Buck M, Freedman L, Bohorquez J, Rutkove S, Zaidman C. Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials. Ann Clin Transl Neurol. 2020 Jan;7(1):4-14. doi: 10.1002/acn3.50958. Epub 2019 Dec 25.
PubMed 31876124
Study at a glance
From the registry record, last updated by the sponsor on 11 Aug 2016. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- A Device for Rapid, Painless, Bedside Muscle Evaluation of Children
- ClinicalTrials.gov ID
- NCT02340923
- Acronym / sponsor ID
- PED-003-2014
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Duchenne Muscular Dystrophy, EIM
- Study type
- Observational
- Recruitment status
- Completed
- Enrolment
- 327 participants (actual)
- Lead sponsor
- Skulpt, Inc.Industry (pharma, biotech, devices)
- Collaborators
- National Institute of Neurological Disorders and Stroke (NINDS)
- Study start
- Jan 2015
- Primary completion
- Jun 2016 (actual)
- Study completion
- Aug 2016 (actual)
- First posted
- 19 Jan 2015 (estimated)
- 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 protocol is to perform Electrical Impedance Myography (EIM) testing on healthy children and children with duchenne muscular dystrophy so as to develop a new, convenient tool for the office based assessment of children with a wide variety of neuromuscular conditions.
Study design
- Observational model
- Cohort
- Time perspective
- Cross-sectional
- Target follow-up
- 12 Months
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Healthy Subjects | – | –Healthy subjects aged 0-18 years old. Subjects cannot have a presence or past history of a neurological disorder or other disease that would be expected to substantially impact health. |
| Duchenne Muscular Dystrophy Subjects | – | –Male subjects aged 0-18 years old with genetic or histopathologic diagnosis of duchenne muscular dystrophy, or signs and symptoms of DMD and genetic or histopathologic diagnosis in a family member. Additionally, subjects cannot have the presence of a superimposed neuromuscular or other medical condition that substantially impacts the individual's health or ability to cooperate. |
Who may qualify
- Ages
- Up to 18 years
- Sex
- Male only
- Healthy volunteers
- Accepted
- Age groups
- Child, Adult
- Study population
- Duchenne Muscular Dystrophy Subjects
Key inclusion criteria (3 listed)
- 1. Age 0-18
- 2. Male
- 3. Genetic or histopathologic diagnosis of duchenne muscular dystrophy, or signs and symptoms of DMD and genetic or histopathologic diagnosis in a family member.
Key exclusion criteria (3 listed)
- 1. Age over 18
- 2. Female
- 3. Presence of a superimposed neuromuscular or other medical condition that substantially impacts the individual's health or ability to cooperate.
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
Electrical Impedance Myography Measurements
Time frame: Baseline (all subjects) & 3 months, 6 months, 1 year for returning subjects only
Up to 11 muscles will be measured using EIM technology. These muscles include: Right Lateral Deltoid, Right Biceps, Right Triceps, Right Wrist Flexors, Right Wrist Extensors, Right Vastus Lateralis, Right Tibialis Anterior, Right Gastrocnemius, Right Gluteus Medius, Right Biceps Femoris, and Right Thoracic Parapinal.
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 11 Aug 2016.
- Derived from this study: PubMed 31876124Leitner ML, Kapur K, Darras BT, Yang M, Wong B, Dalle Pazze L, Florence J, Buck M, Freedman L, Bohorquez J, Rutkove S, Zaidman C. Electrical impedance myography for reducing sample size in Duchenne muscular dystrophy trials. Ann Clin Transl Neurol. 2020 Jan;7(1):4-14. doi: 10.1002/acn3.50958. Epub 2019 Dec 25.
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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. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites 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, run by Skulpt, Inc. at 5 sites 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 3 inclusion and 3 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: finished, as of the sponsor's last update on 11 Aug 2016. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites 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?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Electrical Impedance Myography Measurements (time frame: Baseline (all subjects) & 3 months, 6 months, 1 year for returning subjects only). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
On the ClinicalTrials.gov record for NCT02340923, which also lists 1 linked publication.
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 11 Aug 2016.