Duchenne Muscular DystrophyPhase 1/2NCT01207908
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
A Phase 1/2 study testing IGF-1 in people with Duchenne Muscular Dystrophy, run by Children's Hospital Medical Center, Cincinnati at 1 site in United States.
Trial summary
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD). Read more about this study
- Who is it for?
- Ages 5 years and older, men only
- Conditions the study is for
- Duchenne Muscular Dystrophy
- What is being tested?
- IGF-1
- Type of treatment
- Injection or infusion
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 44 participants
- Where
- 1 siteUnited States
- Study dates
- Nov 2010to Jun 2013
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
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 5 years and older
- Men only
- No healthy volunteers
- Child, Adult, Older adult
Who may be able to join
- DMD diagnosed with mutational testing and/or complete absence of dystrophin on muscle biopsy
- Proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
- Male
- Age > 5 years of age
- Bone maturation (assess by bone age x-ray): </= 11 years of age
- Daily GC (prednisone or deflazacort) therapy for > 12 months
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- Ambulatory
- Informed consent
- Willingness and ability to comply with all protocol requirements and procedures
Who cannot join
- Current or prior treatment with growth hormone or IGF-1 therapy
- Non-ambulatory
- Pubertal (based on clinical Tanner staging examination)
- Congestive cardiac failure
- History of intracranial hypertension
- Daytime ventilatory dependence (non-invasive or tracheostomy)
Show all 10Show fewer
- Concomitant therapy - any other medications/supplements that would be considered, in the opinion of the investigators, to affect muscle function, need to have been started 3 months prior to enrollment
- Patients enrolled in other clinical drug trials
- Any physical or mental conditions which may, in the investigators'opinions, render the subject unable to complete the tasks of the study appropriately
- There will be no selection by ethnicity
Trial locations and contacts
1 site in 1 country.
United States1 site
Cincinnati Children's Hospital Medical Center
Cincinnati, Ohio, 45229
About this study
Official title: IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy
The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
Read the detailed description
Detailed Description:
DMD is a progressive degenerative muscle disorder for which there is no current cure. Glucocorticoids (GC) are often used to improve motor function and survival but have significant side effects such as growth failure, weight gain, insulin resistance and osteoporosis. IGF-1 stimulates both the proliferation and differentiation of skeletal muscle cells and is thus important for muscle repair and regeneration. IGF-1 offers potential as a therapeutic agent for DMD as it may improve or preserve motor function and reduce GC side effects such as growth failure and insulin resistance.
- Conditions
- Duchenne Muscular Dystrophy
- Being tested
- IGF-1
- Keywords
- DMD, Duchenne Muscular Dystrophy, Insulin like growth factor, 6 minute walk test
- 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.
IGF-1Drug
Also called Increlex (mecasermin [rDNA origin] injection)
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
| Study group | Role | What they receive |
|---|---|---|
| IGF-1 | Experimental | IGF-1 plus standard steroid treatment Receives: Drug: IGF-1 |
| Standard steroid treatment alone | No intervention | Standard daily steroid treatment for DMD |
What is being measured
The primary outcome is the main question the study is designed to answer.
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Outcome Measure Data Table shows change of 6-Minute Walk Distance at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
Measured: 6 months
Secondary outcomes (2)
Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Outcome Measure Data Table shows change of height velocity at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
Measured: 6 months
Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Outcome Measure Data Table shows change of North Star Ambulatory Assessment (NSAA) score at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
The NSAA is a 17-item scale that grades performance of various functional skills on a scale from 0 (unable), 1 (complete independently but with modifications), and 2 (complete without compensation).
The range of NSAA score is from 0 to 34. The higher score indicates better motor function.
Measured: 6 months
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted23 Sep 2010 (estimated)
- Study startNov 2010
- Primary completionOct 2012
- Study completionJun 2013
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Duchenne Muscular DystrophyStudies of the same condition, open and under way.
- More trials run by Children's Hospital Medical Center, CincinnatiThe sponsor's other registered studies.
- 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
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind
- Who is masked
- Outcomes assessor
- Enrolment
- 44 participants (actual)
Sponsor and investigators
- Lead sponsor
- Children's Hospital Medical Center, Cincinnati
- Sponsor type
- University, hospital or other
- Collaborators
- Ipsen, Charley's Fund
- Responsible party
- Sponsor
- Investigators
- Meilan Rutter, MD, Principal investigator, Children's Hospital Medical Center, Cincinnati
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT01207908
- Sponsor's protocol ID
- 2010-1491
- Status last verified
- Dec 2020
- First submitted
- 22 Sep 2010
- Last updated
- 20 Jan 2021
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
Study at a glance
From the registry record, last updated by the sponsor on 20 Jan 2021. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy
- ClinicalTrials.gov ID
- NCT01207908
- Acronym / sponsor ID
- 2010-1491
- Conditions
- Duchenne Muscular Dystrophy
- Keywords
- DMD, Duchenne Muscular Dystrophy, Insulin like growth factor, 6 minute walk test
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 44 participants (actual)
- Lead sponsor
- Children's Hospital Medical Center, CincinnatiUniversity, hospital or other
- Collaborators
- Ipsen, Charley's Fund
- Study start
- Nov 2010 (actual)
- Primary completion
- Oct 2012 (actual)
- Study completion
- Jun 2013 (actual)
- First posted
- 23 Sep 2010 (estimated)
- Results
- Posted 20 Jan 2021
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 determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
Detailed description
Detailed Description:
DMD is a progressive degenerative muscle disorder for which there is no current cure. Glucocorticoids (GC) are often used to improve motor function and survival but have significant side effects such as growth failure, weight gain, insulin resistance and osteoporosis. IGF-1 stimulates both the proliferation and differentiation of skeletal muscle cells and is thus important for muscle repair and regeneration. IGF-1 offers potential as a therapeutic agent for DMD as it may improve or preserve motor function and reduce GC side effects such as growth failure and insulin resistance.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Single blind (outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| IGF-1 | Experimental | Drug: IGF-1IGF-1 plus standard steroid treatment |
| Standard steroid treatment alone | No intervention | –Standard daily steroid treatment for DMD |
Who may qualify
- Ages
- 5 years and older
- Sex
- Male only
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
Key inclusion criteria (9 listed)
- DMD diagnosed with mutational testing and/or complete absence of dystrophin on muscle biopsy
- Proximal pelvic girdle weakness (Gower's maneuver, difficulty with arising from floor and going up steps)
- Male
- Age > 5 years of age
- Bone maturation (assess by bone age x-ray): </= 11 years of age
Key exclusion criteria (10 listed)
- Current or prior treatment with growth hormone or IGF-1 therapy
- Non-ambulatory
- Pubertal (based on clinical Tanner staging examination)
- Congestive cardiac failure
- History of intracranial hypertension
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| IGF-1Also: Increlex (mecasermin [rDNA origin] injection)IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months. | Drug | IGF-1 |
Primary outcome
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Time frame: 6 months
Outcome Measure Data Table shows change of 6-Minute Walk Distance at 6 months versus baseline in each arm. The Statistical Analysis section shows the 6-month difference between the 2 arms (control minus IGF-1).
2 secondary outcomes
Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Time frame: 6 months
Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Time frame: 6 months
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 20 Jan 2021.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
- Genetics Home ReferenceLink provided by the sponsor.
- Duchenne and Becker Muscular DystrophyLink provided by the sponsor.
- Muscular DystrophyLink provided by the sponsor.
- MedlineLink provided by the sponsor.
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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.
Meilan Rutter, MD principal investigator
Children's Hospital Medical Center, Cincinnati
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing IGF-1 in people with Duchenne Muscular Dystrophy, run by Children's Hospital Medical Center, Cincinnati 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 9 inclusion and 10 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 20 Jan 2021. 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.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 20 Jan 2021). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
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?
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline (time frame: 6 months). 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 20 Jan 2021.