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| Name | Class |
|---|---|
| Ipsen | INDUSTRY |
| Charley's Fund | OTHER |
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The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| IGF-1 | Experimental | IGF-1 plus standard steroid treatment |
|
| Standard steroid treatment alone | No Intervention | Standard daily steroid treatment for DMD |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IGF-1 | Drug | IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months. |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 6 months |
| Measure | Description | Time Frame |
|---|---|---|
| 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). | 6 months |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Meilan Rutter, MD | Children's Hospital Medical Center, Cincinnati | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Cincinnati Children's Hospital Medical Center | Cincinnati | Ohio | 45229 | United States |
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| Label | URL |
|---|---|
| Genetics Home Reference | View source |
| Duchenne and Becker Muscular Dystrophy | View source |
| Muscular Dystrophy |
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| ID | Title | Description |
|---|---|---|
| FG000 | IGF-1 | IGF-1 plus standard steroid treatment IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months. |
| FG001 | Standard Steroid Treatment Alone | Once daily steroid treatment (Deflazacort or Prednisone) administered orally for DMD. |
| Title | Milestones | Reasons Not Completed | ||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | IGF-1 | IGF-1 plus standard steroid treatment IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months. |
| BG001 | Standard Steroid Treatment Alone |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | ||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | 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). | Posted | Mean | Standard Deviation | meters | 6 months |
|
6 months
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | IGF-1 | IGF-1 plus standard steroid treatment IGF-1: IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months. |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Intracranial hypertension | Nervous system disorders | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Headache | Nervous system disorders | Systematic Assessment |
Small sample size and short duration, Open-label study.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Dr. Meilan Rutter | Cincinnati Children's Hospital Medical Center | 513-636-4744 | meilan.rutter@cchmc.org |
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| ID | Term |
|---|---|
| D020388 | Muscular Dystrophy, Duchenne |
| ID | Term |
|---|---|
| D009136 | Muscular Dystrophies |
| D020966 | Muscular Disorders, Atrophic |
| D009135 | Muscular Diseases |
| D009140 | Musculoskeletal Diseases |
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| ID | Term |
|---|---|
| D007334 | Insulin-Like Growth Factor I |
| C000604197 | mecasermin |
| D007267 | Injections |
| ID | Term |
|---|---|
| D013002 | Somatomedins |
| D000096764 | Insulin-Like Peptides |
| D036341 | Intercellular Signaling Peptides and Proteins |
| D010455 | Peptides |
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| 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. |
| 6 months |
| Medline | View source |
Standard daily steroid treatment for DMD
| BG002 | Total | Total of all reporting groups |
| Years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
|
|
|
| Secondary | 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). | Posted | Mean | Standard Deviation | cm/year | 6 months |
|
|
|
|
| Secondary | 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. | Posted | Mean | Standard Deviation | score on a scale | 6 months |
|
|
|
|
| 0 |
| 22 |
| 1 |
| 22 |
| 20 |
| 22 |
| EG001 | Standard Steroid Treatment Alone | Standard daily steroid treatment for DMD | 0 | 22 | 0 | 22 | 21 | 22 |
| Nausea and vomiting | Gastrointestinal disorders | Systematic Assessment |
|
| seizure | Nervous system disorders | Systematic Assessment |
|
| papilledema | Nervous system disorders | Systematic Assessment |
|
| stinging at injection site | Skin and subcutaneous tissue disorders | Systematic Assessment | The control group was not considered at risk for this adverse event because they did not receive any injections. Therefore the denominator here is zero. |
|
| fever | General disorders | Systematic Assessment |
|
| rash and skin welt | Skin and subcutaneous tissue disorders | Systematic Assessment |
|
| lipohypertrophy | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Foot pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Finger injury | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Leg injury | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Finger infection | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Leg pain | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Arm weakness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
| Leg numbness | Musculoskeletal and connective tissue disorders | Systematic Assessment |
|
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| D009468 | Neuromuscular Diseases |
| D009422 | Nervous System Diseases |
| D040181 | Genetic Diseases, X-Linked |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D000602 | Amino Acids, Peptides, and Proteins |
| D001798 | Blood Proteins |
| D011506 | Proteins |
| D001685 | Biological Factors |
| D004333 | Drug Administration Routes |
| D004358 | Drug Therapy |
| D013812 | Therapeutics |