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Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

Primary Purpose

Duchenne Muscular Dystrophy

Status
Completed
Phase
Phase 1
Locations
United States
Study Type
Interventional
Intervention
IGF-1
Sponsored by
Children's Hospital Medical Center, Cincinnati
About
Eligibility
Locations
Arms
Outcomes
Full info

About this trial

This is an interventional treatment trial for Duchenne Muscular Dystrophy focused on measuring DMD, Duchenne Muscular Dystrophy, Insulin like growth factor, 6 minute walk test

Eligibility Criteria

5 Years - undefined (Child, Adult, Older Adult)MaleDoes not accept healthy volunteers

Inclusion Criteria:

  • 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
  • Ambulatory
  • Informed consent
  • Willingness and ability to comply with all protocol requirements and procedures

Exclusion Criteria:

  • 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)
  • 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

Sites / Locations

  • Cincinnati Children's Hospital Medical Center

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

No Intervention

Arm Label

IGF-1

Standard steroid treatment alone

Arm Description

IGF-1 plus standard steroid treatment

Standard daily steroid treatment for DMD

Outcomes

Primary Outcome Measures

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).

Secondary Outcome Measures

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).
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.

Full Information

First Posted
September 22, 2010
Last Updated
December 28, 2020
Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
Ipsen, Charley's Fund
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1. Study Identification

Unique Protocol Identification Number
NCT01207908
Brief Title
Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy
Official Title
IGF-1 Therapy and Muscle Function in Duchenne Muscular Dystrophy
Study Type
Interventional

2. Study Status

Record Verification Date
December 2020
Overall Recruitment Status
Completed
Study Start Date
November 2010 (Actual)
Primary Completion Date
October 2012 (Actual)
Study Completion Date
June 2013 (Actual)

3. Sponsor/Collaborators

Responsible Party, by Official Title
Sponsor
Name of the Sponsor
Children's Hospital Medical Center, Cincinnati
Collaborators
Ipsen, Charley's Fund

4. Oversight

Data Monitoring Committee
Yes

5. Study Description

Brief Summary
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.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study
Duchenne Muscular Dystrophy
Keywords
DMD, Duchenne Muscular Dystrophy, Insulin like growth factor, 6 minute walk test

7. Study Design

Primary Purpose
Treatment
Study Phase
Phase 1, Phase 2
Interventional Study Model
Parallel Assignment
Masking
Outcomes Assessor
Allocation
Randomized
Enrollment
44 (Actual)

8. Arms, Groups, and Interventions

Arm Title
IGF-1
Arm Type
Experimental
Arm Description
IGF-1 plus standard steroid treatment
Arm Title
Standard steroid treatment alone
Arm Type
No Intervention
Arm Description
Standard daily steroid treatment for DMD
Intervention Type
Drug
Intervention Name(s)
IGF-1
Other Intervention Name(s)
Increlex (mecasermin [rDNA origin] injection)
Intervention Description
IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.
Primary Outcome Measure Information:
Title
Difference in 6-Minute Walk Distance Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Description
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).
Time Frame
6 months
Secondary Outcome Measure Information:
Title
Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Description
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).
Time Frame
6 months
Title
Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline
Description
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.
Time Frame
6 months

10. Eligibility

Sex
Male
Minimum Age & Unit of Time
5 Years
Accepts Healthy Volunteers
No
Eligibility Criteria
Inclusion Criteria: 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 Ambulatory Informed consent Willingness and ability to comply with all protocol requirements and procedures Exclusion Criteria: 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) 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
Overall Study Officials:
First Name & Middle Initial & Last Name & Degree
Meilan Rutter, MD
Organizational Affiliation
Children's Hospital Medical Center, Cincinnati
Official's Role
Principal Investigator
Facility Information:
Facility Name
Cincinnati Children's Hospital Medical Center
City
Cincinnati
State/Province
Ohio
ZIP/Postal Code
45229
Country
United States

12. IPD Sharing Statement

Links:
URL
http://ghr.nlm.nih.gov/
Description
Genetics Home Reference
URL
http://ghr.nlm.nih.gov/condition/duchenne-and-becker-muscular-dystrophy
Description
Duchenne and Becker Muscular Dystrophy
URL
http://www.nlm.nih.gov/medlineplus/musculardystrophy.html
Description
Muscular Dystrophy
URL
http://www.nlm.nih.gov/medlineplus/
Description
Medline

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Safety and Efficacy Study of IGF-1 in Duchenne Muscular Dystrophy

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