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

NCT01207908 | Completed Phase 1 Results DMC

• 1 other identifier: 2010-1491
• Study Type: interventional
• Target: 44
• Locations: 1 country
• Sites: 1

Brief Summary

The purpose of this study is to determine whether IGF-1 therapy improves or preserves muscle function in Duchenne Muscular Dystrophy (DMD).

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; Duchenne Muscular Dystrophy; Insulin like growth factor; 6 minute walk test

Outcome Measures

Primary Outcomes (1)

• 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

Secondary Outcomes (2)

• Difference in Height Velocity Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline

  6 months

• Difference in North Star Ambulatory Assessment (NSAA) Score Between Groups (Control Minus IGF-1) for Change at 6 Months Versus Baseline

  6 months

Study Arms (2)

IGF-1

EXPERIMENTAL

IGF-1 plus standard steroid treatment

Drug: IGF-1

Standard steroid treatment alone

NO INTERVENTION

Standard daily steroid treatment for DMD

Interventions

IGF-1 DRUG

IGF-1 will be administered once daily by subcutaneous injection every morning with breakfast. Duration 6 months.

Also known as: Increlex (mecasermin [rDNA origin] injection)

IGF-1

Eligibility Criteria

• Age: 5 Years+
• Sex: male
• Healthy Volunteers: No
• Age Groups: Child (0-17), Adult (18-64), Older Adult (65+)

You may qualify if:

• 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

You may not qualify if:

• 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

Sponsors & Collaborators

• Children's Hospital Medical Center, Cincinnati: lead
• Ipsen: collaborator
• Charley's Fund: collaborator

Study Sites (1)

Cincinnati Children's Hospital Medical Center

Cincinnati, Ohio, 45229, United States

Location

Related Links

• Genetics Home Reference
• Duchenne and Becker Muscular Dystrophy
• Muscular Dystrophy
• Medline

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Interventions

Insulin-Like Growth Factor I; mecasermin; Injections

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Intervention Hierarchy (Ancestors)

Somatomedins; Insulin-Like Peptides; Intercellular Signaling Peptides and Proteins; Peptides; Amino Acids, Peptides, and Proteins; Blood Proteins; Proteins; Biological Factors; Drug Administration Routes; Drug Therapy; Therapeutics

Limitations and Caveats

Small sample size and short duration, Open-label study.

Results Point of Contact

• Title: Dr. Meilan Rutter
• Organization: Cincinnati Children's Hospital Medical Center

meilan.rutter@cchmc.org

513-636-4744

Study Officials

• Meilan Rutter, MD

  Children's Hospital Medical Center, Cincinnati

  PRINCIPAL INVESTIGATOR

Publication Agreements

• PI is Sponsor Employee: Yes

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: SINGLE
• Who Masked: OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: OTHER
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: September 22, 2010
• First Posted: September 23, 2010
• Study Start: November 1, 2010
• Primary Completion: October 1, 2012
• Study Completion: June 1, 2013
• Last Updated: January 20, 2021
• Results First Posted: January 20, 2021

Record last verified: 2020-12

Locations

US (1)