IPLEX™ Myotonic Dystrophy Study
NOW ENROLLING PATIENTS
Insmed is developing a drug, rhIGF-I/rhIGFBP-3 (sometimes referred to as mecasermin rinfabate or IPLEX™), which has shown promising results in an open-label study in patients with myotonic dystrophy (DM1).
Study Purpose
To further our understanding of the effects of rhIGF-I/rhIGFBP-3, we are conducting a randomized, placebo-controlled, double-blind clinical study in 60 patients with DM1. Patients will receive rhIGF-I/rhIGFBP-3 or placebo for 24 weeks and will undergo assessments to determine the effect of rhIGF-I/rhIGFBP-3 on muscle strength, endurance, ambulation, cognitive functioning, gastrointestinal symptoms, and general health status.
Study Details
Study participants will be required to visit the study site 8 times during the study (screening visit, baseline visit and a visit every 4 weeks during the 24 week treatment period). Procedures will include physical examinations, electrocardiograms (ECGs), muscle testing, endurance and ambulation testing, questionnaires, cognitive testing, blood draws and DM1 genetic testing. The study procedures will be provided free of charge, and participant compensation and/or reimbursement for expenses may be available.
Recruitment Period
Recruitment opened in December 2007 and is expected to remain open until 60 patients are enrolled.
Eligibility Requirements
- Able and willing to give informed consent
- Diagnosis of DM1 (not congenital), confirmed by DM1 genetic mutation
- Age 21-65 years
- Does not have insulin requiring diabetes or type 2 diabetes requiring oral anti-diabetic agents
- Does not have a current diagnosis or history of malignancy
- Does not have any other medical condition(s) that may pose a risk to the participant or interfere with the study objectives (e.g., untreated thyroid disease, major psychiatric illness, symptomatic liver or kidney disease, untreatable hypertension)
Site Study Locations
- Orange, California
- Sacramento, California
- Kansas City, Kansas
- Baltimore, Maryland
- Minneapolis, Minnesota
- St. Louis, Missouri
- Rochester, New York
- Columbus, Ohio
- Portland, Oregon
- Galveston, Texas
- San Antonio, Texas
- Salt Lake City, Utah
Contact Us
For more information about the study, please contact Insmed at clinicaltrials@insmed.com. Or by Phone at 804-565-3130.
