Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

Learn more about:
Related Clinical Trial
Effects of a 12-week Strength Training Program in Women With Myotonic Dystrophy Type 1 DMCRN-02-001: Assessing Pediatric Endpoints in DM1 Myotonic Dystrophy Type 1 and Resistance Exercise Phase 1/2 Study to Evaluate Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of AOC 1001 Administered Intravenously to Adult Myotonic Dystrophy Type 1 (DM1) Patients Biomarker Development for Muscular Dystrophies Extracellular RNA Biomarkers of Myotonic Dystrophy Symptoms and Outcome Measures for Upper- Limb Function in Myotonic Dystrophy Type 1 Safety and Efficacy of Pitolisant on Excessive Daytime Sleepiness and Other Non-Muscular Symptoms in Patients With Myotonic Dystrophy Type 1 Poor Neck Proprioception May Cause Balance Deficits in Myotonic Dystrophy 1 Myotonic Dystrophy – Vascular and Cognition Effect of MYODM on Quality of Life, Fatigue and Hypersomnia in Patients With Myotonic Dystrophy Type 1 Open Label Study in Adolescents and Children With Myotonic Disorders European Home Mechanical Ventilation Registry Effects of SomatoKine (Iplex)Recombinant Human Insulin-like Growth Factor-1/Recombinant Human Insulin-like Growth Factor-binding Protein-3 (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1) Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1 A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1 Sleep Breathing Disorders, a Main Trigger for Cardiac ARythmias in Type I Myotonic Dystrophy ? Safety, Tolerability and Pharmacokinetics of ERX-963 in Adults With Myotonic Dystrophy Type 1 Effects of a 12-week Strength Training Program in Men With Myotonic Dystrophy Type 1 Efficacy and Safety of DHEA for Myotonic Dystrophy Study of Tideglusib in Adolescent and Adult Patients With Myotonic Dystrophy Effects of a Multiple Component Training Program on Muscles in Adults With Myotonic Dystrophy Type 1 Ventilatory Response After Non Invasive Ventilation in Type 1 Myotonic Dystrophy Clinical Efficacy Trial of Mexiletine for Myotonic Dystrophy Type 1 DM-IMT – Controlled, Randomized, Three-arm Intervention Study on the Safety and Efficacy of Regular Respiratory Muscle Training in Patients With Myotonic Dystrophy Type 1 Venous Thromboembolism in DM1 Postural Spirometry Changes in Ambulatory Myotonic Dystrophy Patients Factors Associated With Hypoventilation in the Myotonic Dystrophy, Progressive Profile Over 5 Years PhenoDM1 (Myotonic Dystrophy Type 1 Natural History Study) Children’s Health Research Institute(CHRI), Stanford Lucile Packard Children Hospital (LPCH) Protocol on Myotonic Dystrophy Establishing Biomarkers and Clinical Endpoints in Myotonic Dystrophy Type 1 (END-DM1) Observational Prolonged Trial in Myotonic Dystrophy Type 1 Venous Thromboembolism in Myotonic Dystrophy Type 1 Myotonic Dystrophy Type 1 Aerobic Exercise Study Multicenter Observational Study of Myotonic Dystrophy Type 1 DM1 Heart Registry – DM1 Respiratory Registry

Brief Title

Safety and Efficacy Study of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) In Myotonic Dystrophy Type 1

Official Title

A Placebo Controlled, Randomized, Double-Blind Phase II Clinical Trial to Evaluate Tolerability, Safety and Efficacy Endpoints After Administration of Recombinant Human Insulin-Like Growth Factor-I/Recombinant Human Insulin-Like Growth Factor Binding Protein-3 (rhIGF-I/rhIGFBP-3) for 24 Weeks in Adults With Myotonic Dystrophy Type 1

Brief Summary

      To investigate the effects of rhIGF-I/rhIGFBP-3 treatment for 24 weeks on endurance,
      ambulation, cognitive functioning, insulin resistance, lipid levels, muscle function and
      strength, pain, gastrointestinal functioning, and quality of life endpoints in DM1 patients
    

Detailed Description

      Efficacy Measures:

      Endurance, Ambulation, Cognitive function, Insulin resistance, Cholesterol and triglycerides,
      Muscle function and strength, Pain, Gastrointestinal function, Quality of life

      MINIMUM INCLUSION CRITERIA

        1. A diagnosis of DM1, confirmed by DM1 genetic mutation

        2. Age 21 to 65 years (inclusive)

        3. Ability to walk 30 feet - assistance with cane and/or leg bracing permitted

        4. Able to self-administer study medication by subcutaneous injection or caregiver is
           available to administer study medication
    

Study Phase

Phase 2

Study Type

Interventional


Primary Outcome

Change From Baseline to Week 24 in Distance Walked as Assessed by the Six-minute Walk Test (6MWT) Distance


Condition

Myotonic Dystrophy Type 1

Intervention

rhIGF-I/rhIGFBP-3

Study Arms / Comparison Groups

 rhIGF-I/rhIGFBP-3
Description:  

Publications

* Includes publications given by the data provider as well as publications identified by National Clinical Trials Identifier (NCT ID) in Medline.

Recruitment Information


Recruitment Status

Drug

Estimated Enrollment

69

Start Date

December 2007

Completion Date

December 29, 2008

Primary Completion Date

December 29, 2008

Eligibility Criteria

        Inclusion Criteria (list is not inclusive):

          -  A diagnosis of DM1, confirmed by DM1 genetic mutation

          -  Ability to walk 30 feet - assistance with cane and/or leg bracing permitted

          -  Able to self-administer study medication by subcutaneous injection or caregiver is
             available to administer study medication

        Exclusion Criteria (list is not inclusive):

          -  Congenital DM1

          -  Weight greater than 100 kg or body mass index greater than 30 kg/m2

          -  Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone,
             investigational agent within 60 days of screening

          -  Current diagnosis or history of malignancy expect for surgically cured skin cancer or
             pilomatricoma

          -  Changes in lipid lowering medications during the 3 months prior to screening

          -  Diaphragmatic weakness such that patients are unable to tolerate the supine position,
             or swallowing impairment such that patients are unable to maintain nutrition without
             use of gastrostomy.

          -  Major psychiatric illness (major depression, bipolar disorder or schizophrenia) within
             twelve months of screening

          -  History of non-compliance with other therapies
      

Gender

All

Ages

21 Years - 65 Years

Accepts Healthy Volunteers

No

Contacts

Richard Moxley, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00577577

Organization ID

INSM-110-1001


Responsible Party

Sponsor

Study Sponsor

Insmed Incorporated

Collaborators

 Muscular Dystrophy Association

Study Sponsor

Richard Moxley, M.D., Study Chair, University of Rochester Neuromuscular Disease Center


Verification Date

December 2021