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)

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Brief Title

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)

Official Title

Effects of SomatoKine (Iplex) (rhIGF-I/rhIGFBP-3) in Myotonic Dystrophy Type 1 (DM1)

Brief Summary

      The aim of this study is to investigate the safety and feasibility of daily subcutaneous
      injections of recombinant IGF1 complexed with IGF binding protein 3 (SomatoKine-INSMED) as a
      treatment for muscle wasting and weakness in myotonic dystrophy type 1.
    


Study Phase

Phase 1/Phase 2

Study Type

Interventional


Primary Outcome

The Number of Study Participants Who Safely Tolerated Somatokine


Condition

Myotonic Dystrophy

Intervention

SomatoKine/IPLEX

Study Arms / Comparison Groups

 Cohort 1- Two Escalating Doses of Iplex
Description:  0.5 and 1.0 mg/kg/day

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

17

Start Date

November 2005

Completion Date

May 2008

Primary Completion Date

May 2008

Eligibility Criteria

        Inclusion Criteria:

          -  A clinical diagnosis of DM-1 according to accepted clinical research criteria.23 The
             clinical research criteria require each of the following: (1) clinically evident
             myotonia; (2) muscle weakness in a characteristic distribution (distal predominant);
             and (3) similar findings in a first degree relative.

          -  Age 21 to 60 years (inclusive).

          -  Ability to walk 30 feet without assistance (cane and leg bracing is permitted).

          -  Weakness of sufficient severity to justify treatment and provide a reasonable
             opportunity to observe a therapeutic effect. At the eligibility evaluation, eligible
             patients must show both of the following:

               1. muscle strength in a distal muscle group (ankle dorsiflexors or deep flexors of
                  the fingers) which is less than or equal to grade 4 (Medical Research Council
                  grade).

               2. muscle strength in a proximal or mid-limb muscle group (flexors or extensors of
                  the knee, elbow, shoulder, or hip) which is which is greater than or equal to 4-
                  (Medical Research Council grade).

          -  For patients that are not within driving distance to Rochester, a local health care
             provider in their area must be able to complete their home visits.

          -  Competent, willing, and able to give informed consent.

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

        Exclusion Criteria:

          -  Congenital DM-1. Congenital disease constitutes ~10% of all cases of DM-1. Early in
             life, the weakness in individuals with congenital DM-1 derives from maldevelopment of
             skeletal muscle rather than muscle degeneration. Later in life, these individuals are
             also subject to the added effects of a wasting process similar to classical DM-1.
             However, it is difficult to determine which of these phenomena are mainly to blame for
             weakness in a particular patient. Furthermore, more than 75% of patients with
             congenital DM-1 have mental retardation.

          -  Prior treatment with glucocorticoids, anabolic steroids, testosterone, growth hormone,
             or IGF-I within 1 year of entry; or any investigational agent within 60 days of entry.

          -  Any history of malignancy except for surgically cured skin cancer or pilomatricoma
             (benign tumor of the hair follicle that is associated with DM-1).

          -  Women of childbearing potential who are not using effective birth control; women who
             are pregnant or lactating.

          -  Medical illness which would prevent assessment of muscle strength or function. This
             exclusion would include individuals with orthopedic, cardiac, or pulmonary disorders
             which preclude proper positioning on the myometry testing table, or restrict their
             ability to tolerate repeated maximum muscle contractions.

          -  Known allergy to tetracycline.

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

          -  Symptomatic liver or kidney disease, insulin requiring diabetes or type 2 diabetes
             requiring oral anti-diabetic agents.

          -  Untreated thyroid disease (hypo or hyperthyroidism)

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

          -  History of non-compliance with other therapies.

          -  Drug or alcohol abuse within 12 months of enrollment.

          -  In men, evidence of a mass lesion on clinical examination by their primary care
             physician within twelve months prior to entry into the study (specifically prostate or
             testicular mass on clinical exam or other signs of mass lesion) or evidence of mass
             lesion on chest x-ray. In men 50 years of age or older, prostate specific antigen
             (PSA) elevation above normal.

          -  In women, evidence for mass lesion on clinical examination by their primary care
             physician or gynecologist (specifically breast & pelvic exam) within 12 months of
             entry into the study or evidence of mass lesion on chest x-ray. In women 40 years of
             age or older, evidence of mass lesion on mammogram. Women with Gail Scores > 1.7 will
             be excluded due to their increased risk of developing cancer.

          -  Atrial fibrillation/flutter; 2nd or 3rd degree heart block without pacemaker treatment

          -  Weight greater than 100 kilograms(kg).

          -  Body Mass Index greater than 30.

          -  History of bleeding diathesis or use of anticoagulant medications. Patients taking
             nonsteroidal anti-inflammatory agents will be asked to discontinue these medications 3
             days prior to muscle biopsy.
      

Gender

All

Ages

21 Years - 60 Years

Accepts Healthy Volunteers

Accepts Healthy Volunteers

Contacts

Richard T. Moxley, III, M.D., , 

Location Countries

United States

Location Countries

United States

Administrative Informations


NCT ID

NCT00233519

Organization ID

5 U54 NS048843-03 A


Responsible Party

Principal Investigator

Study Sponsor

University of Rochester

Collaborators

 National Institute of Neurological Disorders and Stroke (NINDS)

Study Sponsor

Richard T. Moxley, III, M.D., Principal Investigator, University of Rochester


Verification Date

June 2012