Trial Search Results

A Safety andTolerability Study of Multiple Doses of ISIS-DMPKRx in Adults With Myotonic Dystrophy Type 1

This study will test the safety, tolerability, and pharmacokinetics of multiple escalating doses of ISIS-DMPKRx administered subcutaneously to adult patients with DM1.

Stanford is currently not accepting patients for this trial.

Lead Sponsor:

Ionis Pharmaceuticals, Inc.

Stanford Investigator(s):


  • Drug: IONIS-DMPKRx
  • Drug: Placebo


Phase 1/Phase 2


Inclusion Criteria:

   1. Must have given written informed consent (signed and dated) and any authorizations
   required by local law and be able to comply with all study requirements

   2. Males or females aged 20 to 55 years old at the time of informed consent

   3. Satisfy the following:

      1. Females: non-pregnant and non-lactating, surgically sterile, post menopausal,
      abstinent, or if engaged in sexual relations of child-bearing potential, subject
      is using an acceptable contraceptive method from the time of signing the informed
      consent until at least 14 weeks after the last dose of Study Drug.

      2. Males: surgically sterile, abstinent or if engaged in sexual relations with a
      female of child-bearing potential, the subject must be using an acceptable
      contraceptive method from the time of signing the informed consent form until at
      least 14 weeks after the last dose of Study Drug.

   4. BMI <35.0 kg/m2

   5. Genetic confirmation of DM1 with DMPK CTG repeat length ‚Č• 100

   6. Onset of DM1 symptoms after the age of 12

   7. Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds, in
   the opinion of the Investigator

   8. Ambulatory (orthoses allowed, canes and walkers not allowed) and able to walk at least
   25 meters at screening

Exclusion Criteria:

   1. Clinically significant abnormalities in medical history (e.g., previous acute coronary
   syndrome within 6 months of screening, major surgery within 3 months of screening) or
   physical examination

   2. Clinically significant abnormalities in screening laboratory values that would render
   the subject unsuitable for inclusion

   3. Active infection requiring systemic antiviral or antimicrobial therapy that will not
   be completed prior to Study Day 1

   4. Unwilling or unable to comply with study procedures (e.g., muscle biopsies), including
   follow-up, as specified by this protocol, or unwillingness to cooperate fully with the

   5. Known history of or previous positive test for human immunodeficiency virus (HIV),
   hepatitis C, or chronic hepatitis B

   6. Active malignancy or history within last 5 years, except for basal or squamous cell
   carcinoma of the skin, carcinoma in situ of the cervix that has been successfully
   treated, or pilomatricoma

   7. Treatment with another investigational drug, biologic agent, or device within one
   month of screening, or 5 half-lives of investigational agent, whichever is longer; any
   history of previous treatment with an oligonucleotide (including siRNA)

   8. Recent history of or current drug or alcohol abuse

   9. History of bleeding tendency or ongoing oral anticoagulation

10. Developmental delay, intellectual disability, or significant behavioral
   neuropsychiatric manifestations

11. Thyroid dysfunction that is untreated (if on thyroid hormone replacement therapy, need
   to have adequate and stable replacement over the previous 6 months)

12. Implanted device for the treatment of cardiac problems (i.e., pacemaker or

13. Clinically significant abnormal ECG or echocardiogram, or significant symptoms of
   cardiac dysfunction at Screening

14. Have a seizure disorder

15. If being treated with testosterone, on a stable replacement dose (i.e., for

16. Treatment with corticosteroids within 8 weeks prior to the first dose of Study Drug

17. History of hypersensitivity to local anesthetics to be used in the biopsy procedure or
   components thereof

18. Treatment with anti-myotonia medication within 30 days prior to screening. May
   include, but not be limited to: Phenytoin, Carbamazepine, Procainamide, Disopyramide,
   Nifedipine, Acetazolamide, Clomipramine, Imipramine, Amytriptiline, Taurine, Quinine,

19. Have any condition, which, in the opinion of the investigator would make the subject
   unsuitable for inclusion, or could interfere with the subject participating in or
   completing the study

Ages Eligible for Study

20 Years - 55 Years

Genders Eligible for Study


Not currently accepting new patients for this trial

Contact Information

Stanford University
School of Medicine
300 Pasteur Drive
Stanford, CA 94305
Shirley Paulose
Not Recruiting