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



Status:Completed
Healthy:No
Age Range:20 - 55
Updated:6/27/2018
Start Date:December 2014
End Date:August 2016

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A Phase 1/2a Blinded, Placebo-Controlled Study to Assess the Safety, Tolerability, and Dose-range Finding of Multiple Ascending Doses of ISIS 598769 Administered Subcutaneously to Adult Patients 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.

This is a Phase 1/2a multicenter, blinded, placebo-controlled study of ISIS-DMPK Rx in adult
patients with DM1.

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 ≥ 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
Investigator

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
defibrillator)

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
hypogonadism)

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,
Mexiletine

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
We found this trial at
8
sites
60 Crittenden Blvd # 70
Rochester, New York 14642
(585) 275-2121
Principal Investigator: Richard Moxley III, MD
Phone: 585-276-4611
University of Rochester The University of Rochester is one of the country's top-tier research universities....
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707 North Broadway
Baltimore, Maryland 21205
443-923-9200
Principal Investigator: Doris Leung, MD
Phone: 443-923-2697
Kennedy Krieger Institute While not officially part of Johns Hopkins Medicine, Kennedy Krieger Institute is...
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Baltimore, MD
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Gainesville, Florida 32610
(352) 392-3261
Principal Investigator: S. H. Subramony, MD
Phone: 352-273-7732
University of Florida The University of Florida (UF) is a major, public, comprehensive, land-grant, research...
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201 Presidents Circle
Salt Lake City, Utah 84108
801) 581-7200
University of Utah Research is a major component in the life of the U benefiting...
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281 W. Lane Ave
Columbus, Ohio 43210
(614) 292-6446
Principal Investigator: John Kissel, MD
Phone: 614-293-4098
Ohio State University The Ohio State University’s main Columbus campus is one of America’s largest...
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Houston, Texas 77030
Principal Investigator: Ericka Simpson, MD
Phone: 713-441-3057
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3901 Rainbow Blvd
Kansas City, Kansas 66160
(913) 588-5000
Principal Investigator: Richard Barohn, MD
Phone: 913-945-9934
University of Kansas Medical Center The University of Kansas Medical Center serves Kansas through excellence...
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300 Pasteur Dr
Stanford, California 94305
(650) 723-4000
Principal Investigator: John Day, MD
Phone: 650-724-3792
Stanford Univ Med Ctr The Medical Center is uniquely advantaged by its location on the...
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