Full Phase I Drug Trial Recruiting for Myotonic Dystrophy (DM1)

This week ISIS pharmaceuticals announced the commencement of recruiting for Phase I of the Myotonic Dystrophy Drug Trial. This is tremendous news for the myotonic dystrophy community. The promise of scientific research to define the DNA site, identify the cause of the disease,  then begin to develop treatments that would reverse the problems in the cell are culminating  to the promise of hope for all that suffer from this horrible affliction.

If you want to participate in this study you must act quickly and contact the centers that are conducting the research. Find the closest location and contact the person via email or phone. You must meet all (meaning all) the qualifications for the trial. The full information is on CLINICALTRIALS

The study is a phase 1 checking for safety and dosage (how much of the drug gets into the blood) that remains in the blood. The information that they are evaluating is whether the drug causes any advserse or bad reactions in the people in the study. Half of the people will get the drug and the other 1/2 will get a placebo (Salt water injection most likely). The study is scheduled to start in December and last for about 24 weeks

The drug was studied in mice that were created to simulate myotonic dystrophy and shows a remarkable ability to reverse the disease in these mice. It is unknown how it will affect humans but I am hopeful it will have a positive impact.

So take action immediately if you want to be considered as a potential participant. Only a very small number of people will be enrolled in the study and only half of these will get the drug!

United States, California

Stanford University Medical Center Not yet recruiting
Stanford, California, United States, 94305
Contact: Shirley Paulose 650-724-3792 spaulose@stanford.edu
Principal Investigator: John Day, MD

United States, Florida
University of Florida Not yet recruiting
Gainesville, Florida, United States, 32610
Contact: Phuong Deleyrolle 352-273-7732 pdeleyrolle@neurology.ufl.edu
Principal Investigator: Tetsuo Ashizawa, MD

United States, Kansas
University of Kansas Medical Center Not yet recruiting
Kansas City, Kansas, United States, 66103
Contact: Nicole Jenci 913-945-9934 njenci@kumc.edu
Principal Investigator: Richard Barohn, MD

United States, Maryland
Kennedy Krieger Institute Not yet recruiting
Baltimore, Maryland, United States, 21205
Contact: Genila Bibat 443-923-2697 bibat@kennedykrieger.org
Principal Investigator: Kathryn Wagner, MD

United States, New York
University of Rochester Not yet recruiting
Rochester, New York, United States, 14642
Contact: Jeanne Deckdebrun 585-276-4611 Jeanne_Dekdebrun@urmc.rochester.edu
Principal Investigator: Richard Moxley III, MD

United States, Ohio
The Ohio State University Not yet recruiting
Columbus, Ohio, United States, 43221
Contact: Filiz Muharrem 614-688-7837 Filiz.Muharrem@osumc.edu
Principal Investigator: John Kissel, MD

United States, Utah

University of Utah Recruiting
Salt Lake, Utah, United States, 84132
Contact: Melissa Dixon 801-585-7606 missy.dixon@genetics.utah.edu
Principal Investigator: Nicholas Johnson, MD

Terms and Conditions to participate in the Study. You must meet all the conditions to be elibible for the drug trial…

Inclusion Criteria:

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

Satisfy the following:
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.
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.
BMI Genetic confirmation of DM1 with DMPK CTG repeat length ≥ 100
Onset of DM1 symptoms after the age of 12
Clinically apparent myotonia equivalent to hand opening time of at least 2 seconds, in the opinion of the Investigator
Ambulatory (orthoses allowed, canes and walkers not allowed) and able to walk at least 25 meters at screening

Exclusion Criteria:

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
Clinically significant abnormalities in screening laboratory values that would render the subject unsuitable for inclusion
Active infection requiring systemic antiviral or antimicrobial therapy that will not be completed prior to Study Day 1
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
Known history of or previous positive test for human immunodeficiency virus (HIV), hepatitis C, or chronic hepatitis B
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
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)
Recent history of or current drug or alcohol abuse
History of bleeding tendency or ongoing oral anticoagulation
Developmental delay, intellectual disability, or significant behavioral neuropsychiatric manifestations
Thyroid dysfunction that is untreated (if on thyroid hormone replacement therapy, need to have adequate and stable replacement over the previous 6 months)
Implanted device for the treatment of cardiac problems (i.e., pacemaker or defibrillator)
Clinically significant abnormal ECG or echocardiogram, or significant symptoms of cardiac dysfunction at Screening
Have a seizure disorder
If being treated with testosterone, on a stable replacement dose (i.e., for hypogonadism)
Treatment with corticosteroids within 8 weeks prior to the first dose of Study Drug
History of hypersensitivity to local anesthetics to be used in the biopsy procedure or components thereof
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
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

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