The purpose of this study is to learn about the effects of an investigational medicine, PGN-EDODM1, to see how safe and tolerable multiple administrations of PGN-EDODM1 are for people with myotonic dystrophy type 1 (DM1) compared to placebo.
Study Type
INTERVENTIONAL
Allocation
RANDOMIZED
Purpose
TREATMENT
Masking
QUADRUPLE
Enrollment
24
Administered by intravenous (IV) infusion
Administered by intravenous (IV) infusion
University of Calgary
Calgary, Alberta, Canada
RECRUITINGOttawa Hospital Research Institute (OHRI)
Ottawa, Ontario, Canada
RECRUITINGCIUSSS du Saguenay-Lac-Saint-Jean
Chicoutimi, Quebec, Canada
Safety and tolerability as assessed by number of participants with Adverse Events (AEs)
Time frame: Baseline through Day 112
Maximum Observed Plasma Drug Concentration (Cmax) of PGN-EDODM1
Time frame: Day 1 through Day 84
Time to Maximum Observed Plasma Drug Concentration (Tmax) of PGN-EDODM1
Time frame: Day 1 through Day 84
Apparent Terminal Half-Life (t½) of PGN-EDODM1
Time frame: Day 1 through Day 84
Area Under the Concentration-time Curve of PGN-EDODM1
Time frame: Day 1 through Day 84
Change in splicing index in skeletal muscle tissue
Time frame: Baseline through Day 91
Change in myotonia as measured by video Hand Opening Time (vHOT)
Time frame: Baseline through Day 112
Hand grip strength
by dynamometer
Time frame: Baseline through Day 112
Change in mobility as measured by 10 meter walk/run time
Time frame: Baseline through Day 112
This platform is for informational purposes only and does not constitute medical advice. Always consult a qualified healthcare professional.
Montreal Neurological Institute
Montreal, Quebec, Canada
RECRUITINGSalford Royal Hospital
Salford, England, United Kingdom
RECRUITINGUniversity College London Hospitals NHS Foundation Trust
London, UK, United Kingdom
RECRUITINGNewcastle Upon Tyne Hospitals
Newcastle upon Tyne, United Kingdom
RECRUITING