Phase
Condition
Muscular Dystrophy
Treatment
Placebo
AOC 1001 (del-desiran)
Clinical Study ID
Ages 16-65 All Genders
Study Summary
Eligibility Criteria
Inclusion
Key Inclusion Criteria:
Clinical and genetic diagnosis (CTG repeat ≥ 100) of DM1
Ability to walk independently (orthoses and ankle braces allowed) for at least 10meters at screening
Exclusion
Key Exclusion Criteria:
Breastfeeding, pregnancy, or intent to become pregnant during the study
Unwilling or unable to comply with contraceptive requirements
Abnormal lab values, conditions or diseases that would make the participantunsuitable for the study
Diabetes that is not adequately controlled
History of decompensated heart failure within 3 months of screening. Participantswith preexisting pacemaker/ICD are not excluded.
Body Mass Index > 35 kg/m2 at Screening
Recently treated with an investigational drug or biological agent
Treatment with anti-myotonic medication within 5 half-lives or 14 days of baseline,whichever is longer, prior to baseline.
Note: Additional protocol defined Inclusion and Exclusion criteria apply
Study Design
Study Description
Connect with a study center
The Ottawa Hospital
Ottawa, Ontario K1Y 4E9
CanadaSite Not Available
The Ottawa Hospital
Ottawa 6094817, Ontario 6093943 K1Y 4E9
CanadaSite Not Available
CSSS de Chicoutimi
Chicoutimi, Quebec G7H 5H6
CanadaSite Not Available
Montreal Neurological Institute
Montreal, Quebec H3A 2B4
CanadaSite Not Available
Montreal Neurological Institute
Montreal 6077243, Quebec 6115047 H3A 2B4
CanadaSite Not Available
Aarhus University Hospital
Aarhus N, 8200
DenmarkSite Not Available
Aarhus University Hospital
Aarhus N 11746890, 8200
DenmarkSite Not Available
Rigshospitalet
Copenhagen 2618425, 2100
DenmarkSite Not Available
Rigshospitalet
Kobenhavn, 2100
DenmarkSite Not Available
Centre de Reference des Maladies Neuromusculaires et de la SLA - AP-HM Hopital de La Timone
Marseille, 13005
FranceSite Not Available
AP-HP Hopital Pitie-Salpetriere
Paris, 75013
FranceSite Not Available
AP-HP Hopital Pitie-Salpetriere
Paris 2988507, 75013
FranceSite Not Available
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich, 80336
GermanySite Not Available
Klinikum der Ludwig-Maximilians-Universitaet Muenchen
Munich 2867714, 80336
GermanySite Not Available
Beaumont Hospital
Dublin, D09 C562
IrelandSite Not Available
Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milan 6951411, 20162
ItalySite Not Available
Fondazione Serena Onlus - Centro Clinico NeMO Milano
Milano, 20162
ItalySite Not Available
Fondazione Policlinico Universitario A. Gemelli IRCCS - Universita Cattolica del Sacro Cuore
Rome, 00168
ItalySite Not Available
Aomori Hospital
Aomori-shi, Aomori 038-1331
JapanSite Not Available
Aomori Hospital
Aomori 2130658, Aomori 2130656 038-1331
JapanSite Not Available
National Hospital Organization Osaka Toneyama Medical Center
Osaka, 560-8552
JapanSite Not Available
Osaka University Hospital
Osaka, 565-0871
JapanSite Not Available
National Hospital Organization Osaka Toneyama Medical Center
Osaka 1853909, 560-8552
JapanSite Not Available
Osaka University Hospital
Osaka 1853909, 565-0871
JapanSite Not Available
National Center of Neurology and Psychiatry
Tokyo, 187-8551
JapanSite Not Available
National Center of Neurology and Psychiatry
Tokyo 1850147, 187-8551
JapanSite Not Available
National Center of Neurology and Psychiatry
Toyko, 187-8551
JapanSite Not Available
Maastricht University Medical Center
Maastricht, 6229 HX
NetherlandsSite Not Available
Maastricht University Medical Center
Maastricht 2751283, 6229 HX
NetherlandsSite Not Available
Stichting Radboud Universitair Medisch Centrum
Nijmegen, 6525 GA
NetherlandsSite Not Available
Stichting Radboud Universitair Medisch Centrum
Nijmegen 2750053, 6525 GA
NetherlandsSite Not Available
Hospital Universitario Donostia
Donostia / San Sebastian 3110044, 20014
SpainSite Not Available
Hospital Universitario Donostia
San Sebastian, 20014
SpainSite Not Available
St. Georges University Hospitals NHS Foundation Trust
London, SW17 0QT
United KingdomSite Not Available
University College London Hospital
London, NW1 2BU
United KingdomSite Not Available
St. Georges University Hospitals NHS Foundation Trust
London 2643743, SW17 0QT
United KingdomSite Not Available
University College London Hospital
London 2643743, NW1 2BU
United KingdomSite Not Available
Royal Hallamshire Hospital
Sheffield, S10 2JF
United KingdomSite Not Available
University of California, Los Angeles (UCLA)
Los Angeles, California 90095
United StatesSite Not Available
University of California, San Diego (UCSD)
San Diego, California 92093
United StatesSite Not Available
Stanford University
Stanford, California 94305
United StatesSite Not Available
Stanford University
Stanford 5398563, California 5332921 94305
United StatesSite Not Available
University of Colorado
Denver, Colorado 80045
United StatesSite Not Available
University of Colorado
Denver 5419384, Colorado 5417618 80045
United StatesSite Not Available
University of Florida
Gainesville, Florida 32608
United StatesSite Not Available
University Research Center of South Florida
Tampa, Florida 33612
United StatesSite Not Available
University of Florida
Gainesville 4156404, Florida 4155751 32608
United StatesSite Not Available
University Research Center of South Florida
Tampa 4174757, Florida 4155751 33612
United StatesSite Not Available
Indiana University (IU)
Indianapolis, Indiana 46202
United StatesSite Not Available
Indiana University (IU)
Indianapolis 4259418, Indiana 4921868 46202
United StatesSite Not Available
Kansas University Medical Center
Kansas City, Kansas 66205
United StatesSite Not Available
Kansas University Medical Center
Kansas City 4273837, Kansas 4273857 66205
United StatesSite Not Available
Kennedy Krieger Institute
Baltimore, Maryland 21205
United StatesSite Not Available
Kennedy Krieger Institute
Baltimore 4347778, Maryland 4361885 21205
United StatesSite Not Available
Beth Israel Deaconess Medical Center
Boston, Massachusetts 02215
United StatesSite Not Available
Beth Israel Deaconess Medical Center
Boston 4930956, Massachusetts 6254926 02215
United StatesSite Not Available
University of Minnesota
Minneapolis, Minnesota 55455
United StatesSite Not Available
University of Minnesota
Minneapolis 5037649, Minnesota 5037779 55455
United StatesSite Not Available
University of Rochester Medical Center
Rochester, New York 14642
United StatesSite Not Available
University of Rochester Medical Center
Rochester 5134086, New York 5128638 14642
United StatesSite Not Available
Duke University Medical Center
Durham, North Carolina 27708
United StatesSite Not Available
Wake Forest
Winston-Salem, North Carolina 27157
United StatesSite Not Available
Duke University Medical Center
Durham 4464368, North Carolina 4482348 27708
United StatesSite Not Available
Wake Forest
Winston-Salem 4499612, North Carolina 4482348 27157
United StatesSite Not Available
University of Cincinnati Gardner Neuroscience Institute
Cincinnati, Ohio 45219
United StatesSite Not Available
Ohio State University
Columbus, Ohio 43221
United StatesSite Not Available
University of Cincinnati Gardner Neuroscience Institute
Cincinnati 4508722, Ohio 5165418 45219
United StatesSite Not Available
Ohio State University
Columbus 4509177, Ohio 5165418 43221
United StatesSite Not Available
University of Pennsylvania
Philadelphia, Pennsylvania 19104
United StatesSite Not Available
University of Pennsylvania
Philadelphia 4560349, Pennsylvania 6254927 19104
United StatesSite Not Available
University of Texas Southwestern Medical Center
Dallas, Texas 75390
United StatesSite Not Available
Houston Methodist Neurological Institute
Houston, Texas 77030
United StatesSite Not Available
Houston Methodist Neurological Institute
Houston 4699066, Texas 4736286 77030
United StatesSite Not Available
Virginia Commonwealth University
Richmond, Virginia 23298
United StatesSite Not Available
Virginia Commonwealth University
Richmond 4781708, Virginia 6254928 23298
United StatesSite Not Available
University of Washington
Seattle, Washington 98104
United StatesSite Not Available
University of Washington
Seattle 5809844, Washington 5815135 98104
United StatesSite Not Available

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