Myotonic dystrophy (DM1) Nervous System Diseases
Conditions
Interventions
DT-818 will be administered in an open-label fashion to healthy participants (Cohort 1) and then to participants with myotonic dystrophy type 1 (DM1, Cohorts 2-6). Each sequential Cohort will receive
Sponsors
Design Therapeutics, Inc.
Eligibility
Sex/Gender
All
Age
18 Years to 65 Years
Inclusion criteria
Inclusion criteria: Healthy Volunteers (Cohort 1) 1. Male or female participants, 18–55 years of age, inclusive. 2. Medically healthy with no clinically significant abnormalities based on medical history, physical exam, vital signs, ECG, and clinical laboratory tests at screening. 3. Negative tests for drugs of abuse, alcohol, hepatitis B surface antigen, hepatitis C antibody, and HIV. 4. Willing and able to comply with study restrictions and provide written informed consent. 5. Women of childbearing potential must use highly effective contraception from screening through the end of the study; men must use contraception and refrain from sperm donation for the same period. DM1 Participants (Cohort 2 and beyond) 1. Adults 18–65 years of age with genetically confirmed Myotonic Dystrophy Type 1 (DM1). 2. Stable clinical status and medications for =1 month prior to screening. 3. Able and willing to comply with study visits, assessments, and treatment requirements. 4. Provides written informed consent (and caregiver consent, if applicable).
Exclusion criteria
Exclusion criteria: 1. History or presence of any clinically significant cardiovascular, hepatic, renal, gastrointestinal, respiratory, hematologic, or neurologic disease (other than DM1 for patient cohorts). 2. Clinically significant ECG findings per protocol defined limits 3. Abnormal laboratory values at screening or per protocol-defined laboratory limits. 4. Participation in another investigational drug or device study within 90 days prior to dosing. 5. Positive test for alcohol or drugs of abuse at screening or admission. 6. Pregnant or breastfeeding women. 7. Any condition that, in the opinion of the investigator, would make the participant unsuitable for the study.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| Safety of DT-818 measured using data collected from electronic Case Report Forms (CRF), including incidence of Treatment Emergent Adverse Events (TEAEs), including Serious Adverse Events (SAEs), from enrollment to end of study | — |
Secondary
| Measure | Time frame |
|---|---|
| Characterization of the PK profile of DT-818 measured using plasma PK parameters, including but not limited to AUClast, AUCinf, Cmax, t1/2, from first dose to end of study, from enrollment to end of study | — |
Countries
Australia, Canada, New Zealand
Contacts
Public ContactDesign Therapeutics Study Manager
Outcome results
None listed