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First in Human SAD/MAD Safety and PK Study with Adult DMD Safety and PK Cohort
This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy adult volunteers (HVs) and adult participants with DMD to determine safety, tolerability, pharmacokinetics and pharmacodynamics.
Study details:
This is a first-in-human (FIH), Phase 1 study of orally administered SAT-3247 in healthy volunteers (HVs) and adult participants with DMD. The study will be conducted in 4 parts. Part A is a Single Ascending Dose (SAD) part that will enroll approximately 40 HVs in up to 5 dose cohorts.
Each participant will receive a single oral dose of SAT 3247 or matched placebo on Day 1; each cohort will receive a higher dose than the prior cohort. Part B is a Multiple Ascending Dose (MAD) part that will enroll approximately 32 HVs in up to 4 sequential dose cohorts. Each participant will receive a daily oral dose of SAT-3247 or matched placebo on Day 1 to Day 7 each cohort will receive a higher dose than the prior cohort.
Part C will assess the effect of food on the PK of SAT-3247 in a fixed sequence, crossover design. The dose to be tested will be determined by the Safety Review Committee (SRC) following review of safety, tolerability, and available PK and PD data from Part A. Approximately 8 healthy participants who completed Part A at the anticipated dose level (in a fasted state) will crossover into a subsequent fed cohort and receive a single dose of the same randomized IP at the same dose level that they received in Part A but, following a high fat meal.
Part D is a open-label Multiple Dose cohort that will comprise 10 adult males with genetically confirmed DMD. Each participant will receive SAT-3247 once daily for 5 consecutive days of each of 4 weeks.
Eligibility criteria
Researchers look for people who fit a certain description, called eligibility criteria. See if you qualify.
Inclusion criteria
Exclusion criteria
Eligibility
Age eligible for study : 18 and older
Healthy volunteers accepted : Yes
Gender eligible for study: Male
Things to know
Study dates
Study start: 2024-08-21
Primary completion: 2025-03-01
Study completion finish: 2025-03-01
Study type
TREATMENT
Phase
EARLY_PHASE1
Trial ID
NCT06565208
Intervention or treatment
DRUG: SAT-3247
DRUG: matched placebo
Conditions
- • Duchenne Muscular Dystrophy
Find a site
Closest Location:
CMAX
Research sites nearby
Select from list below to view details:
CMAX
Adelaide, South Australia, Australia
Veritus
Bayswater, Victoria, Australia
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Participant Group/Arm | Intervention/Treatment |
---|---|
EXPERIMENTAL: SAT-3247
| DRUG: SAT-3247
|
PLACEBO_COMPARATOR: Placebo
| DRUG: matched placebo
|
What is the study measuring?
Primary outcome
Primary Outcome Measure | Primary Outcome Description | Primary Outcome Time Frame |
---|---|---|
Incidence and severity of treatment emergent adverse events | Safety and tolerability of SAT-3247 as compared to placebo | Part A: Day 1-3; Part B: Day 1-8; Part C: Day 1-3; Part D: Day 1-28 |
Secondary outcome
Secondary Outcome Measure | Secondary Outcome Description | Secondary Outcome Time Frame |
---|---|---|
Serum plasma Pharmacokinetics of SAT-3247 | Plasma PK parameters including Cmax, Tmax, AUC, terminal half-life, CL/F, Vz/F, and ratios of Cmax and AUC | Part A: Day 1-3; Part B: Day 1-8; Part C: Day 1-3; Part D: Day 1-28 |
Frequently Asked Questions
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