First in Human SAD/MAD Safety and PK Study With Adult DMD Safety and PK Cohort

NCT06565208 | Completed Early Phase 1

• 1 other identifier: SAT-3247-CL-101
• Study Type: interventional
• Target: 77
• Locations: 1 country
• Sites: 2

Brief Summary

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.

Conditions

Duchenne Muscular Dystrophy

Keywords

DMD; muscle regeneration

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• Serum plasma Pharmacokinetics of SAT-3247

  Part A: Day 1-3; Part B: Day 1-8; Part C: Day 1-3; Part D: Day 1-28

Study Arms (2)

SAT-3247

EXPERIMENTAL

SAT-3247 is an oral tablet that is a potent, muscle penetrant, small molecule inhibitor of AAK1; inhibition of AAK1 rescues perturbed asymmetric division of satellite stem cells, resulting in increased muscle regeneration in animal models of DMD. In vitro and in vivo animal pharmacology studies have demonstrated the efficacy of SAT-3247 in improving muscle strength and the necessary target coverage to maximize functional muscle improvement. Part A: Participants will receive one oral dose of SAT-3247 in accord with cohort assignment (1) 10 mg, (2) 50 mg, (3) 150 mg, (4) 300 mg, (5) 400 mg Part B: Participants will receive one oral dose of SAT-3247 daily for seven days in accord with cohort assignment (1) 60 mg, (2) 120 mg, (3)180 mg, (4) 240 mg Part C: Participants will receive one oral dose of SAT-3247 150 mg, following completion of Part A at the same dose Part D: All participants will receive one SAT-3247 60 mg dose once daily for 5 consecutive days of each of 4 weeks

Drug: SAT-3247

Placebo

PLACEBO COMPARATOR

Part A: Participants will receive one oral dose of matched placebo in accord with cohort assignment of (1) 10 mg, (2) 50 mg, (3) 150 mg, (4) 300 mg, (5) 400 mg Part B: Participants will receive one oral dose of matched placebo daily for seven days in accord with cohort assignment (1) 60 mg, (2) 120 mg, (3)180 mg, (4) 240 mg Part C: Participants will receive one oral dose of matched placebo 150 mg, following completion of Part A at the same dose

Drug: matched placebo

Interventions

SAT-3247 DRUG

SAT-3247 is an oral tablet that is a potent, muscle penetrant, small molecule inhibitor of AAK1; inhibition of AAK1 rescues perturbed asymmetric division of satellite stem cells, resulting in increased muscle regeneration in animal models of DMD. In vitro and in vivo animal pharmacology studies have demonstrated the efficacy of SAT-3247 in improving muscle strength and the necessary target coverage to maximize functional muscle improvement.

Also known as: SAT3247, AAK1 inhibitor

SAT-3247

matched placebo DRUG

matched placebo

Also known as: placebo

Placebo

Eligibility Criteria

• Age: 18 Years - 40 Years
• Gender Eligibility Details: adult DMD patients assigned male at birth
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Able and willing to provide written informed consent after the nature of the study has been explained and prior to the commencement of any study procedures.
• Considered reliable and capable of adhering to the protocol and able and willing to attend the necessary visits to the study site according to the judgment of the PI or designee.
• Male patients ≥18 to ≤ 40 years (inclusive at the time of informed consent), or considered an adult able to consent to participate in a clinical study in the jurisdiction in which the study is being conducted.
• Non-smoker and must not have used any tobacco or cannabis products within 2 months prior to Screening.
• Has a definitive diagnosis of DMD based on documented clinical findings and prior genetic testing with a confirmed mutation in the DMD gene.
• BMI ≥ 18.0 kg/m2 and ≤ 32.0 kg/m2 and weight ≥ 50 kg at Screening.
• Stable dose of systemic glucocorticosteroids, heart medications, and/or other supportive medications, vitamins and supplements according to the standard of care for DMD for 3 months prior to the Screening visit and for the duration of the study. Participants that are not receiving glucocorticosteroids are also eligible, but must refrain from initiating glucocorticosteroid treatment for the duration of the trial.
• Agree to abstain from donating blood or blood products during the study and for up to 3 months after the administration of the IP.

You may not qualify if:

• Underlying psychological condition or history of any mental illness that, in the opinion of the PI or designee, would make it unlikely for the participant to comply with the protocol or complete the study per protocol.
• Presence of acute or chronic illness or history of chronic illness sufficient to invalidate the patient's participation in the study or make it unnecessarily hazardous in the judgment of the PI.
• Any clinically significant medical, surgical, or psychiatric abnormality that, in the judgment of the Investigator, is likely to interfere with study compliance, the safe participation of the subject or the assessment of safety or efficacy.
• Any surgical procedures (eg, stomach bypass) or medical condition that might affect absorption of medicines.
• Has donated blood within 60 days of IP administration or donated plasma within 7 days of IP administration or experienced loss of blood ≥500 mL within 2 months of IP administration.
• Fever (body temperature \>38°C) or symptomatic viral or bacterial infection within 2 weeks prior to Screening.
• Poor pill swallowing ability as determined by PI.
• Presence or history of severe allergic or anaphylactic reactions, or sensitivity to the IP or its constituents.
• History of relevant atopy including any confirmed significant allergic reactions against any drug, or multiple drug allergies (non-active hay fever is acceptable).
• History of malignancy, except for non-melanoma skin cancer excised more than 2 years prior to Screening and cervical intraepithelial neoplasia that has been successfully cured more than 5 years prior to Screening.
• Abnormal ECG findings at Screening and or Day -1 that are considered by the PI or designee to be clinically significant.
• QT value, measured at Screening visit, greater than 450 msecs (male) on 12-lead ECG, using Fridericia's formula (QTcF) for correction.
• Pulse ≤ 45 or ≥ 100 beats per minute (bpm); systolic blood pressure ≤ 90 mmHg or ≥ 160 mmHg, or diastolic blood pressure ≤ 50 mmHg or \> 95 mmHg at Screening.
• History or presence of a condition associated with significant immunosuppression.
• History of life-threatening infection (eg, meningitis).

Sponsors & Collaborators

• Satellos Bioscience, Inc.: lead

Study Sites (2)

CMAX

Adelaide, South Australia, 5000, Australia

Location

Veritus

Bayswater, Victoria, 3153, Australia

Location

MeSH Terms

Conditions

Muscular Dystrophy, Duchenne

Condition Hierarchy (Ancestors)

Muscular Dystrophies; Muscular Disorders, Atrophic; Muscular Diseases; Musculoskeletal Diseases; Neuromuscular Diseases; Nervous System Diseases; Genetic Diseases, X-Linked; Genetic Diseases, Inborn; Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: early phase 1
• Allocation: RANDOMIZED
• Masking: NONE
• Masking Details: Participants, Sponsor and Investigator are masked during Parts A-C. Part D is an open-label cohort.
• Purpose: TREATMENT
• Intervention Model: SEQUENTIAL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Model Details: Single ascending dose cohorts and multiple ascending dose cohorts

Study Record Dates

• First Submitted: August 19, 2024
• First Posted: August 21, 2024
• Study Start: August 21, 2024
• Primary Completion: April 28, 2025
• Study Completion: April 28, 2025
• Last Updated: May 15, 2025

Record last verified: 2025-05

Data Sharing

• IPD Sharing: Will not share

Locations

AU (2)