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Trial details imported from ClinicalTrials.gov

For full trial details, please see the original record at https://clinicaltrials.gov/study/NCT06565208




Registration number
NCT06565208
Ethics application status
Date submitted
19/08/2024
Date registered
21/08/2024

Titles & IDs
Public title
First in Human SAD/MAD Safety and PK Study with Adult DMD Safety and PK Cohort
Scientific title
Phase 1, Randomized, Double-blind, Placebo-controlled, Staggered, Parallel, Single and Multiple Ascending Dose and Food Effect Study to Evaluate the Safety and PK of Oral SAT-3247 in Healthy Volunteers and Participants with DMD
Secondary ID [1] 0 0
SAT-3247-CL-101
Universal Trial Number (UTN)
Trial acronym
Linked study record

Health condition
Health condition(s) or problem(s) studied:
Duchenne Muscular Dystrophy 0 0
Condition category
Condition code
Musculoskeletal 0 0 0 0
Other muscular and skeletal disorders
Human Genetics and Inherited Disorders 0 0 0 0
Other human genetics and inherited disorders
Neurological 0 0 0 0
Other neurological disorders

Intervention/exposure
Study type
Interventional
Description of intervention(s) / exposure
Treatment: Drugs - SAT-3247
Treatment: Drugs - matched placebo

Experimental: SAT-3247 - 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

Placebo comparator: Placebo - 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


Treatment: Drugs: SAT-3247
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.

Treatment: Drugs: matched placebo
matched placebo
Intervention code [1] 0 0
Treatment: Drugs
Comparator / control treatment
Control group

Outcomes
Primary outcome [1] 0 0
Incidence and severity of treatment emergent adverse events
Timepoint [1] 0 0
Part A: Day 1-3; Part B: Day 1-8; Part C: Day 1-3; Part D: Day 1-28
Secondary outcome [1] 0 0
Serum plasma Pharmacokinetics of SAT-3247
Timepoint [1] 0 0
Part A: Day 1-3; Part B: Day 1-8; Part C: Day 1-3; Part D: Day 1-28

Eligibility
Key inclusion criteria
Parts A-C enroll healthy volunteers; only entry criteria for Part D are described below.

Part D

* 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.

Part D
Minimum age
18 Years
Maximum age
40 Years
Sex
Males
Can healthy volunteers participate?
Yes
Key exclusion criteria
* 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).
* Infections requiring parenteral antibiotics within 6 months prior to Screening.
* Positive test for hepatitis C antibody (HCV), hepatitis B surface antigen (HBsAg), human immunodeficiency virus (HIV) antibody.
* Vaccination with a vaccine within 28 days prior to the first administration of IP.
* Creatine kinase > ULN or ALP, AST, bilirubin, and/or ALT >1.5 × ULN at Screening.
* Anticipated change to prescription medication, over the counter medications, vitamins, supplements, and/or herbal remedies during the course of the trial.
* Anything that the PI or designee considers would jeopardize the safety of the participant, prevent complete participation in the study (including the possibility that the participant will not cooperate with the requirements of the protocol) or compromise interpretation of the study data.
* An employee, consultant, and/or immediate family member (ie, first degree relative, spouse, adoptees, or legal dependents) of the site, Sponsor, or the CRO.

Study design
Purpose of the study
Treatment
Allocation to intervention
Randomised controlled trial
Procedure for enrolling a subject and allocating the treatment (allocation concealment procedures)
Methods used to generate the sequence in which subjects will be randomised (sequence generation)
Masking / blinding
Open (masking not used)
Who is / are masked / blinded?



Intervention assignment
Other
Other design features
Phase
Phase 0
Type of endpoint/s
Statistical methods / analysis

Recruitment
Recruitment status
Recruiting
Data analysis
Reason for early stopping/withdrawal
Other reasons
Date of first participant enrolment
Anticipated
Actual
21/08/2024
Date of last participant enrolment
Anticipated
Actual
Date of last data collection
Anticipated
1/03/2025
Actual
Sample size
Target
82
Accrual to date
Final
Recruitment in Australia
Recruitment state(s)
SA,VIC
Recruitment hospital [1] 0 0
CMAX - Adelaide
Recruitment hospital [2] 0 0
Veritus - Bayswater
Recruitment postcode(s) [1] 0 0
5000 - Adelaide
Recruitment postcode(s) [2] 0 0
3153 - Bayswater

Funding & Sponsors
Primary sponsor type
Commercial sector/industry
Name
Satellos Bioscience, Inc.
Address
Country

Ethics approval
Ethics application status

Summary
Brief summary
Trial website
Trial related presentations / publications
Public notes

Contacts
Principal investigator
Name 0 0
Address 0 0
Country 0 0
Phone 0 0
Fax 0 0
Email 0 0
Contact person for public queries
Name 0 0
Satellos Medical Information
Address 0 0
Country 0 0
Phone 0 0
870887900
Fax 0 0
Email 0 0
medicalinfo@satellos.com
Contact person for scientific queries

Data sharing statement
Will individual participant data (IPD) for this trial be available (including data dictionaries)?
No
No/undecided IPD sharing reason/comment


What supporting documents are/will be available?

No Supporting Document Provided



Results publications and other study-related documents

No documents have been uploaded by study researchers.


Results not provided in https://clinicaltrials.gov/study/NCT06565208