Healthy volunteers Other
Conditions
Interventions
This study will consist of a double-blind, randomized, placebo-controlled, single ascending dose design in healthy male volunteers. Approximately 32 participants will be enrolled in the study. Single
Sponsors
Entrada Therapeutics, Inc.
Eligibility
Sex/Gender
Male
Inclusion criteria
Inclusion criteria: 1. Male participants aged 18 to 55 years, inclusive, at Screening. 2. Body mass index (BMI) within the range of 18.0 to 32.0 kg/m², inclusive, and a minimum weight of 50.0 kg at Screening. 3. Healthy, in the opinion of the Investigator, based on medical history, physical examination, vital signs, and 12-lead ECG at Screening. 4. Clinical laboratory test results within the laboratory reference range at Screening; or, if out of range, were not clinically relevant and were acceptable to the Investigator. 5. Negative for drugs of abuse and alcohol tests at Screening and at admission on Day -1. 6. Participants with female partners who are of childbearing-potential must agree to use a highly effective method of contraception (e.g., hormonal contraception) for the duration of the study. 7. Participants able to speak, understand, read, and write English fluently to understand the nature of the study, provide written informed consent, and allow for the completion of all study assessments. 8. Capable of giving signed informed consent which includes compliance with the requirements and restrictions listed in the informed consent form (ICF) and in the protocol. 9. Participants willing and able to abide by all study requirements and restrictions.
Exclusion criteria
Exclusion criteria: 1. Current or prior history of any of the following: a. Any illness or medical disorder or currently under evaluation for an illness or medical disorder. b. Difficulty with blood collection and/or poor venous access for the purposes of phlebotomy. c. History of solid organ transplantation. d. Malignancy within the 5 years prior to Screening with the exception of specific cancers that are cured by surgical resection (e.g., basal cell skin cancer). Participants under evaluation for possible malignancy are not eligible. e. History of significant drug allergy, such as anaphylaxis. f. History of renal disease, including recurrent urinary tract infections. g. Any personal or first-degree family history of genetic kidney disease. 2. Screening ECG with clinically significant abnormalities (arrhythmias, PR > 220 msec, QRS complex > 120 msec, QT interval corrected for heart rate using Fridericia’s method [QTcF] > 450 msec). a. If the ECG is out of range, the Investigator may obtain 2 additional triplicate readings, so that up to 3 consecutive assessments are made. If the ECG data are out of range at each of these 3 consecutive assessments or based on the mean of the 3 readings, the participant is not eligible for study participation. 3. Known family history or known presence of long QT syndrome. 4. Clinically relevant hypertension (systolic blood pressure [BP] > 140 mmHg and/or diastolic BP > 90 mmHg, and heart rate > 100 bpm) at the time of Screening. a. BP may be repeated as a triplicate up to 2 additional times. If individual values are outside the reference range at each of these 3 consecutive assessments or based on the mean of the 3 readings, the participant is not eligible for study participation. 5. eGFR 6.5%) at the time of Screening. 7. Positive for hepatitis B surface antigen (HBsAg), hepatitis C virus (HCV) antibody, or human immunodeficiency virus (HIV)-1 and -2 tests at Screening. 8. A positive COVID-19 test at the time of Screening or Day -1. 9. Participants receiving any prescription, over-the-counter, or herbal medications in the 14 days prior to Day -1. 10. Participants who have received any agents with nephrotoxic potential in the 30 days prior to Day -1. 11. Participants that used an investigational drug in any clinical trial within 30 days or 5 half-lives, whichever is longer, of Day 1. 12. Participants that have previously received exon skipping therapy. 13. Participants with prolonged coagulation times at the time of Screening, and/or participants with any contraindications to needle muscle biopsy, including history of keloid scarring, bleeding disorders, use of anticoagulants, or routine use of aspirin per the Investigator’s discretion.
Design outcomes
Primary
| Measure | Time frame |
|---|---|
| 1. Incidence and severity of treatment-emergent adverse events (TEAEs) and serious adverse events (SAEs) at Day 1 to end of study 2. Incidence of abnormalities in laboratory parameters, electrocardiogram (ECG) parameters, vital sign measurements, and physical examinations at Day 1 to end of study | — |
Secondary
| Measure | Time frame |
|---|---|
| 1. Plasma PK of ENTR-601-44 and PMO-0235a including but not limited to: maximum concentration (Cmax,) area under the curve (AUClast), and the half-life (t½) at Day 1 to end of study 2. Amounts of ENTR-601-44 and PMO-0235a excreted in urine at Day 1 to end of study | — |
Countries
England, United Kingdom
Contacts
Public ContactProject Management Department
Outcome results
None listed