Status and phase
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Treatments
About
This is the first study in humans of ELX-02, an advanced synthetic aminoglycoside optimized as a translational read-through drug (TRID) for the treatment of genetic conditions caused by nonsense. mutations. This is a classical Phase 1a study designed as a randomized, double-blinded, placebo-controlled, single dose escalation to evaluate the safety, tolerability and pharmacokinetics of ELX-02 in healthy adult volunteers.
Enrollment
Sex
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Volunteers
Inclusion and exclusion criteria
Inclusion Criteria
Subjects must meet all of the following inclusion criteria to be eligible for enrollment in the study:
Be able and willing to provide written Informed Consent indicating that the subject has been informed of all pertinent aspects of the study.
Be willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.
Healthy female subjects and/or male subjects who, at the time of Screening, are between the ages of 18 and 45 years, inclusive.
Healthy is defined as no clinically relevant abnormalities identified by a detailed medical history, full physical examination, including blood pressure and pulse rate measurements, 12-lead ECG and clinical laboratory tests.
Female subjects of non childbearing potential must meet at least one of the following criteria: Postmenopausal status, defined as: cessation of regular menses for at least 12 consecutive months with no alternative pathological or physiological cause; post-menopausal status will be confirmed by a serum FSH level, Have undergone a documented hysterectomy and/or bilateral oophorectomy, Have medically confirmed ovarian failure Female subjects of non-childbearing potential must agree to undergo pregnancy test at Screening.
All other female subjects (including females with tubal ligations) will be considered to be of childbearing potential and may be enrolled if they have negative pregnancy tests on Screening and admission days and agree to use a reliable method of contraception for two weeks (14 days) before and four weeks (28 days) after dosing.
Female subjects of childbearing potential must agree to undergo repeated pregnancy tests.
Reliable methods of contraception include: Abstention from sexual intercourse, Established use of oral, inserted, injected, implanted or transdermal hormonal methods of contraception (provided the subject plans to remain on the same treatment throughout the entire study and has been using that hormonal contraceptive for an adequate period of time to ensure effectiveness), Copper-containing intrauterine device
Male subjects must be willing to use an effective method of contraception during the course of the study until 28 days after drug administration. These include condom, having undergone a vasectomy or abstain from sexual intercourse.
Be on no medications with potential to impair renal function, e.g., NSAIDs, or with ototoxic potential, e.g., quinine or salicylates.
Non-smoking and no use of any tobacco or nicotine products (by declaration) for a period of at least 6 months prior to Screening visit.
Normal renal function (MDRD GFR > 60 mL/min) based on serum creatinine concentration.
Negative HIV, HBsAg or HCV Ab serology tests at Screening.
No personal history of hearing loss, tinnitus, vertigo, imbalance and unsteadiness.
Normal Screening auditory and vestibular assessments.
Body Mass Index (BMI) of 19.0 to 30.0 Kg/m2.
Exclusion Criteria
Subjects with any of the following characteristics/conditions will not be included in the study:
Primary purpose
Allocation
Interventional model
Masking
42 participants in 2 patient groups, including a placebo group
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Data sourced from clinicaltrials.gov
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