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Safety, Tolerability, Pharmacokinetics of ELX-02 in Healthy Adult Volunteers

This study is not yet open for participant recruitment.
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Verified June 2016 by Eloxx Pharmaceuticals, Inc.
Sponsor:
Collaborator:
Cato Research
Information provided by (Responsible Party):
Eloxx Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier:
NCT02807961
First received: June 10, 2016
Last updated: June 20, 2016
Last verified: June 2016
  Purpose
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.

Condition Intervention Phase
Genetic Diseases Nonsense Mutations Drug: ELX-02 Drug: Placebo Phase 1

Study Type: Interventional
Study Design: Allocation: Randomized
Intervention Model: Parallel Assignment
Masking: Double Blind (Participant, Care Provider, Investigator, Outcomes Assessor)
Primary Purpose: Treatment
Official Title: Phase 1a, Randomized, Double-blinded, Placebo-controlled, Single-dose Escalation Study to Evaluate the Safety, Tolerability and Pharmacokinetics of ELX-02 in Healthy Adult Volunteers

Further study details as provided by Eloxx Pharmaceuticals, Inc.:

Primary Outcome Measures:
  • Change from baseline in vitals signs [ Time Frame: 0-10 days ]
  • Change from baseline in chemistry parameters [ Time Frame: 0-10 days ]
  • Change from baseline in hematological parameters [ Time Frame: 0-10 days ]
  • Change from baseline in incidence of adverse events [ Time Frame: 0-10 days ]
  • Change from baseline in parameters of renal function [ Time Frame: 0-10 days ]
  • Change from baseline in parameters of auditory function [ Time Frame: 0-10 days ]
  • Change from baseline in parameters of vestibular function [ Time Frame: 0-10 days ]

Estimated Enrollment: 52
Study Start Date: August 2016
Estimated Study Completion Date: February 2017
Estimated Primary Completion Date: January 2017 (Final data collection date for primary outcome measure)
Arms Assigned Interventions
Placebo Comparator: Placebo
Placebo comparator arm
Drug: Placebo
Placebo comparator
Active Comparator: Active treatment
ELX-02, active comparator
Drug: ELX-02
Synthetic aminoglycoside

  Eligibility

Ages Eligible for Study:   18 Years to 55 Years   (Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   Yes
Criteria

Subjects must meet all of the following criteria to participate in this study.

  1. Be able and willing to provide written Informed Consent indicating that the subject has been informed of all pertinent aspects of the study.
  2. Be willing and able to comply with all scheduled visits, treatment plan, laboratory tests, and other study procedures.
  3. Healthy female subjects and/or male subjects who, at the time of screening, are between the ages of 18 and 55 years, inclusive.

    Healthy is defined as no clinically relevant abnormalities identified by a detailed medical history, full physical examination, including BP and pulse rate measurement, 12-lead electrocardiogram (ECG) and clinical laboratory tests.

  4. Female subjects of non-childbearing potential must meet at least one of the following criteria:

    • Achieved postmenopausal status, defined as follows: 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 follicle-stimulating hormone level;
    • Have undergone a documented hysterectomy and/or bilateral oophorectomy;
    • Have medically confirmed ovarian failure.

    All other female subjects (including females with tubal ligations) will be considered to be of childbearing potential and Female subjects of childbearing potential and partners of male subjects must use highly effective methods of contraception for 14 days before and 28 days after drug administration. These 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.;
    • Female barrier contraceptives (e.g., female condom, diaphragm with a spermicide) plus male condom.
  5. 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 at Screening and Day -1 and agree to use a reliable method of contraception for 14 days before and 28 days after the study. Female subjects of childbearing potential must agree to undergo repeated pregnancy tests.
  6. Male subject must be willing to use an highly effective method of contraception. They must agree to use a condom consistently and correctly, during the course of the study until 28 days after drug administration. These include having undergone a vasectomy or abstain from sexual intercourse.
  7. Be on no medications with potential to impair renal function, e.g., non-steroidal anti-inflammatory drugs, or with ototoxic potential, e.g., quinine or salicylates.
  8. 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.
  9. Normal renal function (glomerular filtration rate >70 mL/min) based on creatinine plasma concentration and the Modification of Diet in Renal Disease equation for estimated glomerular filtration rate.
  10. Negative human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab) serology tests at screening.
  11. No history of alcohol or DOA. Negative urine test for drugs of abuse (DOA) and alcohol breath test at Screening and Day -1.
  12. No personal or familial history (or current) of hearing loss, tinnitus, vertigo, imbalance and unsteadiness.
  13. No findings in baseline audiometry and vestibular assessment tests.
  14. Body Mass Index (BMI) of 19.0 to 30.0 kg/m2 (extremes included); and a total body weight >50 kg (110 lbs).

Subjects who meet any of the following criteria are not eligible for this study:

  1. Subjects who are investigational site staff members directly involved in the conduct of the study and their family members, site staff members otherwise supervised by the Investigator, or subjects who are the Sponsor employees directly involved in the conduct of the study.
  2. Participation in another clinical trial within 6 months prior to dosing (calculated from the previous study's last dosing day). If the previous trial involved agents with delayed effects or prolonged metabolism, a 12 months interval is required.
  3. Evidence or history of clinically relevant hematological, renal, endocrine, pulmonary, gastrointestinal, cardiovascular, hepatic, psychiatric, neurologic, or allergic disease (including drug allergies). This includes any acute or chronic medical or psychiatric condition or laboratory abnormality that may increase the risk associated with study participation or investigational drug administration or may interfere with the interpretation of study results and, in the judgment of the Investigator, would make the subject inappropriate for entry into this study.
  4. A positive urine drug screen (cannabinoids, amphetamines, benzodiazepines, and opiates).
  5. History of regular alcohol consumption exceeding 7 drinks/week for females or 14 drinks/week for males (1 drink = 150 mL [5 ounces] of wine or 360 mL [12 ounces] of beer or 45 mL [1.5 ounces] of hard liquor) within 6 months of Screening.
  6. Screening supine BP ≥140 mm Hg (systolic) or ≥90 mm Hg (diastolic), following at least 5 min of supine rest.
  7. Screening supine 12-lead ECG demonstrating QTcF >450 msec for men and >470 msec for women, or a QRS interval >120 msec.
  8. Subjects with ANY abnormalities in clinical laboratory tests at screening, considered by the study physician as clinically relevant. In particular, subjects with alanine aminotransferase (ALT), aspartate aminotransferase (AST) and total bilirubin ≥1.5 upper limit of normal will be excluded. Abnormal tests may be confirmed by a single repeat.
  9. Pregnant or breastfeeding female subjects; male subjects able to father children and female subjects of childbearing potential who are unwilling or unable to use a highly effective method of contraception as outlined in this protocol for the duration of the study and for at least 14 days before and 28 days after the last dose of study medication.
  10. Use of prescription or nonprescription drugs and dietary supplements within 30 days or 5 half-lives (whichever is longer) prior to the administration of study medication. As an exception, acetaminophen/paracetamol may be used at doses of ≤2 g/day. Limited use of non-prescription medications that are not believed to affect subject safety or the overall results of the study may be permitted on a case-by-case basis following approval by the Sponsor.
  11. Herbal supplements that have not been discontinued at least 30 days prior to administration of the study medication.
  12. Subjects who donated blood or received blood or plasma derivatives in the three months preceding study medication administration.
  13. Unwilling or unable to comply with the restrictions described in this protocol.
  14. Known relevant allergy or hypersensitivity to any drug and/or to any of the excipients of the investigational drug.
  15. Subjects with an inability to communicate well with the Investigator and clinical site staff (e.g., language problem, poor mental development).
  16. Subjects with any acute medical situation (e.g., acute infection) within 48 h of study start, which is considered of significance by the Investigator.
  Contacts and Locations
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the Contacts provided below. For general information, see Learn About Clinical Studies.

Please refer to this study by its ClinicalTrials.gov identifier: NCT02807961

Contacts
Contact: Pedro Huertas, MD, PhD 978.394.5700 pedro@eloxxpharma.com

Sponsors and Collaborators
Eloxx Pharmaceuticals, Inc.
Cato Research
  More Information

Responsible Party: Eloxx Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT02807961     History of Changes
Other Study ID Numbers: EL-001
Study First Received: June 10, 2016
Last Updated: June 20, 2016

Keywords provided by Eloxx Pharmaceuticals, Inc.:
Translational read through

ClinicalTrials.gov processed this record on July 24, 2017