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Genetic DiseasesPhase 1NCT02807961

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

A Phase 1 study testing ELX-02 in people with Genetic Diseases and Nonsense Mutations, run by Eloxx Pharmaceuticals, Inc..

Terminated

Stopped early. The study ended before it was planned to and will not resume.

Official record
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Trial summary

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. Read more about this study

Who is it for?
Ages 18 to 45 yearsHealthy volunteers can take part
Conditions the study is for
Genetic Diseases, Nonsense Mutations
What is being tested?
ELX-02
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
42 participants
Where
No sites listed yet
Study dates
Jul 2016to May 2017
Phase
Phase 1
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 to 45 years
  • All sexes
  • Healthy volunteers accepted
  • Adult

Who may be able to join

  • 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.
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  • 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.

Who cannot join

  • Subjects with any of the following characteristics/conditions will not be included in the study:
  • 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.
  • Participation in another clinical trial within 6 months prior to dosing (calculated from the previous study's last doing day). If the previous trial involved agents with delayed effects or prolonged metabolism, a 12 months interval is required.
  • 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 ELX-02 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.
  • A positive urine drug screen (cannabinoids, amphetamines, benzodiazepines, and opiates), at Screening and on admission.
  • History of regular alcohol consumption (by declaration) exceeding 7 drinks/week for females or 14 drinks/week for males (1 drink = 150 mL of wine or 360 mL of beer or 45 mL of hard liquor) within 6 months of Screening. A positive alcohol breath test on admission.
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  • Screening supine blood pressure (BP) ≥ 140 mm Hg (systolic) or ≥ 90 mm Hg (diastolic), following at least 5 minutes of supine rest. If BP exceeds protocol-required limits, BP measurement should be repeated for up to two more times and the last BP value should be used to determine the subject's eligibility.
  • Screening supine 12-lead ECG demonstrating QTc >450 msec for men or >470 msec for women, or a QRS interval >120 msec.
  • Subjects with ANY abnormalities in clinical laboratory tests at Screening, considered by the Investigator as clinically relevant. In particular, subjects with ALT, AST and total bilirubin ≥ 1.5 upper limit of normal will be excluded.
  • Pregnant or breastfeeding female subjects.
  • Use of prescription or nonprescription drugs, vitamins, herbal and/or dietary supplements within 7 days or 5 half-lives (whichever is longer) prior to administration of ELX-02. As an exception, acetaminophen/paracetamol may be used at doses of ≤ 2 g/day. Aspirin and NSAIDS should not be administered within 1 week of study dose. 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.
  • Subjects who donated blood or received blood or plasma derivatives in the 3 months preceding study drug administration.
  • Known allergy or hypersensitivity to any drug and/or to any of the excipients of ELX-02.
  • Subjects with an inability to communicate well with the Investigators and CRC staff (e.g., language problem, poor mental development).
  • Subjects with any acute medical situation (e.g. acute infection) within 48 hours prior to Day 1, which is considered of significance by the Principal Investigator.
  • Subjects with any history of ear disease or surgeries, dizziness or ongoing tinnitus.
  • Subjects with any abnormality on physical examination by an ENT specialist at screening that indicates the presence of a vestibular pathology, conductive hearing loss or balance problem.
  • Subjects with audiometry results as follows: any pure-tone threshold >25dB, inter-ear difference in any frequency of ≥ 20dB, word recognition score (discrimination) <92%.
  • Abnormal ENG/VNG [as indicated by a cumulative response of <12 degrees to caloric testing (response to warm and cold stimulation united) in either ear] or abnormal c/v HIT.
  • DHI-H score >16
  • THI-H score >14

About this study

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

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.

Being tested
ELX-02, Placebo
Keywords
Translational read through
Medical subject headings
Genetic Diseases, Inborn

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • ELX-02Drug

    Synthetic aminoglycoside

  • PlaceboDrug

    Placebo comparator

Study groups in NCT02807961
Study groupRoleWhat they receive
PlaceboPlacebo comparatorPlacebo comparator arm
Receives: Drug: Placebo
Active treatmentActive comparatorELX-02, active comparator
Receives: Drug: ELX-02

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Adverse Events

    Incidence and characteristics of adverse events occurring following single doses of ELX-02

    Measured: 0-10 days

  2. Pharmacokinetics

    The following PK parameters will be calculated based on ELX-02 plasma concentrations: Cmax, Tmax, AUC24h, AUCinf, AUC144h, t½, mean residence time (MRT), volume of distribution (Vd), clearance (CL), absolute bioavailability (F) and an estimation of ELX-02 dose linearity of PK parameters. The following parameters will be calculated based on ELX-02 urine concentrations: urinary mass excretion of ELX-02 (in mass and %dose) and cumulated urinary excretion (in % dose) for each collection interval and total renal clearance. The following parameters will be calculated from the individual urine drug concentrations: Ae, Ae0-12h, Ae12-24h, Ae24-48h, Ae0-48h, fe.

    Measured: 0-10 days

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted21 Jun 2016 (estimated)
  2. Study startJul 2016
  3. Primary completionFeb 2017
  4. Study completionMay 2017

Why it stopped: Transferred study to Antwerp, Belgium

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 1
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Parallel groups
Masking
Quadruple blind
Who is masked
Participant, Care provider, Investigator, Outcomes assessor
Enrolment
42 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02807961
Sponsor's protocol ID
EL-001
Status last verified
Aug 2017
First submitted
10 Jun 2016
Last updated
22 Oct 2019
Results posted
No
Data monitoring committee
Yes

Questions about this trial

What is this clinical trial studying?

A Phase 1 study testing ELX-02 in people with Genetic Diseases and Nonsense Mutations, run by Eloxx Pharmaceuticals, Inc..

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 17 inclusion and 21 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: stopped early, as of the sponsor's last update on 22 Oct 2019. Status can change between updates, so confirm with the study contact before making plans.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. Some participants in this study may receive a placebo. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this phase mean?

Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study was stopped early (reason given: Transferred study to Antwerp, Belgium). Results are not available in the source record.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Adverse Events (time frame: 0-10 days). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02807961.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Oct 2019.

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