Phase 1 Study of ELX-02 in Healthy Adults

NCT03292302 | Completed Phase 1 DMC

• 1 other identifier: EL-006
• Study Type: interventional
• Target: 18
• Locations: 1 country
• Sites: 1

Brief Summary

Phase 1 Single Ascending Dose Study in Normal Healthy Volunteers

Conditions

Genetic Disease; Nonsense Mutation

Keywords

Translational read through

Outcome Measures

Primary Outcomes (10)

• Adverse Events

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

  0-10 days

• Pharmacokinetics: Maximum plasma concentration (Cmax)

  Cmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

• Pharmacokinetics: Time at which Cmax occurs (tmax)

  tmax will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

• Pharmacokinetics: Area under the plasma concentration-time curve calculated from time of administration to time 24h (AUC24h)

  AUC24h will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

• Pharmacokinetics: Area under the plasma concentration-time curve calculated from time of administration to time 48h (AUC48h)

  AUC48h will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

• Pharmacokinetics: Area under the concentration-time curve from time 0 extrapolated to infinity (AUCinf)

  AUCinf will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

• Pharmacokinetics: Mean residence time (MRT)

  MRT will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

• Pharmacokinetics: Elimination half-life (t1/2)

  t1/2 will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

• Pharmacokinetics: Volume of distribution (Vd/F)

  Vd/F will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

• Pharmacokinetics: Clearance (CL/F)

  CL/F will be determined for ELX-02 and an estimation of ELX-02 dose proportionality of PK parameters.

  0-10 days

Study Arms (2)

Placebo Comparator Arm

PLACEBO COMPARATOR

Placebo

Drug: Placebo

Active treatment

ACTIVE COMPARATOR

ELX-02

Drug: ELX-02

Interventions

Placebo DRUG

Placebo comparator

Placebo Comparator Arm

ELX-02 DRUG

Synthetic aminoglycoside

Active treatment

Eligibility Criteria

• Age: 18 Years - 45 Years
• Sex: all
• Healthy Volunteers: Yes
• Age Groups: Adult (18-64)

You may qualify if:

• Be able and willing to provide written Informed Consent indicating that the subject has been informed of all pertinent aspects of the study.
• Healthy female subjects and 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 (BP) and pulse rate measurement, 12-lead electrocardiogram (ECG), and clinical laboratory tests.
• Female subjects of nonchildbearing 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 may be enrolled if they have negative pregnancy tests at screening and admission day and agree to use a highly effective method of contraception for 14 days before study drug administration and 28 days after study drug administration. Female subjects of childbearing potential must agree to undergo repeated pregnancy tests. For highly effective methods of contraception include see Section 6.5.1.
• Male subjects must be willing to use an effective method of contraception. They must agree to use a condom consistently and correctly, during the course of the study until 28 days after study drug administration.
• Not using any prescription medication and dietary supplements within 30 days or 5 half-lives (whichever is longer) prior to the administration of study drug administration, except for contraceptives - nor be taking any over-the-counter (OTC) herbal or medicinal products. As an exception, acetaminophen/paracetamol may be used at doses of ≤ 2 g/day. Aspirin and non steroidal anti inflammatory drugs (NSAIDs) should not be administered within 1 week of study dose.
• 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.
• Be on no medication with potential to impair renal function (e.g., NSAIDs) or with ototoxic potential (e.g., quinine, salicylates, aminoglycosides).
• Normal renal function (glomular filtration rate \>60 mL/min) based on creatinine plasma concentration and the Modification of Diet in Renal Disease equation for estimated glomular filtration rate. Subjects with lower Modification of Diet in Renal Disease (MDRD) clearance can be included on the condition that they have a normal 24h creatinine clearance (determined by a 24h urine collection).
• Negative human immunodeficiency virus (HIV), hepatitis B surface antigen (HBsAg), hepatitis C virus antibody (HCV Ab) serology tests at screening.
• No history of alcohol or drug of abuse (DOA). Negative urine test for DOA and alcohol breath test at screening and Day -1.
• No personal history (or current) or hereditary hearing loss, persistent tinnitus, persistent vertigo, persistent imbalance, and persistent unsteadiness.

Sponsors & Collaborators

• Eloxx Pharmaceuticals, Inc.: lead
• SGS Life Sciences, a division of SGS Belgium NV: collaborator

Study Sites (1)

SGS Life Sciences, Clinical Pharmacology Unit

Antwerp, Belgium

Location

Related Links

• Eloxx Pharmaceuticals Website

MeSH Terms

Conditions

Genetic Diseases, Inborn

Interventions

ELX-02

Condition Hierarchy (Ancestors)

Congenital, Hereditary, and Neonatal Diseases and Abnormalities

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: RANDOMIZED
• Masking: QUADRUPLE
• Who Masked: PARTICIPANT, CARE PROVIDER, INVESTIGATOR, OUTCOMES ASSESSOR
• Purpose: TREATMENT
• Intervention Model: PARALLEL
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: August 30, 2017
• First Posted: September 25, 2017
• Study Start: September 26, 2017
• Primary Completion: December 15, 2017
• Study Completion: December 15, 2017
• Last Updated: August 22, 2023

Record last verified: 2019-10

Locations

BE (1)