Working…
COVID-19 is an emerging, rapidly evolving situation.
Get the latest public health information from CDC: https://www.coronavirus.gov.

Get the latest research information from NIH: https://www.nih.gov/coronavirus.
ClinicalTrials.gov
ClinicalTrials.gov Menu

A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04126473
Recruitment Status : Recruiting
First Posted : October 15, 2019
Last Update Posted : November 5, 2020
Sponsor:
Information provided by (Responsible Party):
Eloxx Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE August 16, 2019
First Posted Date  ICMJE October 15, 2019
Last Update Posted Date November 5, 2020
Actual Study Start Date  ICMJE November 5, 2019
Estimated Primary Completion Date May 14, 2021   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: October 28, 2020)
  • AEs associated with different dose levels of ELX-02 [ Time Frame: From the time of first dosing through the follow-up visit, an average of approximately 9 weeks ]
  • Area under the plasma concentration curve from time zero to to 24 hours (AUC0-24) [ Time Frame: Day 1 of treatment periods 1, 2, 3, and 4 ]
    Full PK profile 12 blood samples up to 24 hours
  • Maximum observed plasma concentration (Cmax) [ Time Frame: Day 1 of treatment periods 1, 2, 3, and 4 ]
    Full PK profile 12 plasma samples over 24 hours
Original Primary Outcome Measures  ICMJE
 (submitted: October 11, 2019)
  • AEs associated with different dose levels of ELX-02 [ Time Frame: From the time of first dosing through the follow-up visit, an average of approximately 9 weeks ]
  • Area under the plasma concentration curve from time zero to 24 hours (AUC0-24) [ Time Frame: Day 1 of treatment periods 1, 2, 3, and 4 ]
    Full PK profile 12 blood samples over 24 hours
  • Maximum observed plasma concentration (Cmax) [ Time Frame: Day 1 of treatment periods 1, 2, 3, and 4 ]
    Full PK profile 12 plasma samples over 12 hours
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: October 11, 2019)
  • Observed plasma concentration at 1 hour post dose (C1h) [ Time Frame: Days 1, 2 and 7 of treatment periods 1-3, Days 1, 2, 7, and 14 of treatment period 4 ]
    Sparse sampling, blood sampling only, pre-dose and 1 hour post-dose
  • Changes from baseline in forced expiratory volume (FEV1) [ Time Frame: Screening, Days 1 & 7 of treatment periods 1-3, and Days 1, 7, and 14 of treatment period 4, and 4 weeks following last dose ]
  • Changes from baseline in forced vital capacity (FVC) [ Time Frame: Screening, Days 1 & 7 of treatment periods 1-3, and Days 1, 7, and 14 of treatment period 4, and 4 weeks following last dose ]
  • Changes from baseline in forced expiratory flow at 25-75% (FEF25-75) [ Time Frame: Screening, Days 1 & 7 of treatment periods 1-3, and Days 1, 7, and 14 of treatment period 4, and 4 weeks following last dose ]
  • Changes in baseline sweat chloride concentration [ Time Frame: Screening, Days 1 & 7 of treatment periods 1-3, and Days 1, 7, and 14 of treatment period 4, and 4 weeks following last dose ]
Original Secondary Outcome Measures  ICMJE Same as current
Current Other Pre-specified Outcome Measures Not Provided
Original Other Pre-specified Outcome Measures Not Provided
 
Descriptive Information
Brief Title  ICMJE A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
Official Title  ICMJE A Phase 2 Open Label Study to Evaluate the Safety, Tolerability, Pharmacokinetics and Pharmacodynamics of Multiple Dose Levels of Subcutaneously Administered ELX-02 in Patients With Cystic Fibrosis With at Least One G542X Allele
Brief Summary

This is a Phase 2 open label study to evaluate the safety, tolerability, PK, and PD of multiple dose levels of SC administered ELX-02 in patients with CF with at least one G542X allele.

Up to 16 patients will be enrolled in the trial; up 4 patients will be homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X and any Class 1 or Class 2 mutation excluding F508del.

Each patient will receive 4 escalating doses as follows:

  • 0.3 mg/kg per day SC
  • 0.75 mg/kg per day SC
  • 1.5 mg/kg per day SC
  • An individualized dose, as high as 3.0 mg/kg per day SC, based upon the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests
Detailed Description Not Provided
Study Type  ICMJE Interventional
Study Phase  ICMJE Phase 2
Study Design  ICMJE Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Condition  ICMJE Cystic Fibrosis
Intervention  ICMJE Drug: ELX-02
ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaryotic ribosomal selective glycoside (ERSG).
Study Arms  ICMJE Experimental: ELX-02
Eukaryotic ribosomal selective glycoside (ERSG)
Intervention: Drug: ELX-02
Publications * Not Provided

*   Includes publications given by the data provider as well as publications identified by ClinicalTrials.gov Identifier (NCT Number) in Medline.
 
Recruitment Information
Recruitment Status  ICMJE Recruiting
Estimated Enrollment  ICMJE
 (submitted: October 11, 2019)
16
Original Estimated Enrollment  ICMJE Same as current
Estimated Study Completion Date  ICMJE June 15, 2021
Estimated Primary Completion Date May 14, 2021   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

Patients must meet the following criteria to participate in this study:

  1. Males and females age 18 years and above in Germany and Israel; in countries where permitted, males and females age 16 years and above
  2. A confirmed diagnosis of nmCF with a documented G542X mutation, homozygote, or compound heterozygote with one of the specified mutations. For heterozygotes, one mutation has to be G542X, and the second mutation could be and Class 1 or Class 2 mutation, excluding F508del. Patients with one G542X allele and a second allele that is not in the above list may be potentially allowed but only after discussion on a case by case basis with and written approval from the Sponsor.
  3. Documented SCC ≥ 60 mEq/L
  4. FEV1 ≥ 40% predicted normal for age, gender and height at Screening (Knudson Equation)
  5. Body Mass Index (BMI) of 19.0 to 30.0 kg/m2 (inclusive).

Patients with any of the following characteristics/conditions will not be included in the study:

  1. Participation in clinical study including administration of any investigational drug or device in the last 30 days or 5 half-lives (whichever is longer) prior to investigational product dosing in the current study
  2. History of any organ transplantation
  3. Major surgery within 180 days (6 months) of Screening
  4. Patients without documented prior aminoglycoside exposure who have a mitochondrial mutation that has been shown to increase sensitivity to aminoglycosides
  5. Known allergy to any aminoglycoside
  6. Patients with any abnormality at ENT screening, that indicates the presence of a vestibular toxicity associated with prior exposure to aminoglycosides.
  7. Dizziness Handicap Inventory (DHI)-H score at screening >16
  8. Patients receiving CFTR modulators within 2 months of study treatment
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 16 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE
Contact: Eloxx Pharmaceuticals 1-781-577-5300 CTI@eloxxpharma.com
Listed Location Countries  ICMJE Germany,   Israel
Removed Location Countries Australia
 
Administrative Information
NCT Number  ICMJE NCT04126473
Other Study ID Numbers  ICMJE EL-004
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: No
Studies a U.S. FDA-regulated Device Product: No
IPD Sharing Statement  ICMJE Not Provided
Responsible Party Eloxx Pharmaceuticals, Inc.
Study Sponsor  ICMJE Eloxx Pharmaceuticals, Inc.
Collaborators  ICMJE Not Provided
Investigators  ICMJE Not Provided
PRS Account Eloxx Pharmaceuticals, Inc.
Verification Date November 2020

ICMJE     Data element required by the International Committee of Medical Journal Editors and the World Health Organization ICTRP