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A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With 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: NCT04135495
Recruitment Status : Recruiting
First Posted : October 22, 2019
Last Update Posted : September 29, 2020
Sponsor:
Information provided by (Responsible Party):
Eloxx Pharmaceuticals, Inc.

Brief Summary:

This is a Phase 2 open-label, dose-escalation 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.

In total, up to eight patients will be enrolled in the trial; up to 4 patients will be homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X and with 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 on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests.

Condition or disease Intervention/treatment Phase
Cystic Fibrosis Drug: ELX-02 Phase 2

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Study Type : Interventional  (Clinical Trial)
Estimated Enrollment : 8 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: 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
Actual Study Start Date : November 25, 2019
Estimated Primary Completion Date : January 15, 2021
Estimated Study Completion Date : February 15, 2021

Resource links provided by the National Library of Medicine

MedlinePlus related topics: Cystic Fibrosis

Arm Intervention/treatment
Experimental: ELX-02
Eukaryotic ribosomal selective glycoside (ERSG)
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 eukaroyotic ribosomal selective glycoside (ERSG)




Primary Outcome Measures :
  1. 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 ]
  2. Area under the plasma concentration curve from time zero to 24 hours (AUC0-24h) [ Time Frame: Day 1 of treatment periods 1, 2, 3, and 4 ]
    Full PK profile 12 blood samples over 24 hours

  3. Maximum observed plasma concentration (Cmax) [ Time Frame: Day 1 of treatment periods 1, 2, 3, and 4 ]
    Full PK profile 12 blood samples over 24 hours


Secondary Outcome Measures :
  1. 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

  2. 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 ]
  3. 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 ]
  4. 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 ]
  5. Changes in baseline sweat chloride concentration [ Time Frame: Screening, Day 7 of treatment periods 1-3, and Day 1, 7 and 14 of treatment period 4, and 4 weeks following last dose ]


Information from the National Library of Medicine

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, Learn About Clinical Studies.


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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No
Criteria

Inclusion Criteria:

  1. Males and females age 18 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 has to be any Class 1 or Class 2 mutation excluding F508del. Patients with one G542X allele and a second allele that is not a Class 1 or Class 2 mutation 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
  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 a lower BMI may be entered into the study at the discretion of the investigator following consultation with the Sponsor.

Exclusion Criteria:

  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 must be >16.
  8. Patients receiving CFTR modulators within 2 months of study treatment

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04135495


Contacts
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Contact: Eloxx Pharmaceuticals 7815775300 CTI@eloxxpharma.com

Locations
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United States, California
Long Beach Memorial Recruiting
Long Beach, California, United States, 90806
Contact: Jimmy Johannes, MD    614-722-4756    Terri.Johnson@nationwidechildrens.org   
Contact: Candice Evans    562-933-5607    cevans@memorialcare.org   
Principal Investigator: Jimmy Johannes, MD         
Stanford School of Medicine Recruiting
Palo Alto, California, United States, 94305
Contact: Carlos Milla, MD    650-493-5193    cmilla@stanford.edu   
Contact: Monica Elazar    650 - 493-5193    monica8@stanford.edu   
Principal Investigator: Carlos Milla, MD         
United States, Colorado
National Jewish Health Recruiting
Denver, Colorado, United States, 80206
Contact: Jennifer Taylor-Cousar, MD    303-270-2764    taylor-cousarj@njhealth.org   
Contact: Alexandra Wilson    303-270-2517    wilsona@njhealth.org   
Principal Investigator: Jennifer Taylor-Cousar, MD         
United States, Maryland
Johns Hopkins Active, not recruiting
Baltimore, Maryland, United States, 21287
United States, Massachusetts
Boston Children's Hospital Recruiting
Boston, Massachusetts, United States, 02451
Contact: Ahmet Uluer, MD    617-355-1834    ahmet.uluer@childrens.harvard.edu   
Contact: Julia Giancola    617-355-6665    Julia.Giancola@childrens.harvard.edu   
United States, Ohio
Nationwide Children's Hospital Recruiting
Columbus, Ohio, United States, 43205
Contact: Karen McCoy, MD    614-722-4766    Karen.McCoy@nationwidechildrens.org   
Contact: Terri Johnson    614-722-4756    Terri.Johnson@nationwidechildrens.org   
Principal Investigator: Karen McCoy, MD         
United States, Texas
Baylor College of Medicine Recruiting
Houston, Texas, United States, 77030
Contact: Tara Barto, MD    832-824-1508    tara.barto@bcm.edu   
Contact: Nicoline Schaap    832-822-4252    nxschaap@texaschildrens.org   
Principal Investigator: Tara Barto, MD         
Sponsors and Collaborators
Eloxx Pharmaceuticals, Inc.
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Responsible Party: Eloxx Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT04135495    
Other Study ID Numbers: EL-012
First Posted: October 22, 2019    Key Record Dates
Last Update Posted: September 29, 2020
Last Verified: September 2020

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Eloxx Pharmaceuticals, Inc.:
cystic fibrosis
ELX-02
G542X allele
eukaryotic ribosomal selective glycoside
Additional relevant MeSH terms:
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Cystic Fibrosis
Fibrosis
Pathologic Processes
Pancreatic Diseases
Digestive System Diseases
Lung Diseases
Respiratory Tract Diseases
Genetic Diseases, Inborn
Infant, Newborn, Diseases