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A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis

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. Read our disclaimer for details.
 
ClinicalTrials.gov Identifier: NCT04069260
Recruitment Status : Terminated (Due to study design limitations, this study has been discontinued and will not proceed with the 2nd cohort as contemplated in the original protocol.)
First Posted : August 28, 2019
Last Update Posted : May 27, 2020
Sponsor:
Information provided by (Responsible Party):
Eloxx Pharmaceuticals, Inc.

Tracking Information
First Submitted Date  ICMJE August 16, 2019
First Posted Date  ICMJE August 28, 2019
Last Update Posted Date May 27, 2020
Actual Study Start Date  ICMJE August 2, 2019
Actual Primary Completion Date December 17, 2019   (Final data collection date for primary outcome measure)
Current Primary Outcome Measures  ICMJE
 (submitted: August 23, 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 10 weeks ]
  • Area under the plasma concentration curve from time zero to 24 hours (AUC0-24h) [ Time Frame: Day 1 of treatment periods 1, 2, and 3 ]
    Full PK profile 12 blood samples over 24 hours
  • Maximum observed plasma concentration (Cmax) [ Time Frame: Day 1 of treatment periods 1, 2, and 3 ]
    Full PK profile 12 blood samples over 24 hours
  • Observed plasma concentration at 1 hour post dose (C1h) [ Time Frame: Days 1, 2, 5, and 7 of treatment periods 1-2; Days 1, 2, 5, 7, 10, and 14 of treatment period 3 ]
    Sparse sampling, blood sampling only, pre-dose and 1 hour post dose
  • Amount of ELX-02 excreted in urine from 0 to 24 hours (Ae24h) [ Time Frame: Day 1 of treatment periods 1, 2, and 3 ]
    6 urine collections over 24 hours
  • Renal clearance on Day 1 (Ae24h/plasma AUC0-24h) [ Time Frame: Day 1 of treatment periods 1, 2, and 3 ]
    6 urine collections over 24 hours
Original Primary Outcome Measures  ICMJE Same as current
Change History
Current Secondary Outcome Measures  ICMJE
 (submitted: August 23, 2019)
Changes from baseline in WBC cystine levels [ Time Frame: Screening; Days 1, 2, 5, and 7 of treatment periods 1-2; Days 1, 2, 5, 7, 10, and 14 of treatment period 3, and 4-week safety follow-up ]
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 of ELX-02 in Patients With Nephropathic Cystinosis
Official Title  ICMJE A Phase 2, Single Center, Open-Label, Multiple Dose Escalation Study to Evaluate the Safety, Tolerability, Pharmacokinetics, and Pharmacodynamics of Daily Subcutaneously Administered ELX-02 in Patients With Nephropathic Cystinosis Bearing One or More CTNS Gene (Cystinosin) Nonsense Mutations
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 cystinosis with nonsense mutation in at least one allele.

Six patients will be enrolled in the trial.

The study will comprise of the following periods for each patient:

  • A screening period of up to 6 weeks
  • A total treatment period of 4 weeks
  • A safety follow-up period of 4 weeks after the last treatment

Each patient will receive three escalating doses as follows:

  • Treatment period 1: ELX-02 0.5 mg/kg SC daily for 7 days (total dose not to exceed 3.5 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 47.5 µg*h/mL)
  • Treatment period 2: ELX-02 1.0 mg/kg SC daily for 7 days (total dose not to exceed 7.0 mg/kg for this week; the daily dose will be individualized to achieve the target weekly exposure of about 95 µg*h/mL)
  • Treatment period 3: ELX-02 2.0 mg/kg SC daily for 14 days (total dose not to exceed 14 mg/kg for these two weeks; the daily dose will be individualized to achieve the target weekly exposure of about 190 µg*h/mL)
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
  • Genetic Disease
  • Nonsense Mutation
  • Cystinosis
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 Terminated
Actual Enrollment  ICMJE
 (submitted: May 22, 2020)
3
Original Estimated Enrollment  ICMJE
 (submitted: August 23, 2019)
6
Actual Study Completion Date  ICMJE December 17, 2019
Actual Primary Completion Date December 17, 2019   (Final data collection date for primary outcome measure)
Eligibility Criteria  ICMJE

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

  1. Male or female patients who, at the time of screening, are 18 years of age or older (Cohort 1) or ≥12 years of age (Cohort 2)
  2. A diagnosis of nephropathic cystinosis and biallelic CTNS mutations, including at least one nonsense mutation
  3. Patients should have a mild to moderate disease estimated glomerular filtration rate ≥40 mL/min/1.73m2 using the Chronic Kidney Disease Epidemiology Collaboration CKD-EPI formula
  4. Body mass index of 19.0 to 30.0 kg/m2. Patients with a lower BMI may be entered into the study at the discretion of the Investigator following consultation with the Sponsor
  5. Renal transplant permitted with stable graft function (serum creatinine) for 3 months prior to Screening

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. Concomitant use of cysteamine bitartrate from 7 days prior to baseline until 7 days following final administration ELX-02
  3. An average systolic blood pressure and/or diastolic blood pressure ≥95th percentile for sex, age, and height on 3 or more occasions during the screening period
  4. Patients without documented prior aminoglycoside exposure who have a mitochondrial mutation that has been shown to increase sensitivity to aminoglycosides
  5. Known relevant allergy or hypersensitivity to aminoglycosides
Sex/Gender  ICMJE
Sexes Eligible for Study: All
Ages  ICMJE 12 Years and older   (Child, Adult, Older Adult)
Accepts Healthy Volunteers  ICMJE No
Contacts  ICMJE Contact information is only displayed when the study is recruiting subjects
Listed Location Countries  ICMJE Canada
Removed Location Countries  
 
Administrative Information
NCT Number  ICMJE NCT04069260
Other Study ID Numbers  ICMJE EL-003
Has Data Monitoring Committee Not Provided
U.S. FDA-regulated Product
Studies a U.S. FDA-regulated Drug Product: Yes
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
Principal Investigator: Paul Goodyer, MD McGill University
PRS Account Eloxx Pharmaceuticals, Inc.
Verification Date December 2019

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