Status:
TERMINATED
A Phase 2 Study of ELX-02 in Patients With Nephropathic Cystinosis
Lead Sponsor:
Eloxx Pharmaceuticals, Inc.
Conditions:
Genetic Disease
Nonsense Mutation
Eligibility:
All Genders
12+ years
Phase:
PHASE2
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...
Eligibility Criteria
Inclusion
- Patients must meet all of the following criteria to participate in this study:
- 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)
- A diagnosis of nephropathic cystinosis and biallelic CTNS mutations, including at least one nonsense mutation
- 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
- 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
- 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:
- 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
- Concomitant use of cysteamine bitartrate from 7 days prior to baseline until 7 days following final administration ELX-02
- 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
- Patients without documented prior aminoglycoside exposure who have a mitochondrial mutation that has been shown to increase sensitivity to aminoglycosides
- Known relevant allergy or hypersensitivity to aminoglycosides
Exclusion
Key Trial Info
Start Date :
August 2 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
December 17 2019
Estimated Enrollment :
3 Patients enrolled
Trial Details
Trial ID
NCT04069260
Start Date
August 2 2019
End Date
December 17 2019
Last Update
May 27 2020
Active Locations (1)
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1
McGill University Health Center
Montreal, Quebec, Canada, H3H 2R9