Status:
COMPLETED
A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele
Lead Sponsor:
Eloxx Pharmaceuticals, Inc.
Conditions:
Cystic Fibrosis
Eligibility:
All Genders
18+ years
Phase:
PHASE2
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 with and without ivacaftor in patients with CF wi...
Eligibility Criteria
Inclusion
- Males and females age 18 years and above
- 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 or phenotypically similar nonsense allele, and the second mutation has to be any Class 1 or Class 2 mutation. Patients with one G542X allele or phenotypically similar nonsense 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.
- Documented SCC ≥60 mEq
- FEV1 ≥40% predicted normal for age, gender and height at Screening (Knudson Equation)
- 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
- 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
- History of any organ transplantation
- Major surgery within 180 days (6 months) of Screening
- Patients without documented prior aminoglycoside exposure who have a mitochondrial mutation that has been shown to increase sensitivity to aminoglycosides
- Known allergy to any aminoglycoside
- Patients with any abnormality at ENT screening, that indicates the presence of a vestibular toxicity associated with prior exposure to aminoglycosides.
- Dizziness Handicap Inventory (DHI)-H score at screening must be \>16.
- Patients receiving CFTR modulators within 2 months of study treatment
Key Trial Info
Start Date :
November 25 2019
Trial Type :
INTERVENTIONAL
Allocation :
ACTUAL
End Date :
October 3 2022
Estimated Enrollment :
17 Patients enrolled
Trial Details
Trial ID
NCT04135495
Start Date
November 25 2019
End Date
October 3 2022
Last Update
November 22 2022
Active Locations (10)
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1
Long Beach Memorial
Long Beach, California, United States, 90806
2
Stanford School of Medicine
Palo Alto, California, United States, 94305
3
National Jewish Health
Denver, Colorado, United States, 80206
4
Johns Hopkins
Baltimore, Maryland, United States, 21287