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

• Register: ClinicalTrials.gov
• Last refreshed on: 28 August 2023
• Main ID: NCT04126473
• Date of registration: 16/08/2019
• Prospective Registration: Yes
• Primary sponsor: Eloxx Pharmaceuticals, Inc.
• Public title: A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
• Scientific 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
• Date of first enrolment: November 5, 2019
• Target sample size: 17
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/ct2/show/NCT04126473
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 2

Countries of recruitment

Australia, Germany, Israel

Key inclusion & exclusion criteria

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 or phenotypically similar
nonsense mutation, homozygote, or compound heterozygote with one of the specified
mutations. For heterozygotes, one mutation has to be G542X or phenotypically similar
nonsense mutation, and the second mutation could be and Class 1 or Class 2 mutation.
Patients with one G542X or phenotypically similar nonsense 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



Age minimum: 16 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Cystic Fibrosis

Intervention(s)

Drug: Ivacaftor

Drug: ELX-02

Primary Outcome(s)

Peak observed plasma concentration (Cpeak) over time [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 at 30 min and 1 hour post-dose]

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]

Trough observed plasma concentrations (Cpredose) over time [Time Frame: Days 1, 2 and 7 of treatment periods 1-3, Days 1, 2, 7 and 14 of treatment period 4, sparse blood sampling at pre-dose]

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]

Maximum observed plasma concentration (Cmax) on Day 1 [Time Frame: Day 1 of treatment periods 1, 2, 3, and 4]

Secondary Outcome(s)

Changes from baseline in percent predicted forced vital capacity (ppFVC) [Time Frame: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4]

Changes from baseline in sweat chloride concentration [Time Frame: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4]

Changes from baseline in percent predicted forced expiratory flow at 25-75% (ppFEF25-75) [Time Frame: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4]

Changes from baseline in percent predicted forced expiratory volume (ppFEV1) [Time Frame: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4]

Secondary ID(s)

EL-004

Source(s) of Monetary Support

Please refer to primary and secondary sponsors