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Nonsense Mutation Suppressor

ELX-02 for Cystic Fibrosis

Phase 2
Waitlist Available
Research Sponsored by Eloxx Pharmaceuticals, Inc.
Eligibility Criteria Checklist
Specific guidelines that determine who can or cannot participate in a clinical trial
Must have
Be older than 18 years old
Timeline
Screening 3 weeks
Treatment Varies
Follow Up days 1, 2, and 7 of treatment periods 1-3; days 1, 2, 7, and 14 of treatment period 4, sparse blood sampling at 30 min and 1 hour post dose
Awards & highlights

Study Summary

This trial is testing a new drug, ELX-02, to see if it is safe and effective in treating patients with cystic fibrosis (CF). The trial will enroll up to 16 patients, who will receive escalating doses of ELX-02 given by injection under the skin. The trial will also test whether adding the drug ivacaftor to ELX-02 improves its safety and effectiveness.

Eligible Conditions
  • Cystic Fibrosis

Timeline

Screening ~ 3 weeks
Treatment ~ Varies
Follow Up ~days 1, 2, and 7 of treatment periods 1-3; days 1, 2, 7, and 14 of treatment period 4, sparse blood sampling at 30 min and 1 hour post dose
This trial's timeline: 3 weeks for screening, Varies for treatment, and days 1, 2, and 7 of treatment periods 1-3; days 1, 2, 7, and 14 of treatment period 4, sparse blood sampling at 30 min and 1 hour post dose for reporting.

Treatment Details

Study Objectives

Outcome measures can provide a clearer picture of what you can expect from a treatment.
Primary outcome measures
AEs associated with different dose levels of ELX-02
Area under the plasma concentration curve from time zero to 24 hours (AUC0-24h)
Maximum observed plasma concentration (Cmax) on Day 1
+2 more
Secondary outcome measures
Changes from baseline in percent predicted forced expiratory flow at 25-75% (ppFEF25-75)
Changes from baseline in percent predicted forced expiratory volume (ppFEV1)
Changes from baseline in percent predicted forced vital capacity (ppFVC)
+1 more

Side effects data

From 2019 Phase 1 trial • 62 Patients • NCT03309605
83%
Injection site reaction
25%
Injection site induration
17%
Diarrhea
17%
Headache
8%
Skin wound
8%
Injection site pruritus
8%
Ear discomfort
8%
Injection site pain
8%
Gastroenteritis
8%
Nasopharyngitis
8%
Cough
8%
Dysuria
8%
Dry mouth
8%
Erythema
8%
Back pain
100%
80%
60%
40%
20%
0%
Study treatment Arm
Cohort 3 and Cohort 5
Cohort 7
Cohort 1
Cohort 2
Cohort 4 and Cohort 6
All Placebo

Trial Design

1Treatment groups
Experimental Treatment
Group I: ELX-02Experimental Treatment2 Interventions
Eukaryotic ribosomal selective glycoside (ERSG)
Treatment
First Studied
Drug Approval Stage
How many patients have taken this drug
Ivacaftor
2013
Completed Phase 3
~2950
ELX-02
2019
Completed Phase 2
~140

Find a Location

Who is running the clinical trial?

Eloxx Pharmaceuticals, Inc.Lead Sponsor
7 Previous Clinical Trials
174 Total Patients Enrolled
1 Trials studying Cystic Fibrosis
17 Patients Enrolled for Cystic Fibrosis

Frequently Asked Questions

These questions and answers are submitted by anonymous patients, and have not been verified by our internal team.
Recent research and studies
clinicaltrials.govStudy
A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele
2019. "A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele". ClinicalTrials.gov. https://clinicaltrials.gov/ct2/show/NCT04135495.
~3 spots leftby Apr 2025
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