A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele
Public ClinicalTrials.gov record NCT04135495. Field values are reproduced from the official study page; the official ClinicalTrials.gov record remains the source of truth for eligibility, enrollment, and contact information.
Data is sourced from official ClinicalTrials.gov public API records. Always review the official ClinicalTrials.gov record for the latest information.
Official 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
Brief summary
Reproduced verbatim from the official ClinicalTrials.gov record. Not medical advice.
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 with at least one G542X allele. In total, up to 16 patients will be enrolled in the trial; up to 4 patients will be homozygotes for G542X, and the remaining patients will be compound heterozygotes with one G542X or phenotypically similar nonsense allele and any Class 1 or Class 2 mutation. Each patient will receive up to 5 escalating doses as follows: * ELX-02 0.3 mg/kg per day SC * ELX-02 0.75 mg/kg per day SC * ELX-02 1.5 mg/kg per day SC * An individualized dose of ELX-02, as high as 3.0 mg/kg per day SC, based on the patients observed safety and tolerability, PK at previous doses and the results of laboratory tests. * ELX-02 1.5 mg/kg per day SC plus 150 mg ivacaftor every 12 bid
Study identification
- NCT ID
- NCT04135495
- Recruitment status
- Completed
- Study type
- Interventional
- Phase
- Phase 2
- Enrollment
- 17 participants
Conditions and interventions
Conditions
Eligibility (public fields only)
- Age range
- 18 Years and older
- Sex
- All
- Healthy volunteers
- Healthy volunteers not accepted
This page does not interpret eligibility. Detailed inclusion and exclusion criteria are on the official ClinicalTrials.gov record.
Study timeline
- Start date
- Nov 24, 2019
- Primary completion
- Oct 2, 2022
- Completion
- Oct 2, 2022
- Last update posted
- Nov 21, 2022
2019 – 2022
United States locations
- U.S. sites
- 7
- U.S. states
- 6
- U.S. cities
- 7
| Facility | City | State | ZIP | Site status |
|---|---|---|---|---|
| Long Beach Memorial | Long Beach | California | 90806 | — |
| Stanford School of Medicine | Palo Alto | California | 94305 | — |
| National Jewish Health | Denver | Colorado | 80206 | — |
| Johns Hopkins | Baltimore | Maryland | 21287 | — |
| Boston Children's Hospital | Boston | Massachusetts | 02451 | — |
| Nationwide Children's Hospital | Columbus | Ohio | 43205 | — |
| Baylor College of Medicine | Houston | Texas | 77030 | — |
Site contact phone numbers, emails, and investigator names are intentionally not displayed here. Open the official ClinicalTrials.gov record for site contact information.
Non-U.S. locations
This page focuses on the U.S. directory. The official record also lists 3 non-U.S. sites.
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About this trial record page
- What this page shows
- Public field values for ClinicalTrials.gov record NCT04135495, including study identification, conditions, interventions, eligibility (age, sex, healthy volunteer), timeline, and U.S. site list.
- What this page does not do
- No medical advice, eligibility judgments, treatment recommendations, study quality scoring, or AI-generated medical summaries. No site contact phone numbers, emails, or investigator names.
- Where the data comes from
- Sourced from the official ClinicalTrials.gov public API. The official record is the source of truth.
- Last refresh
- Last update posted Nov 21, 2022 · Synced Sep 22, 2026
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Open the official record
The complete protocol, eligibility criteria, and contact information for NCT04135495 live on ClinicalTrials.gov.