Cystic FibrosisPhase 2NCT04135495
A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele
A Phase 2 study testing ELX-02 and Ivacaftor in people with Cystic Fibrosis, run by Eloxx Pharmaceuticals, Inc. at 10 sites in 2 countries.
Trial 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 with at least one G542X allele. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- ELX-02 and Ivacaftor
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 17 participants
- Where
- 10 sitesin 2 countries
- Study dates
- 25 Nov 2019to 3 Oct 2022
- Phase
- Phase 2
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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
Trial locations and contacts
10 sites in 2 countries.
10 sites in 2 countries
United States7 sites
Johns Hopkins
Baltimore, Maryland, 21287
Boston Children's Hospital
Boston, Massachusetts, 02451
Nationwide Children's Hospital
Columbus, Ohio, 43205
National Jewish Health
Denver, Colorado, 80206
Baylor College of Medicine
Houston, Texas, 77030
Long Beach Memorial
Long Beach, California, 90806
Stanford School of Medicine
Palo Alto, California, 94305
Canada3 sites
Foothills Hospital Calgary (University of Calgary)
Calgary, Alberta, T2N 4N1
The University of Montreal Health Centre
Montreal, Quebec, H2X0A9
St. Michael's Hospital
Toronto, Ontario, M5B-1W8
About this study
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
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.
Read more
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
- Conditions
- Cystic Fibrosis
- Being tested
- ELX-02, Ivacaftor
- Keywords
- cystic fibrosis, ELX-02, G542X allele, eukaryotic ribosomal selective glycoside
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
ELX-02Drug
ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaroyotic ribosomal selective glycoside (ERSG)
IvacaftorDrug
CFTR potentiator
| Study group | Role | What they receive |
|---|---|---|
| ELX-02 | Experimental | Eukaryotic ribosomal selective glycoside (ERSG) Receives: Drug: ELX-02; Drug: Ivacaftor |
What is being measured
The primary outcome is the main question the study is designed to answer.
AEs associated with different dose levels of ELX-02
Measured: From the time of first dosing through the follow-up visit, an average of approximately 9 weeks
Area under the plasma concentration curve from time zero to 24 hours (AUC0-24h)
Full PK profile 8 blood samples over 24 hours
Measured: Day 1 of treatment periods 1, 2, 3, and 4
Maximum observed plasma concentration (Cmax) on Day 1
Full PK profile 8 blood samples over 24 hours
Measured: Day 1 of treatment periods 1, 2, 3, and 4
Peak observed plasma concentration (Cpeak) over time
Measured: 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
Trough observed plasma concentration (Cpredose) over time
Measured: Days 1, 2 and 7 of treatment periods 1-3, Days 1, 2, 7 and 14 of treatment period 4, sparse sampling at pre-dose
Secondary outcomes (4)
Changes from baseline in sweat chloride concentration
Measured: 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)
Measured: 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 vital capacity (ppFVC)
Measured: 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)
Measured: From baseline to Day 7 of treatment periods 1-3, and Days 7 and 14 of treatment period 4
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted22 Oct 2019
- Study start25 Nov 2019
- Primary completion3 Oct 2022
- Study completion3 Oct 2022
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 2
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 17 participants (actual)
Sponsor and investigators
- Lead sponsor
- Eloxx Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04135495
- Sponsor's protocol ID
- EL-012
- Status last verified
- Feb 2022
- First submitted
- 16 Aug 2019
- Last updated
- 22 Nov 2022
- Results posted
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 22 Nov 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04135495
- Acronym / sponsor ID
- EL-012
- Conditions
- Cystic Fibrosis
- Keywords
- cystic fibrosis, ELX-02, G542X allele, eukaryotic ribosomal selective glycoside
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Eloxx Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 25 Nov 2019 (actual)
- Primary completion
- 3 Oct 2022 (actual)
- Study completion
- 3 Oct 2022 (actual)
- First posted
- 22 Oct 2019 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| ELX-02 | Experimental | Drug: ELX-02, Drug: IvacaftorEukaryotic ribosomal selective glycoside (ERSG) |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (5 listed)
- 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.
Key exclusion criteria (8 listed)
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| ELX-02ELX-02 is a small molecule, new chemical entity being developed for the treatment of genetic diseases caused by nonsense mutations. ELX-02 is a eukaroyotic ribosomal selective glycoside (ERSG) | Drug | ELX-02 |
| IvacaftorCFTR potentiator | Drug | ELX-02 |
Primary outcome
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
Area under the plasma concentration curve from time zero to 24 hours (AUC0-24h)
Time frame: Day 1 of treatment periods 1, 2, 3, and 4
Full PK profile 8 blood samples over 24 hours
Maximum observed plasma concentration (Cmax) on Day 1
Time frame: Day 1 of treatment periods 1, 2, 3, and 4
Full PK profile 8 blood samples over 24 hours
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 blood sampling at 30 min and 1 hour post dose
Trough observed plasma concentration (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 sampling at pre-dose
4 secondary outcomes
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 volume (ppFEV1)
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 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 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
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 22 Nov 2022.
Related trials for Cystic Fibrosis
RecruitingObservational study
Natural History of Bronchiectasis
Bronchiectasis, Cystic Fibrosis
RecruitingPhase 2
Phase 2 Study to Assess the Safety and Efficacy of ANG003
Exocrine Pancreatic Insufficiency (EPI), Cystic Fibrosis (CF)
Recruiting
Diagnostic Value of Oropharyngeal Suction and Effect of Respiratory Physiotherapy on the Detection of Microorganisms in the Pediatric Patient
Cystic Fibrosis (CF), Bronchiectasis, Non-Cystic Fibrosis
Not yet recruitingObservational study
Vascular Health and Function in Cystic Fibrosis
Cystic Fibrosis, Endothelial Dysfunction
RecruitingObservational study
Natural History of Noncirrhotic Portal Hypertension
Cystic Fibrosis, Immunologic Deficiency Syndrome
Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
10 sites in 2 countries: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 2 study testing ELX-02 and Ivacaftor in people with Cystic Fibrosis, run by Eloxx Pharmaceuticals, Inc. at 10 sites in 2 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 8 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 22 Nov 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 10 sites in United States, Canada. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
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). The study lists 5 primary outcomes in all. It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 22 Nov 2022.