Cystic FibrosisPhase 2NCT04126473
A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele
A Phase 2 study testing ELX-02 and Ivacaftor in people with Cystic Fibrosis, run by Eloxx Pharmaceuticals, Inc. at 9 sites in 3 countries.
Trial summary
This is a Phase 2 open label 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 or phenotypically similar nonsense allele. Read more about this study
- Who is it for?
- Ages 16 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
- 9 sitesin 3 countries
- Study dates
- 5 Nov 2019to 6 Apr 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 16 years and older
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
Patients must meet the following criteria to participate in this study:
- Males and females age 18 years and above in Germany and Israel; in countries where permitted, males and females age 16 years and above
- 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.
- Documented SCC ≥ 60 mEq/L
- 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 any of the following characteristics/conditions will not be included in the study:
- 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 >16
- Patients receiving CFTR modulators within 2 months of study treatment
Trial locations and contacts
9 sites in 3 countries.
9 sites in 3 countries
Israel4 sites
Carmel Medical Center
Haifa
Hadassah Medical Center
Jerusalem
Schneider Children's Medical Center
Petach Tikvah
Safra Children's Hospital - Chaim Sheba Medical Center
Ramat Gan
Australia3 sites
The Royal Adelaide Hospital
Adelaide, South Australia
The Royal Prince Alfred Hospital
Camperdown, New South Whales, 2050
The Alfred Hospital
Melbourne, Victoria
Germany2 sites
Universitätsmedizin Essen Ruhrlandklinik
Essen, North Rhine-Westphalia, 45239
Universitätsklinikum Frankfurt
Frankfurt
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 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 or phenotypically similar nonsense allele.
Up to 16 patients will be enrolled in the trial; up 4 patients will be homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X or phenotypically similar nonsense mutation and any Class 1 or Class 2 mutation.
Read more
Each patient will receive 5 escalating doses as follows:
- 0.3 mg/kg per day SC
- 0.75 mg/kg per day SC
- 1.5 mg/kg per day SC
- An individualized dose, as high as 3.0 mg/kg per day SC, based upon 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
- aminoglycoside, Nonsense Mutation, Translational read through
- 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 eukaryotic 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 (AUC0-24)
Full PK profile 8 blood samples up to 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 sampling, blood sampling at 30 min and 1 hour post-dose
Trough observed plasma concentrations (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 blood 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 posted15 Oct 2019
- Study start5 Nov 2019
- Primary completion6 Apr 2022
- Study completion6 Apr 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
- NCT04126473
- Sponsor's protocol ID
- EL-004
- Status last verified
- Jul 2021
- First submitted
- 16 Aug 2019
- Last updated
- 21 Aug 2023
- Results posted
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 21 Aug 2023. 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
- NCT04126473
- Acronym / sponsor ID
- EL-004
- Conditions
- Cystic Fibrosis
- Keywords
- aminoglycoside, Nonsense Mutation, Translational read through
- Phase
- Phase 2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 17 participants (actual)
- Lead sponsor
- Eloxx Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Study start
- 5 Nov 2019 (actual)
- Primary completion
- 6 Apr 2022 (actual)
- Study completion
- 6 Apr 2022 (actual)
- First posted
- 15 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 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 or phenotypically similar nonsense allele.
Up to 16 patients will be enrolled in the trial; up 4 patients will be homozygotes to G542X, and the remaining patients will be compound heterozygotes with G542X or phenotypically similar nonsense mutation and any Class 1 or Class 2 mutation.
Each patient will receive 5 escalating doses as follows:
- 0.3 mg/kg per day SC
- 0.75 mg/kg per day SC
- 1.5 mg/kg per day SC
- An individualized dose, as high as 3.0 mg/kg per day SC, based upon 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
- 16 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
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 eukaryotic 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 (AUC0-24)
Time frame: Day 1 of treatment periods 1, 2, 3, and 4
Full PK profile 8 blood samples up to 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 sampling, blood sampling at 30 min and 1 hour post-dose
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
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 21 Aug 2023.
- Eloxx Pharmaceuticals WebsiteLink provided by the sponsor.
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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.
9 sites in 3 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 9 sites in 3 countries.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
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 21 Aug 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 9 sites in Israel, Australia, Germany. 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 21 Aug 2023.