Trial record 1 of 2 for:
ELX-02 | Cystic FIbrosis
A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD of ELX-02 in Cystic Fibrosis Patients With G542X Allele
|
The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Know the risks and potential benefits of clinical studies and talk to your health care provider before participating. Read our disclaimer for details. |
| ClinicalTrials.gov Identifier: NCT04135495 |
|
Recruitment Status :
Recruiting
First Posted : October 22, 2019
Last Update Posted : February 16, 2022
|
Sponsor:
Eloxx Pharmaceuticals, Inc.
Information provided by (Responsible Party):
Eloxx Pharmaceuticals, Inc.
- Study Details
- Tabular View
- No Results Posted
- Disclaimer
- How to Read a Study Record
Brief 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.
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
| Condition or disease | Intervention/treatment | Phase |
|---|---|---|
| Cystic Fibrosis | Drug: ELX-02 Drug: Ivacaftor | Phase 2 |
| Study Type : | Interventional (Clinical Trial) |
| Estimated Enrollment : | 16 participants |
| Allocation: | N/A |
| Intervention Model: | Single Group Assignment |
| Masking: | None (Open Label) |
| Primary Purpose: | Treatment |
| 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 |
| Actual Study Start Date : | November 25, 2019 |
| Estimated Primary Completion Date : | July 31, 2022 |
| Estimated Study Completion Date : | July 31, 2022 |
Resource links provided by the National Library of Medicine
MedlinePlus Genetics related topics:
Cystic fibrosis
MedlinePlus related topics:
Cystic Fibrosis
Drug Information available for:
Ivacaftor
| Arm | Intervention/treatment |
|---|---|
|
Experimental: ELX-02
Eukaryotic ribosomal selective glycoside (ERSG)
|
Drug: ELX-02
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) Drug: Ivacaftor CFTR potentiator |
Primary Outcome Measures :
- 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 ]
Secondary Outcome Measures :
- 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 ]
Information from the National Library of Medicine
Choosing to participate in a study is an important personal decision. Talk with your doctor and family members or friends about deciding to join a study. To learn more about this study, you or your doctor may contact the study research staff using the contacts provided below. For general information, Learn About Clinical Studies.
| Ages Eligible for Study: | 18 Years and older (Adult, Older Adult) |
| Sexes Eligible for Study: | All |
| Accepts Healthy Volunteers: | No |
Criteria
Inclusion Criteria:
- 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.
Exclusion Criteria:
- 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
Information from the National Library of Medicine
To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.
Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT04135495
Contacts
| Contact: Eloxx Pharmaceuticals | 7815775300 | CTI@eloxxpharma.com |
Locations
| United States, California | |
| Long Beach Memorial | Recruiting |
| Long Beach, California, United States, 90806 | |
| Contact: Jimmy Johannes, MD 614-722-4756 Terri.Johnson@nationwidechildrens.org | |
| Contact: Candice Evans 562-933-5607 cevans@memorialcare.org | |
| Principal Investigator: Jimmy Johannes, MD | |
| Stanford School of Medicine | Recruiting |
| Palo Alto, California, United States, 94305 | |
| Contact: Carlos Milla, MD 650-493-5193 cmilla@stanford.edu | |
| Contact: Monica Elazar 650 - 493-5193 monica8@stanford.edu | |
| Principal Investigator: Carlos Milla, MD | |
| United States, Colorado | |
| National Jewish Health | Recruiting |
| Denver, Colorado, United States, 80206 | |
| Contact: Jennifer Taylor-Cousar, MD 303-270-2764 taylor-cousarj@njhealth.org | |
| Contact: Alexandra Wilson 303-270-2517 wilsona@njhealth.org | |
| Principal Investigator: Jennifer Taylor-Cousar, MD | |
| United States, Maryland | |
| Johns Hopkins | Recruiting |
| Baltimore, Maryland, United States, 21287 | |
| Contact: Peter Mogayzel, MD 443-287-8983 pmogayze@jhmi.edu | |
| Contact: Britany Zeglin, CCRC 443.287.8983 Bzeglin@jhmi.edu | |
| Principal Investigator: Peter Mogayzel, MD | |
| United States, Massachusetts | |
| Boston Children's Hospital | Recruiting |
| Boston, Massachusetts, United States, 02451 | |
| Contact: Ahmet Uluer, MD 617-355-1834 ahmet.uluer@childrens.harvard.edu | |
| Contact: Julia Giancola 617-355-6665 Julia.Giancola@childrens.harvard.edu | |
| United States, Ohio | |
| Nationwide Children's Hospital | Recruiting |
| Columbus, Ohio, United States, 43205 | |
| Contact: Karen McCoy, MD 614-722-4766 Karen.McCoy@nationwidechildrens.org | |
| Contact: Terri Johnson 614-722-4756 Terri.Johnson@nationwidechildrens.org | |
| Principal Investigator: Karen McCoy, MD | |
| United States, Texas | |
| Baylor College of Medicine | Completed |
| Houston, Texas, United States, 77030 | |
| Canada, Alberta | |
| Foothills Hospital Calgary (University of Calgary) | Recruiting |
| Calgary, Alberta, Canada, T2N 4N1 | |
| Contact: Michael Parkins, MD mdparkin@ucalgary.ca | |
| Contact: Claire Smith 403-944-3694 Clare.Smith@albertahealthservices.ca | |
| Principal Investigator: Michael Parkins, MD | |
| Canada, Ontario | |
| St. Michael's Hospital | Not yet recruiting |
| Toronto, Ontario, Canada, M5B-1W8 | |
| Contact: Elizabeth Tullis, MD Elizabeth.Tullis@unityhealth.to | |
| Contact: Katherine Griffin 416-864-5406 Katherine.Griffin@unityhealth.to | |
| Principal Investigator: Elizabeth Tullis, MD | |
| Canada, Quebec | |
| The University of Montreal Health Centre | Recruiting |
| Montreal, Quebec, Canada, H2X0A9 | |
| Contact: François Tremblay, MD f.tremblay@umontreal.ca | |
| Contact: Nadia Beaudoin 514 890-8000 ext 14635 nadia.beaudoin.chum@ssss.gouv.qc.ca | |
Sponsors and Collaborators
Eloxx Pharmaceuticals, Inc.
| Responsible Party: | Eloxx Pharmaceuticals, Inc. |
| ClinicalTrials.gov Identifier: | NCT04135495 |
| Other Study ID Numbers: |
EL-012 |
| First Posted: | October 22, 2019 Key Record Dates |
| Last Update Posted: | February 16, 2022 |
| Last Verified: | February 2022 |
| Studies a U.S. FDA-regulated Drug Product: | Yes |
| Studies a U.S. FDA-regulated Device Product: | No |
Keywords provided by Eloxx Pharmaceuticals, Inc.:
|
cystic fibrosis ELX-02 G542X allele eukaryotic ribosomal selective glycoside |
Additional relevant MeSH terms:
|
Cystic Fibrosis Fibrosis Pathologic Processes Pancreatic Diseases Digestive System Diseases Lung Diseases Respiratory Tract Diseases |
Genetic Diseases, Inborn Infant, Newborn, Diseases Ivacaftor Chloride Channel Agonists Membrane Transport Modulators Molecular Mechanisms of Pharmacological Action |