NCT04126473

A Phase 2 Study to Evaluate the Safety, Tolerability, PK and PD in Cystic Fibrosis Patients With at Least 1 G542X Allele

Completed Phase 2 Last updated 21 August 2023

What this trial tests

Phase 2 trial testing ELX-02 in Cystic Fibrosis in 17 participants. Completed in 6 April 2022.

Timeline

5 November 2019

Primary endpoint
6 April 2022

6 April 2022

Quick facts

Lead sponsor	Eloxx Pharmaceuticals, Inc.
Phase	Phase 2
Status	Completed
Study type	INTERVENTIONAL
Allocation	na
Design	single group
Masking	none
Primary purpose	treatment
Enrollment	17
Start date	5 November 2019
Primary completion	6 April 2022
Estimated completion	6 April 2022
Sites	9 locations across Germany, Israel, Australia

Drugs / interventions tested

• ELX-02 — full drug profile →
• Ivacaftor (IVACAFTOR) — full drug profile →

Conditions studied

• Cystic Fibrosis — all drugs for Cystic Fibrosis →

Sponsor

Eloxx Pharmaceuticals, Inc.

Who can join

16 and older, any sex, with Cystic Fibrosis. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. CFTR Modulators: The Changing Face of Cystic Fibrosis in the Era of Precision Medicine.
   Lopes-Pacheco M. · · 2019 · cited 349× · PMID 32153386 · DOI 10.3389/fphar.2019.01662
2. Nonsense suppression therapies in human genetic diseases.
   Martins-Dias P, Romão L. · · 2021 · cited 70× · PMID 33751142 · DOI 10.1007/s00018-021-03809-7
3. Pharmacological Modulation of Ion Channels for the Treatment of Cystic Fibrosis.
   Pinto MC, Silva IAL, Figueira MF, Amaral MD, et al · · 2021 · cited 37× · PMID 34326672 · DOI 10.2147/jep.s255377
4. One Size Does Not Fit All: The Past, Present and Future of Cystic Fibrosis Causal Therapies.
   Ensinck MM, Carlon MS. · · 2022 · cited 24× · PMID 35740997 · DOI 10.3390/cells11121868
5. Pharmaceuticals Promoting Premature Termination Codon Readthrough: Progress in Development.
   Li S, Li J, Shi W, Nie Z, et al · · 2023 · cited 21× · PMID 37371567 · DOI 10.3390/biom13060988
6. Treatment of Pulmonary Disease of Cystic Fibrosis: A Comprehensive Review.
   Girón Moreno RM, García-Clemente M, Diab-Cáceres L, Martínez-Vergara A, et al · · 2021 · cited 20× · PMID 33922413 · DOI 10.3390/antibiotics10050486
7. NanoLuc reporters identify <i>COL4A5</i> nonsense mutations susceptible to drug-induced stop codon readthrough.
   Omachi K, Kai H, Roberge M, Miner JH. · · 2022 · cited 15× · PMID 35243249 · DOI 10.1016/j.isci.2022.103891
8. Modulation of Ion Transport to Restore Airway Hydration in Cystic Fibrosis.
   Reihill JA, Douglas LEJ, Martin SL. · · 2021 · cited 13× · PMID 33810137 · DOI 10.3390/genes12030453