Clinical Trials

Phase 2

Cystic Fibrosis Clinical Trial Program

Our Our Phase 2 cystic fibrosis clinical trial program for ELX-02 is being conducted at leading global investigator sites in Europe, Israel and the United States. In the U.S., the Cystic Fibrosis Foundation (“CF Foundation”) is providing funding for a portion of the trial and we have formed a joint program advisory group with the CF Foundation focused on the development of ELX-02 for cystic fibrosis. In the U.S., the Cystic Fibrosis Therapeutics Development Network (“TDN”) has sanctioned the Phase 2 study protocol which is being conducted at TDN member sites. In Europe, the protocol has been sanctioned by the European Cystic Fibrosis Society Clinical Trial Network which has given our European/Israel trial a “high priority” ranking. The identifiers for these trials are: NCT04126473 and NCT04135495.

Professor Eitan Kerem, M.D., is supporting the global clinical trial as a Senior Medical Consultant to Eloxx and was formerly the Head of the Division of Pediatrics, Children’s Hospital, Hadassah Medical Center in Israel. For the U.S. trial, Dr. Ahmet Uluer, Director of the Adult Cystic Fibrosis Program at the Boston Children’s Hospital/Brigham and Women’s Hospital CF Center, is the lead study investigator.

Our Phase 2 clinical trials are targeting up to 16 cystic fibrosis patients with a G542X mutation on one or both alleles. Patients with one G542X allele and a second minimally functional allele (frequently identified as class 1 or class 2), that is included in the list of accepted 2nd alleles may be eligible for participation in these trials. This may include patients with an F508del mutation who are not receiving a CFTR modulator. Patients with other minimally functional second alleles may be allowed to enter the trial, but only after discussion on a case by case basis with the clinical site principal investigator and approval from the Sponsor.

If you are a physician, or a patient that may be interested in participating in one of these trials, in addition to (Identifiers: NCT04126473 and NCT04135495), see the following documents for additional information or contact us at

> Patient Brochure (English)

> Patient Brochure (Hebrew)

> Patient Brochure (German)

> Patient Brochure (Arabic)

> Patient Brochure (Russian)


  • Targeting up to 16 CF patients with a G542X mutation on one or both alleles
  • Intra-patient dose escalation
  • 4 increasing doses of ELX-02 ranging from 0.3 up to 3.0 mg/kg/day

Primary Outcome Measures

  • Safety, tolerability, and pharmacokinetics

Secondary Outcome Measures

  • Pharmacodynamic changes from baseline in sweat chloride levels and FEV1


  • Enrollment open at sites in Europe, Israel and the United States

Orphan Drug Designation and Advocacy Sanction

  • Orphan drug designation granted in U.S. and Europe
  • Partial funding provided by the CF Foundation, sanctioned by the TDN and with a high priority ranking by ECFS-CTN
Trial Program Design

Dose 1: 7 Days

0.3 mg/kg SC QD

Dose 2: 7 Days

0.75 mg/kg SC QD

Dose 3: 7 Days

1.5 mg/kg SC QD

Dose 4: 14 Days

Up to 3 mg/kg SC QD