HOPE-1 (Hydration for Optimal Pulmonary Effectiveness)

NCT03229252

NOW ENROLLING*

The Phase II clinical trial of our investigational treatment for cystic fibrosis, SPX-101 Inhalation Solution, is currently recruiting adult participants.

HOPE-1 (SPX-101-CF-201) Clinical Trial Design

A randomized, double-blind, placebo-controlled phase II study to evaluate the efficacy and safety of SPX-101 Inhalation Solution in subjects with cystic fibrosis is underway in Canada, the United Kingdom, France, and Portugal. Additional clinical sites are pending in Italy.

This study will evaluate the efficacy, safety and tolerability of SPX-101 Inhalation Solution for 28 days in adults 18 – 50 years of age who have a confirmed diagnosis of cystic fibrosis. All CFTR mutations are eligible for this study.

HOPE-1 will include three arms:

• Placebo inhalation solution twice daily for 28 days

• SPX-101 Inhalation Solution (Low Dose) twice daily for 28 days

• SPX-101 Inhalation Solution (High Dose) twice daily for 28 days

Participants must meet the inclusion and exclusion criteria detailed below

Inclusion Criteria Adult

  • males and non-pregnant, non-lactating females 18 – 50 years of age
  • Patients with a confirmed diagnosis of CF, all mutations included
  • ppFEV1 between 40.0% and 70.0%
  • Patients with stable CF lung disease

Exclusion Criteria

  • Patients with significant unstable co-morbidities within 28 days of screening
  • Has received an investigational drug within 28 days of screening

HOPE-1 Outcome Measures

Primary Outcome Measures

  • Change in percent predicted FEV1

Secondary Outcome Measures

  • Number of participants with adverse events
  • Change from baseline through Day 28 in clinical laboratory tests
     Chemistry, Hematology, Urinalysis

“The Clinical Trial Network was pleased to review this important protocol and to approve it for inclusion in the CTN with its highest priority rating. We look forward to working with Spyryx Biosciences to continue the development of this important potential treatment.”

– Dr. Tim Lee, Director of the Clinical Trials Network (CTN) of the European Cystic Fibrosis Society

A portion of the funding for this study was generously provided by CFFT Therapeutics, the development arm of the CF Foundation.

What is SPX-101?

SPX-101 is a therapeutic peptide that binds to epithelial sodium channels (ENaC) on the airway surface, causing a decrease in channel density by cellular internalization. This novel mechanism durably reduces absorption so that fluid is retained on the airway surface. Enhancing fluid levels restores hydration to the resident mucus and promotes its clearance by airway cilia, a process called mucociliary clearance. SPX-101 is in clinical development to treat CF-related lung disease for all people with this disease, regardless of their specific CFTR mutation.

 What is cystic fibrosis (CF)?

CF is a genetic disease that impacts the body’s ability to regulate the flow of salt and water across epithelial membranes, resulting in loss of fluid volume. The respiratory tract is one of the most profoundly affected, causing dehydration-induced lung disease that begins with depleted airway surface liquid and accumulations of dry, sticky mucus and leads to chronic inflammation, recurrent infections, and reduced ability to breathe.