EMPIRE CF: A Phase 2 Study to Evaluate the Efficacy, Safety, and Tolerability of CTX-4430 in Adult Cystic Fibrosis (CF) Patients

Celtaxsys

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: Placebo; Drug: CTX-4430

Study type: Interventional

Funder types: Other

Identifiers

NCT02443688

CTX-4430-CF-201

Details and patient eligibility

About

This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF.

Full description

This study is a Phase 2, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of CTX-4430 administered once-daily for 48 weeks for treatment of CF. A total of 195 pulmonary CF patients that meet all the inclusion and no exclusion criteria and provide written informed consent will be randomized to receive 50 mg CTX-4430, 100 mg CTX-4430, or placebo in a 1:1:1 ratio. Follow-up visits will be conducted approximately every 4 weeks from Week 4 to Week 52 (4 weeks after completion of treatment).

Enrollment: 200 patients

Sex: All

Ages: 18 to 30 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Forced expiratory volume at one second (FEV1) ≥50 percent predicted at Screening
• At least 1 pulmonary exacerbation in the 12 months before Screening

Exclusion criteria

• Pregnant or nursing women
• Medical condition that is unstable, could be adversely impacted by participation in the study, or could impact assessment of the study results
• History of organ transplantation
• History of alcoholism or drug abuse within 2 years before Screening
• Regular use of a high-dose NSAID within 60 days before Screening

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Triple Blind

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200 participants in 3 patient groups, including a placebo group

50 mg CTX-4430

Experimental group

Description:

Once daily oral capsule for 48 weeks

Treatment:

Drug: CTX-4430

100 mg CTX-4430

Experimental group

Description:

Once daily oral capsule for 48 weeks

Treatment:

Drug: CTX-4430

Matching Placebo

Placebo Comparator group

Description:

Once daily oral capsule for 48 weeks

Treatment:

Drug: Placebo