Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF) (CLEAN-CF)

Parion Sciences

Status and phase: Completed; Phase 2

Conditions: Cystic Fibrosis

Treatments: Drug: Hypertonic Saline; Drug: Saline; Drug: P-1037

Study type: Interventional

Funder types: Industry

Identifiers

NCT02343445

PS-G201

Details and patient eligibility

About

The goal of the study is to evaluate the safety and tolerability of P-1037 and to determine whether the combination of P-1037 with hypertonic saline or P-1037 alone has a greater effect on lung function in patients with CF than placebo (0.17% saline).

Enrollment: 142 patients

Sex: All

Ages: 12 to 80 years old

Volunteers: No Healthy Volunteers

Inclusion criteria

• Male or female, 12 years of age or older.
• Diagnosis of cystic fibrosis as determined by the 1997 CF Consensus criteria (NIH Consensus Statement, 1997)
• Non-smoker
• FEV1 at Screening Visit 1 between 40% and 90%
• Stable regimen of CF medications and chest physiotherapy for the 28 days prior to Screening. Must be willing to discontinue use of hypertonic saline for the duration of the study.
• Clinically stable for at least 2 weeks
• All females of child-bearing potential must have a negative serum pregnancy test and if sexually active must agree to practice a highly effective form of contraception throughout the study and for 28 days after the last dose of study medication.

Exclusion criteria

• History of any organ transplantation or any significant disease or disorder

• Use of diuretics (including amiloride) or renin-angiotensin antihypertensive drugs or trimethoprim in the 28 days prior to Screening

• History of significant intolerance to inhaled hypertonic saline, as determined by the Investigator

• Known hypersensitivity to the study drug or amiloride

• Any clinically significant laboratory abnormalities at Screening Visit 1 as judged by the investigator, or any of the following:

  • Potassium ≥ 5 milliequivalent per Liter (mEq/L)
  • Abnormal renal function
  • Abnormal liver function, defined as ≥ 3 x upper limit of normal (ULN)
  • Hemoglobin level < 10.0 g/dL

• Female who is pregnant or lactating

• History of sputum or throat swab culture yielding Burkholderia species or Mycobacterium abscessus within 2 years of screening

• Has previously participated in an investigational trial involving administration of any investigational compound or use of an investigational device with 28 days prior to Screening

• Currently being treated with any ivacaftor containing regimen

Trial design

Primary purpose: Treatment

Allocation: Randomized

Interventional model: Parallel Assignment

Masking: Quadruple Blind

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142 participants in 4 patient groups, including a placebo group

P-1037 in Hypertonic Saline (HS)

Experimental group

Description:

P-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID

Treatment:

Drug: P-1037

Drug: Hypertonic Saline

P-1037 in Saline

Experimental group

Description:

P-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID

Treatment:

Drug: Saline

Drug: P-1037

Saline

Placebo Comparator group

Description:

Placebo (0.17% saline) BID

Treatment:

Drug: Saline

Hypertonic Saline

Sham Comparator group

Description:

Hypertonic saline (4.2% saline) BID

Treatment:

Drug: Hypertonic Saline