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P-1037

Phase 2

Cystic Fibrosis | Small molecule | Respiratory |Vertex Pharmaceuticals Incorporated|Last Updated: Jul 26, 2021

Success Probability

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Market & Valuation

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Trial Design

RandomizedDouble-BlindPLACEBO_CONTROLLEDBiomarker
Total Trials1
Total Enrollment142

FDA Designations

No designations recorded

Clinical trial landscape

P-1037 · 1 trial · 1 indication

Phase 2 1
NCT02343445Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)Cystic Fibrosis
COMPLETED142 Analytics
PHASE2COMPLETED
Clearing Lungs With Epithelial Sodium Channel (ENaC) Inhibition in Cystic Fibrosis (CF)
Cystic FibrosisUnlock trial analytics

Study Endpoints

Primary Endpoints

Safety (number of adverse events of P-1037 in treatment groups)
Day 15

number of adverse events of P-1037 in treatment groups

Safety [change in forced expiratory volume in one second (FEV1) from predosing to 1 hour post dosing]
Day 1

change in FEV1 from predosing to 1 hour post dosing with P-1037 in 4.2 % hypertonic saline and P-1037 in 0.17% saline

Secondary Endpoints

Absolute change in FEV1
Day 15
Effect of P-1037 on other forced vital capacity (FVC) measures (change in FVC from baseline (immediately before first dose) to same time of day on Day 15)
Day 15
Effect of P-1037 on Cystic Fibrosis Questionnaire - Revised (CFQ-R)
Day 15
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Study Design & Arms

AllocationRANDOMIZED
MaskingQUADRUPLE
ModelPARALLEL
PurposeTREATMENT

Treatment Arms

ArmTypeDescription
P-1037 in Hypertonic Saline (HS)EXPERIMENTALP-1037 Solution for Inhalation, 85 μg twice daily (BID) (28.3 μg/mL) in hypertonic saline (4.2% saline) BID
P-1037 in SalineEXPERIMENTALP-1037 Solution for Inhalation, 85 μg BID (28.3 μg/mL) in 0.17% saline BID
SalinePLACEBO_COMPARATORPlacebo (0.17% saline) BID
Hypertonic SalineSHAM_COMPARATORHypertonic saline (4.2% saline) BID

Interventions

NameTypeDescription
P-1037DRUGP-1037 is a novel ENaC inhibitor
Hypertonic SalineDRUG4.2% saline solution
SalineDRUG0.17% saline solution
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Eligibility Criteria

Age Range12 Years to 80 Years
SexALL
Healthy VolunteersNo
Study Sites33

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 ...

Countries:United States
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Frequently asked questions about P-1037

What is P-1037 used for?

P-1037 is an investigational small molecule being developed for the treatment of cystic fibrosis. It is designed to address the underlying respiratory complications of the disease. The drug is currently in clinical development and has not been approved by regulatory authorities.

What does P-1037 target?

P-1037 targets the epithelial sodium channel (ENaC) in the lungs. By inhibiting this channel, the drug aims to reduce sodium absorption and improve airway surface hydration in patients with cystic fibrosis. This mechanism is being studied as a potential therapeutic approach for the condition.

Who makes P-1037?

P-1037 is being developed by Vertex Pharmaceuticals Incorporated, a biopharmaceutical company traded on the NASDAQ under the ticker symbol VRTX. Vertex is conducting clinical research to evaluate the safety and efficacy of P-1037 in patients with cystic fibrosis.

What phase is P-1037 in?

P-1037 is in Phase 2 clinical development. It is an investigational drug and has not received FDA approval. The Phase 2 trial has been completed, and the results will inform whether the drug advances to later-stage studies.

What clinical trials is P-1037 in?

P-1037 has been studied in one completed Phase 2 clinical trial, identified as NCT02343445. This randomized, double-blind, placebo-controlled study enrolled 142 participants with cystic fibrosis in the United States. The trial evaluated the effects of ENaC inhibition on lung function and other outcomes.

Is P-1037 the same as other cystic fibrosis drugs?

P-1037 is a distinct investigational compound developed by Vertex Pharmaceuticals. It is not the same as other approved cystic fibrosis drugs, which target different molecular pathways. P-1037 specifically inhibits the epithelial sodium channel, a unique mechanism being explored for the disease.