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CYstic Fibrosis bacterioPHage Study at Yale (CYPHY)

Primary Purpose

Cystic Fibrosis

Status

Active

Phase

Phase 1

Locations

United States

Study Type

Interventional

Intervention

Standard Dose YPT-01

Placebo

About this trial

This is an interventional treatment trial for Cystic Fibrosis focused on measuring Pseudomonas, bacteriophage, phage

Eligibility Criteria

18 Years - undefined (Adult, Older Adult)All SexesDoes not accept healthy volunteers

Inclusion Criteria:

Capable of giving signed informed consent;
Stated willingness to comply with all study procedures and availability for the duration of the study;
Age ≥18;
CF diagnosis based upon genetics, sweat chloride testing, or clinical manifestations;
Able to provide repeated induced sputum samples;
Able to use a nebulizer;
PsA culture positive on one occasions within past 2 years and in sputum at screening visit;
FEV1 >40%;
Clinically stable lung disease, defined as no decrease in FEV1 >10% or pulmonary exacerbations in the 4 weeks prior to screening;
If on CF modulator therapy (e.g., ivacaftor, ivacaftor/elexacaftor/tezacaftor), then subject remains on the same modulator therapy for at least 2 months prior to enrollment;
For females of reproductive potential: use of effective contraception for at least 1 month prior to screening and agreement to use 2 methods of effective contraception during study participation and for an additional 6 weeks after the end of YPT-01 administration;
Males of non-reproductive potential (e.g., documented congenital bilateral absence of vas deferens) or males of reproductive potential (e.g., non-vasectomized males or males vasectomized less than 120 days prior to study start) that agree to use condoms with spermicide while engaging in sexual activity or be sexually abstinent.

Exclusion Criteria:

History of solid organ transplant (e.g., lung or liver);
Severe neutropenia, as defined by absolute neutrophil count (ANC) of < 500 per microliter;
No YPT-01 phage identified that effectively targets sputum PsA;
Treatment for pulmonary exacerbation within the prior 4 weeks;
Change in pulmonary medications within the prior 4 weeks;
Subjects who are pregnant, who intend to become pregnant, or who do not wish to use contraception;
Subjects who are breastfeeding;
Participation in another clinical research study concurrently or within the prior 2 months;
Known allergy to soy, egg, yeast, or meat.
Any genetic or acquired (including medication-induced) immunocompromised condition, beyond the level of immunocompromise typically associated with CF and its management.

Sites / Locations

Yale New Haven Hospital

Arms of the Study

Arm 1

Arm 2

Arm Type

Experimental

Active Comparator

Arm Label

Phage therapy

Placebo

Arm Description

Participants will be randomized to receive 3mL phage therapy, nebulized daily for 7 days.

Participants will be randomized to receive the 3mL placebo, nebulized daily for 7 days.

Outcomes

Primary Outcome Measures

Reduction in Sputum Bacterial Culture

Reduction in sputum bacterial culture titers of Pseudomonas as measured by colony forming units/mL at day 14

Secondary Outcome Measures

Change in Lung Function

Change in lung function [percent predicted forced expiratory volume in 1 second (FEV1pp)] in subjects randomized to phage therapy and placebo from screening to day 14, 21, 28, and 56

Change in the Rate of Pulmonary Exacerbations

Compare the rates of pulmonary exacerbations between subjects randomized to phage therapy versus placebo during the first 56 days of the study

Change in the Rate of Hospitalization

Compare the rates of hospitalizations between subjects randomized to phage therapy versus placebo during the first 56 days of the study

Change in the Rate of Acute Antibiotic Use

Compare the rates of acute antibiotic use between subjects randomized to phage therapy versus placebo during the first 56 days of the study

Patient's Quality of Life

Compare the changes in subject-reported quality of life, using the CFQR, between subjects randomized to phage therapy versus placebo during the first 56 days of the study. This survey consists of 50 questions that relate to a subjects clinical condition and mental health. Administration time takes 10 minutes

Full Information

NCT ID

NCT04684641

First Posted

December 18, 2020

Last Updated

February 2, 2023

Sponsor

Yale University

1. Study Identification

Unique Protocol Identification Number

NCT04684641

Brief Title

CYstic Fibrosis bacterioPHage Study at Yale (CYPHY)

Official Title

CYstic Fibrosis bacterioPHage Study at Yale (CYPHY): A Single-site, Randomized, Double-blind, Placebo-controlled Study of Bacteriophage Therapy YPT-01 for Pseudomonas Aeruginosa Infections in Adults With Cystic Fibrosis

Study Type

Interventional

2. Study Status

Record Verification Date

February 2023

Overall Recruitment Status

Active, not recruiting

Study Start Date

March 29, 2021 (Actual)

Primary Completion Date

September 1, 2022 (Actual)

Study Completion Date

March 2023 (Anticipated)

3. Sponsor/Collaborators

Responsible Party, by Official Title

Principal Investigator

Name of the Sponsor

Yale University

4. Oversight

Studies a U.S. FDA-regulated Drug Product

Yes

Studies a U.S. FDA-regulated Device Product

Data Monitoring Committee

Yes

5. Study Description

Brief Summary

This is a Phase 2 study with primary objective of looking whether YPT-01 phage therapy reduces sputum bacterial load in cystic fibrosis subjects with Pseudomonas aeruginosa. In addition, study evaluates the safety profile of phage therapy in this patient population.

Detailed Description

This is a prospective, randomized, placebo-controlled, double-blinded, single-site study of Yale Phage Therapy (YPT) 01 in cystic fibrosis subjects with chronic Pseudomonas aeruginosa airway infections. The study has 2 parallel arms of phage therapy and placebo, with all study materials GMP-manufactured. The purpose of this study is to demonstrate efficacy and safety of inhaled (nebulized) phage therapy YPT-01. Clinically stable subjects who have confirmed diagnosis of CF with PsA in sputum cultures on at least two occasions within past year, and in sputum at screening visit, will be recruited into this study. An open-label extension is available for subjects in the placebo group to receive YPT-01 following completion of blinded portion of the study. Nov 2022 study ended enrollment after 8 subjects. The Double-Blind Randomized portion of the study was closed and the Open-Label Extension was opened.

6. Conditions and Keywords

Primary Disease or Condition Being Studied in the Trial, or the Focus of the Study

Cystic Fibrosis

Keywords

Pseudomonas, bacteriophage, phage

7. Study Design

Primary Purpose

Treatment

Study Phase

Phase 1, Phase 2

Interventional Study Model

Parallel Assignment

Masking

ParticipantCare ProviderInvestigatorOutcomes Assessor

Allocation

Randomized

Enrollment

8 (Actual)

8. Arms, Groups, and Interventions

Arm Title

Phage therapy

Arm Type

Experimental

Arm Description

Participants will be randomized to receive 3mL phage therapy, nebulized daily for 7 days.

Arm Title

Placebo

Arm Type

Active Comparator

Arm Description

Participants will be randomized to receive the 3mL placebo, nebulized daily for 7 days.

Intervention Type

Drug

Intervention Name(s)

Standard Dose YPT-01

Other Intervention Name(s)

Phage Therapy

Intervention Description

Participants will be randomized to receive the standard dose of phage therapy YPT-01.

Intervention Type

Other

Intervention Name(s)

Placebo

Intervention Description

Participants will be randomized to receive the placebo.

Primary Outcome Measure Information:

Title

Reduction in Sputum Bacterial Culture

Description

Reduction in sputum bacterial culture titers of Pseudomonas as measured by colony forming units/mL at day 14

Time Frame

Day 14

Secondary Outcome Measure Information:

Title

Change in Lung Function

Description

Change in lung function [percent predicted forced expiratory volume in 1 second (FEV1pp)] in subjects randomized to phage therapy and placebo from screening to day 14, 21, 28, and 56

Time Frame

Screening, day 14, day 21, day 28, and day 56

Title

Change in the Rate of Pulmonary Exacerbations

Description

Compare the rates of pulmonary exacerbations between subjects randomized to phage therapy versus placebo during the first 56 days of the study

Time Frame

Baseline, day 56

Title

Change in the Rate of Hospitalization

Description

Compare the rates of hospitalizations between subjects randomized to phage therapy versus placebo during the first 56 days of the study

Time Frame

Baseline, day 56

Title

Change in the Rate of Acute Antibiotic Use

Description

Compare the rates of acute antibiotic use between subjects randomized to phage therapy versus placebo during the first 56 days of the study

Time Frame

Baseline, day 56

Title

Patient's Quality of Life

Description

Time Frame

Baseline, day 56

10. Eligibility

Sex

All

Minimum Age & Unit of Time

18 Years

Accepts Healthy Volunteers

Eligibility Criteria

Inclusion Criteria: Capable of giving signed informed consent; Stated willingness to comply with all study procedures and availability for the duration of the study; Age ≥18; CF diagnosis based upon genetics, sweat chloride testing, or clinical manifestations; Able to provide repeated induced sputum samples; Able to use a nebulizer; PsA culture positive on one occasions within past 2 years and in sputum at screening visit; FEV1 >40%; Clinically stable lung disease, defined as no decrease in FEV1 >10% or pulmonary exacerbations in the 4 weeks prior to screening; If on CF modulator therapy (e.g., ivacaftor, ivacaftor/elexacaftor/tezacaftor), then subject remains on the same modulator therapy for at least 2 months prior to enrollment; For females of reproductive potential: use of effective contraception for at least 1 month prior to screening and agreement to use 2 methods of effective contraception during study participation and for an additional 6 weeks after the end of YPT-01 administration; Males of non-reproductive potential (e.g., documented congenital bilateral absence of vas deferens) or males of reproductive potential (e.g., non-vasectomized males or males vasectomized less than 120 days prior to study start) that agree to use condoms with spermicide while engaging in sexual activity or be sexually abstinent. Exclusion Criteria: History of solid organ transplant (e.g., lung or liver); Severe neutropenia, as defined by absolute neutrophil count (ANC) of < 500 per microliter; No YPT-01 phage identified that effectively targets sputum PsA; Treatment for pulmonary exacerbation within the prior 4 weeks; Change in pulmonary medications within the prior 4 weeks; Subjects who are pregnant, who intend to become pregnant, or who do not wish to use contraception; Subjects who are breastfeeding; Participation in another clinical research study concurrently or within the prior 2 months; Known allergy to soy, egg, yeast, or meat. Any genetic or acquired (including medication-induced) immunocompromised condition, beyond the level of immunocompromise typically associated with CF and its management.

Overall Study Officials:

First Name & Middle Initial & Last Name & Degree

Jonathan Koff, MD

Organizational Affiliation

Yale University

Official's Role

Study Director

First Name & Middle Initial & Last Name & Degree

Benjamin Chan, PhD

Organizational Affiliation

Yale University

Official's Role

Principal Investigator

Facility Information:

Facility Name

Yale New Haven Hospital

City

New Haven

State/Province

Connecticut

ZIP/Postal Code

06520

Country

United States

12. IPD Sharing Statement

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CYstic Fibrosis bacterioPHage Study at Yale (CYPHY)

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