CYstic Fibrosis bacterioPHage Study at Yale (CYPHY)

NCT ID: NCT04684641

Last Updated: 2023-11-18

Basic Information

• Recruitment Status: COMPLETED
• Clinical Phase: PHASE1/PHASE2
• Total Enrollment: 8 participants
• Study Classification: INTERVENTIONAL
• Study Start Date: 2021-03-29
• Study Completion Date: 2023-06-22

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.

Conditions

Cystic Fibrosis

Study Design

• Allocation Method: RANDOMIZED
• Intervention Model: PARALLEL
• Primary Study Purpose: TREATMENT
• Blinding Strategy: QUADRUPLE

Study Groups

Phage therapy

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

Group Type: EXPERIMENTAL

Standard Dose YPT-01

Intervention Type: DRUG

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

Placebo

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

Group Type: ACTIVE_COMPARATOR

Placebo

Intervention Type: OTHER

Participants will be randomized to receive the placebo.

Interventions

Standard Dose YPT-01

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

Intervention Type: DRUG

Placebo

Participants will be randomized to receive the placebo.

Intervention Type: OTHER

Other Intervention Names

Phage Therapy

Eligibility Criteria

Inclusion Criteria

1. Capable of giving signed informed consent;

2. Stated willingness to comply with all study procedures and availability for the duration of the study;

3. Age ≥18;

4. CF diagnosis based upon genetics, sweat chloride testing, or clinical manifestations;

5. Able to provide repeated induced sputum samples;

6. Able to use a nebulizer;

7. PsA culture positive on one occasions within past 2 years and in sputum at screening visit;

8. FEV1 >40%;

9. Clinically stable lung disease, defined as no decrease in FEV1 >10% or pulmonary exacerbations in the 4 weeks prior to screening;

10. 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;

11. 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;

12. 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

1. History of solid organ transplant (e.g., lung or liver);

2. Severe neutropenia, as defined by absolute neutrophil count (ANC) of < 500 per microliter;

3. No YPT-01 phage identified that effectively targets sputum PsA;

4. Treatment for pulmonary exacerbation within the prior 4 weeks;

5. Change in pulmonary medications within the prior 4 weeks;

6. Subjects who are pregnant, who intend to become pregnant, or who do not wish to use contraception;

7. Subjects who are breastfeeding;

8. Participation in another clinical research study concurrently or within the prior 2 months;

9. Known allergy to soy, egg, yeast, or meat.

10. Any genetic or acquired (including medication-induced) immunocompromised condition, beyond the level of immunocompromise typically associated with CF and its management.

• Minimum Eligible Age: 18 Years
• Eligible Sex: ALL
• Accepts Healthy Volunteers: No

Sponsors

Yale University

OTHER

Sponsor Role: lead

Responsible Party

Jonathan Koff

Director, Adult Cystic Fibrosis Program

Responsibility Role: PRINCIPAL_INVESTIGATOR

Principal Investigators

Jonathan Koff, MD

Role: STUDY_DIRECTOR

Yale University

Benjamin Chan, PhD

Role: PRINCIPAL_INVESTIGATOR

Yale University

Locations

Yale New Haven Hospital

New Haven, Connecticut, United States

Countries

United States

Provided Documents

Document Type: Study Protocol and Statistical Analysis Plan

View Document

Other Identifiers

20-004179

Identifier Type: OTHER_GRANT

Identifier Source: secondary_id

2000029160

Identifier Type: -

Identifier Source: org_study_id