Cystic FibrosisPhase 1/2NCT04684641
CYstic Fibrosis bacterioPHage Study at Yale (CYPHY)
A Phase 1/2 study testing Standard Dose YPT-01 in people with Cystic Fibrosis, run by Yale University at 1 site in United States.
Trial 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. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Cystic Fibrosis
- What is being tested?
- Standard Dose YPT-01
- Could I receive a placebo?
- Yes, some participants may receive a placebo
- How many people
- 8 participants
- Where
- 1 siteUnited States
- Study dates
- 29 Mar 2021to 22 Jun 2023
- Phase
- Phase 1/2
- Are there results?
- Yes, posted in the registrySummary in the doctors' view
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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;
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- 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.
Who cannot join
- 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;
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- 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.
Trial locations and contacts
1 site in 1 country.
United States1 site
Yale New Haven Hospital
New Haven, Connecticut, 06520
About this study
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
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.
Read the 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
- Being tested
- Standard Dose YPT-01, Placebo
- Keywords
- Pseudomonas, bacteriophage, phage
- Medical subject headings
- Cystic Fibrosis, Pseudomonas Infections
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
Standard Dose YPT-01Drug
Also called Phage Therapy
Participants will be randomized to receive the standard dose of phage therapy YPT-01.
PlaceboOther
Participants will be randomized to receive the placebo.
| Study group | Role | What they receive |
|---|---|---|
| Phage therapy | Experimental | Participants will be randomized to receive 3mL phage therapy, nebulized daily for 7 days. Receives: Drug: Standard Dose YPT-01 |
| Placebo | Active comparator | Participants will be randomized to receive the 3mL placebo, nebulized daily for 7 days. Receives: Other: Placebo |
What is being measured
The primary outcome is the main question the study is designed to answer.
Change in Sputum Bacterial Culture
Change in sputum bacterial culture titers of Pseudomonas as measured by colony forming units/mL at day 14
Measured: Day 14
Secondary outcomes (5)
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
Measured: Screening, day 14, day 21, day 28, and day 56
Difference 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
Measured: Baseline, day 56
Difference 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
Measured: Baseline, day 56
Difference 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
Measured: Baseline, day 56
Patient's Quality of Life
Changes in subject-reported quality of life, using the Cystic Fibrosis Questionnaire Revised (CFQ-R) Teen/Adult, from baseline to Day 56. This survey consists of 50 questions that relate to a subjects clinical condition and mental health. Scores for each domain range from 0 to 100, with higher scores indicating a higher patient-reported quality of life with regard to the domain being evaluated.
Measured: Baseline, day 56
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted24 Dec 2020
- Study start29 Mar 2021
- Primary completion26 May 2023
- Study completion22 Jun 2023
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Posted resultsParticipant flow, outcome measures and adverse events reported by the sponsor.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- More trials run by Yale UniversityThe sponsor's other registered studies.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1/2
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind
- Who is masked
- Participant, Care provider, Investigator, Outcomes assessor
- Enrolment
- 8 participants (actual)
Sponsor and investigators
- Lead sponsor
- Yale University
- Sponsor type
- University, hospital or other
- Responsible party
- Principal investigator, Jonathan Koff, Yale University
- Investigators
- Jonathan Koff, MD, Study director, Yale UniversityBenjamin Chan, PhD, Principal investigator, Yale University
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT04684641
- Sponsor's protocol ID
- 2000029160
- Other IDs
- 20-004179
- Status last verified
- Oct 2023
- First submitted
- 18 Dec 2020
- Last updated
- 18 Nov 2023
- Results posted
- Yes, on ClinicalTrials.gov
- Data monitoring committee
- Yes
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 18 Nov 2023. Recruitment status is as the sponsor reported it: confirm with the study team.
- 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
- ClinicalTrials.gov ID
- NCT04684641
- Acronym / sponsor ID
- 2000029160
- Conditions
- Cystic Fibrosis
- Keywords
- Pseudomonas, bacteriophage, phage
- Phase
- Phase 1/2
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 8 participants (actual)
- Lead sponsor
- Yale UniversityUniversity, hospital or other
- Study start
- 29 Mar 2021 (actual)
- Primary completion
- 26 May 2023 (actual)
- Study completion
- 22 Jun 2023 (actual)
- First posted
- 24 Dec 2020 (actual)
- Results
- Posted 3 Mar 2023
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Primary purpose
- Treatment
- Allocation
- Randomised
- Intervention model
- Parallel groups
- Masking
- Quadruple blind (participant, care provider, investigator, outcomes assessor)
- Number of arms
- 2
| Arm | Type | Receives |
|---|---|---|
| Phage therapy | Experimental | Drug: Standard Dose YPT-01Participants will be randomized to receive 3mL phage therapy, nebulized daily for 7 days. |
| Placebo | Active comparator | Other: PlaceboParticipants will be randomized to receive the 3mL placebo, nebulized daily for 7 days. |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (12 listed)
- 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;
Key exclusion criteria (10 listed)
- 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;
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| Standard Dose YPT-01Also: Phage TherapyParticipants will be randomized to receive the standard dose of phage therapy YPT-01. | Drug | Phage therapy |
| PlaceboParticipants will be randomized to receive the placebo. | Other | Placebo |
Primary outcome
Change in Sputum Bacterial Culture
Time frame: Day 14
Change in sputum bacterial culture titers of Pseudomonas as measured by colony forming units/mL at day 14
5 secondary outcomes
Change in Lung Function
Time frame: Screening, day 14, day 21, day 28, and day 56
Difference in the Rate of Pulmonary Exacerbations
Time frame: Baseline, day 56
Difference in the Rate of Hospitalization
Time frame: Baseline, day 56
Difference in the Rate of Acute Antibiotic Use
Time frame: Baseline, day 56
Patient's Quality of Life
Time frame: Baseline, day 56
Results and safety
Loading the posted results from ClinicalTrials.gov…
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 18 Nov 2023.
- Posted resultsParticipant flow, baseline, all outcome measures and adverse events.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jonathan Koff, MD study director
Yale University
Benjamin Chan, PhD principal investigator
Yale University
1 site in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1/2 study testing Standard Dose YPT-01 in people with Cystic Fibrosis, run by Yale University at 1 site in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 12 inclusion and 10 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 18 Nov 2023. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 1 site in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this combined phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
Yes. Results are posted in the registry (first posted 3 Mar 2023). A summary is in the For doctors view, and the full results are on ClinicalTrials.gov.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Change in Sputum Bacterial Culture (time frame: Day 14). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 18 Nov 2023.