NCT06262282

Brief Summary

About 10 people with cystic fibrosis (CF) and persistent Nontuberculosis mycobacteria (NTM) infection despite treatment will be screened to find out if their NTM infection has at least one mycobacteriophage that is effective in killing the mycobacteria. Individuals who are found to have at least one phage will be offered assistance in pursuing FDA approval for treatment via expanded-access Individual New Drug (IND) for compassionate-use. They will receive phage treatment for 1 year along with their guideline-based antibiotics for NTM. Individuals who are not identified as having a phage match will be followed as they continue to receive guideline based antibiotic therapy for 1 year. All subjects, including those who do not have a phage match will continue to be observed for the duration of the study, or about 1 year.

Trial Health

75
On Track

Trial Health Score

Automated assessment based on enrollment pace, timeline, and geographic reach

Enrollment
10

participants targeted

Target at below P25 for all trials

Timeline
33mo left

Started Feb 2024

Longer than P75 for all trials

Geographic Reach
1 country

18 active sites

Status
enrolling by invitation

Health score is calculated from publicly available data and should be used for screening purposes only.

Trial Relationships

Click on a node to explore related trials.

Study Timeline

Key milestones and dates

Study Progress46%
Feb 2024Dec 2028

First Submitted

Initial submission to the registry

February 4, 2024

Completed
1 day until next milestone

Study Start

First participant enrolled

February 5, 2024

Completed
11 days until next milestone

First Posted

Study publicly available on registry

February 16, 2024

Completed
2.9 years until next milestone

Primary Completion

Last participant's last visit for primary outcome

December 31, 2026

Expected
2 years until next milestone

Study Completion

Last participant's last visit for all outcomes

December 31, 2028

Last Updated

May 21, 2025

Status Verified

May 1, 2025

Enrollment Period

2.9 years

First QC Date

February 4, 2024

Last Update Submit

May 20, 2025

Conditions

Keywords

cystic fibrosisphagemycobacteriophage

Outcome Measures

Primary Outcomes (1)

  • Adherence to therapy

    Proportion who adhere to the POSTSTAMP protocol based on number of respiratory cultures obtained per year, withdrawals or major deviations from protocol.

    Comparing the year of phage therapy to the year prior to start of phage therapy

Secondary Outcomes (8)

  • Phage susceptibility

    At the time of enrollment

  • Culture conversion

    Any 12 month interval from the start of phage therapy to end of follow-up, an average of about 2 years.

  • Tolerance of treatment

    From enrollment through study completion, an average of about 24-30 months.

  • Clinical Response (pulmonary function testing)

    From enrollment through study completion, an average of about 24-30 months.

  • Microbiologic response to phage

    A year interval from month 6 of treatment to month 18 following start of phage will be compared to the year prior to start of phage.

  • +3 more secondary outcomes

Study Arms (2)

People with cystic fibrosis who are receiving phage treatment for NTM.

People with cystic fibrosis who have identified at least one phage effective against their NTM infection and are receiving treatment with the phage. These subjects will remain on guideline based NTM antibiotic treatment and their standard CF care. They will receive therapeutic phage twice daily for 1 year.

Biological: mycobacteriophage

People with cystic fibrosis who are not receiving phage treatment for NTM.

People with cystic fibrosis who have not identified any phage effective against their NTM infection and are not receiving phage treatment. These subjects will continue with guideline based NTM antibiotic treatment for their NTM disease and standard CF care.

Interventions

mycobacteriophage phage that has been found effective in killing participants NTM infection

People with cystic fibrosis who are receiving phage treatment for NTM.

Eligibility Criteria

Age6 Years+
Sexall
Healthy VolunteersNo
Age GroupsChild (0-17), Adult (18-64), Older Adult (65+)
Sampling MethodNon-Probability Sample
Study Population

Participants with a diagnosis of CF who meet all of the inclusion and none of the exclusion criteria will be eligible for participation in this study.

You may qualify if:

  • Written informed consent (and assent when applicable) obtained from participant or participant's legal representative
  • Enrolled in the CFF Patient Registry (CFF PR)
  • Be willing to adhere to study procedures in the context of clinical care, and other protocol requirements
  • Male or female participant ≥ 6 years of age at enrollment who are able to reliably expectorate sputum and/or willing to undergo sputum induction (if necessary)
  • Diagnosis of CF consistent with the 2017 CFF Guidelines
  • NTM pulmonary disease on treatment with guideline-based antibiotics for \>12 months without consistent conversion of airway cultures to negative.
  • Physician intention to treat NTM with phage therapy (if susceptible)
  • Be willing and able to continue guideline-based antibiotics for NTM concurrent with phage.
  • Documentation of a sufficient number of NTM cultures with a sufficient proportion of positive cultures in the interval 12 months prior to initiation of phage to allow for a within-subject power ≥0.80 to detect a difference in the percent positive NTM cultures in the interval 6-18 months following initiation of phage.

You may not qualify if:

  • Pregnant or breastfeeding
  • Prior or ongoing phage therapy for the species of NTM under consideration.
  • History of solid organ or hematological transplantation
  • Has any other condition that, in the opinion of the Site Investigator/designee, would preclude informed consent or assent, make study participation unsafe, complicate interpretation of study outcome data, or otherwise interfere with achieving the study objectives

Contact the study team to confirm eligibility.

Sponsors & Collaborators

Study Sites (18)

University of Alabama

Birmingham, Alabama, 35233, United States

Location

Children's Hospital of Los Angeles

Los Angeles, California, 90027, United States

Location

University of California

San Diego, California, 92103, United States

Location

Childrens Hospital Colorado

Denver, Colorado, 80045, United States

Location

National Jewish Health

Denver, Colorado, 80206, United States

Location

University of Florida

Gainesville, Florida, 32610, United States

Location

Northwestern University

Chicago, Illinois, 60611, United States

Location

John Hopkins University

Baltimore, Maryland, 21218, United States

Location

Boston Childrens Hospital

Boston, Massachusetts, 02115, United States

Location

University of Michigan

Ann Arbor, Michigan, 48109, United States

Location

Dartmouth Hitchcock Medical Center

Lebanon, New Hampshire, 03756, United States

Location

Columbia University

New York, New York, 10032, United States

Location

University of North Carolina at Chapel Hill

Chapel Hill, North Carolina, 27599, United States

Location

Nationwide Children's Hospital

Columbus, Ohio, 43215, United States

Location

University of Pittsburgh Medical Center

Pittsburgh, Pennsylvania, 15213, United States

Location

Southwestern Medical Center at Dallas

Dallas, Texas, 75390, United States

Location

University of Vermont

Burlington, Vermont, 05401, United States

Location

University of Washington

Seattle, Washington, 98195, United States

Location

Biospecimen

Retention: SAMPLES WITH DNA

NTM isolates will be sent to the National Jewish Health (NJH) laboratory for additional testing and will be stored for future research. Specimens will be banked for future research use (blood, sputum, urine, and NTM isolates- including any previous isolates that might be available or previously sent to NJH for analysis). The specimens will be used to learn more about NTM Disease and to learn more about CF.

MeSH Terms

Conditions

Cystic FibrosisMycobacterium Infections, NontuberculousMycobacterium Infections

Condition Hierarchy (Ancestors)

Pancreatic DiseasesDigestive System DiseasesLung DiseasesRespiratory Tract DiseasesGenetic Diseases, InbornCongenital, Hereditary, and Neonatal Diseases and AbnormalitiesInfant, Newborn, DiseasesActinomycetales InfectionsGram-Positive Bacterial InfectionsBacterial InfectionsBacterial Infections and MycosesInfections

Study Officials

  • Jerry Nick, MD

    National Jewish Health

    PRINCIPAL INVESTIGATOR

Study Design

Study Type
observational
Observational Model
COHORT
Time Perspective
PROSPECTIVE
Sponsor Type
OTHER
Responsible Party
PRINCIPAL INVESTIGATOR
PI Title
Director, Adult Cystic Fibrosis Program

Study Record Dates

First Submitted

February 4, 2024

First Posted

February 16, 2024

Study Start

February 5, 2024

Primary Completion (Estimated)

December 31, 2026

Study Completion (Estimated)

December 31, 2028

Last Updated

May 21, 2025

Record last verified: 2025-05

Locations