Clinical trial NCT06262282: Mycobacteriophage Treatment of Non-tuberculosis Mycobacteria
Official title: A Prospective Standardized Assessment of People With Cystic Fibrosis and Non-tuberculosis Mycobacteria Pulmonary Disease Undergoing Treatment With Mycobacteriophage (POSTSTAMP)
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Cystic Fibrosis
Nontuberculous Mycobacterial Lung Disease
Nontuberculous Mycobacterium Infection
Mycobacterium Infections
Mycobacterium; Pulmonary
Keywords: cystic fibrosis
phage
mycobacteriophage
Lead sponsor: National Jewish Health
Sponsor class: OTHER
Collaborators: name: Cystic Fibrosis Foundation
First posted: 2024-02-16
Enrollment: 10
Sex: ALL
Minimum age (years): 6
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: COHORT
Time perspective: PROSPECTIVE
Sampling method: NON_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.

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.

Detailed description: About 10 people with cystic fibrosis and NTM infection with positive sputum cultures after a minimum of 12 months of guideline-based therapy will be screened to find out if their NTM infection has at least one mycobacteriophage that is known to be effective against the NTM. Individuals who have been found to have at least one effective phage will be offered assistance in pursuing FDA approval for phage treatment through a compassionate-use Individual New Drug (IND). These subjects 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 continue to receive guideline based antibiotic therapy. All subjects, receiving phage or not, will be observed and assessed, including collection of specimens, to evaluate response to treatment.

Eligibility criteria: Inclusion Criteria:

1. Written informed consent (and assent when applicable) obtained from participant or participant's legal representative
2. Enrolled in the CFF Patient Registry (CFF PR)
3. Be willing to adhere to study procedures in the context of clinical care, and other protocol requirements
4. 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)
5. Diagnosis of CF consistent with the 2017 CFF Guidelines
6. NTM pulmonary disease on treatment with guideline-based antibiotics for \>12 months without consistent conversion of airway cultures to negative.
7. Physician intention to treat NTM with phage therapy (if susceptible)
8. Be willing and able to continue guideline-based antibiotics for NTM concurrent with phage.
9. 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.

Exclusion Criteria:

1. Pregnant or breastfeeding
2. Prior or ongoing phage therapy for the species of NTM under consideration.
3. History of solid organ or hematological transplantation
4. 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

Primary outcomes:
measure: Adherence to therapy
timeFrame: Comparing the year of phage therapy to the year prior to start of phage therapy
Secondary outcomes:
measure: Phage susceptibility
timeFrame: At the time of enrollment
measure: Culture conversion
timeFrame: Any 12 month interval from the start of phage therapy to end of follow-up, an average of about 2 years.
measure: Tolerance of treatment
timeFrame: From enrollment through study completion, an average of about 24-30 months.
measure: Clinical Response (pulmonary function testing)
timeFrame: From enrollment through study completion, an average of about 24-30 months.
measure: Microbiologic response to phage
timeFrame: 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.
measure: Clinical response (BMI)
timeFrame: From enrollment through study completion, an average of about 24-30 months.
measure: Clinical response (CFQR)
timeFrame: From enrollment through study completion, an average of about 24-30 months.
measure: Clinical Response (antibiotic courses for non- NTM exacerbations)
timeFrame: From enrollment through study completion, an average of about 24-30 months.
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: University of Alabama
state: Alabama
city: Los Angeles
country: United States
facility: Children's Hospital of Los Angeles
state: California
city: San Diego
country: United States
facility: University of California
state: California
city: Denver
country: United States
facility: Childrens Hospital Colorado
state: Colorado
city: Denver
country: United States
facility: National Jewish Health
state: Colorado
city: Gainesville
country: United States
facility: University of Florida
state: Florida
city: Chicago
country: United States
facility: Northwestern University
state: Illinois
city: Baltimore
country: United States
facility: John Hopkins University
state: Maryland
city: Boston
country: United States
facility: Boston Childrens Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: University of Michigan
state: Michigan
city: Lebanon
country: United States
facility: Dartmouth Hitchcock Medical Center
state: New Hampshire
city: New York
country: United States
facility: Columbia University
state: New York
city: Chapel Hill
country: United States
facility: University of North Carolina at Chapel Hill
state: North Carolina
city: Columbus
country: United States
facility: Nationwide Children's Hospital
state: Ohio
city: Pittsburgh
country: United States
facility: University of Pittsburgh Medical Center
state: Pennsylvania
city: Dallas
country: United States
facility: Southwestern Medical Center at Dallas
state: Texas
city: Burlington
country: United States
facility: University of Vermont
state: Vermont
city: Seattle
country: United States
facility: University of Washington
state: Washington
Study officials:
affiliation: National Jewish Health
name: Jerry Nick, MD
role: PRINCIPAL_INVESTIGATOR
References: