Mycobacteriophage Treatment of Non-tuberculosis Mycobacteria
For patients and families
In plain language
An automatic summary of structured registry data. It is an orientation aid, not a substitute for the official protocol or a physician assessment.
- What is being studied
- The protocol lists: mycobacteriophage.
- Who it may be relevant to
- Registry conditions: Cystic Fibrosis, Nontuberculous Mycobacterial Lung Disease, Nontuberculous Mycobacterium Infection, Mycobacterium Infections. Basic parameters: from 6 years · All.
- What needs checking
- Age, condition and sex are only basic indicators. Prior treatment, laboratory values and other mandatory requirements appear in the eligibility criteria below.
- Where it takes place
- United States
- Next step
- Save the trial, show it to the treating physician, and confirm current recruitment with the study center. Costs, documents and travel →
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Official title
A Prospective Standardized Assessment of People With Cystic Fibrosis and Non-tuberculosis Mycobacteria Pulmonary Disease Undergoing Treatment With Mycobacteriophage (POSTSTAMP)
Overview
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.
Interventions
- Biological mycobacteriophage
mycobacteriophage phage that has been found effective in killing participants NTM infection
Primary outcome measures
- Adherence to therapy [Time frame: Comparing the year of phage therapy to the year prior to start of phage therapy]
Secondary outcome measures (8)
- Phage susceptibility [Time frame: At the time of enrollment]
- Culture conversion [Time frame: Any 12 month interval from the start of phage therapy to end of follow-up, an average of about 2 years.]
- Tolerance of treatment [Time frame: From enrollment through study completion, an average of about 24-30 months.]
- Clinical Response (pulmonary function testing) [Time frame: From enrollment through study completion, an average of about 24-30 months.]
- Microbiologic response to phage [Time frame: 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.]
- Clinical response (BMI) [Time frame: From enrollment through study completion, an average of about 24-30 months.]
- Clinical response (CFQR) [Time frame: From enrollment through study completion, an average of about 24-30 months.]
- Clinical Response (antibiotic courses for non- NTM exacerbations) [Time frame: From enrollment through study completion, an average of about 24-30 months.]
Eligibility criteria
Inclusion criteria
- 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.
Exclusion criteria
- 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
Criteria are shown verbatim from the registry (in English). Final eligibility is always assessed by the study center.
Healthy volunteers: No
Study design
- Observational model
- Cohort
Study locations
United States · 18 centers
- University of Alabama — Birmingham
- Children's Hospital of Los Angeles — Los Angeles
- University of California — San Diego
- Childrens Hospital Colorado — Denver
- National Jewish Health — Denver
- University of Florida — Gainesville
- Northwestern University — Chicago
- John Hopkins University — Baltimore
- … and 10 more centers
Identifiers
NCT: NCT06262282 · NTM-OB-17 (PART C)