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| Name | Class |
|---|---|
| Cystic Fibrosis Foundation | OTHER |
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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.
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.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| 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. |
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| 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. |
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| mycobacteriophage | Biological | mycobacteriophage phage that has been found effective in killing participants NTM infection |
|
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
| Measure | Description | Time Frame |
|---|---|---|
| Phage susceptibility | Proportion of participants with NTM infection susceptible to phage | At the time of enrollment |
| Culture conversion | Proportion of participants with >12 months of consecutive negative cultures with no subsequent positive cultures. |
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Inclusion Criteria:
Exclusion Criteria:
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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.
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| Name | Affiliation | Role |
|---|---|---|
| Jerry Nick, MD | National Jewish Health | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University of Alabama | Birmingham | Alabama | 35233 | United States | ||
| Children's Hospital of Los Angeles |
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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.
| Any 12 month interval from the start of phage therapy to end of follow-up, an average of about 2 years. |
| Tolerance of treatment | Proportion requiring antibiotic course change due to intolerance or lack of microbiological conversion (i.e. eradication from sputum) | From enrollment through study completion, an average of about 24-30 months. |
| Clinical Response (pulmonary function testing) | Within subject change in forced expiratory volume at one second (FEV1) with increase indicating improvement in lung function or FEV1 decrease indicating decline in lung function. | From enrollment through study completion, an average of about 24-30 months. |
| Microbiologic response to phage | Within-subject change in percent positive (%pos) cultures following phage initiation (or identification that no phage is available), compared to the interval prior to phage initiation (or identification that no phage is available): the period of antibiotic treatment without 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. |
| Clinical response (BMI) | Within subject change in BMI with decline in BMI as sign of decline or worse outcome. | From enrollment through study completion, an average of about 24-30 months. |
| Clinical response (CFQR) | Within subject change in Cystic Fibrosis Questionnaire -Research (CFQR) score, with higher score indicating worse symptoms. | From enrollment through study completion, an average of about 24-30 months. |
| Clinical Response (antibiotic courses for non- NTM exacerbations) | Proportion of non-NTM exacerbations compared to NTM exacerbations. | From enrollment through study completion, an average of about 24-30 months. |
| Los Angeles |
| California |
| 90027 |
| United States |
| University of California | San Diego | California | 92103 | United States |
| Childrens Hospital Colorado | Denver | Colorado | 80045 | United States |
| National Jewish Health | Denver | Colorado | 80206 | United States |
| University of Florida | Gainesville | Florida | 32610 | United States |
| Northwestern University | Chicago | Illinois | 60611 | United States |
| John Hopkins University | Baltimore | Maryland | 21218 | United States |
| Boston Childrens Hospital | Boston | Massachusetts | 02115 | United States |
| University of Michigan | Ann Arbor | Michigan | 48109 | United States |
| Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | United States |
| Columbia University | New York | New York | 10032 | United States |
| University of North Carolina at Chapel Hill | Chapel Hill | North Carolina | 27599 | United States |
| Nationwide Children's Hospital | Columbus | Ohio | 43215 | United States |
| University of Pittsburgh Medical Center | Pittsburgh | Pennsylvania | 15213 | United States |
| Southwestern Medical Center at Dallas | Dallas | Texas | 75390 | United States |
| University of Vermont | Burlington | Vermont | 05401 | United States |
| University of Washington | Seattle | Washington | 98195 | United States |
| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| D009165 | Mycobacterium Infections, Nontuberculous |
| D009164 | Mycobacterium Infections |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
| D030342 | Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |
| D000193 | Actinomycetales Infections |
| D016908 | Gram-Positive Bacterial Infections |
| D001424 | Bacterial Infections |
| D001423 | Bacterial Infections and Mycoses |
| D007239 | Infections |
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