A Prospective Standardized Assessment of People With Cystic Fibrosis and Non-tuberculosis Mycobacteria Pulmonary Disease Undergoing Treatment With Mycobacteriophage (POSTSTAMP)
试验速览
- 阶段
- 不适用
- 状态
- Enrolling By Invitation
- 入组人数
- 10
- 试验地点
- 18
- 主要终点
- Adherence to therapy
研究概览
简要总结
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.
详细描述
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.
研究设计
- 研究类型
- Observational
- 观察模型
- Cohort
- 时间视角
- Prospective
入排标准
- 年龄范围
- 6 Years 至 —（Child, Adult, Older Adult）
- 性别
- All
- 接受健康志愿者
- 否
入选标准
- •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.
排除标准
- •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
研究组 & 干预措施
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.
干预措施: mycobacteriophage (Biological)
结局指标
主要结局
Adherence to therapy
时间窗: Comparing the year of phage therapy to the year prior to start of phage therapy
Proportion who adhere to the POSTSTAMP protocol based on number of respiratory cultures obtained per year, withdrawals or major deviations from protocol.
次要结局
- Tolerance of treatment(From enrollment through study completion, an average of about 24-30 months.)
- Clinical response (BMI)(From enrollment through study completion, an average of about 24-30 months.)
- Culture conversion(Any 12 month interval from the start of phage therapy to end of follow-up, an average of about 2 years.)
- Phage susceptibility(At the time of enrollment)
- Clinical Response (pulmonary function testing)(From enrollment through study completion, an average of about 24-30 months.)
- Clinical response (CFQR)(From enrollment through study completion, an average of about 24-30 months.)
- Clinical Response (antibiotic courses for non- NTM exacerbations)(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.)
研究者
Jerry A. Nick, M.D.
Director, Adult Cystic Fibrosis Program
National Jewish Health