Clinical trial NCT02419989: PATIENCE Trial: Prospective Algorithm for Treatment of NTM in Cystic Fibrosis
Official title: Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis
Study type: OBSERVATIONAL
Overall status: ENROLLING_BY_INVITATION
Conditions: Cystic Fibrosis
Keywords: Nontuberculous Mycobacteria
Lead sponsor: National Jewish Health
Sponsor class: OTHER
Collaborators: name: Cystic Fibrosis Foundation
name: Children's Hospital Colorado
First posted: 2015-04-17
Enrollment: 70
Sex: ALL
Minimum age (years): 6
Maximum age (years): 99
Standard ages: CHILD
ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Observational model: CASE_ONLY
Time perspective: PROSPECTIVE
Sampling method: NON_PROBABILITY_SAMPLE
Study population: Male and female subjects with CF age 6 years and older with a confirmed diagnosis of NTM disease who are initiating treatment for NTM.

Brief summary: Isolation of nontuberculous mycobacteria (NTM) from the sputum of individuals with CF is an increasingly common finding, and the lack of an evidenced-based approach to treatment of NTM disease has been identified as one of the greatest unmet needs within the CF community. Current evidence predicts that the prevalence of NTM will remain relatively high in the CF population. Approaches to NTM disease treatment differ widely between centers, and expected outcomes are not known. This study is observational and follows current best practices. The study will help define response to treatment, and collect relevant data associated with treatment of NTM disease to build a framework for future therapeutic trials.


Eligibility criteria: Inclusion Criteria:

1. Previous participation in PREDICT and diagnosis of NTM disease for the same species of NTM
2. Intention to treat for NTM disease for M. avium complex or M. abscessus complex
3. Written informed consent (and assent when applicable) obtained from participant or participant's legal representative
4. Signed informed consent to participate in data submission to the CFF Patient Registry
5. Be willing and able to initiate treatment for NTM and to adhere to study procedures in the context of clinical care, and other protocol requirements

Exclusion Criteria:

1. Pregnant or breastfeeding
2. 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: Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol
timeFrame: 12 months following end of antibiotic treatment
Secondary outcomes:
measure: Duration and combinations of NTM treatment regimens received
timeFrame: At end of treatment interval
measure: Proportion requiring antibiotic course change due to intolerance or lack of microbiological conversion (i.e. eradication from sputum)
timeFrame: At end of treatment interval
measure: Proportion who complete ≥ 12 months of treatment since first negative culture
timeFrame: At end of treatment interval
measure: Proportion eradicating NTM defined by ≥ 12 months of negative cultures since end of treatment
timeFrame: 12 months following end of treatment
measure: Variability between sites for NTM eradication success
timeFrame: 12 months following end of treatment
measure: Time to treatment discontinuation due to treatment success
timeFrame: 12 months following end of treatment
measure: Clinical outcomes (FEV1, growth parameters, CFQ-R)
timeFrame: 12 months following end of treatment
Other outcomes:
Locations:
city: Birmingham
country: United States
facility: CFF Pediatric Program, 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 San Diego
state: California
city: Aurora
country: United States
facility: Children's 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: CFF Adult Program, Johns Hopkins University
state: Maryland
city: Boston
country: United States
facility: Boston Children's Hospital, Brigham & Women's Hospital
state: Massachusetts
city: Ann Arbor
country: United States
facility: CFF Pediatric Program 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: CFF Pediatric Program, Columbia University
state: New York
city: Chapel Hill
country: United States
facility: CFF Adult Program, 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
state: Pennsylvania
city: Dallas
country: United States
facility: CFF Adult Program, The University of Texas Southwestern Medical Center at Dallas
state: Texas
city: Burlington
country: United States
facility: University of Vermont Medical Center
state: Vermont
city: Seattle
country: United States
facility: CFF Pediatric Program Seattle Children's Hospital
state: Washington
city: Seattle
country: United States
facility: CFF Adult Program, University of Washington
state: Washington
Study officials:
affiliation: National Jewish Health
name: Jerry A Nick, MD
role: PRINCIPAL_INVESTIGATOR
affiliation: Children's Hospital Colorado
name: Stacey Martiniano, MD
role: PRINCIPAL_INVESTIGATOR
References: