Clinical trial NCT02073409: PREDICT Trial: PRospective Evaluation of NTM Disease 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: 2014-02-27
Enrollment: 200
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 who have a positive sputum culture 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 the diagnosis 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 diagnosis differ widely between centers. This study is observational and follows current best practices. The study will help standardize the diagnosis and collect relevant data associated with the diagnosis of NTM disease to build a framework for future therapeutic trials.


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 and able 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
5. Diagnosis of CF consistent with the 2017 CFF Guidelines
6. NTM positive for a species or sub-species in the 2 years prior to enrollment that has never been treated.
7. Willing to discontinue chronic azithromycin use for the duration of the study

Exclusion Criteria:

1. Prior or ongoing antibiotic treatment of the same NTM species or sub-species for which the patient is being considered for this study
2. History of solid organ or hematological transplantation
3. 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 meeting the diagnosis of NTM disease
timeFrame: 12 months intervals following study enrollment
Secondary outcomes:
measure: Adherence to PREDICT protocol: number of respiratory cultures obtained per participant per year, withdrawals and major deviations from protocol
timeFrame: 12 months intervals following study enrollment
measure: Incidence and prevalence of NTM species/subspecies by geographical region
timeFrame: 12 months intervals following study enrollment
measure: Proportion of eligible NTM positive individuals with CF enrolling in PREDICT by site
timeFrame: 12 months intervals following study enrollment
measure: Proportion of PREDICT participants with NTM disease diagnosis who enroll in Part B PATIENCE
timeFrame: 12 months intervals following study enrollment
measure: Clinical parameters and outcomes (FEV1, growth parameters, CFQ-R)
timeFrame: 12 months intervals following study enrollment
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 Medical Center
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: