PATIENCE Trial: Prospective Algorithm for Treatment of NTM in Cystic Fibrosis

Cystic Fibrosis Clinical Trial

— PATIENCE  

Official title:

Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis

Clinical Trial Details — Status: Enrolling by invitation

Administrative data

• NCT number: NCT02419989
• Other study ID #: NTM-OB-17 (PATIENCE PART B)
• Status: Enrolling by invitation
• Start date: January 2014
• Est. completion date: March 2025

Study information

• Verified date: March 2024
• Source: National Jewish Health
• Contact: n/a
• Is FDA regulated: No
• Study type: Observational

Clinical Trial 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.

Recruitment information / eligibility

• Status: Enrolling by invitation
• Enrollment: 70
• Est. completion date: March 2025
• Est. primary completion date: March 2025
• Accepts healthy volunteers: No
• Gender: All
• Age group: 6 Years to 99 Years

Eligibility

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

Related Conditions & MeSH terms

• Cystic Fibrosis
• Fibrosis

Locations

Country	Name	City	State
United States	CFF Pediatric Program University of Michigan	Ann Arbor	Michigan
United States	Children's Hospital Colorado	Aurora	Colorado
United States	CFF Adult Program, Johns Hopkins University	Baltimore	Maryland
United States	CFF Pediatric Program, University of Alabama	Birmingham	Alabama
United States	Boston Children's Hospital, Brigham & Women's Hospital	Boston	Massachusetts
United States	University of Vermont Medical Center	Burlington	Vermont
United States	CFF Adult Program, University of North Carolina at Chapel Hill	Chapel Hill	North Carolina
United States	Northwestern University	Chicago	Illinois
United States	Nationwide Children's Hospital	Columbus	Ohio
United States	CFF Adult Program, The University of Texas Southwestern Medical Center at Dallas	Dallas	Texas
United States	National Jewish Health	Denver	Colorado
United States	University of Florida	Gainesville	Florida
United States	Dartmouth Hitchcock Medical Center	Lebanon	New Hampshire
United States	Children's Hospital of Los Angeles	Los Angeles	California
United States	CFF Pediatric Program, Columbia University	New York	New York
United States	University of Pittsburgh	Pittsburgh	Pennsylvania
United States	University of California San Diego	San Diego	California
United States	CFF Adult Program, University of Washington	Seattle	Washington
United States	CFF Pediatric Program Seattle Children's Hospital	Seattle	Washington

Sponsors (3)

Lead Sponsor	Collaborator
National Jewish Health	Children's Hospital Colorado, Cystic Fibrosis Foundation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol		12 months following end of antibiotic treatment
Secondary	Duration and combinations of NTM treatment regimens received		At end of treatment interval
Secondary	Proportion requiring antibiotic course change due to intolerance or lack of microbiological conversion (i.e. eradication from sputum)		At end of treatment interval
Secondary	Proportion who complete = 12 months of treatment since first negative culture		At end of treatment interval
Secondary	Proportion eradicating NTM defined by = 12 months of negative cultures since end of treatment		12 months following end of treatment
Secondary	Variability between sites for NTM eradication success		12 months following end of treatment
Secondary	Time to treatment discontinuation due to treatment success		12 months following end of treatment
Secondary	Clinical outcomes (FEV1, growth parameters, CFQ-R)		12 months following end of treatment