Cystic FibrosisPATIENCENCT02419989
PATIENCE Trial: Prospective Algorithm for Treatment of NTM in Cystic Fibrosis
An observational study of Cystic Fibrosis, run by National Jewish Health at 19 sites in United States.
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. Read more about this study
- Who is it for?
- Ages 6 to 99 years
- Conditions the study is for
- Cystic Fibrosis
- How many people
- 70 participants
- Where
- 19 sitesUnited States
- Study dates
- Jan 2014to Mar 2025
- Phase
- Observational
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 6 to 99 years
- All sexes
- No healthy volunteers
- Child, Adult, Older adult
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.
Who may be able to join
- Previous participation in PREDICT and diagnosis of NTM disease for the same species of NTM
- Intention to treat for NTM disease for M. avium complex or M. abscessus complex
- Written informed consent (and assent when applicable) obtained from participant or participant's legal representative
- Signed informed consent to participate in data submission to the CFF Patient Registry
- 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
Who cannot join
- Pregnant or breastfeeding
- 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
Trial locations and contacts
19 sites in 1 country.
19 sites in 1 country
United States19 sites
CFF Pediatric Program University of Michigan
Ann Arbor, Michigan, 48109
Children's Hospital Colorado
Aurora, Colorado, 80045
CFF Adult Program, Johns Hopkins University
Baltimore, Maryland, 21205
CFF Pediatric Program, University of Alabama
Birmingham, Alabama, 35233
Boston Children's Hospital, Brigham & Women's Hospital
Boston, Massachusetts
University of Vermont Medical Center
Burlington, Vermont
CFF Adult Program, University of North Carolina at Chapel Hill
Chapel Hill, North Carolina, 27517
Northwestern University
Chicago, Illinois
Nationwide Children's Hospital
Columbus, Ohio
CFF Adult Program, The University of Texas Southwestern Medical Center at Dallas
Dallas, Texas, 75390-8558
National Jewish Health
Denver, Colorado, 80206
University of Florida
Gainesville, Florida
Dartmouth Hitchcock Medical Center
Lebanon, New Hampshire
Children's Hospital of Los Angeles
Los Angeles, California
CFF Pediatric Program, Columbia University
New York, New York, 10032
University of Pittsburgh
Pittsburgh, Pennsylvania
University of California San Diego
San Diego, California
CFF Pediatric Program Seattle Children's Hospital
Seattle, Washington, 98105
CFF Adult Program, University of Washington
Seattle, Washington, 98195
About this study
Official title: Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis
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.
- Conditions
- Cystic Fibrosis
- Keywords
- Nontuberculous Mycobacteria
- Medical subject headings
- Cystic Fibrosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
| Study group | Role | What they receive |
|---|---|---|
| CF patients | Male and female subjects with CF age 6 years and older who meet diagnostic criteria for NTM disease through participation in the PREDICT (Part A) study who are being offered NTM treatment. |
What is being measured
The primary outcome is the main question the study is designed to answer.
Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol
Measured: 12 months following end of antibiotic treatment
Secondary outcomes (7)
Duration and combinations of NTM treatment regimens received
Measured: At end of treatment interval
Proportion requiring antibiotic course change due to intolerance or lack of microbiological conversion (i.e. eradication from sputum)
Measured: At end of treatment interval
Proportion who complete ≥ 12 months of treatment since first negative culture
Measured: At end of treatment interval
Proportion eradicating NTM defined by ≥ 12 months of negative cultures since end of treatment
Measured: 12 months following end of treatment
Variability between sites for NTM eradication success
Measured: 12 months following end of treatment
Time to treatment discontinuation due to treatment success
Measured: 12 months following end of treatment
Clinical outcomes (FEV1, growth parameters, CFQ-R)
Measured: 12 months following end of treatment
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted17 Apr 2015 (estimated)
- Study startJan 2014
- Primary completionMar 2025 (estimated)
- Study completionMar 2025 (estimated)
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Cystic FibrosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Observational
- Observational model
- Case only
- Time perspective
- Prospective
- Enrolment
- 70 participants (estimated)
Sponsor and investigators
- Lead sponsor
- National Jewish Health
- Sponsor type
- University, hospital or other
- Collaborators
- Cystic Fibrosis Foundation, Children's Hospital Colorado
- Responsible party
- Principal investigator, Jerry A. Nick, M.D., National Jewish Health
- Investigators
- Jerry A Nick, MD, Principal investigator, National Jewish HealthStacey Martiniano, MD, Principal investigator, Children's Hospital Colorado
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT02419989
- Sponsor's protocol ID
- NTM-OB-17 (PATIENCE PART B)
- Acronym
- PATIENCE
- Status last verified
- Mar 2024
- First submitted
- 9 Dec 2014
- Last updated
- 2 Apr 2024
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- No
- FDA-regulated device
- No
Study at a glance
From the registry record, last updated by the sponsor on 2 Apr 2024. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Prospective Evaluation of a Standardized Approach to Diagnosis (PREDICT) and Treatment (PATIENCE) of Nontuberculous Mycobacteria Disease in Cystic Fibrosis
- ClinicalTrials.gov ID
- NCT02419989
- Acronym / sponsor ID
- PATIENCE / NTM-OB-17 (PATIENCE PART B)
- Conditions
- Cystic Fibrosis
- Keywords
- Nontuberculous Mycobacteria
- Study type
- Observational
- Recruitment status
- Unknown status
- Enrolment
- 70 participants (estimated)
- Lead sponsor
- National Jewish HealthUniversity, hospital or other
- Collaborators
- Cystic Fibrosis Foundation, Children's Hospital Colorado
- Study start
- Jan 2014 (actual)
- Primary completion
- Mar 2025 (estimated)
- Study completion
- Mar 2025 (estimated)
- First posted
- 17 Apr 2015 (estimated)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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.
Study design
- Observational model
- Case only
- Time perspective
- Prospective
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| CF patients | – | –Male and female subjects with CF age 6 years and older who meet diagnostic criteria for NTM disease through participation in the PREDICT (Part A) study who are being offered NTM treatment. |
Who may qualify
- Ages
- 6 to 99 years
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Child, Adult, Older adult
- 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.
Key inclusion criteria (5 listed)
- Previous participation in PREDICT and diagnosis of NTM disease for the same species of NTM
- Intention to treat for NTM disease for M. avium complex or M. abscessus complex
- Written informed consent (and assent when applicable) obtained from participant or participant's legal representative
- Signed informed consent to participate in data submission to the CFF Patient Registry
- 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
Key exclusion criteria (2 listed)
- Pregnant or breastfeeding
- 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
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Primary outcome
Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol
Time frame: 12 months following end of antibiotic treatment
7 secondary outcomes
Duration and combinations of NTM treatment regimens received
Time frame: At end of treatment interval
Proportion requiring antibiotic course change due to intolerance or lack of microbiological conversion (i.e. eradication from sputum)
Time frame: At end of treatment interval
Proportion who complete ≥ 12 months of treatment since first negative culture
Time frame: At end of treatment interval
Proportion eradicating NTM defined by ≥ 12 months of negative cultures since end of treatment
Time frame: 12 months following end of treatment
Variability between sites for NTM eradication success
Time frame: 12 months following end of treatment
Time to treatment discontinuation due to treatment success
Time frame: 12 months following end of treatment
Clinical outcomes (FEV1, growth parameters, CFQ-R)
Time frame: 12 months following end of treatment
Results and safety
Results are not available in the source record.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 2 Apr 2024.
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
Jerry A Nick, MD principal investigator
National Jewish Health
Stacey Martiniano, MD principal investigator
Children's Hospital Colorado
19 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
An observational study of Cystic Fibrosis, run by National Jewish Health at 19 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 5 inclusion and 2 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: status not confirmed, as of the sponsor's last update on 2 Apr 2024. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 19 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
Are results available for this study?
Results are not available in the source record.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Proportion who adhere to the protocol based on: number of respiratory cultures obtained per year, withdrawals or major deviations from protocol (time frame: 12 months following end of antibiotic treatment). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 2 Apr 2024.