A Prospective Study to Evaluate Biological and Clinical Effects of Significantly Corrected CFTR Function in Infants and Young Children (BEGIN)

Enrollment in this study is closed.

The purpose of this study is to assess the impacts of highly effective modulator therapy on different health measures that are affected by CF.

Primary Investigator: Jillian Sullivan, MD

Research Coordinator: Julie Sweet, CCRC 

• Children <10 years old with a diagnosis of CF
• Visits are timed with standard of care CF clinical visits, assessments include sweat chloride testing, blood, urine, stool and throat swab biospecimen collection, spirometry, optional bronchoalveolar lavage (BAL)
• Compensation: up to $700

Sinus Disease in Young Children with Cystic Fibrosis (BEGIN-S) 

Enrollment in this study is closed.

The purpose of this study is to assess the impacts of modulator therapy on sinuses in children with CF using sinus MRI 

Primary Investigator: Jillian Sullivan, MD

Research Coordinator: Julie Sweet, CCRC 

• Children <10 years old with a diagnosis of CF
• Sinus MRI, questionnaires
• Compensation: up to $300

Streamlined Treatment of Pulmonary Exacerbations in Pediatrics Randomized Control Trial (STOP PEDS RCT) 

The purpose of this study is to evaluate the efficacy of tailored therapy vs immediate antibiotics during a CF pulmonary exacerbation. 

Primary Investigator: Thomas Lahiri, MD 

Coordinator: Vanessa Marascio, MSc, MPH 

• Patients diagnosed with CF aged 3 to <19, able to access the internet/receive text messages
• Visits are timed with standard clinical visits, weekly symptom questionnaires, multiple breath washout testing, respiratory illness follow up
• Compensation: up to $685.00.

A Phase III open label extension study of Next-generation CFTR corrector VX121 in triple combination with d-Iva and Tezacaftor in cystic fibrosis patients

This study is closed to enrollment.

The purpose of this study is to evaluate the long-term safety and efficacy of VX121 triple combo therapy for use in children age 6 years and older with CF 

Primary Investigator: Thomas Lahiri, MD 

Coordinator: Julie Sweet, CCRC and Vanessa Marascio, MSc, MPH 

• Individuals diagnosed with CF age 6 years and older who have completed the preceding VX21-121-105 study.
• 192-week treatment period with a 28-day follow-up, assessments include sweat chloride testing, blood tests, EKG, lung function tests, other biospecimen collection
• Compensation: up to $1272.00.

A Phase III open label extension study of Next-generation CFTR corrector VX121 in triple combination with d-Iva and Tezacaftor in cystic fibrosis patients

This study is closed to enrollment.

The purpose of this study is to evaluate the efficacy of VX121 triple combo therapy for use in CF in children aged 2-5 years old.

Primary Investigator: Thomas Lahiri, MD 

Coordinator: Vanessa Marascio, MSc, MPH 

• Individuals diagnosed with CF aged 2-5 years old homozygous for F508del or heterozygous with 1 CFTR mutation that is responsive to triple combination therapy
• 96-week treatment period with a 28 day follow-up, assessments include sweat chloride testing, blood tests, EKG, lung function tests, other biospecimen collection
• Compensation: up to $990.00

The purpose of this study is to develop a standard diagnosis path for nontuberculous mycobacteria disease and a treatment algorithm in CF patients 

Primary Investigator: Zachary Weintraub, MD 

Coordinator: Vanessa Marascio, MSc, MPH 

• Patients diagnosed with CF, at least one NTM+ culture in the 2 years prior to enrolling that hasn’t been treated
• Visits are timed with standard clinical visits. Questionnaires, and annual biospecimens (blood, urine, sputum) are collected
• Compensation: $60 baseline visit, $15 research questionnaire, $30 research specimen

 The purpose of this study is to assess and evaluate potential sources of NTM acquisition  

Primary Investigator: Charlotte Teneback, MD  

Coordinator: Vanessa Marascio, MSc, MPH 

• All CF patients with an NTM+ culture
• No visits required, signing consent to use positive cultures in research  
• Compensation: none

This study is closed to enrollment.

 The purpose of this study is to evaluate the efficacy of VX121 triple combo therapy for use in CF   

Primary Investigator: Charlotte Teneback, MD  

Coordinator: Julie Sweet, CCRC 

• Individuals diagnosed with CF aged 12 years and older homozygous for F508del or heterozygous with 1 CFTR mutation that is responsive to triple combination therapy
• 144-week treatment period with a 28-day follow-up, assessments include sweat chloride testing, blood tests, EKG, lung function tests, other biospecimen collection   
• Compensation: up to $1815

The purpose of this study is to evaluate the safety and efficacy of new CFTR modulator VX-828 in triple combination with tezacaftor and deutivacaftor in adults diagnosed with CF   

Primary Investigator: Charlotte Teneback, MD  

Coordinator: Julie Sweet, CCRC 

• Adults with cystic fibrosis aged 18 and older and F508del/MF mutations, FEV1 must be 40+%
• Study treatment period of 28 days with visits 4-7 days apart, study drug wash out period of 52 days    
• Compensation: up to $2350

The purpose of this study is to evaluate the safety and efficacy of two, 5-day IV gallium infusions in adults with CF who culture NTM    

Primary Investigator: Zachary Weintraub, MD   

Coordinator: Julie Sweet, CCRC 

• Adults with CF 18 years and older who have 2 consecutive positive NTM cultures for same species in the absence of NTM disease diagnosis
• Two series of daily infusions of IV gallium over the course of 5 days each, spirometry, questionnaires, blood and sputum biospecimen collection    
• Compensation: up to $1620