Skip Navigation

Cystic Fibrosis Research Program

On This Page:

The Johns Hopkins CF program is actively engaged in many different research studies covering everything from the genetics underlying cystic fibrosis, the diagnosis of CF, symptom management in CF, adherence to CF treatments, improving lung transplant outcomes in CF, and numerous clinical trials of CF therapies. We have been involved in most major trials of new therapeutic agents for CF over the past two decades. Some ongoing studies are highlighted below. Because there are new clinical trials in CF starting frequently, you can find the latest trials at the CF Foundations website

If you are interested in learning more about CF research at Johns Hopkins you can contact Mary Rykiel at 410-614-1409 or

Johns Hopkins Lung Transplant Research Project List

View complete list of lung transplant studies.

  • We have conducted a series of cohort studies aimed at determining the prevalence of pain in adults and adolescents with CF and determining the impact of pain on clinical outcomes in CF. Future aims include optimizing the assessment of pain in CF and designing interventions to alleviate pain for individuals with CF.

    Team: Sarah Allgood, Noah Lechtzin

    Funding: NIH

  • This series of studies utilizes data from the United Network for Organ Sharing (UNOS) and the Cystic Fibrosis Foundation Patient Registry to determine how various risk factors such as renal function and lung allocation score impact clinical outcome after lung transplantation.

    Team: Christian Merlo, Errol Bush, Pali Shah

  • This study aims to use the CF Foundation Patient Registry to determine which aspects of guideline recommended care are most important for improving clinical outcomes in CF.

    Team: Rebecca Dezube, Kristin Riekert, Noah Lechtzin

  • The adult CF group has been involved with many phase 1, phase 2, and phase 3 clinical trials of new treatments for CF. Recent and ongoing studies are below:

    • A Phase 3, Randomized, Double-blind, Controlled Study Evaluating the Efficacy and Safety of VX-659 Combination Therapy in Subjects With Cystic Fibrosis Who Are Heterozygous for the F508del Mutation and a Minimal Function Mutation
      • PI: Noah Lechtzin
      • Funding: Vertex Pharmaceuticals
    • A Phase Ib Randomized Blinded Placebo-Controlled, Cross-Over Study to Assess the Effect of AZD5634 on Mucociliary Clearance as Well as Safety, Tolerability, and Pharmacokinetic Parameters
      • PI: Noah Lechtzin
      • Funding: Astra Zeneca
    • Phase 2a study of Galapagos GLPG2222 in adults with CF
      • PI: Natalie West
      • Funding: Galapagos
    • Phase 2 Study of VX-445 combination drug in people with cystic fibrosis
      • PI: Christian Merlo
      • Funding: Vertex Pharmaceuticals
    • IGNITE: Safety and effectiveness of gallium nitrate in adults with cystic fibrosis
      • PI: Noah Lechtzin
      • Funding: FDA and CFF
    • Phase 2 Study of lenabasum in people with CF ages 12 and older
      • PI: Natalie West
      • Funding: Corbus
  • This study aims to validate the Barriers to Adherence Screener (BAS) and the Adherence Barriers Questionnaire- Cystic Fibrosis (ABQ-CF) to help identify challenges that people with CF face in getting treatments done.

    Team: Kristin Riekert, Noah Lechtzin

    Funding: Funding CF Foundation

  • The aim of these studies is to standardize non-tuberculous mycobacterium diagnostic approaches and then to standardize treatment regimens for individuals who require therapy.

    Team: Keira Cohen, Noah Lechtzin

    Funding: CF Foundation

    • STOP 2: Treatment of pulmonary exacerbations in people with CF.

    This study aims to determine the optimal duration of antibiotic therapy for treating CF pulmonary exacerbations.

    • PI: Natalie West
    • Funding: CF Foundation
    • Biomarkers of CF Exacerbations

    This is a prospective cohort study aimed at measuring novel biomarkers in blood and exhaled breath condensate with the goal of identifying markers that will identify patients who will have better responses to treatment than others.

    • PI: Natalie West
    • Funding: CF Foundation
  • This clinical trial aims to determine if a multifaceted antimicrobial approach can eradicate MRSA from respiratory cultures from people with CF.

    Team: Mark Jennings, Rebecca Dezube, Michael Boyle

    Funding: CF Foundation

  • This is a series of observational studies aimed to determine if there a sex differences in presentation, treatment and outcomes in CF Pulmonary Exacerbations.

    Team: Kristina Montemayor, Natalie West, Noah Lechtzin

    Funding: Pending

  • The Johns Hopkins Adherence Research Center serves as the Data Management Core for the STRC and the Hopkins Pediatric and Adult Centers are participating centers. The mission of the STRC is to facilitate the clinical study of interventions to improve adherence and CF disease self-management in order to optimize health outcomes and quality of life. There are several ongoing studies.

    Team: Kristin Riekert, Noah Lechtzin

    Funding: Funding CF Foundation

  • The aim of this study is to determine if oral azithromycin interferes with the antimicrobial effectiveness of inhaled tobramycin.

    Team: Peter Mogayzel, Noah Lechtzin

    Funding: CF Foundation

  • The project aims to test and validate a wearable sensor that provides real time testing of sweat chloride. Sweat chloride is a marker of CFTR function and has been shown to decrease in response to CFTR modulator drugs. This study will follow serial sweat measurements after initiation of CFTR modulator drugs in order to gain insight into the time course of sweat chloride changes and how these vary around dosing of medications.

    Team: Mark Jennings, Garry Cutting, DH Choi

    Funding: CF Foundation

Clinical Research

Clinical Research

  • Phase 1, 2, & 3 clinical trials of novel therapeutic agents
  • Interventions to improve adherence in CF
  • Assessments of Pain in CF
  • Optimizing CF Exacerbation Treatment
  • Observational Study of Non-tuberculous mycobacteria
  • Improving pre & post-transplant care for CF
  • Studies of outcomes and interventions for MRSA infection
  • Evaluating sex difference in exacerbation treatment and outcomes
  • Evaluation of a continuous sweat chloride monitor
  • Comparative effectiveness research of CF Guideline care

Patient Resources/Useful Links

Cystic Fibrosis Physicians


I want to...

back to top button