It is essential for clinicians to remain informed about emerging clinical trial opportunities and resulting data that may inform therapeutic strategies, affect clinical guidelines, expand treatment options, and ultimately improve patient quality of life.
The following clinical trials preview features six US-based clinical trials focusing on cystic fibrosis with upcoming anticipated completion dates. The ranges of anticipated primary completion dates range from June 2026 through December 2030. The following trials are arranged in order of their start date.
Quick View Table of Cystic Fibrosis Clinical Trials
| Study Title | Phase | Recruiting? | Estimated Enrollment | Start Date | Estimated Primary Completion |
|---|---|---|---|---|---|
| Assessing Tenapanor as a Treatment of CF-related Constipation | 3 | Recruiting | 25 | June 2025 | June 2026 |
| Ensuring Access to Optimal Therapy in CF: The ENACT Study (ENACT) | 4 | Recruiting | 95 | June 2025 | December 2030 |
| Maralixibat in Patients With Cystic Fibrosis and Constipation | 2,3 | Recruiting | 20 | April 2025 | June 2027 |
| Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older | 3 | Enrolling by Invitation | 174 | August 2023 | July 2029 |
| A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy | 3 | Active, Not Recruiting | 822 | November 2022 | October 2026 |
| BEGIN Novel ImagiNG Biomarkers (BEGINNING) | 4 | Recruiting | 44 | May 2022 | November 2028 |
Featured Cystic Fibrosis Clinical Trials
Clinical Trial Name: Assessing Tenapanor as a Treatment of CF-related Constipation
- Sponsor: Massachusetts General Hospital
- Goal: Understand tenapanor as a treatment for cystic fibrosis-related constipation (CFrC) in CF patients.
- Intervention: Tenapanor
- Primary Outcome Measure: Increase in SBM frequency.
- Time Frame: Patients will record SBM frequency over the four-week treatment period.
- Study Details
- Source
Ensuring Access to Optimal Therapy in CF: The ENACT Study (ENACT)
- Sponsor: Arkansas Children's Hospital Research Institute
- Goal: Examining the action and effects of several new drugs in the treatment of cystic fibrosis in children. In addition, several genetic factors are examined.
- Interventions: Elexacaftor / Ivacaftor / Tezacaftor; therapeutic drug monitoring
- Primary Outcome Measure: Concentration (ng/mL).
- Time Frame: One time assessment for observational part of the study, up to six times (six months or more) for the therapeutic drug monitoring pilot and feasibility study.
- Study Details
- Source
Clinical Trial Name: Maralixibat in Patients With Cystic Fibrosis and Constipation
- Sponsor: Children's Hospital Los Angeles
- Goal: Determine whether Maralixibat improves stool consistency to a Bristol Stool Scale score >4. The secondary objective is to assess changes in ease of defecation using standardized questionnaires.
- Interventions: Maralixibat 9.5 MG/ML [Livmarli]
- Primary Outcome Measures: Change in stool consistency measured by the Bristol Stool Scale.
- Time Frame: Baseline to four weeks.
- Study Details
- Source
Evaluation of Long-Term Safety and Efficacy of Vanzacaftor/Tezacaftor/Deutivacaftor in Cystic Fibrosis Participants 1 Year of Age and Older
- Sponsor: Vertex Pharmaceuticals Incorporated
- Goal: Evaluate the long-term safety, tolerability, and efficacy of vanzacaftor/tezacaftor/deutivacaftor (VNZ/TEZ/D-IVA) in participants with cystic fibrosis (CF).
- Intervention: VNZ/TEZ/D-IVA
- Primary Outcome Measures: Part A: Safety and tolerability as assessed by number of participants with adverse events (AEs); Part B: Safety and tolerability as assessed by number of participants with adverse events (AEs).
- Time Frame: From baseline up to week 100; From baseline up to week 196.
- Study Details
- Source
A Study Evaluating the Long-term Safety and Efficacy of VX-121 Combination Therapy
- Sponsor: Vertex Pharmaceuticals Incorporated
- Goal: Evaluate the long-term safety, tolerability, and efficacy of VX-121/tezacaftor/deutivacaftor (VX-121/TEZ/D-IVA) in participants with cystic fibrosis.
- Intervention: VX-121/TEZ/D-IVA
- Primary Outcome Measure: Safety and tolerability as assessed by number of participants with adverse events (AEs) and serious adverse events (SAEs).
- Time Frame: From baseline up to week 148.
- Study Details
- Source
BEGIN Novel ImagiNG Biomarkers (BEGINNING)
- Sponsor: Children's Hospital Medical Center, Cincinnati
- Goal: Determine the treatment effect of triple-combination therapy in 6-8 year olds after presumed FDA approval, using rapid structural and functional pulmonary and abdominal MRI (UTE and 129Xe).
- Intervention: 129Xe
- Primary Outcome Measures: Ventilation defect percentage change from baseline; Pancreas volume.
- Time Frames: 1 year; 1 year.
- Study Details
- Source
Related Clinical Guidance:
- Diagnosis of Cystic Fibrosis
- Society: Cystic Fibrosis Foundation
- Date Published: February 2017
- Cystic Fibrosis Evidence-Based Nutrition Recommendations
- Society: Academy of Nutrition and Dietetics
- Date Published: May 2020
- Best Practices in the Management of the Gastrointestinal Aspects of Cystic Fibrosis
- Society: Cystic Fibrosis Foundation
- Date Published: November 2017
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