A study to evaluate the safety, tolerability, and efficacy of galicaftor/navocaftor/ABBV-119 combination therapy in subjects with cystic fibrosis

 Cystic Fibrosis / Posted 2 months ago

Cystic Fibrosis (CF) is a rare, life-threatening, genetic disease that affects the lungs and digestive system, significantly impairing the quality of life, with those affected having a median age of death at 40. The main objective of this study is to assess how safe and effective is the combination therapy of galicaftor/navocaftor/ABBV-119 in adult participants with CF.

 

Galicaftor/navocaftor/ABBV-119 combination therapy is being developed as an investigational drug for the treatment of CF. Study doctors place participants in 1 of the 4 groups, called treatment arms. Each group receives a different treatment. Around 90 adult participants with a diagnosis of CF will be enrolled in the study around approximately 35 sites worldwide.

Participants in arm 1 will receive oral capsules of galicaftor/navocaftor dual combination for 28 days followed by galicaftor/navocaftor/ABBV-119 triple combination for 28 days. Participants in arms 2 and 3 will receive the galicaftor/navocaftor/ABBV-119 triple combination or placebo for 28 days. Participants in arm 4 will receive galicaftor/navocaftor/ABBV-119 triple combination therapy for 28 days. For all study arms, galicaftor, navocaftor, will be given once daily and ABBV-119 twice a day.

  • Inclusion Criteria :
    • 1. Confirmed clinical diagnosis of CF, and genotype homozygous for the F508del CFTR mutation for Cohort 1 and Cohort 3, heterozygous for F508del CFTR mutation and a minimal function mutation for Cohort 2 and Cohort 3.
    • 2. ppFEV1 ≥40% and ≤90% of predicted normal for age, gender, and height (Global Lung Function Initiative [GLI] equations) at Screening.
    • 3. No clinically significant laboratory values at Screening that would pose undue risk for the subject or interfere with safety assessments (per the investigator).
    • 4. Absence of clinically significant abnormality detected on ECG regarding rate, rhythm, or conduction (e.g., QT interval corrected for heart rate using Fridericia's formula [QTcF] should be <450 msec for males and <460 msec for females).
    • 5. Stable pulmonary status, i.e., no respiratory infections or exacerbations requiring a change in therapy (including antimicrobials) or causing an acute decline in ppFEV1 of >10% from usual ppFEV1 level within 4 weeks.
    • 6. SwCl at screening visit must be ≥60 mmol/L for Cohort 1 and Cohort 2, and this criterion does not apply to Cohort 3.
    • 7. No history of diseases aggravated or triggered by ultraviolet radiation and no history of abnormal reaction photosensitivity or photoallergy to sunlight, or artificial source of intense light, especially ultraviolet light.
  • Exclusion Criteria :
    • 1. Cirrhosis with or without portal hypertension (e.g., splenomegaly, esophageal varices) or history of clinically significant liver disease
    • 2. History of malignancy within past 5 years (except for excised basal cell carcinoma of the skin with no recurrence, or treated carcinoma in situ of the cervix with no recurrence)
    • 3. Recent (within the past 6 months) history of drug or alcohol abuse that might preclude adherence to the protocol, in the opinion of the investigator
    • 4. Smoking or vaping tobacco or cannabis products within 6 months before Screening
    • 5. History of solid organ or hematopoietic transplantation
    • 6. History of known sensitivity to any component of the study drug
    • 7. Need for supplemental oxygen while awake, or >2 L/minute while sleeping.
    • 8. Evidence of active SARS-CoV-2 infection. If a subject has signs/symptoms suggestive of SARS CoV-2 infection, they should undergo molecular (e.g., polymerase chain reaction [PCR]) testing to rule out SARS-CoV-2 infection.
  • Study end date : December 2022
  • Wales-Based Study Contact : Please speak to your clinician
  • Principal Investigator : Dr Daniel Peckham (St James University Hospital, Leeds)
Contact details

All Wales Adult Cystic Fibrosis Centre, Penlan RoadUniversity Hospital Llandough,Cardiff,CF64 2XX  Show Phone Number Daniel.Peckham@nhs.net cavuhb.nhs.wales

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