A Study of the Safety and Effectiveness of Inhaled Mannitol for Adult Patients with Cystic Fibrosis

Overview

  • Study type

    Interventional
  • Study phase

    III
  • Study IDs

  • Describes the nature of a clinical study. Types include:

    • Observational study — observes people and measures outcomes without affecting results.
    • Interventional study (clinical trial) — studies new tests, treatments, drugs, surgical procedures or devices.
    • Medical records research — uses historical information collected from medical records of large groups of people to study how diseases progress and which treatments and surgeries work best.
  • During the early phases (phases 1 and 2), researchers assess safety, side effects, optimal dosages and risks/benefits. In the later phase (phase 3), researchers study whether the treatment works better than the current standard therapy. They also compare the safety of the new treatment with that of current treatments. Phase 3 trials include large numbers of people to make sure that the result is valid. There are also less common very early (phase 0) and later (phase 4) phases. Phase 0 trials are small trials that help researchers decide if a new agent should be tested in a phase 1 trial. Phase 4 trials look at long-term safety and effectiveness, after a new treatment has been approved and is on the market.

  • Site IRB
    • Rochester, Minnesota: 15-007817
    NCT ID: NCT02134353
    Sponsor Protocol Number: DPM-CF-303

About this study

The purpose of this study is to provide evidence of the safety and effectiveness of inhaled mannitol for adult patients with cystic fibrosis.

 

Participation eligibility

Participant eligibility includes age, gender, type and stage of disease, and previous treatments or health concerns. Guidelines differ from study to study, and identify who can or cannot participate. If you need assistance understanding the eligibility criteria, please contact the study team.

See eligibility criteria

Inclusion Criteria

  • Have given written informed consent to participate in this trial in accordance with local regulations
  • Have a confirmed diagnosis of cystic fibrosis (positive sweat chloride value ≥ 60 mEq/L) and/or genotype with two identifiable mutations consistent with CF, accompanied by one or more clinical features consistent with the CF phenotype)
  • At least 18 years old
  • Have FEV1 > 40 % and < 90% predicted (using NHanes III [1])
  • Able to perform all the techniques necessary to measure lung function
  • Adherent with maintenance therapies (antibiotics and or rhDNase), if used, for at least 80% of the time in the two weeks prior to visit 1
  • If rhDNase and/or maintenance antibiotic are being used treatment must have been established at least 1 month prior to screening visit 0.
  • The subject should remain on the rhDNase and / or maintenance antibiotics for the duration of the trial.
  • The subject should not start treatment with rhDNase or maintenance antibiotics during this trial

Exclusion Criteria

  • Investigators, site personnel directly affiliated with this trial, or their immediate families. Immediate family is defined as a spouse, parent, child or sibling, whether biologically or legally adopted
  • Considered "terminally ill" or eligible for lung transplantation
  • Have had a lung transplant
  • Used a maintenance nebulized hypertonic saline in the 2 weeks prior to visit 1
  • Have had a significant episode of hemoptysis (> 60 mL) in the three months prior to visit 0
  • Have had a myocardial infarction in the three months prior to visit 0
  • Have had a cerebral vascular accident in the three months prior to visit 0
  • Have had major ocular surgery in the three months prior to visit 0
  • Have had major abdominal, chest or brain surgery in the three months prior to visit 0
  • Have a known cerebral, aortic or abdominal aneurysm
  • Are breast feeding or pregnant, or plan to become pregnant while in the trial
  • Are using an unreliable form of contraception (female subjects at risk of pregnancy only)
  • Are participating in another investigative drug trial, parallel to, or within 4 weeks of screening visit 0
  • Have a known allergy to mannitol
  • Are using non-selective oral beta blockers
  • Have uncontrolled hypertension -i.e. systolic BP > 190 and / or diastolic BP > 100
  • Have a condition or in a situation which in the Investigator's opinion may put the subject at significant risk, may confound results or may interfere significantly with the subject's participation in the trial
  • Have a failed or incomplete MTT at trial entry

Participating Mayo Clinic locations

Study statuses change often. Please contact us for help.

Mayo Clinic Location Status

Rochester, Minn.

Mayo Clinic principal investigator

Mark Wylam, M.D.

Closed for enrollment

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CLS-20201314

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