Could a Sugar-Blocking drug help cystic fibrosis lungs?
NCT ID NCT02325362
First seen Jun 26, 2026 · Last updated Jun 27, 2026 · Updated 1 time
Summary
This study tested whether miglustat, a drug that blocks certain sugar processing, can improve salt movement in the noses of adults with cystic fibrosis who have the F508del gene mutation. Sixteen participants took miglustat for two weeks and a placebo for two weeks, with a washout period in between. The goal was to see if miglustat could restore function of the faulty CFTR protein.
What this could mean
Our plain-language read of the trial. This is informational only, not medical advice or a prediction.
- Active substance
- miglustat
- What this could lead to
- If it works, this could point toward a treatment that helps cystic fibrosis patients with the F508del mutation breathe easier.
- What could go wrong
- This is a very small, early study with only 16 people. It may not show clear benefit, and miglustat can cause side effects like diarrhea or tremors.
This is an AI summary of the original study and may miss details. Read our disclaimer.
Study facts
What this study's own registry entry says, in plain language.
- Phase
-
Phase 2/3
Runs two stages together: whether the treatment works, then large-scale confirmation.
- Participants
-
16 people
The number who actually took part.
- Started
-
Mar 2015
- Finished
-
Apr 2017
- Lead sponsor
-
Other sponsor
The registry's catch-all category, for sponsors it does not file as a company, a government agency, or a research network.
Who can take part
This study's own entry requirements. Only the study team can say for certain whether you qualify.
- Ages
-
18 years and older
- Sex
-
Anyone
- Healthy volunteers
-
Not accepted
This study is not open to healthy volunteers. The entry requirements below say who it is open to.
Show the full entry requirements Hide the full entry requirements
Copied word for word from the study's registry entry, so the wording is the study team's rather than ours.
Inclusion Criteria: Inclusion criteria at screening visit (Visit 1): * Aged 18 years and older * Male or female * Women of childbearing potential must: * have a negative serum pregnancy test at Visit 1 * agree to use from Visit 1 until 3 months after the last study drug intake a reliable method of contraception * Male patients accepting for the duration of the study and for 3 months thereafter to use a condom * Homozygous for the F508del mutation as confirmed by genetic testing * Sweat chloride ≥ 60 mmol/L * Basal nasal potential difference (NPD) ≤ -30.0 mV (equal to or more electrically negative than -30.0 mV) and total chloride secretion (TCS) ≥ - 5.0 mV for at least one nostril. However, if it is possible to analyze both nostrils, the total chloride secretion (TCS) is to be ≥ - 5.0 mV (equal to or more electrically positive than - 5.0 mV) in both nostrils. * FEV1 ≥ 25% of predicted * Able to comply with all protocol requirements * Signed informed consent prior to any study-mandated procedure Inclusion criteria at randomization visit (Visit 2): * Women of child-bearing potential must have a negative urine pregnancy test * Basal nasal potential difference (NPD) ≤ - 30.0 mV (equal to or more electrically negative than - 30.0 mV) and total chloride secretion (TCS) ≥ - 5.0 mV for at least one nostril. However, if it is possible to analyze both nostrils, the total chloride secretion (TCS) is to be ≥ - 5.0 mV (equal to or more electrically positive than - 5.0 mV) in both nostrils. Exclusion Criteria: * Any condition prohibiting the correct measurement of the NPD such as upper respiratory tract infection * Acute upper or lower respiratory tract infection requiring antibiotic intervention within 2 weeks of screening * Lung transplant recipient or patient on a lung transplant waiting list * Any modification in regular treatments (new treatment initiated or discontinued treatment) or modification in dosing within 2 weeks prior to start of Period 1 * Moderate/Severe renal impairment (creatinine clearance \< 70 mL/min as per Cockroft and Gault) * Systemic corticosteroids (\> 10 mg/day prednisone or equivalent) within 14 days prior to screening and up to start of study * Women who are breast-feeding, pregnant, or who plan to become pregnant during the course of the study * History of significant lactose intolerance * Presence of clinically significant diarrhoea (\> 3 liquid stools per day for \> 7 days) without definable cause within one month prior to screening * Any known factor or disease that might interfere with treatment compliance, study conduct or interpretation of the results such as drug or alcohol dependence or psychiatric disease * Active or passive smoking * Hypersensitivity to Miglustat or any excipients * Planned treatment or treatment with another investigational drug or therapy (e.g., gene therapy) within one month prior to randomization * Known concomitant life-threatening disease with a life expectancy \< 12 months * Indication against Isuprel® (Isoproterenol) including heart diseases.
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Conditions
The condition(s) this trial relates to.
As listed by the trial registrant
The condition terms exactly as the trial's registrant entered them.
Contacts and locations
Locations
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Assistance publique-Hôpitaux de Paris, Hôpital Cochin
Paris, 75014, France
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Other studies related to the condition(s) this trial covers.
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