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Cystic FibrosisPhase 2/3MIGLUSTAT-CFNCT02325362

Effect of Miglustat on the Nasal Potential Difference in Patients With Cystic Fibrosis Homozygous for the F508del Mutation

A Phase 2/3 study of Cystic Fibrosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.

Completed

Finished. The study has ended and is not enrolling. Results may be posted on the official record.

Official record
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Trial summary

The purpose of this study is to demonstrate that Miglustat restores the function of the cystic fibrosis transmembrane conductance regulator (CFTR) in adult patients with cystic fibrosis homozygous for the F508del mutation. Read more about this study

Who is it for?
Ages 18 years and older
Conditions the study is for
Cystic Fibrosis
Could I receive a placebo?
Yes, some participants may receive a placebo
How many people
16 participants
Where
1 siteFrance
Study dates
17 Mar 2015to 3 Apr 2017
Phase
Phase 2/3
Are there results?
None posted in the registry

Key requirements

Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.

  • Ages 18 years and older
  • All sexes
  • No healthy volunteers
  • Adult, Older adult

Who may be able to join

  • 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
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  • 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
  • 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.

Who cannot join

  • 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
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  • 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.

Trial locations and contacts

1 site in 1 country.

France1 site

  • Assistance publique-Hôpitaux de Paris, Hôpital Cochin

    Paris, 75014

Other trials in France

About this study

Official title: Single Center, Double-blind, Randomized, Placebo-controlled, Two-period/Two-treatment Crossover, Proof-of-mechanism Study Investigating the Effect of Miglustat on the Nasal Potential Difference in Adult Patients With Cystic Fibrosis Homozygous for the F508del Mutation

The purpose of this study is to demonstrate that Miglustat restores the function of the cystic fibrosis transmembrane conductance regulator (CFTR) in adult patients with cystic fibrosis homozygous for the F508del mutation.

Read the detailed description

The aims of this study are:

  1. To determine whether Miglustat can restore the function of the CFTR protein in adult patients with cystic fibrosis homozygous for the F508del mutation
  2. To evaluate the safety, tolerability and pharmacokinetics of Miglustat in adult patients with cystic fibrosis homozygous for the F508del mutation.
  3. To investigate pharmacokinetic-pharmacodynamic of Miglustat in adult patients with cystic fibrosis homozygous for the F508del mutation.
Conditions
Cystic Fibrosis
Being tested
Miglustat ; placebo, Placebo ; Miglustat
Keywords
Cystic fibrosis, CFTR, F508del mutation, Miglustat, Total Chloride Secretion, Nasal Potential Difference
Medical subject headings
Cystic Fibrosis

What participants receive

What participants receive. In a randomised study each person is assigned to one group by chance.

  • Miglustat ; placeboDrug

    For this 2 x 2 (2 periods /2 treatments) crossover design each patient will receive Miglustat during the first period (2 weeks), following by a wash out period(14 days (up to 4 weeks)), then Placebo during the second period (2 weeks). 30 days follow-up will be carried out after end-of-treatment of the second period.

  • Placebo ; MiglustatDrug

    For this 2 x 2 (2 periods /2 treatments) crossover design each patient will receive Placebo during the first period (2 weeks), following by wash out period (14 days (up to 4 weeks)), then Miglustat during the second period (2 weeks). 30 days follow-up will be carried out after end of treatment of the second period.

Study groups in NCT02325362
Study groupRoleWhat they receive
Miglustat then placeboExperimental10 patients will received Miglustat then the placebo
Receives: Drug: Miglustat ; placebo
Placebo then MiglustatExperimental10 patients will received Placebo then Miglustat
Receives: Drug: Placebo ; Miglustat

What is being measured

The primary outcome is the main question the study is designed to answer.

  1. Mean TCS in mV

    TCS (Total Chloride Secretion) is the sum of responses in nasal potential difference (NPD) calculated as the mean of the right and left nostril measurements for each patient

    Measured: day 1

  2. Mean TCS in mV

    TCS (Total Chloride Secretion) is the sum of responses in nasal potential difference (NPD) calculated as the mean of the right and left nostril measurements for each patient

    Measured: Day 14

Secondary outcomes (21)

  1. TCS difference in mV

    TCS difference is calculated as the change in measurements of TCS for the right and left nostrils independently for each patient.

    Measured: day 1

  2. TCS difference in mV

    TCS difference is calculated as the change in measurements of TCS for the right and left nostrils independently for each patient.

    Measured: Day 14

  3. Percentage of patients with a TCS response to treatment ≤ - 5 mV

    The percentage of patients with a TCS response to treatment defined as a difference in TCS from baseline to end-of-treatment ≤ -5mV

    Measured: day 1

  4. Percentage of patients with a TCS response to treatment ≤ - 5 mV

    The percentage of patients with a TCS response to treatment defined as a difference in TCS from baseline to end-of-treatment ≤ -5mV

    Measured: day 14

  5. Percentage of patients with a TCS at end-of-treatment ≤ - 5 mV

    The percentage of patients with a TCS response at end-of-treatment ≤ -5mV

    Measured: day 1

  6. Percentage of patients with a TCS at end-of-treatment ≤ - 5 mV

    The percentage of patients with a TCS response at end-of-treatment ≤ -5mV

    Measured: day 14

  7. Change of basal NPD in mV

    Basal NPD at end-of-treatment minus basal NPD at baseline

    Measured: day 1

  8. Change of basal NPD in mV

    Basal NPD at end-of-treatment minus basal NPD at baseline

    Measured: day 14

  9. Change of the response in NPD after superfusion with amiloride

    NPD after superfusion with amiloride at end-of-treatment minus NPD after superfusion with amiloride at baseline

    Measured: day 1

  10. Change of the response in NPD after superfusion with amiloride

    NPD after superfusion with amiloride at end-of-treatment minus NPD after superfusion with amiloride at baseline

    Measured: day 14

  11. Change of the response in NPD after superfusion with a chloride-free buffer in the presence of amiloride

    NPD after superfusion with a chloride-free buffer in the presence of amiloride at end-of-treatment minus NPD after superfusion with a chloride-free buffer in the presence of amiloride at baseline

    Measured: day 1

  12. Change of the response in NPD after superfusion with a chloride-free buffer in the presence of amiloride

    NPD after superfusion with a chloride-free buffer in the presence of amiloride at end-of-treatment minus NPD after superfusion with a chloride-free buffer in the presence of amiloride at baseline

    Measured: day 14

  13. Wilschanski's index change

    Wilschanski's index is defined as (exposant(response to Chloride-free and isoproterenol/response amiloride)): Wilschanski's index at end-of-treatment minus Wilschanski's at baseline

    Measured: day 1

  14. Wilschanski's index change

    Wilschanski's index is defined as (exposant(response to Chloride-free and isoproterenol/response amiloride)): Wilschanski's index at end-of-treatment minus Wilschanski's at baseline

    Measured: day 14

  15. Sweat chloride concentration in mmol/L

    Sweat chloride concentration at end-of-treatment minus sweat chloride concentration at baseline

    Measured: day 1

  16. Sweat chloride concentration in mmol/L

    Sweat chloride concentration at end-of-treatment minus sweat chloride concentration at baseline

    Measured: day 14

  17. FEV1 (in % of predicted)

    Pulmonary function FEV1: mean Forced expiry volume in 1 second. FEV1 at end-of-treatment minus FEV1 at baseline

    Measured: day 1

  18. FEV1 (in % of predicted)

    Pulmonary function FEV1: mean Forced expiry volume in 1 second. FEV1 at end-of-treatment minus FEV1 at baseline

    Measured: day 14

  19. Change in electrochemical skin conductance

    Electrochemical skin conductance at end-of-treatment minus electrochemical skin conductance at baseline

    Measured: day 1

  20. Change in electrochemical skin conductance

    Electrochemical skin conductance at end-of-treatment minus electrochemical skin conductance at baseline

    Measured: day 14

  21. Number of cells expressing CFTR at the cell membrane (in %percentage)

    Percentage of nasal cells expressing CFTR at the cell membrane as assessed by immunochemistry and confocal microscopy

    Measured: day 14

Timeline

From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.

  1. First posted25 Dec 2014 (estimated)
  2. Study start17 Mar 2015
  3. Primary completion3 Apr 2017
  4. Study completion3 Apr 2017

Resources

More details

For doctors and researchers: how the study is designed, who runs it, and its registry IDs.

Study design
Study type
Interventional
Phase
Phase 2/3
Primary purpose
Treatment
Allocation
Randomised
Intervention model
Crossover
Masking
Triple blind
Who is masked
Participant, Care provider, Investigator
Enrolment
16 participants (actual)
IDs, dates and publications
ClinicalTrials.gov ID
NCT02325362
Sponsor's protocol ID
P120703
Other IDs
2013-000497-29
Acronym
MIGLUSTAT-CF
Status last verified
Oct 2025
First submitted
10 Dec 2014
Last updated
20 Nov 2025
Results posted
No
Data monitoring committee
No
Individual data sharing
No

Questions about this trial

What is this clinical trial studying?

A Phase 2/3 study of Cystic Fibrosis, run by Assistance Publique - Hôpitaux de Paris at 1 site in France.

The full registered description is in the About this study section and the official record.

Who may be eligible to take part?

The sponsor lists 14 inclusion and 15 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.

Is this clinical trial currently recruiting?

The record says: finished, as of the sponsor's last update on 20 Nov 2025. Status can change between updates, so confirm with the study contact before making plans.

Where is the study taking place?

The record lists 1 site in France. Whether a site is taking part right now should be confirmed with that site. See every site.

What are the possible benefits and risks?

They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.

What does this combined phase mean?

Phase 2 studies give the treatment to a larger group to see whether it has the intended effect and to keep checking safety.

Phase 3 studies compare the treatment with the current standard treatment or a placebo in a large group, to confirm how well it works and watch for side effects.

Not every study follows the same path, and some studies (observational studies, device studies) have no phase.

Are results available for this study?

The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.

How can a doctor refer a patient or contact the research team?

The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.

What does the primary outcome measure?

Mean TCS in mV (time frame: day 1). The study lists 2 primary outcomes in all. It is what the study was designed to measure, not a finding.

Where can I read the original study information?

On the ClinicalTrials.gov record for NCT02325362.

Does a listing mean the treatment is approved or proven to work?

No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.

How often is this information updated?

AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 20 Nov 2025.

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