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| ID | Type | Description | Link |
|---|---|---|---|
| 2013-000497-29 | EudraCT Number |
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| Name | Class |
|---|---|
| Actelion | INDUSTRY |
| CRCM (Centres de Ressources et de Compétences de la Mucoviscidose) | UNKNOWN |
| URC-CIC Paris Descartes Necker Cochin | OTHER |
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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.
The aims of this study are:
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| Miglustat then placebo | Experimental | 10 patients will received Miglustat then the placebo |
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| Placebo then Miglustat | Experimental | 10 patients will received Placebo then Miglustat |
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| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| Miglustat ; placebo | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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 | day 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 | Day 14 |
| Measure | Description | Time Frame |
|---|---|---|
| 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. | day 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. |
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Inclusion Criteria:
Inclusion criteria at screening visit (Visit 1):
Aged 18 years and older
Male or female
Women of childbearing potential must:
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):
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Isabelle FAJAC, MD, PhD. | Assistance Publique - Hôpitaux de Paris | Study Director |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Assistance publique-Hôpitaux de Paris, Hôpital Cochin | Paris | 75014 | France |
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| ID | Term |
|---|---|
| D003550 | Cystic Fibrosis |
| ID | Term |
|---|---|
| D010182 | Pancreatic Diseases |
| D004066 | Digestive System Diseases |
| D008171 | Lung Diseases |
| D012140 | Respiratory Tract Diseases |
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| ID | Term |
|---|---|
| C059896 | miglustat |
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| Placebo ; Miglustat | Drug | 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. |
|
| Day 14 |
| 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 | day 1 |
| 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 | day 14 |
| 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 | day 1 |
| 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 | day 14 |
| Change of basal NPD in mV | Basal NPD at end-of-treatment minus basal NPD at baseline | day 1 |
| Change of basal NPD in mV | Basal NPD at end-of-treatment minus basal NPD at baseline | day 14 |
| 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 | day 1 |
| 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 | day 14 |
| 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 | day 1 |
| 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 | day 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 | day 1 |
| 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 | day 14 |
| Sweat chloride concentration in mmol/L | Sweat chloride concentration at end-of-treatment minus sweat chloride concentration at baseline | day 1 |
| Sweat chloride concentration in mmol/L | Sweat chloride concentration at end-of-treatment minus sweat chloride concentration at baseline | day 14 |
| FEV1 (in % of predicted) | Pulmonary function FEV1: mean Forced expiry volume in 1 second. FEV1 at end-of-treatment minus FEV1 at baseline | day 1 |
| FEV1 (in % of predicted) | Pulmonary function FEV1: mean Forced expiry volume in 1 second. FEV1 at end-of-treatment minus FEV1 at baseline | day 14 |
| Change in electrochemical skin conductance | Electrochemical skin conductance at end-of-treatment minus electrochemical skin conductance at baseline | day 1 |
| Change in electrochemical skin conductance | Electrochemical skin conductance at end-of-treatment minus electrochemical skin conductance at baseline | day 14 |
| 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 | day 14 |
| D030342 |
| Genetic Diseases, Inborn |
| D009358 | Congenital, Hereditary, and Neonatal Diseases and Abnormalities |
| D007232 | Infant, Newborn, Diseases |