A Phase 2 interventional study of LAU-7b and Placebo oral capsule in Cystic Fibrosis, sponsored by Laurent Pharmaceuticals Inc.. Completed at 40 sites in 3 countries. Open to participants aged 18 Years and older. Per ClinicalTrials.gov, last updated 2024-10-09.
Sponsored by Laurent Pharmaceuticals Inc. · Phase 2, Interventional, and Treatment
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF.
An International Phase II, double-blind, randomized, placebo-controlled study to evaluate the safety and efficacy of LAU-7b administered once-daily for 6 months for the treatment of CF. All patients will remain on their CF standard-of-care treatments over the trial duration.
The goal for the treatment with LAU-7b in CF is to preserve lung function by reducing the persistent inflammation in the lung and to improve its capacity to defend against resistant bacteria such as Pseudomonas aeruginosa.
The treatment regimen will consist of 6 consecutive "dosing cycles" of 21 days each, spaced by study drug-free periods of 7 days. A total of 136 eligible adult patients with CF will be randomized to receive 300 mg LAU-7b or placebo in a 1:1 ratio. The participation in the study will last about 7 months.
1,619 studies on the registry are indexed under Cystic Fibrosis; 199 are open to participants now.
This study's enrollment of 166 is above the median of 36 across 1,051 interventional studies indexed under Cystic Fibrosis.
Browse Cystic Fibrosis studies →Laurent Pharmaceuticals Inc. is the lead sponsor of 3 studies on the registry; none are open to participants now.
Counted across the registry records on this site, refreshed daily.
Go through the criteria one by one — yes, no or not sure — and take the list to your doctor or the study team. Kept in this browser only.
Exclusion Criteria:
Underlined terms in the criteria open these too. Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
FEV1
From a breathing test (spirometry): how much air you can blow out in the first second. Often given as a percentage of what is expected for your age, height and sex.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Contraception
Ways of preventing pregnancy. “Highly effective” methods fail less than 1% of the time a year when used correctly — for example an IUD or an implant.
Criteria usually say which methods count and how long to keep using them after the last dose, sometimes for male participants too. Some studies accept a partner’s vasectomy if it was confirmed successful and that person is the participant’s only sexual partner.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Bilirubin
A yellow substance made when red blood cells are broken down, which the liver clears. A high level can mean a problem with the liver or the bile ducts.
Criteria often allow more for people with Gilbert’s syndrome, a harmless inherited condition that raises bilirubin.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ULN (upper limit of normal)
The top of the normal range for a blood test, as set by the laboratory that runs it. Each lab prints its own range next to your result.
“≤ 2.5 × ULN” means the result can be up to two and a half times that top value.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
ALT and AST (liver enzymes)
Two enzymes measured in a routine blood test of the liver. Higher levels can mean the liver is under strain or damaged. SGPT and SGOT are older names for ALT and AST.
Criteria usually give a limit as a multiple of the upper limit of normal (ULN), often higher when cancer has spread to the liver.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Corticosteroids (steroids)
Anti-inflammatory medicines such as prednisone, prednisolone and dexamethasone — not the anabolic steroids used for muscle.
Studies differ in what they allow: check which steroid medicines, routes (tablets, injections, creams, inhalers, eye drops) and doses this study accepts.
Explained by clinicaltrials.gg, not part of the registry record. The study team decides who can take part.
Active drug fenretinide (as LAU-7b capsules)
Drug: LAU-7b
Placebo oral capsule (as inactive capsules identical to active arm)
Drug: Placebo oral capsule
LAU-7b will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.
Also known as: fenretinide
Placebo will be administered orally once-a-day with the first meal of the day as cycles of 21 days on, 7 days off, for a total of 6 planned cycles.
Also known as: Placebo
Absolute Change in Percent Predicted Forced Expiratory Volume in 1 Second (FEV1%)
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph. The outcome measure is presented using the Least Squares Mean at the Week 24 time point and the Least Squares Mean of all post-baseline time points through Week 24 averaged.
Time frame: From baseline to 24 weeks
Summary of Treatment Emergent Adverse Events With ≥ 10% Incidence
This was assessed through adverse event monitoring at all visits, including spontaneously reported events and those obtained through serial probing of the subjects, and from safety laboratory tests
Time frame: From Baseline to 28 weeks
The Proportion of Patients Achieving Normalization of the Arachidonic Acid, Docosahexaenoic Acid and Their Ratio in Phospholipids
Assessed through 4 blood sampling occasions during the trial. Plasma samples were analyzed using a validated LC/MS method and corrected for phospholipid content. Highest proportion of normalization during treatment was determined versus analyte ranges obtained from a group of 20 healthy, non-CF individuals.
Time frame: From baseline to 28 weeks
The Absolute Change in FEV1 Percent Predicted at 3, 7, 11, 15, 24 and 28 Weeks Into the Trial
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph.
Time frame: From baseline to 3, 7, 11, 15, 24 and 28 weeks into the trial
The Relative (%) Change in FEV1 Percent Predicted at 3, 7, 11, 15, 24 and 28 Weeks Into the Trial
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph.
Time frame: From baseline to 3, 7, 11, 15, 24 and 28 weeks into the trial
The Time to First Protocol-Defined Pulmonary Exacerbation
Reports of IV antibiotics-treated pulmonary exacerbations during the trial that meet the Fuch's criteria and after the first treatment cycle.
Time frame: From baseline to 28 weeks
The Number Per Subject of Protocol-Defined Pulmonary Exacerbations (PEx) During the Trial
The number per subject of Protocol-Defined IV antibiotics-treated pulmonary exacerbations (events) during the trial that meet the Fuch's criteria. Also presented are the number per subject of IV antibiotics-treated pulmonary exacerbations and combined number per subject of IV- or Oral antibiotics-treated pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
Time frame: From baseline to 28 weeks
The Time to First Change and Usage of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
The time to first change and usage of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
Time frame: From baseline to 28 weeks
Usage (Number of Antibiotic Treatments) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Usage (number of antibiotic treatments per subject) of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
Time frame: From baseline to 28 weeks
Usage (Days) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin)
Usage (days) of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
Time frame: From baseline to 28 weeks
The Change From Baseline of Systemic Markers of Inflammation in Blood
This was assessed through scheduled blood sampling during the trial on three occasions. Both ITT and PP populations results presented. Samples were analyzed using validated analytical methods.
Time frame: Change from baseline to Week 24
The Change From Screening of the Body Weight
This was assessed through serial weighing during the trial. Measurements performed at clinical sites using calibrated balances.
Time frame: From screening to 28 weeks
The Change From Screening of the Body Mass Index (BMI)
This was assessed through serial weighing during the trial and calculation of BMI. Measurements performed at clinical sites using calibrated balances.
Time frame: From screening to 28 weeks
The Overall Change From Screening of the Pseudomonas Aeruginosa Density (Colony Forming Units) in the Sputum
This was assessed through induced sputum (and spontaneously obtained during COVID-19 pandemic) on 3 occasions during the trial. Samples were analyzed at a central laboratory. An area under the curve (AUC from baseline to Week 24 inclusive) of colony forming unit/mL is calculated.
Time frame: From screening to Week 24
The Impact (From Baseline) on Overall Health, Daily Life, Perceived Well-being and Symptoms Measured With the Cystic Fibrosis Questionnaire-Revised (CFQ-R)
This was assessed through administration of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at four planned times during the trial. The CFQ-R respiratory sub-score (range 0-100) was extracted and analyzed. The Minimum Clinically Important Difference (MCID) for the respiratory sub-score is 4 units. A higher score means a better outcome.
Time frame: From baseline to 24 weeks
The Change in Metabolipidomic Profile and in Markers of Oxidative Stress in Blood
This was assessed through serial blood sampling during the trial. Samples were analyzed using validated methods at specialized laboratories.
Time frame: From baseline to 24 weeks
The Change in Metabolipidomic Profile in Blood, the Systemic Markers of Inflammation in Blood, the FEV1, the Body Weight and Calculated BMI
Only in patients who experience a pulmonary exacerbation requiring IV antibiotics, this was to be assessed prior to- and after receiving an IV antibiotic course.
Time frame: From baseline to 28 weeks
The Change From Baseline of Systemic Bone Formation and Resorption Biomarkers
This was assessed through blood sampling on 2 occasions during the trial at Baseline and Week 24.
Time frame: Baseline and 24 weeks
The Change From Baseline of Bone Mineral Density
This was assessed through Lumbar spine bone mineral density measured on 2 occasions during the trial at Baseline and at Week 28 in a subset of sites and on a voluntary basis. Bone mineral density in g/cm2 is then normalized and expressed as a Z-score distribution according to age and gender. A Z-score of 0 represents the population mean for the age and gender category, a -1 value or +1 value means below or above the population mean bone mineral density for the age and gender category, but considered normal. A -2.5 value indicates secondary osteoporosis, a worse outcome.
Time frame: Baseline and 28 weeks
| Milestone | LAU-7b | Placebo |
|---|---|---|
| Started | 83 | 83 |
| Completed | 60 | 71 |
| Not completed | 23 | 12 |
| Withdrew: Adverse event | 0 | 1 |
| Withdrew: Withdrawal by subject | 13 | 5 |
| Withdrew: Drug withdrawn | 3 | 3 |
| Withdrew: Any other reason | 7 | 3 |
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph. The outcome measure is presented using the Least Squares Mean at the Week 24 time point and the Least Squares Mean of all post-baseline time points through Week 24 averaged.
| Change in FEV1 % predicted | LAU-7b | Placebo |
|---|---|---|
| ITT population at Week 24 | -1.1774 (-2.5038 to 0.1490) | -1.9489 (-3.2241 to -0.6738) |
| ITT population through Week 24 | -0.5417 (-1.5226 to 0.4392) | -1.5470 (-2.5304 to -0.5637) |
| PP population through Week 24 | -0.3374 (-1.4987 to 0.8239) | -1.5632 (-2.7333 to -0.3930) |
| ITT subgroup >=70% ppFEV1, at Week 24 | -1.4059 (-3.5506 to 0.7388) | -4.0687 (-6.1289 to -2.0085) |
| ITT subgroup on CFTR modulators, at Week 24 | -0.3336 (-2.2467 to 1.5795) | -1.7247 (-3.2954 to -0.1539) |
| ITT subgroup on ETI, at Week 24 | -0.7406 (-3.7472 to 2.2659) | -1.8709 (-4.1669 to 0.4252) |
This was assessed through adverse event monitoring at all visits, including spontaneously reported events and those obtained through serial probing of the subjects, and from safety laboratory tests
| Participants | LAU-7b | Placebo |
|---|---|---|
| Subjects with at least one TEAE with ≥10% incidence | 74 | 64 |
| Mild highest reported severity | 22 | 27 |
| Moderate highest reported severity | 45 | 43 |
| Severe highest reported severity | 13 | 7 |
| Life threatening | 0 | 0 |
| Infective pulmonary exacerbation of cystic fibrosis | 37 | 32 |
| Cough | 23 | 24 |
| Delayed dark adaptation | 22 | 4 |
| Night blindness | 15 | 2 |
| Headache | 14 | 10 |
| Upper respiratory tract infections | 13 | 8 |
| Hemoptysis | 11 | 13 |
| Diarrhoea | 11 | 6 |
| Fatigue | 10 | 7 |
| Delayed light adaptation | 10 | 2 |
| Dyspnea | 10 | 11 |
| Glare | 9 | 6 |
| Visual impairment | 9 | 2 |
| Sputum increased | 7 | 13 |
Assessed through 4 blood sampling occasions during the trial. Plasma samples were analyzed using a validated LC/MS method and corrected for phospholipid content. Highest proportion of normalization during treatment was determined versus analyte ranges obtained from a group of 20 healthy, non-CF individuals.
| Participants | LAU-7b | Placebo |
|---|---|---|
| Arachidonic acid (AA) normalization | 15 | 25 |
| Docosahexaenoic acid (DHA) normalization | 16 | 18 |
| AA/DHA ratio normalization | 25 | 23 |
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph.
| FEV1 percent predicted change | LAU-7b | Placebo |
|---|---|---|
| Absolute change at Week 3 | -0.243 ± 5.110 | -1.494 ± 4.537 |
| Absolute change at Week 7 | 0.280 ± 4.689 | -0.888 ± 3.968 |
| Absolute change at Week 11 | -0.309 ± 4.268 | -0.871 ± 4.133 |
| Absolute change at Week 15 | -0.508 ± 5.100 | -1.591 ± 4.389 |
| Absolute change at Week 24 | -1.024 ± 4.286 | -1.759 ± 4.533 |
| Absolute change at Week 28 | -1.539 ± 4.808 | -1.413 ± 4.457 |
Standardized, serial FEV1 measurements were performed during the trial by the clinical sites using standardized spirometers. All spirometry measurements were centrally read and validated by the spirometry provider, Vitalograph.
| FEV1 percent predicted change | LAU-7b | Placebo |
|---|---|---|
| Relative (%) change at Week 3 | -1.188 ± 8.632 | -2.341 ± 7.289 |
| Relative (%) change at Week 7 | 0.561 ± 8.348 | -1.550 ± 6.829 |
| Relative (%) change at Week 11 | -0.213 ± 7.124 | -1.277 ± 7.120 |
| Relative (%) change at Week 15 | -0.924 ± 8.963 | -2.745 ± 7.425 |
| Relative (%) change at Week 24 | -1.450 ± 6.966 | -2.652 ± 7.742 |
| Relative (%) change at Week 28 | -2.388 ± 8.275 | -2.085 ± 7.525 |
Reports of IV antibiotics-treated pulmonary exacerbations during the trial that meet the Fuch's criteria and after the first treatment cycle.
| days | LAU-7b | Placebo |
|---|---|---|
| The Time to First Protocol-Defined Pulmonary Exacerbation | NA (NA to NA) | NA (NA to NA) |
The number per subject of Protocol-Defined IV antibiotics-treated pulmonary exacerbations (events) during the trial that meet the Fuch's criteria. Also presented are the number per subject of IV antibiotics-treated pulmonary exacerbations and combined number per subject of IV- or Oral antibiotics-treated pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
| Events per subject | LAU-7b | Placebo |
|---|---|---|
| Protocol-Defined IV-treated PEx events per subject | 0.16 ± 0.398 | 0.10 ± 0.335 |
| All IV-treated PEx events per subject | 0.24 ± 0.458 | 0.18 ± 0.566 |
| All IV- or Oral antibiotics-treated PEx events per subject | 0.53 ± 0.721 | 0.47 ± 0.817 |
The time to first change and usage of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
| days | LAU-7b | Placebo |
|---|---|---|
| The Time to First Change and Usage of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin) | NA (NA to NA) | NA (NA to NA) |
Usage (number of antibiotic treatments per subject) of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
| Number of IV antibiotic treatments | LAU-7b | Placebo |
|---|---|---|
| Usage (Number of Antibiotic Treatments) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin) | 0.53 ± 1.09 | 0.41 ± 1.32 |
Usage (days) of IV antibiotics to treat pulmonary exacerbations during the trial. Excluded are exacerbations occurring during the first treatment cycle.
| Days of IV antibiotics | LAU-7b | Placebo |
|---|---|---|
| Usage (Days) of Antibiotic (Other Than Chronic Inhaled Antibiotics Already Started Prior to Trial or Oral Chronic Azithromycin) | 3.8 ± 7.50 | 3.5 ± 14.94 |
This was assessed through scheduled blood sampling during the trial on three occasions. Both ITT and PP populations results presented. Samples were analyzed using validated analytical methods.
| Change in mg/L | LAU-7b | Placebo |
|---|---|---|
| Absolute change from baseline of C-reactive protein at Week 24 ITT Population | -1.197 ± 7.904 | 2.532 ± 10.650 |
| Absolute change from baseline of calprotectin at Week 24, ITT population | -27 ± 962 | 478 ± 2088 |
| Absolute change from baseline of C-reactive protein at Week 24 PP Population | -1.216 ± 7.987 | 2.532 ± 10.654 |
| Absolute change from baseline of calprotectin at Week 24, PP population | -18 ± 970 | 478 ± 2088 |
This was assessed through serial weighing during the trial. Measurements performed at clinical sites using calibrated balances.
| Change in kg | LAU-7b | Placebo |
|---|---|---|
| The Change From Screening of the Body Weight | -0.43 ± 2.41 | 0.14 ± 1.96 |
This was assessed through serial weighing during the trial and calculation of BMI. Measurements performed at clinical sites using calibrated balances.
| Change in kg/m2 | LAU-7b | Placebo |
|---|---|---|
| The Change From Screening of the Body Mass Index (BMI) | -0.162 ± 0.922 | 0.037 ± 0.706 |
This was assessed through induced sputum (and spontaneously obtained during COVID-19 pandemic) on 3 occasions during the trial. Samples were analyzed at a central laboratory. An area under the curve (AUC from baseline to Week 24 inclusive) of colony forming unit/mL is calculated.
| CFU*weeks/mL | LAU-7b | Placebo |
|---|---|---|
| The Overall Change From Screening of the Pseudomonas Aeruginosa Density (Colony Forming Units) in the Sputum | 17681448107 ± 33050105775 | 10218800226 ± 16808101968 |
This was assessed through administration of the Cystic Fibrosis Questionnaire-Revised (CFQ-R) at four planned times during the trial. The CFQ-R respiratory sub-score (range 0-100) was extracted and analyzed. The Minimum Clinically Important Difference (MCID) for the respiratory sub-score is 4 units. A higher score means a better outcome.
| Change of units on a scale | LAU-7b | Placebo |
|---|---|---|
| The Impact (From Baseline) on Overall Health, Daily Life, Perceived Well-being and Symptoms Measured With the Cystic Fibrosis Questionnaire-Revised (CFQ-R) | -2.485 ± 20.28 | 0.235 ± 11.44 |
This was assessed through serial blood sampling during the trial. Samples were analyzed using validated methods at specialized laboratories.
| umol/L | LAU-7b | Placebo |
|---|---|---|
| 11-Hydroxy Eicosatetraenoic Acid | -0.0195 ± 0.13559 | 0.0187 ± 0.08397 |
| 12-Hydroxy Eicosatetraenoic Acid | -0.00445 ± 0.491341 | -0.01181 ± 0.820744 |
| 15-Hydroxy Eicosapentaenoic Acid | 0.00069 ± 0.025828 | 0.02028 ± 0.059147 |
| 19,20-diHydroxy Docosapentaenoic Acid | 0.16447 ± 0.865788 | 0.06606 ± 0.568695 |
| 8-Hydroxy Eicosatetraenoic Acid | -0.00744 ± 0.093498 | 0.01879 ± 0.088266 |
| Thiobarbituric Acid Reactive Substances | -0.0504 ± 0.79940 | 0.0406 ± 1.19656 |
| Nitrotyrosine (NT3) | -1.57 ± 72.964 | 10.35 ± 146.824 |
Only in patients who experience a pulmonary exacerbation requiring IV antibiotics, this was to be assessed prior to- and after receiving an IV antibiotic course.
No measurements were reported for this outcome.
This was assessed through blood sampling on 2 occasions during the trial at Baseline and Week 24.
| Change in ug/L | LAU-7b | Placebo |
|---|---|---|
| Aminoterminal Propeptide | -5.0 ± 14.90 | -4.5 ± 16.97 |
| C-Telopeptide | -0.012 ± 0.2092 | 0.015 ± 0.1857 |
| Osteocalcin | -1.46 ± 6.181 | 0.57 ± 5.703 |
This was assessed through Lumbar spine bone mineral density measured on 2 occasions during the trial at Baseline and at Week 28 in a subset of sites and on a voluntary basis. Bone mineral density in g/cm2 is then normalized and expressed as a Z-score distribution according to age and gender. A Z-score of 0 represents the population mean for the age and gender category, a -1 value or +1 value means below or above the population mean bone mineral density for the age and gender category, but considered normal. A -2.5 value indicates secondary osteoporosis, a worse outcome.
| Z-score | LAU-7b | Placebo |
|---|---|---|
| The Change From Baseline of Bone Mineral Density | -0.18 ± 0.69 | 0.09 ± 0.28 |
Collected over 28 weeks. Non-serious events are listed at a 5% frequency threshold.
| Group | Deaths | Serious | Other |
|---|---|---|---|
| LAU-7b | 0/83 (0%) | 20/83 (24.1%) | 80/83 (96.4%) |
| Placebo | 0/83 (0%) | 15/83 (18.1%) | 79/83 (95.2%) |
| Event | LAU-7b | Placebo |
|---|---|---|
| Infective pulmonary exacerbation of cystic fibrosisInfections and infestations | 15/83 | 10/83 |
| Distal intestinal obstruction syndromeGastrointestinal disorders | 2/83 | 2/83 |
| AppendicitisInfections and infestations | 2/83 | 0/83 |
| HemoptysisRespiratory, thoracic and mediastinal disorders | 1/83 | 2/83 |
| Loss of visual contrast sensitivityEye disorders | 1/83 | 0/83 |
| ConstipationGastrointestinal disorders | 1/83 | 0/83 |
| Small intestinal obstructionGastrointestinal disorders | 0/83 | 1/83 |
| InfluenzaInfections and infestations | 1/83 | 0/83 |
| Pneumonia staphylococcalInfections and infestations | 1/83 | 0/83 |
| PneumoniaInfections and infestations | 0/83 | 1/83 |
| Event | LAU-7b | Placebo |
|---|---|---|
| CoughRespiratory, thoracic and mediastinal disorders | 23/83 | 24/83 |
| Delayed dark adaptationEye disorders | 22/83 | 4/83 |
| Infective pulmonary exacerbation of cystic fibrosisInfections and infestations | 22/83 | 22/83 |
| Night blindnessEye disorders | 15/83 | 2/83 |
| HeadacheNervous system disorders | 14/83 | 10/83 |
| Upper respiratory tract infectionInfections and infestations | 13/83 | 8/83 |
| HemoptysisRespiratory, thoracic and mediastinal disorders | 11/83 | 13/83 |
| Sputum increasedRespiratory, thoracic and mediastinal disorders | 7/83 | 13/83 |
| DiarrheaGastrointestinal disorders | 11/83 | 6/83 |
| DyspneaRespiratory, thoracic and mediastinal disorders | 10/83 | 11/83 |
| Age, Continuous(years) | LAU-7b | Placebo | Total |
|---|---|---|---|
| Mean | 31.5 ± 10.70 | 33.9 ± 12.95 | 32.7 ± 11.90 |
| Age, Customized(Participants) | LAU-7b | Placebo | Total |
|---|---|---|---|
| <=25 years | 29 | 26 | 55 |
| >25 years | 54 | 57 | 111 |
| Sex: Female, Male(Participants) | LAU-7b | Placebo | Total |
|---|---|---|---|
| Female | 50 | 49 | 99 |
| Male | 33 | 34 | 67 |
| Ethnicity (NIH/OMB)(Participants) | LAU-7b | Placebo | Total |
|---|---|---|---|
| Hispanic or Latino | 7 | 2 | 9 |
| Not Hispanic or Latino | 76 | 81 | 157 |
| Unknown or Not Reported | 0 | 0 | 0 |
| Race (NIH/OMB)(Participants) | LAU-7b | Placebo | Total |
|---|---|---|---|
| American Indian or Alaska Native | 0 | 1 | 1 |
| Asian | 0 | 0 | 0 |
| Native Hawaiian or Other Pacific Islander | 0 | 0 | 0 |
| Black or African American | 2 | 3 | 5 |
| White | 81 | 78 | 159 |
| More than one race | 0 | 0 | 0 |
| Unknown or Not Reported | 0 | 1 | 1 |
| Region of Enrollment(participants) | LAU-7b | Placebo | Total |
|---|---|---|---|
| Canada | 24 | 22 | 46 |
| United States | 48 | 51 | 99 |
| Australia | 11 | 10 | 21 |
| Weight (kg), Mean (SD)(kg) | LAU-7b | Placebo | Total |
|---|---|---|---|
| Mean | 63.88 ± 12.213 | 64.53 ± 14.329 | 64.20 ± 13.277 |
| BMI (kg/m2), Mean (SD)(kg/m2) | LAU-7b | Placebo | Total |
|---|---|---|---|
| Mean | 23.34 ± 3.354 | 23.39 ± 4.099 | 23.36 ± 3.734 |
2 further baseline measures are reported on the registry.
PDFs hosted by the registry, ClinicalTrials.gov.
Plan to share: No
Ran from Nov 2018 to primary completion in Sep 2021, 34 months.
Results were first posted in Oct 2024, 3 years after primary completion.
Of 40 industry-sponsored phase 2 interventional studies under Cystic Fibrosis, half reached primary completion within 15 months of starting (the median), counting those still running; a quarter within 7 and three quarters within 29. This study's 34 months is above that.
Counting every sponsor, half of 57 studies reached it within 21 months; a quarter within 9 and three quarters within 34.
How these figures are worked out, and which studies are left out of them.
This study is completed, as verified in Oct 2024. You cannot join it, but the record below documents what was studied.
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Studies of the drug tested here: Fenretinide
Laurent Pharmaceuticals Inc.