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| ID | Type | Description | Link |
|---|---|---|---|
| 2021-003875-32 | EudraCT Number |
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Prospective, international, randomised, double-blind, placebo controlled, multicentre, parallel group study. Patients will be randomised in a 2:1 allocation ratio to receive either IFB-088 + riluzole 100 mg or placebo + riluzole 100 mg. This clinical trial is an exploratory study, designed to show a signal of efficacy of IFB-088 through ALSFRS-R, MITOS and King's College. Respiratory function will be followed through SVC. Biomarkers and quality of life will also be evaluated throughout the study.
Patients will be treated over a 6-month period. After a screening/consent visit, patients will undergo clinic visits at randomisation (V0), at 2 weeks (V1), and at months 1 (V2), 3 (V3) and 6 (V4). One week after V0, the patient will undergo urine analysis (dipstick)and blood sampling for measurement of creatinine
, as well as blood sampling for measurement of creatinine and calculation of eGFR at months 2, 4 and 5. At the V2 visit, in addition to other assessments, patients will undergo blood sampling for PK measurements and urine sampling for crystalluria examination. Blood and urine chemistry, as well as physical examination and vital signs assessment to assess safety will be performed at each visit for safety purpose and crystalluria examination will be repeated at the follow-up visit, performed one month ± one week after V4.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| IFB-088 50 mg/day + riluzole 100 mg/day | Experimental | The test product, IFB-088, will be administered orally in 50 mg/day dosage consisting of two uptakes of 25 mg each (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). |
|
| placebo + riluzole 100 mg/day | Placebo Comparator | The placebo will be administered orally in two uptakes (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| IFB-088 50mg/day | Drug | Tested product |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety Assessment of IFB-088 50 mg/Day in Patients With Bulbar-onset ALS. Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] |
| from beginning of IMP intake up to 30 days after stopping the intake, an average of 7 months |
| Measure | Description | Time Frame |
|---|---|---|
| Efficacy With Scale : ALSFRS-R (ALS Functional Rating Scale Revised) | ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale Revised) 12 items, clinician rated including 5 choices from normal to disabled. Maximal score: 48, minimal score: 0 (death) | Efficacy scale from baseline to V3 (3 months) and V4 (6 months). |
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Inclusion Criteria:
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Shahram Attarian, Pr | Assistance Publique Hôpitaux de Marseille (APHM) Hospital La Timone Adultes, France | Principal Investigator |
| Giuseppe Lauria, Pr | IRCCS Carlo Besta Institute of Milan, Italy | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Hôpital Neurologique Pierre Wertheimer | Bron | 69677 | France | |||
| APHM Hôpital La Timone Adultes SCE Maladies Neuromusculaires / SLA |
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| ID | Title | Description |
|---|---|---|
| FG000 | Placebo + Riluzole 100 mg/Day | The placebo will be administered orally in two uptakes (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). Placebo: Placebo Riluzole 100mg/day: Standard of care treatment, co-administered with tested product (IFB-088 50mg/day) or placebo |
| FG001 | IFB-088 50 mg/Day + Riluzole 100 mg/Day | The test product, IFB-088, will be administered orally in 50 mg/day dosage consisting of two uptakes of 25 mg each (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). IFB-088 50mg/day: Tested product Riluzole 100mg/day: Standard of care treatment, co-administered with tested product (IFB-088 50mg/day) or placebo |
| Title | Milestones | Reasons Not Completed | |||||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Overall Study |
|
|
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| ID | Title | Description |
|---|---|---|
| BG000 | IFB-088 50 mg/Day + Riluzole 100 mg/Day | The test product, IFB-088, will be administered orally in 50 mg/day dosage consisting of two uptakes of 25 mg each (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). IFB-088 50mg/day: Tested product Riluzole 100mg/day: Standard of care treatment, co-administered with tested product (IFB-088 50mg/day) or placebo |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Continuous | Mean |
| Type | Title | Description | Population Description | Reporting Status | Anticipated Posting Date | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Time Frame | Units Analyzed | Denominator Units Selected | Arm/Group Information | Denominators | Classes | Analyses | |||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| Primary | Safety Assessment of IFB-088 50 mg/Day in Patients With Bulbar-onset ALS. Number of Participants With Treatment-Emergent Adverse Events [Safety and Tolerability] |
| Posted | Count of Participants | Participants | from beginning of IMP intake up to 30 days after stopping the intake, an average of 7 months |
|
7 months, from inclusion to last patient visit, one month after 6 months treatment ending
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| ID | Title | Description | Deaths (Affected) | Deaths (At Risk) | Serious Events (Affected) | Serious Events (At Risk) | Other Events (Affected) | Other Events (At Risk) |
|---|---|---|---|---|---|---|---|---|
| EG000 | Placebo + Riluzole 100 mg/Day | The placebo will be administered orally in two uptakes (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). Placebo: Placebo Riluzole 100mg/day: Standard of care treatment, co-administered with tested product (IFB-088 50mg/day) or placebo |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| COVID 19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Nausea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
Assessment of Body Composition (Exploratory) could not be assessed properly due to late availability of the devices.
Biomarkers analysis performed by the primary laboratory plant lead to important BLQ data, important CV deviations, requiring cautious interpretation of the results.
Additional biomarker analysis were performed in another lab, and were used for post hoc analysis.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| Beatrice Lejeune | InFlectis Bioscience | +33 668919060 | beatricelejeune@inflectisbioscience.com |
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| Type | Includes Protocol | Includes SAP | Includes ICF | Document Label | Document Date | Document Uploaded Date | Document File Name |
|---|---|---|---|---|---|---|---|
| Prot | Yes | No | No | Study Protocol | Apr 8, 2024 | Jun 19, 2025 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Nov 12, 2024 | Jun 19, 2025 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| D016472 | Motor Neuron Disease |
| D019636 | Neurodegenerative Diseases |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D057177 | TDP-43 Proteinopathies |
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| ID | Term |
|---|---|
| D019782 | Riluzole |
| ID | Term |
|---|---|
| D013844 | Thiazoles |
| D013457 | Sulfur Compounds |
| D009930 | Organic Chemicals |
| D052160 | Benzothiazoles |
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Patients will be randomised in a 2:1 allocation ratio to receive either IFB-088 + riluzole 100 mg or placebo + riluzole 100 mg.
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Study double-blind.
| Placebo | Drug | Placebo |
|
| Riluzole 100mg/day | Drug | Standard of care treatment, co-administered with tested product (IFB-088 50mg/day) or placebo |
|
|
| Efficacy With Scale : ALS_MITOS (ALS Milano-Torino Staging) |
ALS_MITOS (Amyotrophic Lateral Sclerosis Milano-Torino Staging) scale scores the total points of points given in 4 domains (movement, swallowing, communicating, breathing), clinician rated. The ALS8MITOS score is determined by the sum of functional score of 1 for each domain, up to 5, being death. The score may go from 0= no functionnal domain lost, up to 5=death (1 to 4 corresponding to the number of domains lost by the patient). |
| baseline, V3 (3 months), V4 (6 months) |
| Efficacy With Scale : King's College Scale (ALS Staging Form) | King's college Scale (King's ALS staging form), clinician rated, 8 items. 0=best, 5=death | Efficacy scale from baseline to 3 months and 6 months. |
| Efficacy Based on Assessment of Respiratory Function (Slow Vital Capacity [SVC]) | Assessment of respiratory function (slow vital capacity [SVC]). | Respiratory function at screening, 3 and 6 months. |
| Efficacy Based on Assessment of Respiratory Function (Arterial Blood Gases [ABG]), PaCO2) | Assessment of respiratory function (Arterial Blood Gases [ABG]): description of PaCO2 (mmHg), at screening, 3 and 6 months. | Respiratory function at screening, 3 and 6 months. |
| Efficacy Based on Assessment of Body Composition (Exploratory) | change of body composition (% of water, muscle, bone in the body) evaluated by bioelectrical impedance | At baseline and 6 months |
| Pharmacokinetic Parameters (Area Under Curve [AUC]) | Area Under the Curve (AUC (0-12h)) of IFB-088 and its metabolite IFB-139. As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 ng.h/mL. | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
| Pharmacokinetic Parameters (Cmax) | Maximum observed plasma concentration (Cmax) of IFB-088 and its metabolite IFB-139. As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 ng/mL. | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
| Pharmacokinetic Parameters (Tmax) | Time at which maximum plasma concentration (Cmax) of IFB-088 and its metabolite IFB-139 is measured As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 h | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
| Pharmacokinetic Parameters (t1/2) | Terminal or apparent terminal half-life (t1/2) of IFB-088 and its metabolite IFB-139. As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 h. | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
| Pharmacokinetic Parameters (Clearance) | IFB-088 Apparent systemic clearance calculation (CL/F) As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 L/h. | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
| Pharmacokinetic Parameters (Vd) | IFB-088 Apparent volume of distribution (Vd). As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 L. | PK parameters will be analysed after 4 weeks of treatment. |
| Biomarkers (TDP-43) | Change in TDP-43 plasmatic concentration from baseline to 6 months, compared to placebo (concentration in pg/mL, technology Simoa®). | At baseline and 6 months. |
| Biomarkers (Neurofilament Light Chain) | Change in neurofilament (NfL) light chain plasmatic concentration from baseline to 6 months, compared to placebo (concentration in pg/mL, technology Simoa®). | At baseline and 6 months. |
| Biomarkers (Inflammation Biomarkers) | Inflammation biomarkers (interleukin [IL]-6, tumour necrosis factor-α [TNFα], interferon γ [IFNγ], IL-1β, IL-8, IL-10, monocyte chemoattractant protein-1 [MCP-1], nerve growth factor [NGF], brain-derived neurotrophic factor [BDNF], vascular endothelial growth factor [VEGF]): (concentration of each biomarker in ng/mL, technology Luminex®)). | All assessed at baseline and V4 visit (6 months). Only GDF15, MCP1, BDNF, and TGFb1 also assessed at V3 visit (3 months) |
| Biomarkers (3-Nitrotyrosine) | 3-Nitrotyrosine (Oxidative stress biomarker): at baseline, 3 and 6 months (concentration in ng/mL, ELISA method). | At baseline, 3 months, and 6 months. |
| Quality of Life With ALSAQ-40 (ALS Assessment Questionnaire) | Change in ALS assessment questionnaire (ALSAQ-40). ALSAQ-40 (Amyotrophic Lateral Sclerosis Assessment Questionnaire) Quality of Life questionnaire 40 items, patient rated including 5 choices from never to always. best=0, worse=100 | QoL will be assessed from baseline to 6 months |
| Efficacy Based on Assessment of Respiratory Function (Arterial Blood Gases [ABG]), PO2 (mmHg) | Assessment of respiratory function (Arterial Blood Gases [ABG]): description of PO2 (mmHg) at screening, 3 and 6 months. | Respiratory function at screening, 3 and 6 months. |
| Efficacy Based on Assessment of Respiratory Function (Arterial Blood Gases [ABG]), HCO3 (mEq/L) | Assessment of respiratory function (Arterial Blood Gases [ABG]): description of HCO3 (mEq/L) at screening, 3 and 6 months. | Respiratory function at screening, 3 and 6 months. |
| Efficacy Based on Assessment of Respiratory Function (Arterial Blood Gases [ABG]), Oxygen Saturation (%) | Assessment of respiratory function (Arterial Blood Gases [ABG]): description of Oxygen saturation (%) at screening, 3 and 6 months. | Respiratory function at screening, 3 and 6 months. |
| Marseille |
| 13385 |
| France |
| CHU de Nantes - Hôpital Laennec | Nantes | 44093 | France |
| CHU de Toulouse - Hôpital Pierre-Paul Riquet | Toulouse | 31059 | France |
| CHU Bretonneau | Tours | 37044 | France |
| Ospedale Civile Sant'Agostino Estense | Baggiovara | 41126 | Italy |
| Centro Clinico NeMO per le Malattie Neuromuscolari | Gussago | 25064 | Italy |
| IRCSS Istituto Neurologico Carlo Besta | Milan | 20133 | Italy |
| Sant'Andrea Hospital Unit of Neuromuscular Disorders | Roma | 00189 | Italy |
| Adverse Event |
|
| Physician Decision |
|
| wrongly randomized |
|
| non-compliance |
|
| BG001 | Placebo + Riluzole 100 mg/Day | The placebo will be administered orally in two uptakes (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). Placebo: Placebo Riluzole 100mg/day: Standard of care treatment, co-administered with tested product (IFB-088 50mg/day) or placebo |
| BG002 | Total | Total of all reporting groups |
| years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race (NIH/OMB) | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| BMI | Median | Standard Deviation | kg/m^2 |
|
| Tobacco smoking, n (%) | Count of Participants | Participants |
|
| OG001 | IFB-088 50 mg/Day + Riluzole 100 mg/Day | The test product, IFB-088, will be administered orally in 50 mg/day dosage consisting of two uptakes of 25 mg each (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). IFB-088 50mg/day: Tested product Riluzole 100mg/day: Standard of care treatment, co-administered with tested product (IFB-088 50mg/day) or placebo |
|
|
| Secondary | Efficacy With Scale : ALSFRS-R (ALS Functional Rating Scale Revised) | ALSFRS-R (Amyotrophic Lateral Sclerosis Functional Rating Scale Revised) 12 items, clinician rated including 5 choices from normal to disabled. Maximal score: 48, minimal score: 0 (death) | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Mean | Standard Deviation | score on a scale | Efficacy scale from baseline to V3 (3 months) and V4 (6 months). |
|
|
|
|
| Secondary | Efficacy With Scale : ALS_MITOS (ALS Milano-Torino Staging) | ALS_MITOS (Amyotrophic Lateral Sclerosis Milano-Torino Staging) scale scores the total points of points given in 4 domains (movement, swallowing, communicating, breathing), clinician rated. The ALS8MITOS score is determined by the sum of functional score of 1 for each domain, up to 5, being death. The score may go from 0= no functionnal domain lost, up to 5=death (1 to 4 corresponding to the number of domains lost by the patient). | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Mean | Standard Deviation | score on a scale | baseline, V3 (3 months), V4 (6 months) |
|
|
|
|
| Secondary | Efficacy With Scale : King's College Scale (ALS Staging Form) | King's college Scale (King's ALS staging form), clinician rated, 8 items. 0=best, 5=death | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Mean | Standard Deviation | score on a scale | Efficacy scale from baseline to 3 months and 6 months. |
|
|
|
|
| Secondary | Efficacy Based on Assessment of Respiratory Function (Slow Vital Capacity [SVC]) | Assessment of respiratory function (slow vital capacity [SVC]). | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). SVC is a respiratory test hence patients suffering from ALS were not always able to perform the test. | Posted | Mean | Standard Deviation | percentage of SVC | Respiratory function at screening, 3 and 6 months. |
|
|
|
|
| Secondary | Efficacy Based on Assessment of Respiratory Function (Arterial Blood Gases [ABG]), PaCO2) | Assessment of respiratory function (Arterial Blood Gases [ABG]): description of PaCO2 (mmHg), at screening, 3 and 6 months. | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Mean | Standard Deviation | millimeters of Mercury | Respiratory function at screening, 3 and 6 months. |
|
|
|
| Secondary | Efficacy Based on Assessment of Body Composition (Exploratory) | change of body composition (% of water, muscle, bone in the body) evaluated by bioelectrical impedance | Not Posted | Jan 2026 | At baseline and 6 months | Participants |
| Secondary | Pharmacokinetic Parameters (Area Under Curve [AUC]) | Area Under the Curve (AUC (0-12h)) of IFB-088 and its metabolite IFB-139. As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 ng.h/mL. | As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 ng.h/mL. | Posted | Geometric Mean | Geometric Coefficient of Variation | ng.h/mL | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
|
|
|
| Secondary | Pharmacokinetic Parameters (Cmax) | Maximum observed plasma concentration (Cmax) of IFB-088 and its metabolite IFB-139. As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 ng/mL. | As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 ng/mL. | Posted | Geometric Mean | Geometric Coefficient of Variation | ng/mL | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
|
|
|
| Secondary | Pharmacokinetic Parameters (Tmax) | Time at which maximum plasma concentration (Cmax) of IFB-088 and its metabolite IFB-139 is measured As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 h | As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 h. | Posted | Mean | Inter-Quartile Range | h | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
|
|
|
| Secondary | Pharmacokinetic Parameters (t1/2) | Terminal or apparent terminal half-life (t1/2) of IFB-088 and its metabolite IFB-139. As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 h. | As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 h. | Posted | Geometric Mean | Geometric Coefficient of Variation | h | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
|
|
|
| Secondary | Pharmacokinetic Parameters (Clearance) | IFB-088 Apparent systemic clearance calculation (CL/F) As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 L/h. | As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 L/h. | Posted | Geometric Mean | Geometric Coefficient of Variation | L/h | PK parameters will be analysed after 4 weeks of treatment. Blood samples were collected at V2 (5 samples/patient: pre-IMP dose, and at one, 2, 4 and 6 hours post dose). |
|
|
|
| Secondary | Pharmacokinetic Parameters (Vd) | IFB-088 Apparent volume of distribution (Vd). As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 L. | As study was double blinded, the analysis was done on all the patients, either receiving IFB-088 or placebo. Dosage of IFB-088 in patients under placebo was equal to 0 L. | Posted | Geometric Mean | Geometric Coefficient of Variation | L | PK parameters will be analysed after 4 weeks of treatment. |
|
|
|
| Secondary | Biomarkers (TDP-43) | Change in TDP-43 plasmatic concentration from baseline to 6 months, compared to placebo (concentration in pg/mL, technology Simoa®). | Not Posted | Jan 2026 | At baseline and 6 months. | Participants |
| Secondary | Biomarkers (Neurofilament Light Chain) | Change in neurofilament (NfL) light chain plasmatic concentration from baseline to 6 months, compared to placebo (concentration in pg/mL, technology Simoa®). | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Geometric Mean | 95% Confidence Interval | pg/mL | At baseline and 6 months. |
|
|
|
|
| Secondary | Biomarkers (Inflammation Biomarkers) | Inflammation biomarkers (interleukin [IL]-6, tumour necrosis factor-α [TNFα], interferon γ [IFNγ], IL-1β, IL-8, IL-10, monocyte chemoattractant protein-1 [MCP-1], nerve growth factor [NGF], brain-derived neurotrophic factor [BDNF], vascular endothelial growth factor [VEGF]): (concentration of each biomarker in ng/mL, technology Luminex®)). | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Geometric Mean | 95% Confidence Interval | ng/mL | All assessed at baseline and V4 visit (6 months). Only GDF15, MCP1, BDNF, and TGFb1 also assessed at V3 visit (3 months) |
|
|
|
| Secondary | Biomarkers (3-Nitrotyrosine) | 3-Nitrotyrosine (Oxidative stress biomarker): at baseline, 3 and 6 months (concentration in ng/mL, ELISA method). | Not Posted | Jan 2026 | At baseline, 3 months, and 6 months. | Participants |
| Secondary | Quality of Life With ALSAQ-40 (ALS Assessment Questionnaire) | Change in ALS assessment questionnaire (ALSAQ-40). ALSAQ-40 (Amyotrophic Lateral Sclerosis Assessment Questionnaire) Quality of Life questionnaire 40 items, patient rated including 5 choices from never to always. best=0, worse=100 | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Mean | Standard Deviation | score on a scale | QoL will be assessed from baseline to 6 months |
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| Secondary | Efficacy Based on Assessment of Respiratory Function (Arterial Blood Gases [ABG]), PO2 (mmHg) | Assessment of respiratory function (Arterial Blood Gases [ABG]): description of PO2 (mmHg) at screening, 3 and 6 months. | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Mean | Standard Deviation | millimeters of Mercury | Respiratory function at screening, 3 and 6 months. |
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| Secondary | Efficacy Based on Assessment of Respiratory Function (Arterial Blood Gases [ABG]), HCO3 (mEq/L) | Assessment of respiratory function (Arterial Blood Gases [ABG]): description of HCO3 (mEq/L) at screening, 3 and 6 months. | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Mean | Standard Deviation | mEq/L (HCO3) | Respiratory function at screening, 3 and 6 months. |
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| Secondary | Efficacy Based on Assessment of Respiratory Function (Arterial Blood Gases [ABG]), Oxygen Saturation (%) | Assessment of respiratory function (Arterial Blood Gases [ABG]): description of Oxygen saturation (%) at screening, 3 and 6 months. | The presented data are on the Full Analysis Set (FAS), in which the number of patients changed over time as some patients were lost between baseline and V3 or V4 (death, consent withdrawal, ...). | Posted | Mean | Standard Deviation | % (O2 sat) | Respiratory function at screening, 3 and 6 months. |
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| 2 |
| 17 |
| 4 |
| 17 |
| 10 |
| 17 |
| EG001 | IFB-088 50 mg/Day + Riluzole 100 mg/Day | The test product, IFB-088, will be administered orally in 50 mg/day dosage consisting of two uptakes of 25 mg each (morning and evening uptakes), as an add-on therapy to riluzole 100 mg. Intervals for dosing should ideally be about 12 hours (± one hour). Tablets will be swallowed with a glass of water 30 minutes before the meal, in fasting condition. Administration of riluzole 100 mg, tablet or suspension, will be at the patient's and/or investigator's choice, as per summary of product characteristics. The daily dose of 100 mg will be taken in two 50 mg doses every 12 hours, at the same time than the IMPs. Patients will be treated for a period of 6 months (26 weeks). IFB-088 50mg/day: Tested product Riluzole 100mg/day: Standard of care treatment, co-administered with tested product (IFB-088 50mg/day) or placebo | 5 | 34 | 8 | 34 | 25 | 34 |
| Device related sepsis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Pneumonia aspiration | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Pneumonia viral | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| Respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| Lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| cardiac arrest | Cardiac disorders | MedDRA (26.0) | Systematic Assessment |
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| cardio-respiratory arrest | Cardiac disorders | MedDRA (26.0) | Systematic Assessment |
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| disease progression | General disorders | MedDRA (26.0) | Systematic Assessment |
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| hospitalisation | Surgical and medical procedures | MedDRA (26.0) | Systematic Assessment |
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| Diarrhoea | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| gastrooesophageal reflux disease | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| abdominal pain | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| dysphagia | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| Abdominal pain upper | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| constipation | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| reflux gastritis | Gastrointestinal disorders | MedDRA (26.0) | Systematic Assessment |
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| hypertension | Vascular disorders | MedDRA (26.0) | Systematic Assessment |
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| flank pain | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
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| pollakiuria | Renal and urinary disorders | MedDRA (26.0) | Systematic Assessment |
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| anxiety | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
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| depression | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
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| mood altered | Psychiatric disorders | MedDRA (26.0) | Systematic Assessment |
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| asthenia | General disorders | MedDRA (26.0) | Systematic Assessment |
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| disease progression | General disorders | MedDRA (26.0) | Systematic Assessment |
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| pyrexia | General disorders | MedDRA (26.0) | Systematic Assessment |
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| atrial fibrillation | Cardiac disorders | MedDRA (26.0) | Systematic Assessment |
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| bradycardia | Cardiac disorders | MedDRA (26.0) | Systematic Assessment |
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| cardiac arrest | Cardiac disorders | MedDRA (26.0) | Systematic Assessment |
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| cardia-respiratory arrest | Cardiac disorders | MedDRA (26.0) | Systematic Assessment |
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| exostosis | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| myalgia | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| spinal pain | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| tendonitis | Musculoskeletal and connective tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| gastrostomy | Surgical and medical procedures | MedDRA (26.0) | Systematic Assessment |
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| cataract operation | Surgical and medical procedures | MedDRA (26.0) | Systematic Assessment |
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| hospitalisation | Surgical and medical procedures | MedDRA (26.0) | Systematic Assessment |
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| alopecia | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| dermatitis contact | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| erythema | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| rash erythematous | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| urticaria | Skin and subcutaneous tissue disorders | MedDRA (26.0) | Systematic Assessment |
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| fall | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| clavicle fracture | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| eschar | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| hand fracture | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| joint injury | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| periorbital haematoma | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| procedural dizziness | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| skin injury | Injury, poisoning and procedural complications | MedDRA (26.0) | Systematic Assessment |
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| respiratory failure | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| respiratory distress | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| hypercapnia | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| laryngospasm | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| lower respiratory tract congestion | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| lung disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| orthopnoea | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| respiratory disorder | Respiratory, thoracic and mediastinal disorders | MedDRA (26.0) | Systematic Assessment |
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| COVID-19 | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| Oral fungal infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| bronchitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| urinary tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| bronchitis fungal | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| cystitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| device related sepsis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| diverticulitis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| gastroenteritis | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| influenza | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| pneumonia | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| pneumonia aspiration | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| pneumonia viral | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| respiratory tract infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
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| sebaceous gland infection | Infections and infestations | MedDRA (26.0) | Systematic Assessment |
|
Not provided
Not provided
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| D001393 |
| Azoles |
| D006573 | Heterocyclic Compounds, 1-Ring |
| D006571 | Heterocyclic Compounds |
| D006574 | Heterocyclic Compounds, 2-Ring |
| D000072471 | Heterocyclic Compounds, Fused-Ring |
| ALSFRS-R V3 visit (3 months) |
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| ALSFRS-R V4 visit (6 months) |
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| post hoc analysis adjusted on baseline NfL on top of previous parameters | ANCOVA | 0.65 | post hoc analysis on the FAS population, adjusted on baseline NfL on top of previous parameters | Mean Difference (Final Values) | 1.5 | Standard Error of the Mean | 3.3 | 2-Sided | 95 | -5.1 | 8.2 | Other | FAS post hoc |
| V3 visit |
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| V4 visit |
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| V3 visit |
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| V4 visit |
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| V3 visit |
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| V4 visit |
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| PaCO2 V3 Visit |
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| PaCO2 V4 visit |
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| V4 visit |
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| GDF15 V3 visit |
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| GDF15 V4 visit |
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| MCP1 baseline |
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| MCP1 V3 visit |
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| MCP1 V4 visit |
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| BDNF baseline |
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| BDNF V3 visit |
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| BDNF V4 visit |
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| TGFb1 baseline |
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| TGFb1 V3 visit |
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| TGFb1 V4 visit |
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| 8-OxoDG baseline |
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| 8-OxoDG V4 visit |
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| FGF21 baseline |
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| FGF21 V4 visit |
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| NGFR/p75ECD baseline |
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| NGFR/p75ECD V4 visit |
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| IL-6 baseline |
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| IL-6 V4 visit |
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| TNFa baseline |
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| TNFa V4 visit |
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| IFNg baseline |
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| IFNg V4 visit |
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| IL1b baseline |
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| IL1b V4 visit |
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| IL8 baseline |
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| IL8 V4 visit |
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| IL10 baseline |
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| IL10 V4 visit |
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| V4 visit |
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| PaO2 V3 Visit |
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| PaO2 V4 Visit |
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| HCO3 V3 visit |
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| HCO3 V4 visit |
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| O2 saturation V3 visit |
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| O2 stauration V4 visit |
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