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Study initially suspended due to COVID-19 Pandemic- treatment stopped - never reopened to recruitment..
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| Name | Class |
|---|---|
| TikoMed AB | INDUSTRY |
| University Hospital Birmingham | OTHER |
| Neuregenix Ltd | UNKNOWN |
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This is a phase II study to determine the safety and tolerability of ILB®, a type of low molecular weight dextran sulfate, in patients with Motor Neurone Disease (MND)/ Amyotrophic Lateral Sclerosis (ALS)
Amyotrophic Lateral Sclerosis (ALS) belongs to a wider group of disorders known as motor neuron diseases and mainly involves the nerve cells (neurons) in the body. Voluntary muscles produce movements like chewing, walking and talking. ALS is caused by gradual deterioration (degeneration) and death of these motor neurons. The disease is progressive, meaning the symptoms get worse over time and most people with ALS die from respiratory failure, usually within 3 to 5 years from when the symptoms first appear. Currently there is no cure for ALS and no effective treatment to halt or reverse the progression of the disease (National Institute of Neurological Disorders and Stroke, Fact Sheet).
The aim of this study is to explore the safety and acceptability of a type of low molecular weight dextran sulfate called ILB®.
The investigators will invite 15 patients to take part from a single centre in the United Kingdom (UK). Participants will be closely monitored for any side-effects; for changes in ALS symptoms and on quality of life during and after the study.
The trial period for patient participation is maximum 56 weeks (12 months), ILB® injections will be administered once weekly for up to a maximum of 48 weeks.
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| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ILB® Arm | Experimental | ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ILB® | Drug | Administration will be weekly subcutaneous injections at a dose of 2mg/kg once per week for up to a maximum of 48 weeks |
|
| Measure | Description | Time Frame |
|---|---|---|
| Safety Assessed by SAEs and AEs - Measured by Incidence | Measured by the number of serious adverse events (SAEs) and adverse events (AEs) using CTCAE grading v4.0. | From informed consent up to 30 days after last administration of trial treatment |
| Safety Assessed by AEs - Summarised by Grade | Grade refers to the severity of the AE as follows: Grade 1 - Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 - Moderate; minimal, local or non-invasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 - Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 - Life-threatening consequences; urgent intervention indicated. Grade 5 - Death related to AE. | From informed consent up to 30 days after last administration of trial treatment |
| Safety Assessed by AEs - Summarised by Relatedness | Relatedness categories: 1 = unrelated, 2 = unlikely to be related, 3 = possibly related, 4 = probably related, 5 = definitely related | From informed consent up to 30 days after last administration of trial treatment |
| Safety Assessed by SAEs - Summarised by Admitting Event Grade | Grade refers to the severity of the admitting event as follows: Grade 1 - Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 - Moderate; minimal, local or non-invasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 - Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 - Life-threatening consequences; urgent intervention indicated. Grade 5 - Death related to AE. |
| Measure | Description | Time Frame |
|---|---|---|
| Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Score Change | Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R). This patient reported outcome measures the subjective well-being of patients. There are 5 scales which are calculated and scored: physical mobility, independence,eating and drinking, communication,emotional functioning. Each is scored between 0-100. An improved condition is represented by a decreasing sub-scale score. These sub scales are then averaged to make a summary index score. The range of the summary index is 0-100 and an improved condition is represented by decreasing summary index score. For each of the sub-scales and summary index. Interpretation is as follows: 0-19 Never or very rarely, 20-39 rarely experience problems, 40-59 sometimes experience problems,60-79 often experience, 80-100 problems (nearly) always or unable to do at all. The scale of the summary score is also 0-100 with analogous interpretation to the sub scales. An increase in score is a worse outcome. |
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Inclusion Criteria:
Patients ≥18 years and who have provided written informed consent to participate in the study
Prior to trial entry patients will have a definite diagnosis of ALS according to El Escorial Criteria. All patients will demonstrate either:
Electrophysiological tests (Electromyography (EMG) / Nerve Conduction Study (NCS)) that supports the diagnosis of Motor Neurone Disease (MND) and to exclude mimic disorders
Forced Vital Capacity (FVC) ≥50% of predicted value for gender, height and age at screening and a mean Sniff Nasal Inspiratory Pressure (SNIP) ≥50% of predicted value for age
Adequate haematological function (Hb≥10g/dl absolute neutrophil count ≥1.5x109/L and a platelet count ≥60 x109/L
International Normalised Ratio (INR) ≤ 1.5, Activated Partial Thromboplastin Time (aPTT) 30 - 40 seconds, Prothrombin Time (PT) 11-13.5 seconds
Patient willing and able to comply with schedule visits, treatment plan and other study procedures.
Patients taking Riluzole must have discontinued treatment ≥28 days prior to study entry (and following consent to take part in the study)
Women Of Child Bearing Potential (WOCBP) who agree to use highly effective means of contraception (as defined in the Heads of Medicines Agencies_Clinical Trials Facilitation Group (HMA_CTFG) guideline (see Appendix 8) and in combination with a barrier contraception method (condom, diaphragm or cap) for the entirety of the study
Exclusion Criteria:
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| Name | Affiliation | Role |
|---|---|---|
| Venkataramanan Srinivasan, MRCP, MRCP | University of Birmingham | Principal Investigator |
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| University Hospitals Birmingham NHS Foundation Trust | Birmingham | West Midlands | B15 2TH | United Kingdom |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 38990927 | Derived | Srinivasan V, Homer V, Barton D, Clutterbuck-James A, Jenkins S, Potter C, Brock K, Logan A, Smith D, Bruce L, Nagy Z, Bach SP. A low molecular weight dextran sulphate, ILB(R), for the treatment of amyotrophic lateral sclerosis (ALS): An open-label, single-arm, single-centre, phase II trial. PLoS One. 2024 Jul 11;19(7):e0291285. doi: 10.1371/journal.pone.0291285. eCollection 2024. |
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Weekly dosing for 10 weeks (in the first instance) during out-patient visits at site. Dosing beyond 10 weeks (initially to 24 weeks and then up to a maximum of up to 48 weeks) was dependent upon a formal review of the patient's eligibility, their wishes and the most suitable treatment options.
Following suspension of the trial due to the COVID-19 pandemic, patients were asked to re-consent for an additional single point long-term remote follow up visit in Quarter 1 of 2021 via video call.
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| ID | Title | Description |
|---|---|---|
| FG000 | ILB® Arm | ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks |
| Title | Milestones | Reasons Not Completed | ||||||||||||||||||
|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|---|
| 10 Week Initial Treatment Period |
|
| ||||||||||||||||||
| Treatment Extension |
|
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| ID | Title | Description |
|---|---|---|
| BG000 | ILB® Arm | ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks |
| Units | Counts |
|---|---|
| Participants |
|
| Title | Description | Population Description | Parameter Type | Dispersion Type | Unit of Measure | Calculate Percentage | Denominator Units Selected | Denominators | Classes |
|---|---|---|---|---|---|---|---|---|---|
| Age, Categorical | Count of Participants |
| 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 Assessed by SAEs and AEs - Measured by Incidence | Measured by the number of serious adverse events (SAEs) and adverse events (AEs) using CTCAE grading v4.0. | Posted | Number | Adverse events | From informed consent up to 30 days after last administration of trial treatment |
|
|
Details of all Adverse Events (AEs) were documented and reported from the participant signing of the informed consent and included the collection of all baseline AEs. AE collection continued until 30 days after the end of treatment visit. Up to 52 weeks. The actual length of AEs collection for each participants was determined by the individual participant length of time on the study.
All AEs, either observed by the Investigator or reported by the subject, were recorded by the Investigator (on an adverse event form) and evaluated. AEs were reviewed using the Common Terminology Criteria for Adverse Events (CTCAE) version 4.0.
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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 | ILB® Arm | ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Generalised muscle weakness | Musculoskeletal and connective tissue disorders | CTCAE V4.0 | Systematic Assessment |
| Term | Organ System | Source Vocabulary | Assessment Type | Notes | Statistical Information |
|---|---|---|---|---|---|
| Not clinically significant abnormal blood creatinine | Blood and lymphatic system disorders | CTCAE V4.0 | Systematic Assessment |
Early termination due to the COVID-19 pandemic, the high-risk status of trial patients, and the frequent hospital based visits, meant that both recruitment to the trial and further treatment was suspended after 11 patients had been recruited. As a result, patients were treated with interrupted and varying numbers of weekly ILB® treatments.
The pharmacokinetic evaluation was limited due to rejection of some poor quality blood samples.
| Title | Organization | Phone | Extension | |
|---|---|---|---|---|
| D3B Trial Mangement Team Leader | CRCTU, University of Birmingham | +44 (0) 121 371 8027 | ALS@trials.bham.ac.uk |
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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 | Nov 25, 2020 | Jul 1, 2024 | Prot_000.pdf |
| SAP | No | Yes | No | Statistical Analysis Plan | Sep 11, 2020 | Jul 18, 2024 | SAP_001.pdf |
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| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| D016472 | Motor Neuron Disease |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D019636 | Neurodegenerative Diseases |
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| From informed consent up to 30 days after last administration of trial treatment |
| Safety Assessed by SAEs - Summarised by Admitting Event Relatedness | Relatedness categories: 1 = unrelated, 2 = unlikely to be related, 3 = possibly related, 4 = probably related, 5 = definitely related | From informed consent up to 30 days after last administration of trial treatment |
| Safety Assessed by SAEs - Summarised by Admitting Event Type | Description of the main event type - primary cause of admission (body system, Adverse event term and grade) | From informed consent up to 30 days after last administration of trial treatment |
| Safety Assessed by SAEs - Summarised by Expectedness | Serious Adverse Events only will be defined as expected or unexpected based on information provided in the Quick Reference Document | From informed consent up to 30 days after last administration of trial treatment |
| Safety Assessed by SAEs - Summarised by Sequelae | Outcome of serious adverse events only: Resolved with sequelae or Resolved without sequelae | From informed consent up to 30 days after last administration of trial treatment |
| Tolerability Assessed by the Incidence of Intolerable Adverse Events | An intolerable adverse event will satisfy all of the following criteria:
Adverse events which are considered unrelated or probably not related will not be classed as intolerable events. | From informed consent up to 30 days after last administration of trial treatment |
| Quantity of Study Drug Administered - Total Drug Administered | Total drug administered over the study period (measured in milligrams) | From baseline to final treatment visit |
| Quantity of Study Drug Administered - Number of Administrations | Numerical count of the number of study drug injections given whilst on the trial | From baseline to final treatment visit |
| Quantity of Study Drug Administered - Number of Interruptions | Numerical count of the number of study drug injections missed whilst on the trial | From baseline to final treatment visit |
| Quantity of Study Drug Administered - Duration of Interruptions | Length of interruptions in weeks between study drug injections for those participants that experienced a treatment interruption whilst on the trial | From baseline to final treatment visit |
| Quantity of Study Drug Administered - Number of Discontinuations | numerical count of patients who have discontinued study drug treatment | From baseline to final treatment visit |
| From baseline to final treatment visit |
| Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change | Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 . A functional rating scale including assessments of communication, mobility, dressing and respiration. the total score range is 0-40. An improved condition is represented by decreasing sub-scale score. Interpretation is as follows: 0 being the best outcome and 40 being the worst. Minimum value is 0 and Maximum value is 40 per time-point / questionnaire completion | From baseline to final treatment visit |
| Urinary p75ECD Change | Urinary p75 extracellular domain (p75ECD) is a biological fluid-based biomarker of ALS disease progression | From baseline to final treatment visit |
| Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration | This outcome measure quantifies the amount of drug detectable in the blood after administration over time | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
| Pharmacokinetics (PK; Tmax) Statistics of ILB® in Plasma Following Administration | Tmax (the time the peak concentration occurred) was calculated to characterise the kinetic profile of ILB® in plasma post-administration | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
| Pharmacokinetics (PK; Cmax) Statistics of ILB® in Plasma Following Administration | Cmax (peak concentration of ILB® in plasma post-administration) was calculated to characterise the kinetic profile of ILB® in plasma post-administration | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
| Pharmacokinetics (PK; AUC0-last) Statistics of ILB® in Plasma Following Administration | AUC0-last (area under the curve time 0 (time of administration) to the last value above the limit of quantification) was calculated to characterise the kinetic profile of ILB® in plasma post-administration | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
| Pharmacokinetics (PK; t1/2) Statistics of ILB® in Plasma Following Administration | t1/2 (terminal half-life of ILB® in plasma post-administration) was calculated to characterise the kinetic profile of ILB® in plasma post-administration | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
| NfL in Plasma Change | Plasma neurofilament light chain (NfL) is a blood-based biomarker for neurodegeneration | from baseline to final treatment visit |
|
| Participants |
|
| Age, Continuous | Mean | Standard Deviation | years |
|
| Sex: Female, Male | Count of Participants | Participants |
|
| Race and Ethnicity Not Collected | Race and Ethnicity were not collected from any participant. | Count of Participants | Participants |
|
| Region of Enrollment | Number | participants |
|
| Time from ALS diagnosis to trial entry (months) | Mean | Standard Deviation | months |
|
| Family history of motor neurone disease (n(%)) | Count of Participants | Participants |
|
| Family history of fronto-temporal dementia (n(%)) | Count of Participants | Participants |
|
|
| Primary | Safety Assessed by AEs - Summarised by Grade | Grade refers to the severity of the AE as follows: Grade 1 - Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 - Moderate; minimal, local or non-invasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 - Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 - Life-threatening consequences; urgent intervention indicated. Grade 5 - Death related to AE. | Posted | Number | number of AEs | From informed consent up to 30 days after last administration of trial treatment |
|
|
|
| Primary | Safety Assessed by AEs - Summarised by Relatedness | Relatedness categories: 1 = unrelated, 2 = unlikely to be related, 3 = possibly related, 4 = probably related, 5 = definitely related | Posted | Number | number of AEs | From informed consent up to 30 days after last administration of trial treatment |
|
|
|
| Primary | Safety Assessed by SAEs - Summarised by Admitting Event Grade | Grade refers to the severity of the admitting event as follows: Grade 1 - Mild; asymptomatic or mild symptoms; clinical or diagnostic observations only; intervention not indicated. Grade 2 - Moderate; minimal, local or non-invasive intervention indicated; limiting age-appropriate instrumental activities of daily living. Grade 3 - Severe or medically significant but not immediately life-threatening; hospitalization or prolongation of hospitalization indicated; disabling; limiting self care activities of daily living. Grade 4 - Life-threatening consequences; urgent intervention indicated. Grade 5 - Death related to AE. | Posted | Number | number of SAEs | From informed consent up to 30 days after last administration of trial treatment |
|
|
|
| Primary | Safety Assessed by SAEs - Summarised by Admitting Event Relatedness | Relatedness categories: 1 = unrelated, 2 = unlikely to be related, 3 = possibly related, 4 = probably related, 5 = definitely related | Posted | Number | number of SAEs | From informed consent up to 30 days after last administration of trial treatment |
|
|
|
| Primary | Safety Assessed by SAEs - Summarised by Admitting Event Type | Description of the main event type - primary cause of admission (body system, Adverse event term and grade) | Posted | Number | number of SAEs | From informed consent up to 30 days after last administration of trial treatment |
|
|
|
| Primary | Safety Assessed by SAEs - Summarised by Expectedness | Serious Adverse Events only will be defined as expected or unexpected based on information provided in the Quick Reference Document | Expectedness of SAEs is only ascertained in the instance of the event being a Serious Adverse Reaction (SAR), therefore as the only SAE was unrelated, expectedness was not applicable | Posted | From informed consent up to 30 days after last administration of trial treatment |
|
|
|
| Primary | Safety Assessed by SAEs - Summarised by Sequelae | Outcome of serious adverse events only: Resolved with sequelae or Resolved without sequelae | Posted | Number | number of SAEs | From informed consent up to 30 days after last administration of trial treatment |
|
|
|
| Primary | Tolerability Assessed by the Incidence of Intolerable Adverse Events | An intolerable adverse event will satisfy all of the following criteria:
Adverse events which are considered unrelated or probably not related will not be classed as intolerable events. | Posted | Number | Number of intolerable adverse events | From informed consent up to 30 days after last administration of trial treatment |
|
|
|
| Primary | Quantity of Study Drug Administered - Total Drug Administered | Total drug administered over the study period (measured in milligrams) | Posted | Median | Inter-Quartile Range | mg | From baseline to final treatment visit |
|
|
|
| Primary | Quantity of Study Drug Administered - Number of Administrations | Numerical count of the number of study drug injections given whilst on the trial | Posted | Median | Inter-Quartile Range | number of drug administrations | From baseline to final treatment visit |
|
|
|
| Primary | Quantity of Study Drug Administered - Number of Interruptions | Numerical count of the number of study drug injections missed whilst on the trial | Posted | Median | Inter-Quartile Range | number of interruptions | From baseline to final treatment visit |
|
|
|
| Primary | Quantity of Study Drug Administered - Duration of Interruptions | Length of interruptions in weeks between study drug injections for those participants that experienced a treatment interruption whilst on the trial | 6 patients experienced a treatment interruption whilst on the trial | Posted | Median | Inter-Quartile Range | weeks | From baseline to final treatment visit |
|
|
|
| Primary | Quantity of Study Drug Administered - Number of Discontinuations | numerical count of patients who have discontinued study drug treatment | Posted | Number | number of treatment discontinuations | From baseline to final treatment visit |
|
|
|
| Secondary | Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Score Change | Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R). This patient reported outcome measures the subjective well-being of patients. There are 5 scales which are calculated and scored: physical mobility, independence,eating and drinking, communication,emotional functioning. Each is scored between 0-100. An improved condition is represented by a decreasing sub-scale score. These sub scales are then averaged to make a summary index score. The range of the summary index is 0-100 and an improved condition is represented by decreasing summary index score. For each of the sub-scales and summary index. Interpretation is as follows: 0-19 Never or very rarely, 20-39 rarely experience problems, 40-59 sometimes experience problems,60-79 often experience, 80-100 problems (nearly) always or unable to do at all. The scale of the summary score is also 0-100 with analogous interpretation to the sub scales. An increase in score is a worse outcome. | Posted | Median | Inter-Quartile Range | score on a scale | From baseline to final treatment visit |
|
|
|
| Secondary | Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change | Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 . A functional rating scale including assessments of communication, mobility, dressing and respiration. the total score range is 0-40. An improved condition is represented by decreasing sub-scale score. Interpretation is as follows: 0 being the best outcome and 40 being the worst. Minimum value is 0 and Maximum value is 40 per time-point / questionnaire completion | Posted | Median | Inter-Quartile Range | change in ALSAQ-40 score from baseline | From baseline to final treatment visit |
|
|
|
| Secondary | Urinary p75ECD Change | Urinary p75 extracellular domain (p75ECD) is a biological fluid-based biomarker of ALS disease progression | Posted | Median | Inter-Quartile Range | ng/mmol creatinine | From baseline to final treatment visit |
|
|
|
| Secondary | Pharmacokinetics (PK; Amount of Detectable Drug) of ILB® in Plasma Following Administration | This outcome measure quantifies the amount of drug detectable in the blood after administration over time | Posted | Mean | Standard Deviation | μg/mL | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
|
|
|
| Secondary | Pharmacokinetics (PK; Tmax) Statistics of ILB® in Plasma Following Administration | Tmax (the time the peak concentration occurred) was calculated to characterise the kinetic profile of ILB® in plasma post-administration | Posted | Mean | Standard Deviation | hours | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
|
|
|
| Secondary | Pharmacokinetics (PK; Cmax) Statistics of ILB® in Plasma Following Administration | Cmax (peak concentration of ILB® in plasma post-administration) was calculated to characterise the kinetic profile of ILB® in plasma post-administration | Posted | Mean | Standard Deviation | μg/mL | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
|
|
|
| Secondary | Pharmacokinetics (PK; AUC0-last) Statistics of ILB® in Plasma Following Administration | AUC0-last (area under the curve time 0 (time of administration) to the last value above the limit of quantification) was calculated to characterise the kinetic profile of ILB® in plasma post-administration | Posted | Mean | Standard Deviation | μg*h/mL | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
|
|
|
| Secondary | Pharmacokinetics (PK; t1/2) Statistics of ILB® in Plasma Following Administration | t1/2 (terminal half-life of ILB® in plasma post-administration) was calculated to characterise the kinetic profile of ILB® in plasma post-administration | The terminal half-life t1/2 could not be calculated for 3 out of the 6 patients as the concentration of ILB® in plasma did not drop below half its maximal value. | Posted | Mean | Standard Deviation | hours | 0.5,1,2,2.5,3,4 and 6 hours post first ILB® administration |
|
|
|
| Secondary | NfL in Plasma Change | Plasma neurofilament light chain (NfL) is a blood-based biomarker for neurodegeneration | Posted | Median | Inter-Quartile Range | ng/L | from baseline to final treatment visit |
|
|
|
| 0 |
| 11 |
| 1 |
| 11 |
| 11 |
| 11 |
| Not clinically significant creatinine kinase, 286, (normal range 30-200) | Blood and lymphatic system disorders | CTCAE V4.0 | Systematic Assessment |
|
| Not clinically significant monocyte count, 0.9, (normal range 0.2 - 0.8) | Blood and lymphatic system disorders | CTCAE V4.0 | Systematic Assessment |
|
| Not clinically significant raised red cell distribution, 14.8 (normal range 11-14) | Blood and lymphatic system disorders | CTCAE V4.0 | Systematic Assessment |
|
| 'Sticky' left eye | Eye disorders | CTCAE V4.0 | Systematic Assessment |
|
| Constipation | Gastrointestinal disorders | CTCAE V4.0 | Systematic Assessment |
|
| Diarrhea | Gastrointestinal disorders | CTCAE V4.0 | Systematic Assessment |
|
| Rectal bleeding | Gastrointestinal disorders | CTCAE V4.0 | Systematic Assessment |
|
| Chesty cough | General disorders | CTCAE V4.0 | Systematic Assessment |
|
| Cold chills down left arm | General disorders | CTCAE V4.0 | Systematic Assessment |
|
| Cramps in the chest and abdomen | General disorders | CTCAE V4.0 | Systematic Assessment |
|
| Discomfort at injection site when touched | General disorders | CTCAE V4.0 | Systematic Assessment |
|
| Patient felt faint | General disorders | CTCAE V4.0 | Systematic Assessment |
|
| Bilateral ear infection | Infections and infestations | CTCAE V4.0 | Systematic Assessment |
|
| Chest infection | Infections and infestations | CTCAE V4.0 | Systematic Assessment |
|
| Common cold | Infections and infestations | CTCAE V4.0 | Systematic Assessment |
|
| Patient visited the dentist and had a filling | Infections and infestations | CTCAE V4.0 | Systematic Assessment |
|
| Bruising | Injury, poisoning and procedural complications | CTCAE V4.0 | Systematic Assessment |
|
| Fall | Injury, poisoning and procedural complications | CTCAE V4.0 | Systematic Assessment |
|
| Bruising to right hip from fall | Injury, poisoning and procedural complications | CTCAE V4.0 | Systematic Assessment |
|
| Bruising to right shoulder following fall | Injury, poisoning and procedural complications | CTCAE V4.0 | Systematic Assessment |
|
| Bruising to the face following fall | Injury, poisoning and procedural complications | CTCAE V4.0 | Systematic Assessment |
|
| Fall up the stairs leading to flat due to increasing leg weakness | Injury, poisoning and procedural complications | CTCAE V4.0 | Systematic Assessment |
|
| Fall while getting out of bed, due to increase in leg weakness | Injury, poisoning and procedural complications | CTCAE V4.0 | Systematic Assessment |
|
| Activated partial thromboplastin time prolonged | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Alanine aminotransferase increased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Alkaline phosphatase increased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Aspartate aminotransferase increased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Blood bilirubin increased | Investigations | CTCAE V4.0 | Systematic Assessment |
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| Cholesterol high | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Hemoglobin increased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Abnormal CRP blood results | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Abnormal eosinophil blood results | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Abnormal glucose levels (3.1, normal results 3.5-11.0) | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Abnormal hdl level, clinically insignificant. | Investigations | CTCAE V4.0 | Systematic Assessment |
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| Abnormal mean cell haemoglobin level, clinically insignificant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Abnormal platelet distribution width result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Abnormal ptt blood result (elevated) | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Abnormal red cell distribution levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Creatinine level decreased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| CRP increased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased aptt result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased basophil count | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased basophil level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased basophil result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased creatinine | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased creatinine levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased creatinine result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased creatinine value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased haematocrit value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased haemoglobin value | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased hdl cholesterol result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased hdl cholesterol value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased hdl cholesterol value -not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased hdl value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased igm result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased monocyte result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased potassium level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased rbc dist width | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased rbc distribution width | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased red blood cell value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Decreased sodium levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated aptt ratio | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated ast blood level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated c- reactive protein blood results | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated calcim level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated calcium level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated ck levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated creatine kinase result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated crp result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated crp value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated eosinophil result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated haematocrit blood level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated hdl cholesterol blood levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated mean cell haemoglobin | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated mean cell hb concentration | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated mean cell hb level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated mean cell hb result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated monocyte result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated red blood cell count | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated sodium levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Elevated total protein result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Hdl cholesterol levels high | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased ck value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased crp level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased eosinophils count - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased esr value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased iga value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased monocyte count - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased neutrophil count - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased platelet dist width | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased platelet dist. width | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased rbc distribution width | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Increased white blood cell value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Low albumin level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Low haematocrit | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Low sodium | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Mean cell hb concentration level elevated | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Prolonged pr interval on ecg | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised ast blood levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised ast levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised blood glucose level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised crp result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised eosinophil count | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised eosinophils | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised hdl cholesterol blood levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised sodium level | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Raised total protein result | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Reduced aptt ratio value - not clinically significant | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Reduced basophil count | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Reduced free thyroxine levels | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Lymphocyte count decreased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Neutrophil count decreased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| White blood cell decreased | Investigations | CTCAE V4.0 | Systematic Assessment |
|
| Back pain | Musculoskeletal and connective tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Fractured nasal bone following fall | Musculoskeletal and connective tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Increased leg weakness leading to inability to stand resulting in admission to hospital | Musculoskeletal and connective tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Muscle spasm in legs | Musculoskeletal and connective tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Occasional spasm in neck when yawning | Musculoskeletal and connective tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Painful left shoulder blade | Musculoskeletal and connective tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Right hip pain | Musculoskeletal and connective tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Headache | Nervous system disorders | CTCAE V4.0 | Systematic Assessment |
|
| Insomnia | Psychiatric disorders | CTCAE V4.0 | Systematic Assessment |
|
| Allergic rhinitis | Respiratory, thoracic and mediastinal disorders | CTCAE V4.0 | Systematic Assessment |
|
| Pneumonia | Respiratory, thoracic and mediastinal disorders | CTCAE V4.0 | Systematic Assessment |
|
| Sore throat | Respiratory, thoracic and mediastinal disorders | CTCAE V4.0 | Systematic Assessment |
|
| Eczema | Skin and subcutaneous tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Rash on abdomen around injection site | Skin and subcutaneous tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
| Rash on both feet | Skin and subcutaneous tissue disorders | CTCAE V4.0 | Systematic Assessment |
|
The Clinical Centre shall not publish or otherwise disseminate the conclusions of the Study, including all or any part of the Results of the Study without the prior written consent of the Sponsor, such consent not to be unreasonably withheld or delayed. Any publication or other dissemination of the conclusions of the Study by the National Health Organisation (NHS) Organisation shall not' occur until the Sponsor has published the conclusions of the Study.
Results published- PubMed ID: 38990927
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |
| Title | Measurements |
|---|
|
| Grade 4 |
|
| Grade 5 |
|
| Probably related |
|
| Definitely related |
|
| Title | Measurements |
|---|
|
| Grade 4 |
|
| Grade 5 |
|
| Probably related |
|
| Definitely related |
|
| Title | Measurements |
|---|---|
|
| Communication |
|
| Emotional functioning |
|
| Summary index score |
|
|
| 2.5 hours |
|
| 3.0 hours |
|
| 4.0 hours |
|
| 6.0 hours |
|