A Safety and Tolerability Study of ILB® in Patients With Amyotrophic Lateral Sclerosis (ALS) (ALS)

June 25, 2025 updated by: University of Birmingham

A Phase II Pilot Single-arm Safety and Tolerability Study of ILB® in Patients With Motor Neurone Disease (MND)/ Amyotrophic Lateral Sclerosis (ALS)

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)

Study Overview

Status

Status

Indicates the current recruitment status or the expanded access status.
Terminated

Conditions

Conditions

The disease, disorder, syndrome, illness, or injury that is being studied. On ClinicalTrials.gov, conditions may also include other health-related issues, such as lifespan, quality of life, and health risks.

Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.

Detailed Description

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.

Study Type

Study Type

Describes the nature of a clinical study. Study types include interventional studies (also called clinical trials), observational studies (including patient registries), and expanded access.
Interventional

Enrollment (Actual)

Enrollment

The number of participants in a clinical study. The "anticipated" enrollment is the target number of participants that the researchers need for the study.
11

Phase

Phase

The stage of a clinical trial studying a drug or biological product, based on definitions developed by the U.S. Food and Drug Administration (FDA). The phase is based on the study's objective, the number of participants, and other characteristics. There are five phases: Early Phase 1 (formerly listed as Phase 0), Phase 1, Phase 2, Phase 3, and Phase 4. Not Applicable is used to describe trials without FDA-defined phases, including trials of devices or behavioral interventions.
  • Phase 2

Contacts and Locations

This section provides the contact details for those conducting the study, and information on where this study is being conducted.

Study Locations

  • United Kingdom
    • West Midlands
      • Birmingham, West Midlands, United Kingdom, B15 2TH
        • University Hospitals Birmingham NHS Foundation Trust

Participation Criteria

Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.

Eligibility Criteria

Eligibility Criteria

The key requirements that people who want to participate in a clinical study must meet or the characteristics they must have. Eligibility criteria consist of both inclusion criteria (which are required for a person to participate in the study) and exclusion criteria (which prevent a person from participating). Types of eligibility criteria include whether a study accepts healthy volunteers, has age or age group requirements, or is limited by sex.

Ages Eligible for Study

18 years and older (Adult, Older Adult)

Accepts Healthy Volunteers

No

Description

Inclusion Criteria:

  1. Patients ≥18 years and who have provided written informed consent to participate in the study

  2. Prior to trial entry patients will have a definite diagnosis of ALS according to El Escorial Criteria. All patients will demonstrate either:

    • presence of Upper Motor Neuron (UMN) (increased tone, brisk reflexes) as well as Lower Motor Neuron (LMN) (weakness,
    • wasting and fasciculation) signs in the bulbar region and at least two of the other spinal regions (cervical, thoracic or lumbosacral) or
    • presence of UMN and LMN signs in all three spinal regions (cervical, thoracic or lumbosacral)
  3. Electrophysiological tests (Electromyography (EMG) / Nerve Conduction Study (NCS)) that supports the diagnosis of Motor Neurone Disease (MND) and to exclude mimic disorders
  4. 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
  5. Adequate haematological function (Hb≥10g/dl absolute neutrophil count ≥1.5x109/L and a platelet count ≥60 x109/L
  6. International Normalised Ratio (INR) ≤ 1.5, Activated Partial Thromboplastin Time (aPTT) 30 - 40 seconds, Prothrombin Time (PT) 11-13.5 seconds
  7. Patient willing and able to comply with schedule visits, treatment plan and other study procedures.
  8. Patients taking Riluzole must have discontinued treatment ≥28 days prior to study entry (and following consent to take part in the study)
  9. 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:

  1. Patients classified as either probable or possible ALS according to El Escorial Criteria.
  2. Subjects in whom other causes of neuromuscular weakness have not been excluded
  3. Assisted ventilation of any type within 3 months before the screening visit or at screening
  4. Patients requiring Radiologically Inserted Gastrostomy (RIG) or Percutaneous Endoscopic Gastroscopy (PEG) feeding
  5. Involvement in any other interventional study involving use of another Investigational Medicinal Product (IMP) or biological product, within 3 months of screening
  6. Any use of antioxidants, edaravone, tirasemtiv or CK-2127107 within 1 month before the screening visit
  7. Any botulinum toxin use within 3 months before the screening visit.
  8. Any form of stem cell or gene therapy for the treatment of amyotrophic lateral sclerosis (ALS)
  9. Neuroimaging of brain and cervical spine with Magnetic Resonance imaging (MRI) indicating compressive myelopathy as an alternate diagnosis
  10. Laboratory examinations including Acetylcholine receptor (AChR) antibodies and Muscle Specific Kinase (MuSK) antibodies to exclude Bulbar onset Myasthenia gravis from Bulbar onset Motor neuron disease as an alternate diagnosis and Antinuclear Antibodies (ANA), Anti-neutrophil cytoplasmic antibodies (ANCA), Extractable Nuclear Antigen (ENA) antibodies, Creatine Kinase (CK), electrophoresis and immunoglobulin indicating an alternate diagnosis for muscle disease like Myositis
  11. Abnormal liver function defined as Aspartate Transaminase (AST) and/or Alanine Transaminase (ALT) >3 times upper limit of normal
  12. Any head trauma, intracranial or spinal surgery within 3 months of trial entry
  13. Patients who have had recurrent falls will be excluded to reduce the risk of intracerebral haemorrhage with this IMP
  14. Current use of an anticoagulant e.g Warfarin, Aspirin, Clopidogrel, any novel anticoagulants (NOAC)s or low molecular weight subcutaneous heparin
  15. Uncontrolled severe hypertension defined as systolic blood pressure (SBP) ≥ 220 mmHg or diastolic blood pressure (DBP) ≥120 mmHg
  16. Current or previous history of heparin-induced thrombocytopenia
  17. Active peptic ulcer disease
  18. Known hypersensitivity to sulphur
  19. Severe liver insufficiency
  20. Patients with evidence of major psychiatric illness, significant cognitive impairment or clinically evident dementia that may interfere with the patients' ability to comply with study procedures
  21. Pulmonary illness (e.g asthma or Chronic Obstructive Pulmonary Disease (COPD)) requiring regular treatment
  22. Patient judged to be actively suicidal by the investigator during 3 months before the screening visit
  23. Subjects with a diagnosis of another neurodegenerative disease (e.g. Parkinson's disease, Alzheimer's disease and Frontotemporal dementia)
  24. Pregnant and/or breastfeeding.

Study Plan

This section provides details of the study plan, including how the study is designed and what the study is measuring.

How is the study designed?

Design Details

  • Primary Purpose: Treatment
  • Allocation: N/A
  • Interventional Model: Single Group Assignment
  • Masking: None (Open Label)

Arms and Interventions

Participant Group / Arm

Participant Group / Arm

A group or subgroup of participants in a clinical trial that receives a specific intervention/treatment, or no intervention, according to the trial's protocol.
Intervention / Treatment

Intervention / Treatment

A process or action that is the focus of a clinical study. Interventions include drugs, medical devices, procedures, vaccines, and other products that are either investigational or already available. Interventions can also include noninvasive approaches, such as education or modifying diet and exercise.
Experimental: ILB® Arm
ILB® subcutaneous injection at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Drug: ILB®
Administration will be weekly subcutaneous injections at a dose of 2mg/kg once per week for up to a maximum of 48 weeks
Other Names:
  • DSSS5
  • TM-500
  • TM-700
  • LMW-DS
  • IBsolvMIR

What is the study measuring?

Primary Outcome Measures

Primary Outcome Measures

In a clinical study's protocol, the planned outcome measure that is the most important for evaluating the effect of an intervention/treatment. Most clinical studies have one primary outcome measure, but some have more than one.
Outcome Measure
Measure Description
Time Frame
Safety Assessed by SAEs and AEs - Measured by Incidence
Time Frame: From informed consent up to 30 days after last administration of trial treatment
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
Time Frame: From informed consent up to 30 days after last administration of trial treatment

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
Time Frame: From informed consent up to 30 days after last administration of trial treatment
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
Time Frame: From informed consent up to 30 days after last administration of trial treatment

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.
From informed consent up to 30 days after last administration of trial treatment
Safety Assessed by SAEs - Summarised by Admitting Event Relatedness
Time Frame: From informed consent up to 30 days after last administration of trial treatment
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
Time Frame: From informed consent up to 30 days after last administration of trial treatment
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
Time Frame: From informed consent up to 30 days after last administration of trial treatment
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
Time Frame: From informed consent up to 30 days after last administration of trial treatment
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
Time Frame: From informed consent up to 30 days after last administration of trial treatment

An intolerable adverse event will satisfy all of the following criteria:

  1. Associated with a serious adverse event or a drug discontinuation of greater than three weeks;
  2. Grade 3, 4 or 5 in severity according to CTCAE version 4;
  3. In the opinion of the Investigator is i) definitely related or ii) probably related or iii) possibly related to the study drug treatment.

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
Time Frame: From baseline to final treatment visit
Total drug administered over the study period (measured in milligrams)
From baseline to final treatment visit
Quantity of Study Drug Administered - Number of Administrations
Time Frame: From baseline to final treatment visit
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
Time Frame: From baseline to final treatment visit
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
Time Frame: From baseline to final treatment visit
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
Time Frame: From baseline to final treatment visit
numerical count of patients who have discontinued study drug treatment
From baseline to final treatment visit

Secondary Outcome Measures

Secondary Outcome Measures

In a clinical study's protocol, a planned outcome measure that is not as important as the primary outcome measure for evaluating the effect of an intervention but is still of interest. Most clinical studies have more than one secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
Amyotrophic Lateral Sclerosis Functional Rating Scale Revised (ALSFRS-R) Score Change
Time Frame: From baseline to final treatment visit
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.
From baseline to final treatment visit
Amyotrophic Lateral Sclerosis Assessment Questionnaire-40 (ALSAQ-40) Score Change
Time Frame: From baseline to final treatment visit
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
Time Frame: From baseline to final treatment visit
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
Time Frame: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® 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
Time Frame: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® 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
Time Frame: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® 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
Time Frame: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® 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
Time Frame: 0.5,1,2,2.5,3,4 and 6 hours post first ILB® 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
Time Frame: from baseline to final treatment visit
Plasma neurofilament light chain (NfL) is a blood-based biomarker for neurodegeneration
from baseline to final treatment visit

Other Outcome Measures

Other Outcome Measures

For clinical trials, a planned measurement described in the protocol that is used to determine the effect of an intervention/treatment on participants. For observational studies, a measurement or observation that is used to describe patterns of diseases or traits, or associations with exposures, risk factors, or treatment. Types of outcome measures include primary outcome measure and secondary outcome measure.
Outcome Measure
Measure Description
Time Frame
HPLC analyses of purine-pyrimidine metabolites (serum)
Time Frame: 48 weeks
Biomarker analysis - exploratory disease status
48 weeks
HPLC analysis of fat-soluble vitamins and antioxidants (serum)
Time Frame: 48 weeks
Biomarker analysis - exploratory disease status
48 weeks
HPLC analyses of amino acids (AA) and amino-group containing compounds (ACCG) (serum)
Time Frame: 48 weeks
Biomarker analysis - exploratory disease status
48 weeks
Spectrophotometric analysis of lactate
Time Frame: 48 weeks
Biomarker analysis - exploratory disease status
48 weeks

Collaborators and Investigators

This is where you will find people and organizations involved with this study.

Sponsor

Sponsor

The organization or person who initiates the study and who has authority and control over the study.
University of Birmingham

Collaborators

Collaborators

An organization other than the sponsor that provides support for a clinical study. This support may include activities related to funding, design, implementation, data analysis, or reporting.
TikoMed AB
University Hospital Birmingham
Neuregenix Ltd

Investigators

Investigators

A researcher involved in a clinical study. Related terms include site principal investigator, site sub-investigator, study chair, study director, and study principal investigator.
  • Principal Investigator: Venkataramanan Srinivasan, MRCP, MRCP, University of Birmingham

Study record dates

These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.

Study Major Dates

Study Start (Actual)

Study Start

The actual date on which the first participant was enrolled in a clinical study. The "anticipated" study start date is the date that the researchers think will be the study start date.
March 29, 2019

Primary Completion (Actual)

Primary Completion

The date on which the last participant in a clinical study was examined or received an intervention to collect final data for the primary outcome measure. Whether the clinical study ended according to the protocol or was terminated does not affect this date. For clinical studies with more than one primary outcome measure with different completion dates, this term refers to the date on which data collection is completed for all the primary outcome measures. The "anticipated" primary completion date is the date that the researchers think will be the primary completion date for the study.
July 28, 2021

Study Completion (Actual)

Study Completion

The date on which the last participant in a clinical study was examined or received an intervention/treatment to collect final data for the primary outcome measures, secondary outcome measures, and adverse events (that is, the last participant's last visit). The "anticipated" study completion date is the date that the researchers think will be the study completion date.
July 28, 2021

Study Registration Dates

First Submitted

First Submitted

The date on which the study sponsor or investigator first submitted a study record to ClinicalTrials.gov. There is typically a delay of a few days between the first submitted date and the record's availability on ClinicalTrials.gov (the first posted date).
September 11, 2018

First Submitted That Met QC Criteria

First Submitted That Met QC Criteria

The date on which the study sponsor or investigator first submits a study record that is consistent with National Library of Medicine (NLM) quality control (QC) review criteria. The sponsor or investigator may need to revise and submit a study record one or more times before NLM's QC review criteria are met. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
October 10, 2018

First Posted (Actual)

First Posted

The date on which the study record was first available on ClinicalTrials.gov after National Library of Medicine (NLM) quality control (QC) review has concluded. There is typically a delay of a few days between the date the study sponsor or investigator submitted the study record and the first posted date.
October 15, 2018

Study Record Updates

Last Update Posted (Actual)

Last Update Posted

The most recent date on which changes to a study record were made available on ClinicalTrials.gov. There may be a delay between when the changes were submitted to ClinicalTrials.gov by the study's sponsor or investigator (the last update submitted date) and the last update posted date.
June 26, 2025

Last Update Submitted That Met QC Criteria

Last Update Submitted That Met QC Criteria

The most recent date on which the study sponsor or investigator submitted changes to a study record that are consistent with National Library of Medicine (NLM) quality control (QC) review criteria. It is the responsibility of the sponsor or investigator to ensure that the study record is consistent with the NLM QC review criteria.
June 25, 2025

Last Verified

Last Verified

The most recent date on which the study sponsor or investigator confirmed the information about a clinical study on ClinicalTrials.gov as accurate and current. If a study with a recruitment status of recruiting; not yet recruiting; or active, not recruiting has not been confirmed within the past 2 years, the study's recruitment status is shown as unknown.
July 1, 2024

More Information

Terms related to this study

Keywords

Additional Relevant MeSH Terms

Other Study ID Numbers

Other Study ID Numbers

Identifiers or ID numbers other than the NCT number that are assigned to a clinical study by the study's sponsor, funders, or others. These numbers may include unique identifiers from other trial registries and National Institutes of Health grant numbers.
  • RG_17-250

Plan for Individual participant data (IPD)

Plan to Share Individual Participant Data (IPD)?

NO

Drug and device information, study documents

Studies a U.S. FDA-regulated drug product

No

Studies a U.S. FDA-regulated device product

No

This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.

Clinical Trials on Motor Neuron Disease

Clinical Trials on ILB®

Search Similar Trials

Sponsors and Collaborators

Medical Conditions

Drug Interventions

Conditions

Rare Diseases

Drug Interventions

Dietary Supplements

Sponsor/Collaborators

Locations

Cookie Settings

We use cookies to ensure that we give you the best experience on our website. For more details carefully read our Privacy and Cookies Policy. ...>>>You can change your preferences or withdraw your consent at any time by deleting the cookies from your website or computer as described in the policy. Please accept it and choose your preferences by ticking the corresponding boxes in the "Manage Settings" section below. Please carefully read our Terms of Service before you proceed with using any part of this website.
«Manage Settings