The ALS Study
Research type
Research Study
Full title
A Phase II pilot safety and tolerability study of ILB in patients with Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS)
IRAS ID
242984
Contact name
Venkataramanan Srinivasan
Contact email
Sponsor organisation
University of Birmingham
Eudract number
2018-000668-28
Duration of Study in the UK
2 years, 6 months, 0 days
Research summary
Research Summary:
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. Neurons receive and send messages from the body to the brain and back to the body and are responsible for controlling voluntary muscle movement. Voluntary muscles produce movements like chewing, walking and talking. ALS is caused by gradual deterioration (degeneration) and death of motor neurons. In ALS, the motor neurons degenerate or die, and stop sending messages to the muscles. Unable to function, the muscles gradually weaken and waste away. Eventually the brain loses its ability to initiate and control voluntary movement.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) https://www.ninds.nih.gov/Disorders/Patient-Caregiver-Education/Fact-Sheets/Amyotrophic-Lateral-Sclerosis-ALS-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.
ILB has been developed by a small Swedish industry company called TikoMed. TikoMed along with colleagues at the University of Birmingham have tested this drug in a number of pre-clinical (animal studies) and in 79 humans (69 healthy volunteers and 10 patients with diabetes). Results of these studies indicate there may be some beneficial effects to using ILB in a number of diseases including ALS. This will be the first study in this patient group.
We will invite 15 patients to take part from a single centre in the UK.
Patients will be closely monitored for any side-effects; for changes in ALS symptoms and on their quality of life during and after the study. At the end of the study we will know if ILB is safe and if it is acceptable to ALS patients.
The ALS study
Our research will answer an important question: will ILB be safe and acceptable for patients with ALS. We will do this by measuring the number and severity of unanticipated experiences or reactions (both serious and non-serious) that occur when patients are treated with ILB, these events will be graded, recorded and closely monitored. Similarly we also hope to be able to assess how acceptable the treatment is to ALS patients. In addition to the safety and tolerability assessments we would hope to be able to describe the effect of ILB on patients ALS symptoms, and on their quality of life.
We don’t know if ILB will have any effect on the patients in this study or ALS patients generally but the information gained from this study will help researchers develop future studies in this disease area.
The study can be broken down into 5 key stages:
Stage 1 Screening tests
Stage 2 The start of treatment, within 14 days of the screening tests
Stage 3 Weekly hospital visits for 10 weeks for safety checks and study injections
Stage 4 End of treatment visit at week 12 with additional tests for safety checks
Stage 5 Follow up – includes visits to hospital at 16, 20 and 24 weeks for safety checks
Trial durationThe trial period for patient participation is 24 weeks (6 months). We hope to recruit all 15 patients over an 8- 12 month period.
The broad timetable for the study is as follows: we plan to recruit the first patient in September/October 2018.
Once the last patient has completed the last visit, the data collection will be closed and data analysed. We anticipate a trial report within 12 months of the last patient completing the study. This will be end of quarter one 2020.
Number of patients
15 patients in totalSummary of Results:
Abstract Purpose of the study: The aim of this trial was to assess the safety and tolerability of ILB®, a drug containing a molecule called dextran sulphate, in patients with Amyotrophic Lateral Sclerosis (ALS).
What was tested: ILB® was tested for safety in a group of patients with ALS in this Phase 2 trial. In a phase 2 trial, a smaller number of patients receive the drug to see if it’s safe and whether it is worthwhile doing a larger trial.
People taking part: 11 adults with ALS in the United Kingdom took part and were recruited over approximately 1 year.
Results: There were no serious side effects associated with ILB® treatment. No intolerable events occurred during the trial and only 1 Serious Adverse Event (SAE) was reported, which was not related to ILB®. The trial ended earlier than planned due to the COVID-19 pandemic and the high-risk status of study participants.
Safety: This phase 2 clinical trial study found that ILB® is safe to use in the patients with ALS at the concentrations tested.REC name
South Central - Oxford B Research Ethics Committee
REC reference
18/SC/0368
Date of REC Opinion
8 Aug 2018
REC opinion
Further Information Favourable Opinion