A study to assess safety and acceptability of a treatment in patients with ALS
- Conditions
- Motor Neurone Disease (MND)/Amyotrophic Lateral Sclerosis (ALS)MedDRA version: 20.0 Level: PT Classification code 10002026 Term: Amyotrophic lateral sclerosis System Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2018-000668-28-GB
- Lead Sponsor
- niversity of Birmingham
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- Not specified
- Target Recruitment
- 15
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 UMN (increased tone, brisk reflexes) as well as 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)
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, aPTT 30 – 40 seconds, 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 for the entirety of the study
Are the trial subjects under 18? no
Number of subjects for this age range: 0
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subjects for this age range 15
F.1.3 Elderly (>=65 years) yes
F.1.3.1 Number of subjects for this age range 0
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 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 Neurone 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 AST and/or 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 Investigational Medicinal Product (IMP)
14. Current use of an anticoagulant e.g Warfarin, Aspirin, Clopidogrel, any novel anticoagulants (NOAC)s or low molecular weight heparin
15. Uncontrolled hypertension
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 patient`s 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, Altzheimers disease and Frontotemporal dementia)
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method