A Phase 2 interventional study of ILB in Amyotrophic Lateral Sclerosis, sponsored by TikoMed AB. Terminated at 1 site in Sweden. Open to participants aged 18 Years to 80 Years. Per ClinicalTrials.gov, last updated 2023-06-12.
Sponsored by TikoMed AB · Phase 2, Interventional, and Treatment
This is a Phase 2a single-centre, open single-arm study in patients with Amyotrophic Lateral Sclerosis (ALS) of intermediate progression rate. Eligible subjects will be administered weekly doses of ILB. A total of 5 subcutaneous (s.c.) doses will be administered at the study clinic.
The study consists of 10 visits; One 2-part screening visit, 5 ILB administration visits, and 3 follow-up visits. Each individual patient's study participation will be 4 months, including the screening and follow-up visits. Fifteen patients are planned to be included.
The primary objective of the study is to evaluate the safety and tolerability of ILB in patients diagnosed with ALS.
ILB is a solution for subcutaneous (s.c.) injection in saline solution. The dose administered will depend on the subject's body weight at the second study visit, prior to the first ILB administration.
No formal sample size calculation has been performed for this study. The proposed sample size is considered sufficient in this early phase 2 development to provide adequate information on the patients. Categorical data will be presented as counts and percentages. Continuous data will be summarised using descriptive statistics.
717 studies on the registry are indexed under Motor Neuron Disease; 137 are open to participants now.
This study's enrollment of 13 is below the median of 35 across 461 interventional studies indexed under Motor Neuron Disease.
Browse Motor Neuron Disease studies →This is the only study on the registry with TikoMed AB as lead sponsor.
Counted across the registry records on this site, refreshed daily.
Exclusion criteria
ILB treatment
Drug: ILB
The investigational medicinal product ILB will be given as single short-term s.c. injections in the abdomen, the thigh or the buttock, in that order of priority. Subjects will be observed for at least 3 hours after injection.The IMP is a sterile, colourless to pale yellow solution for subcutaneous injection.
Frequency, seriousness and intensity of Treatment-emergent Adverse Events (TEAEs)
A TEAE is any adverse event (AE) not present prior to the initiation of IMP administration or any event already present that worsens in either intensity or frequency following exposure to the IMP. AEs (including baseline events) identified using any of the following methods will be recorded: * AEs spontaneously reported by the subject * AEs observed by the Investigator or medical personnel * AEs elicited based on non-leading questions from the Investigator or medical personnel
Time frame: up to 3 months
Change in physical status
A complete physical examination according to clinical praxis will be performed, including assessment of the head, eyes, ears, nose, throat (EENT), cardiac, peripheral vascular, pulmonary, musculoskeletal, neurologic, abdominal, lymphatic and dermatological functions. According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in vital signs - blood pressure
Percent change in blood pressure (mmHg) from baseline at 3 months
Time frame: up to 3 months
Change in electrocardiogram (ECG) recordings
Change in single 12-lead ECG (PQ/PR (ms), QRS (ms), QT (ms) and QTcF (ms)) from baseline at 3 months
Time frame: up to 3 months
Change in vital signs - heart rate
Percent change in heart rate (bpm, beats per minute) from baseline at 3 months
Time frame: up to 3 months
Change in vital signs - body temperatue
Percent change in body temperature (degrees Celsius) from baseline at 3 months
Time frame: up to 3 months
Change in safety laboratory measurements - sodium, potassium, chloride, calcium, glucose (non-fasting)
According to clinical praxis, laboratory tests for sodium, potassium, chloride, calcium, glucose (non-fasting) will be analysed. Unit of measure is mmol/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in safety laboratory measurements - albumin
According to clinical praxis, laboratory test for albumin will be analysed. Unit of measure is g/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in safety laboratory measurements - AST, ALT, CK, alkaline phosphatase
According to clinical praxis, laboratory tests for aspartate amino-transferase (AST), alanine amino-transferase (ALT), creatine kinase (CK) and alkaline phosphatase will be analysed. Unit of measure is micro-kat/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in safety laboratory measurements - creatinine and total bilirubin
According to clinical praxis, laboratory tests for creatinine and total bilirubin will be analysed. Unit of measure is micro-mol/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in safety laboratory measurements - myoglobin
According to clinical praxis, laboratory test for myoglobin will be analysed. Unit of measure is micro-g/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in safety laboratory measurements - CRP
According to clinical praxis, laboratory test for C-reactive protein (CRP) will be analysed. Unit of measure is milli-g/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in hematology laboratory measurements - Hemoglobin and fibrinogen
According to clinical praxis, laboratory tests for hemoglobin (Hb) and fibrinogen will be analysed. Unit of measure is g/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in hematology laboratory measurements - Red blood cell count
According to clinical praxis, laboratory test for blood cell count (RBC) will be analysed. Unit of measure is 10x12/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in hematology laboratory measurements - WBC, platelets, basophils, eosinophils, lymphocytes, monocytes, neutrophils
According to clinical praxis, laboratory tests for white blood cell count (WBC), platelets, basophils, eosinophils, lymphocytes, monocytes and neutrophils will be analysed. Unit of measure is 10x9/L According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in hematology laboratory measurements - APTT
According to clinical praxis, laboratory test for activated partial thromboplastin time (aPTT) will be analysed. Unit of measure is s According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in hematology laboratory measurements - PK-INR
According to clinical praxis, laboratory test for prothrombin kinase international normalized ratio (PK-INR) will be analysed. Unitless measure According to clinical praxis each function is judged as either normal or abnormal, and either clinically significant (CS) or not clinically significant (NCS) Change from baseline at 3 months.
Time frame: up to 3 months
Change in functional rating with Amyotrophic Lateral Sclerosis Functional Rating Scale - Revised (ALSFRS-R)
The ALSFRS-R provides a physician-generated estimate of the patient's degree of functional impairment, which can be evaluated serially to objectively assess any response to treatment or progression of disease. The ALSFRS-R includes questions that ask the physician to rate his/her impression of the patients level of functional impairment in performing one of twelve common tasks. Each task is rated on a five-point scale from 0 = can't do, to 4 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 48=best Absolute change from baseline at 3 months
Time frame: up to 3 months
Change in functional rating with Norris rating scale
The Norris rating scale provides a physician-generated estimate of the patient's degree of functional impairment, which can be evaluated serially to objectively assess any response to treatment or progression of disease. The Norris rating scale includes questions that ask the physician to rate his/her impression of the patients level of functional impairment in performing one of 34 common tasks and bodily functions. Each task or function is rated on a four-point scale from 0 = can't do, to 3 = normal ability. Individual item scores are summed to produce a reported score of between 0=worst and 100=best Absolute change from baseline at 3 months
Time frame: up to 3 months
Change in pulmonary function (FVC) from baseline
Time frame: up to 3 months
Change in Quality of Life (QoL) assessed by visual analogue scale (VAS)
Questionnaire with three questions for patient and next-of-kin self reporting of: * general health status * physical health status * mental health status Each item is rated on a millimeter scale from 0 = very bad, to 100 = very good Absolute change from baseline at 3 months
Time frame: up to 1.5 months
Change in functional rating of autonomous and sensory symptoms
The autonomous and sensory rating scale provides a physician-generated estimate of the patient's degree of functional impairment, which can be evaluated serially to objectively assess any response to treatment or progression of disease. The rating scale includes questions that ask the physician to rate his/her impression of the patients level of impairment in 16 autonomous and sensory functions. Each function is rated on a four-point scale from 0 = not impaired, to 3 = very impaired. Absolute change from baseline at 3 months
Time frame: up to 3 months
Change in maximum plasma concentration (Cmax) of ILB
Time frame: up to 1 month
Change in exposure (Area Under the Curve, AUC) of ILB
Time frame: up to 1 month
Changes in APTT (effect APTT) from day of dosing (day 1)
Time frame: up to 1 month
Change in plasma concentration of hepatocyte growth factor (HGF)
Time frame: up to 1 month
This study is terminated, as verified in Jun 2023. You cannot join it, but the record below documents what was studied.
Get an email when the registry record changes — status, dates, results — or when someone posts here.
Sign in to followQuestions and observations about this study, from anyone following it. Not medical advice, and not a channel to the study team — their contact details are on the registry record.
Sign in to join the discussion. Reading takes no account; posting does. You choose a display name, and a pseudonym is the default.
Nothing here yet. If you are running this trial, taking part in it, or weighing whether to, this is the place to say so.