Not provided
| ID | Type | Description | Link |
|---|---|---|---|
| 2017-005065-47 | EudraCT Number |
Not provided
Not provided
Not provided
Inclusion was stopped with 13 of 15 patients recruited according to protocol. Patients responded with no safety signals. Study stop was due to slow recruitment.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
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.
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Label | Type | Description | Intervention Names |
|---|---|---|---|
| ILB | Experimental | ILB treatment |
|
| Name | Type | Description | Arm Group Labels | Other Names |
|---|---|---|---|---|
| ILB | Drug | 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. |
| Measure | Description | Time Frame |
|---|---|---|
| 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:
| 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. | up to 3 months |
| Change in vital signs - blood pressure | Percent change in blood pressure (mmHg) from baseline at 3 months | 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 | up to 3 months |
| Change in vital signs - heart rate | Percent change in heart rate (bpm, beats per minute) from baseline at 3 months | up to 3 months |
| Change in vital signs - body temperatue |
| Measure | Description | Time Frame |
|---|---|---|
| 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 |
Not provided
Inclusion Criteria:
Exclusion criteria
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
| Facility | Status | City | State | ZIP | Country | Contacts |
|---|---|---|---|---|---|---|
| Sahlgrenska University Hospital | Gothenburg | Sweden |
| PubMed Identifier | Type | Citation | Retractions |
|---|---|---|---|
| 35613082 | Result | Logan A, Nagy Z, Barnes NM, Belli A, Di Pietro V, Tavazzi B, Lazzarino G, Lazzarino G, Bruce L, Persson LI. A phase II open label clinical study of the safety, tolerability and efficacy of ILB(R) for Amyotrophic Lateral Sclerosis. PLoS One. 2022 May 25;17(5):e0267183. doi: 10.1371/journal.pone.0267183. eCollection 2022. | |
| 34442438 |
Not provided
Not provided
Not provided
| ID | Term |
|---|---|
| D000690 | Amyotrophic Lateral Sclerosis |
| ID | Term |
|---|---|
| D013118 | Spinal Cord Diseases |
| D002493 | Central Nervous System Diseases |
| D009422 | Nervous System Diseases |
| D016472 | Motor Neuron Disease |
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
Not provided
|
Percent change in body temperature (degrees Celsius) from baseline at 3 months |
| 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | 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. | up to 3 months |
| 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 | up to 3 months |
| Change in pulmonary function (FVC) from baseline | 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:
Each item is rated on a millimeter scale from 0 = very bad, to 100 = very good Absolute change from baseline at 3 months | 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 | up to 3 months |
| Change in maximum plasma concentration (Cmax) of ILB | up to 1 month |
| Change in exposure (Area Under the Curve, AUC) of ILB | up to 1 month |
| Changes in APTT (effect APTT) from day of dosing (day 1) | up to 1 month |
| Change in plasma concentration of hepatocyte growth factor (HGF) | up to 1 month |
| Lazzarino G, Mangione R, Belli A, Di Pietro V, Nagy Z, Barnes NM, Bruce L, Ropero BM, Persson LI, Manca B, Saab MW, Amorini AM, Tavazzi B, Lazzarino G, Logan A. ILB(R) Attenuates Clinical Symptoms and Serum Biomarkers of Oxidative/Nitrosative Stress and Mitochondrial Dysfunction in Patients with Amyotrophic Lateral Sclerosis. J Pers Med. 2021 Aug 14;11(8):794. doi: 10.3390/jpm11080794. |
| D019636 | Neurodegenerative Diseases |
| D057177 | TDP-43 Proteinopathies |
| D009468 | Neuromuscular Diseases |
| D057165 | Proteostasis Deficiencies |
| D008659 | Metabolic Diseases |
| D009750 | Nutritional and Metabolic Diseases |