A Study to Evaluate the Safety, Tolerability and Efficacy of ILB in Patients With Amyotrophic Lateral Sclerosis
A Single-centre, Open Single-arm Study Where the Safety, Tolerability and Efficacy of Subcutaneously Administered ILB Will be Evaluated in Patients With Amyotrophic Lateral Sclerosis
Lead SponsorTikoMed AB
Indication/ConditionAmyotrophic Lateral Sclerosis
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.
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.
Inclusion Criteria: Willing and able to give written informed consent for participation in the study. Clinical diagnosis of Amyotrophic Lateral Sclerosis (ALS). Male or female patients between 18 to 80 years (inclusive). Forced Vital Capacity (FVC) 65% of predicted value for gender, height and age at screening. Evaluated with ALSFRS-R and Norris clinical rating scales for at least the past 4 weeks before study drug administration. Exclusion criteria Unable to understand information about the study or are expected not to collaborate with the study team. Concurrent serious disease, other than ALS, at the discretion of the nvestigator. Pregnancy. Patients of childbearing potential not willing to use adequate double contraception with less than 1 percentage failure rate after the screening visit until the last visit. Addiction to drugs or alcohol. Confirmed HIV, hepatitis B or hepatitis C. Known bleeding disorders or abnormal bleeding events. Treatment with anticoagulant drugs warfarin and novel oral anticoagulants (NOAC) within the last 14 days prior to screening. Treatment with Riluzole or Lamotrigine within the last 28 days prior to study drug administration. Hypersensitivity to dextran sulfate. Poor venous access. Patients with clinically significant abnormal PK-INR, fibrinogen, von Willebrand factor and activated partial thromboplastin time (APTT) at screening.