Clinical trials in ALS
Birmingham study
This prospective, single-arm, open-label phase II clinical trial of sustained weekly administration of 2 mg/kg ILB® was conducted in a single UK hospital. 11 patients were recruited and treated for up to 38 weeks. There were no treatment terminations or withdrawals.
As a result of the COVID-19 pandemic and the high-risk status of study participants, recruitment and treatment was suspended early in Mar-2020. At the long-term follow-up, three patients had died after the trial was halted, between 53 and 62 weeks after their final ILB® injection. Long-term weekly ILB® injections of 2 mg/kg was well tolerated and had an acceptable safety profile in patients with ALS.
For more information, see the full publication from the trial.
The Gothenburg trial
Thirteen patients with ALS were treated with 5 weekly subcutaneous injections of ILB®. Safety and efficacy outcome measures were recorded weekly during treatment and at regular intervals for a further 70 days.
No deaths, serious adverse events or participant withdrawals occurred during or after ILB® treatment and no significant drug-related changes in blood safety markers were evident, demonstrating safety and tolerability of the drug in this cohort of patients with ALS. The ILB® injection elicited a transient elevation of plasma Hepatocyte Growth Factor, a neurotrophic and myogenic growth factor.
The ALSFRS-R rating improved from 36.31 ± 6.66 to 38.77 ± 6.44 and the Norris rating also improved from 70.61 ± 13.91 to 77.85 ± 14.24 by Day 36. The improvement of functions was associated with a decrease in muscle atrophy biomarkers. These therapeutic benefits decreased 3–4 weeks after the last dosage.
This pilot clinical study demonstrates safety and tolerability of ILB® in patients with ALS. The exploratory biomarker and functional measures must be cautiously interpreted but suggest clinical benefit and have a bearing on the mechanism of action of ILB®.
For more information, see the full publication from the trial.
The Oslo trial
Oslo University Hospital has in 2026 started a randomized controlled trial in ALS comparing ILB® to riluzole, the current European accepted standard of care for ALS. The trial will include 116 patients in a 6-month RCT phase followed by 6 months' open-label extension where patients will get both ILB® and riluzole.
For more information, see the full study protocol.
ILB® early access programme
TikoMed conducts an early access programme for ILB® in ALS.