Pegcetacoplan (EMPAVELI™)

Action: Inhibition of complement, which is involved in inflammation. It was used to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH).

Clinical Trial Status: Phase II

Participants are being recruited.


Action: Inhibition of C3 complement, which is involved in inflammation of the motor endplates at the nerve junction. It is used to treat adult patients with paroxysmal nocturnal hemoglobinuria (PNH). Animal studies have been inconclusive regarding the efficacy of complement inhibition. (Lobsiger et al., 2013; Woodruff et al., 2008)

Clinical trial status: Evaluation of the efficacy and safety of Pegcetacoplan (pegetacoplan) in adult patients Phase II Identifier: NCT04579666 not recruiting

not recruiting

Criteria for selection: Sporadic ALS with onset within 16.5 months (72 weeks) and slow vital capacity >60% of the predicted value

Dosage: Subcutaneous injection twice a week

Side effects: Streptococcus pneumoniae, meningococcus, and Haemophilus influenzae type B (vaccination required)

Note: Labulizumab (Yurtomiris®), an inhibitor of the C5 complement, was discontinued early after an interim analysis of a phase III trial, because it failed to demonstrate efficacy.

Recombinant human HGF protein

Action: Neurotrophic factor. It inhibits progression in rodent models (Ishigaki et al., 2007)

Clinical trial status: A phase II trial consisting of a placebo-controlled double-blind and open-label continuation study to evaluate the efficacy and safety of 6-month intraspinal administration of HGF protein (KP-100IT) using NP022 (intrathecal catheter and subcutaneous implant port). ALS severity of 1 or %FVC ≥70% within 30 months of onset

UMIN Study ID: UMIN000022050

End of entry: The observation date for the last case was completed.

AT-1501 (anti-CD40L) (Tegoprubar)

Action: Suppresses neuroinflammation

Clinical Trial Status: Phase IIa suppressed disease progression compared to that in the controls in the ALS clinical trial data.


Action: Suppresses neuroinflammation by inhibiting the interaction between CD40 ligand (CD40L), a surface antigen of immune cells, and CD40 receptors.

Clinical trial status: Phase IIa completed

Tegoprubart well tolerated, no serious adverse events.

ALSFRS decline was suppressed compared to that in the controls in an ALS clinical trial data set in the ALS PRO-ACT database*.

Dosage: Biweekly intravenous administration

Latozinemab, AL001, GSK4527223

Action: Recombinant human anti-sortilin monoclonal IgG1.

Sortilin is a sorting receptor for intracellular trafficking. It is involved in neurotrophic factors, signal transduction, and lysosomal degradation. It regulates the endocytosis and degradation of progranulin, a major cause of frontotemporal dementia. Progranulin levels are reduced by 50%-70% in this disease. Suppression of sortilin with this drug increases progranulin. 

Clinical Trial Status: Phase 2 for ALS due to C9ORF72 mutation

The primary endpoints are safety, tolerability, pharmacokinetics and pharmacodynamics; plasma and CSF progranulin levels Identifier: NCT04436510

not recruiting

Low-dose aldesleukin

Action:Low dose (ld) IL2 boosted peripheral regulatory T cells (Tregs) and modified inflammation.

Clinical Trial Status: Phase II, completed Identifier: NCT02059759

Criteria for selection: ALS disease duration 24 months, VC 70%, not previously treated with riluzole.

Dosage: Subcutaneous injection in 5-day cycles every 28 days

Results: The primary efficacy outcome was time to death (survival) at 21 months (640 days) post-randomization. Aldesleukin tended to improve survival rates by 19% compared with a placebo, but the findings failed to reach statistical significance. In patients with lower levels of CSF phosphorylated neurofilament heavy chain (biomarker of disease severity), Aldesleukin results in a significant reduction – by 43% – in the risk of death and also slower disease progression in ALSFRS-R scores.



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