<p>NEMO’s activity aims to improve the patient’s quality of life with effective interventions focused on minimizing the damages caused by the disease progression.</p>


Principal investigator

English

Nederlands

Christian Lunetta - Tricals

Specialists

TUDCA (tauroursodeoxycholic acid) is a small molecule that is being explored for its potential as a treatment for amyotrophic lateral sclerosis (ALS). TUDCA may decrease nerve cell death through its ability to act as an antioxidant that prevents toxic reactive oxygen from accumulating inside cells. There may also be an effect on immune function.

TUDCA-ALS is a randomized, double-blind, placebo-controlled study. This means the effect of TUDCA is tested by comparing a group of patients that receive TUDCA to a group of patients receiving a placebo-drug. Patients will be randomized to one of two treatment arms. During the study, both the patient as the research team do not know who is receiving placebo or TUDCA. This will help researchers to assess potential side- and treatment effects of TUDCA.

TUDCA-ALS (TUDCA)

Industry sponsored trial

<h4>An innovative platform trial</h4>
Within an innovative platform design study, we will evaluate the efficacy of multiple promising compounds, all of which have been shown to be safe in patients with ALS and have a high potential for therapeutic efficacy. Patients will be randomized into the three treatment arms based on their genetic profile. The primary aim is to assess the efficacy of the drugs versus placebo on overall survival. A large advantage of this design is that we can decrease the chance of being randomized to placebo, namely in a ratio of 2:1 instead of 1:1. In the future, other compounds may be added to this platform study.
<h4>PreLude -  Lithium carbonate treatment arm</h4>
In the last decade, it has become increasingly clear ALS is not a single disease, but rather a collection of disorders with varying genetic backgrounds but highly similar clinical symptoms. It seems, therefore, increasingly unlikely that we will be able treat all forms of ALS with a single drug. With this in mind, previous findings of lithium as a drug for ALS were analysed again, showing an increased survival probability for UNC13a homozygous patients treated with lithium. This treatment arm aims to confirm the efficacy of lithium in UNC13a homozygous patients.

PreLude (Lithium carbonate)

Platform trial

<h4>An innovative platform trial</h4>
Within an innovative platform design study, we will evaluate the efficacy of multiple promising compounds, all of which have been shown to be safe in patients with ALS and have a high potential for therapeutic efficacy. Patients will be randomized into the three treatment arms based on their genetic profile. The primary aim is to assess the efficacy of the drugs versus placebo on overall survival. A large advantage of this design is that we can decrease the chance of being randomized to placebo, namely in a ratio of 2:1 instead of 1:1. In the future, other compounds may be added to this platform study.

<strong>Lighthouse – Triumeq treatment arm</strong>

Triumeq, a therapy developed for treatment of human immunodeficiency virus (HIV) infection has shown to slow progression on the ALSFRS-R in a Phase IIa trial in 40 patients with ALS, while Triumeq was also demonstrated to be safe and well tolerated. This treatment arm aims to confirm the efficacy of Triumeq as a treatment in patients with ALS in prolonging overall survival time.

Lighthouse (Triumeq)

<h4>An innovative platform trial</h4>
Within an innovative platform design study, we will evaluate the efficacy of multiple promising compounds, all of which have been shown to be safe in patients with ALS and have a high potential for therapeutic efficacy. Patients will be randomized into the three treatment arms based on their genetic profile. The primary aim is to assess the efficacy of the drugs versus placebo on overall survival. A large advantage of this design is that we can decrease the chance of being randomized to placebo, namely in a ratio of 2:1 instead of 1:1. In the future, other compounds may be added to this platform study.
<h4>iAdore - oral edaravone treatment arm</h4>
Edaravone is a formulation that reduces oxidative stress and is currently on the market in Japan and the United States in an intravenous formulation to treat ALS. As the chronic intravenous administration has important logistical drawbacks and safety concerns, an oral formulation of edaravone (TW001) was developed, which can be provided daily. TW001 has been shown to be safe and well-tolerated in three phase I studies. This treatment arm will investigate TW001 as treatment modality to prolong the life-expectancy in patients with ALS.

Centro Clinico Nemo

Contact

Specialists

Christian Lunetta

Current trials

TUDCA-ALS (TUDCA)

PreLude (Lithium carbonate)

Lighthouse (Triumeq)

iAdore (Oral edaravone)