CURRENT RECRUITING TRIALS
CONTACT US
als-cru.neuro@mcgill.ca OR (514) 398-5500
ALS-DARI1 (Darifenacin)
A Study to Assess the Safety, Tolerability, and Pharmacology of Darifenacin in Patients With ALS
Amyotrophic lateral sclerosis (ALS) is a progressive neurological disorder characterized by selective death of upper and lower motor neurons, which leads to severe disability and fatal outcomes. One of the major hallmarks of ALS is the denervation of neuromuscular junctions (NMJs), which is one of the earliest events seen in ALS patients and mouse models of ALS. Under healthy conditions, glial cells called Perisynaptic Schwann Cells (PSCs) have a key role in regulating the stability and maintenance of NMJs, but they only participate in NMJ repair once denervation occurs. Denervation and the subsequent decline in synaptic activity triggers a loss of muscarinic acetylcholine receptors (mAChRs) in the PSC, and the resulting decrease in mAChR-mediated gene expression drives the “repair mode” of the PSC.
In assessing the NMJ under conditions of ALS, a scarcity of process extensions in PSCs was observed for months prior to disease onset in the superoxide dismutase 1 (SOD1) mouse model of ALS, indicating inadequate glial repair. Collectively, these preclinical findings support the hypothesis that dampening glial mAChRs will restore the anticipated “repair” response of PSCs in the NMJ. Hence, the use of a selective M3 muscarinic receptor antagonist, darifenacin, as a disease-modifying therapeutic in familial and sporadic ALS could improve NMJ function, resulting in a beneficial impact on the autonomy and quality of life of ALS patients.
ACTIVE (NOT RECRUITING) TRIALS
ABBVIE CALICO (ABBV-CLS-7262)
ABBV-CLS-7262 is an investigational drug being researched for the treatment of Amyotrophic Lateral Sclerosis. This is a 48-week, 2-part study. Part 1 will be a 4-week, randomized, double-blind, placebo-controlled study; Part 2 will be a 44-week active treatment extension (ATE) during which all subjects will receive ABBV-CLS-7262.
ARGENX REALISE (ARGX-119)
A Safety and Efficacy Study of ARGX-119 in Adult Patients with Amyotrophic Lateral Sclerosis (ALS) (ReALiSe)
A Phase 2a, Double-Blinded, Randomized, Placebo-Controlled, and Active-Treatment Extension Study to Assess the Safety, Tolerability, Efficacy, Pharmacokinetics, and Immunogenicity of ARGX-119 in Participants with Amyotrophic Lateral Sclerosis
This study aims to evaluate the safety of ARGX-119 in adults with ALS. The study will also assess the impact of ARGX-119 on ALS disease outcomes, including muscle function. The study consists of 2 periods: a treatment period when participants will receive one of three ARGX-119 doses or placebo and an extension period when all participants will receive the same dose of ARGX-119. Participation in the study will last up to approximately 100 weeks.
CORCEPT DAZALS (Dazucorilant)
A Phase 2, Multicenter, Randomized, Double-Blind, Placebo-Controlled Study Evaluating Safety and Efficacy of CORT113176 (Dazucorilant) in Patients With Amyotrophic Lateral Sclerosis (DAZALS)
The purpose of this study is to assess the safety and efficacy of CORT113176 (dazucorilant) in patients with Amyotrophic Lateral Sclerosis (ALS).
Eligible ALS patients will be randomized to one of three treatment arms (1:1:1) across North America and Europe for a 24-week double-blind treatment period.
Patients who complete participation (i.e., completed all visits) in the double-blind treatment period will be eligible for participation in a 132-week open-label extension (OLE) study. A daily dose of 300 mg dazucorilant will be used in the 132-week OLE period.
Patients that complete the double-blind treatment period and who do not enter the OLE will enter the 132-week follow-up period.
IONIS ION363-CS1 (FUS ALS)
A Phase 1-3 Study to Evaluate the Efficacy, Safety, Pharmacokinetics and Pharmacodynamics of Intrathecally Administered ION363 in Amyotrophic Lateral Sclerosis patients with Fused in Sarcoma mutations (FUS-ALS)
To evaluate the clinical efficacy of ION363 in clinical functioning and survival in Amyotrophic Lateral Sclerosis (ALS) patients with Fused in Sarcomamutations (FUS-ALS).
KYORIN COMBAT (Ibudilast)
A Phase 2b/3 multicenter, randomized, double-blind, placebo-controlled, parallel group study to evaluate the efficacy, safety and tolerability of MN-166 given to ALS participants for 12 months followed by a 6-month open-label extension phase.
PROJENX PRO-101 (Prosetin)
A Phase 1C study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of prosetin in ALS patients.
Prosetin is a selective, oral, brain-penetrant, MAP4K inhibitor developed by ProJenX co-founders at Columbia University for the treatment of ALS.
Following the discovery that MAP4K inhibition confers potent motor neuron protection across multiple patient stem cell-derived models of ALS, prosetin was optimized for potency against MAP4Ks, efficacy in motor neuron rescue, and preferential distribution to the CNS.
QURALIS QRL-201
The primary objective of this study is to determine the safety and tolerability of multiple doses of QRL-201 in people living with ALS
UPCOMING TRIALS
Study list to come.
The Team – ALS
Dr Angela Genge
Principal Investigator
Dr Oliver Blanchard
Principal Investigator
Sabrina Yusuf
Clinical Research Coordinator
The Team – ALS
Dr Rami Massie
Principal Investigator
Michael Pileggi
Team Lead
Tanya Karyakina
Clinical Research Coordinator
SPOTLIGHT ON OUR RESEARCH
Click to read