CURRENT RECRUITING TRIALS
AP101-02
A Study to Evaluate, Safety, Tolerability, Pharmacodynamic (PD) Markers and Pharmacokinetics (PK) of AP-101 in Participants With Amyotrophic Lateral Sclerosis (ALS)
The purpose of this project is to evaluate the safety, tolerability, PK, and PD of AP-101 in participants with fALS and sALS.
Eligible participants will be randomized to take the study drug or a placebo by intravenous infusion (IV).
This study includes infusions at Day 1 and a maintenance dose at Day 2 and every 3 weeks until week 24. There is a follow-up after an additional 12 weeks.
Biogen 275AS101 (Ataxin) (BIIB105)
The purpose of the study is to look at whether the BIIB105is safe and tolerable in people with ALS or polyQ-ALS. The study will also look at:
- What the body does to BIIB105(called “pharmacokinetics” or PK).
- The concentrations of BIIB105in the cerebrospinal fluid (CSF), which is the fluid around the spinal cord.
- What BIIB105does to the body (called “pharmacodynamics” or PD).
- Whether BIIB105affects the symptoms of ALS.
COMBAT-ALS (ibudilast)
From clinicaltrials.gov:
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.
HIMALAYA (ACT16970 - SAR443820)
Phase 2 Study for SAR443820 in Participants With Amyotrophic Lateral Sclerosis
This is a parallel treatment, Phase 2, randomized, double-blind study to assess the efficacy, safety, tolerability, PK, and PD of twice daily (BID) oral SAR443820 compared with placebo in male and female participants,18 to 80 years of age with ALS followed by an open label, longterm extension period.
Study ACT16970 consists of 2 parts (A and B) as follows:
Part A is a 24week, double blind, placebo controlled part, preceded by a screening period of up to 4 weeks before Day 1.
Part B is an open label, longterm extension period that starts from the end of Part A (Week 24) and continues up to Week 106.
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).
OBSERVATIONAL STUDIES
CAPTURE ALS
The Comprehensive Analysis Platform To Understand, Remedy and Eliminate ALS
This is a prospective, multicenter, longitudinal cohort study investigating the natural history of ALS and related disorders. CAPTURE ALS will collect data and biosamples from participants to create the most comprehensive biological picture of people living with ALS. Through open science, this information collected from participants will be shared anonymously with worldwide ALS researchers to better understand and treat ALS.
For this study, male and female patients and healthy controls aged 18 or older will be recruited at 4 sites across Canada. Patients with ALS or a related disorder including ALS-Frontotemporal Dementia, Primary Lateral Sclerosis and Progressive Muscular Atrophy, or asymptomatic individuals with a known ALS mutation are eligible to participate.
CALS-NIC2 (Biomarkers)
Novel MRI Biomarkers for monitoring disease progression in ALS
From clinicaltrials.gov:
Routine MRI is normal in motor neuron diseases such as ALS. However, advanced MRI techniques can provide an objective measure of degeneration (a “biomarker”) by examining brain structure, wiring, chemistry, and function. We will develop and evaluate novel MRI techniques that could improve our understanding of ALS and provide a means to diagnose it sooner and monitor its progression. Importantly, we expect these techniques to improve how new drugs are tested, which may lead to the more rapid discovery of a treatment for ALS.
Each participant will have 3 MRI scans over a period of 8 months, along with neurological and cognitive evaluations. Study visits will take 2 – 3 hours. MRI is a safe technique that does not involve radiation.
ALS Pharma
Le but de ce projet est de comparer la présence de protèine SOD1 dans le liquide céphalo-rachidien de participants atteints de SLA avec le liquide céphalo-rachidien de volontaires non SLA appariés sur le plan démographique. Pour ce faire, une ponction lombaire doit être pratiquée.
REFINE-ALS
Radicava®/(Edaravone) Findings in Biomarkers From ALS (REFINE-ALS)
The purpose of this study is to find biomarkers that show why edaravone is slowing ALS symptom progression. The data gathered during the study may also be useful for medical professionals and researchers in the future.
For this study, 300 male and female participants aged 18 and older will be recruited at approximately 40 sites.
You are about to start taking edaravone per your standard of care treatment for your ALS treatment. We will ensure that your visits for the clinical trial are scheduled around your standard of care treatment.
The Team – ALS
Dr Angela Genge
Principal Investigator
Vanessa Bertone
ALS Team Lead, Sr. Clin. Research Coord.
Maria Gobbo
Clinical Research Coordinator
The Team – ALS
Dr Rami Massie
Principal Investigator
Helen Fong
Clinical Research Coordinator
L’ÉQUIPE – SLA
UPCOMING TRIALS
Machine Learning in ALS
The purpose of this study is is to develop a tool that allows identification of UMN and LMN features in speech in patients with bulbar dysfunction due to ALS and other MND.
Learning more about characteristics of UMN and LMN symptoms in ALS/MND will help us to better understand the disease, possibly improve time between symptom onset and diagnosis and may result in new assessment and therapy tools.
For this study, 150 male and female participants will be recruited at about 11 ALS/MND clinics throughout Canada and the USA (in Toronto, London, Hamilton, Edmonton, Calgary, Saskatoon, Montreal, Quebec City, Fredericton, Gainesville and Ohio).
MT1186-A04 (oral edaravone extension)
An Open Label Extension Study open to patients who participated in MT1186-A03 for Oral Edaravone
More information to come.
ACTIVE (NOT RECRUITING) TRIALS
ALS Pharma LP Study
Misfolded SOD1 as a Biomarker for ALS
The purpose of this project is to compare the presence of SOD1 in the spinal fluid of participants with ALS against the spinal fluid of demographically matched non-ALS volunteers. In order to do so, a lumbar puncture must be administered.
For more information, please contact the ALS Team.
ANX-005-ALS1
The purpose of this study is to find out if the investigational drug ANX005 is well tolerated and safe in subjects with ALS when given on Day 1, Day 5 or 6 and then every 2 weeks on Week 2, 4, 6, 8 and 10. Another purpose of this study is to find out more about the investigational drug.
For this study, 24 male and female participants will be recruited at about 18 sites worldwide. The participants will be over the age of 18.
Biogen 233AS102 (BIIB067)
From clinicaltrials.gov:
The primary objective of the study is to evaluate the long-term safety and tolerability of BIIB067 in participants with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation (SOD1-ALS). The secondary objective is to evaluate the pharmacokinetic (PK) profile of BIIB067 in participants with SOD1-ALS.
CALICO
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.
FOCUS C9 (WVE-004)
Study of WVE-004 in Patients With C9orf72-associated Amyotrophic Lateral Sclerosis (ALS) or Frontotemporal Dementia (FTD) (FOCUS-C9)
This is a Phase 1b/2a multicenter, randomized, double-blind, placebo-controlled study to evaluate the safety, tolerability, PK, and PD of intrathecal (IT) WVE-004 in adult patients with C9orf72-associated ALS or FTD. To participate in the study, patients must have a documented mutation (GGGGCC [G4C2] repeat expansion) in the first intronic region of the C9orf72 gene and be diagnosed with ALS or FTD.
For this study, 42 individuals between the ages of 18 and 80 will be recruited.
MT 1186-A02 (daily oral edaravone)
A Phase 3b, Multicenter, Randomized, Double-Blind Study to Evaluate Efficacy and Safety of Oral Edaravone Administered for a Period of 48 Weeks in Subjects with Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to test whether an experimental oral liquid form of edaravone (a liquid solution that you will swallow) is effective as treatment against ALS; and if it is safe and well tolerated when given to patients with your illness. The study will compare the efficacy and safety of two different dosing regimens of the oral form of edaravone .
For this study, approximately 380 male and female participants will be recruited at approximately 95 sites. The participants will be aged between 18 and 75 years inclusively.
MT 1186-A03
A Phase 3, Multi-Center, Open-label, Safety Extension Study of Oral Edaravone Administered over 96 Weeks in Subjects with Amyotrophic Lateral Sclerosis (ALS)
The purpose of this study is to evaluate the long-term safety and effectiveness of edaravone in participants with Amyotrophic Lateral Sclerosis (ALS) over 96 weeks.
The results of this study could help Mitsubishi Tanabe Pharma Development America, Inc. acquire Health Canada approval for the oral liquid form of edaravone in the future. This would allow the oral liquid form of edaravone to be used in regular medical practice. The data gathered during the study may also be useful for medical professionals and researchers in the future.
For this study, 100 male and female participants who successfully completed study MT-1186-A01 will be enrolled in this study. The participants will be aged between 18 and 75 years.
REALS-1 (Enoxacin)
A randomized, double-blind, parallel group, single centre, phase 1b/2 study to assess the safety, tolerability, pharmacokinetics and pharmacodynamics of three doses of enoxacin (200mg twice daily, 400mg twice daily and 600mg twice daily) in patients with Amyotrophic Lateral Sclerosis
The purpose of this study is to test the safety and tolerability of enoxacin in patients with ALS.
SPOTLIGHT ON OUR RESEARCH
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