CURRENT RECRUITING TRIALS

AL-S 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 Natalie Saunders

Biogen 233AS101 (BIIB067)

A Phase 1, Placebo-Controlled, Single and Multiple Ascending Dose Study to Evaluate the Safety, Tolerability, and Pharmacokinetics of BIIB067 Administered to Adult Subjects with Amyotrophic Lateral Sclerosis

From clinicaltrials.gov:

The primary objective of the study is to evaluate the safety, tolerability, and pharmacokinetics (PK) of BIIB067 in adults with ALS. The secondary objective is to evaluate the effects of BIIB067 on levels of superoxide dismutase 1 (SOD1) protein in the cerebrospinal fluid (CSF).

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Biogen 233AS102 (BIIB067)

A An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

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.

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Biogen 245AS101 (BIIB078)
A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB078 Administered Intrathecally to Adults with C9ORF72-Associated Amyotrophic Lateral Sclerosis

The purpose of this study is to find out about the safety, tolerability, and pharmacokinetics (PK) of BIIB078and whether it works when given as repeated doses in adult patients with C9ORF72-associated amyotrophic lateral sclerosis (ALS).

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Biogen 245AS102 (BIIB078)
An Extension Study to Assess the Long-Term Safety, Tolerability, Pharmacokinetics, and Effect on Disease Progression of BIIB067 Administered to Previously Treated Adults with Amyotrophic Lateral Sclerosis Caused by Superoxide Dismutase 1 Mutation

The main purpose of this extension study is to find out about the longer-term safety, tolerability and pharmacokinetics (PK) of the study drug in participants with Amyotrophic Lateral Sclerosis caused by a mutation in the super oxide dismutase one gene (SOD1-ALS).

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Biogen 275AS101 (Ataxin) (BIIB105)

A Phase 1 Multiple-Ascending-Dose Study to Assess the Safety, Tolerability, and Pharmacokinetics of BIIB105 Administered Intrathecally to Adults With Amyotrophic Lateral Sclerosis With or Without Poly-CAG Expansion in the Ataxin-2 Gene

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.

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CALS-NIC2 (need healthy controls aged matched to ALS patients)

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.

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COMBAT-ALS (ibudilast)
Evaluation of MN-166 (Ibudilast) for 12 Months Followed by an Open-label Extension for 6 Months in Patients With ALS (COMBAT-ALS)

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.

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CY5019 PEG study (reldesemtiv)
Safety study of oral reldesemtiv administered in subjects with ALS

Inclusion criteria:
• Between the ages of 18 to 80 years old
• With gastrostomy tube in place
• Able to safely swallow whole tablets and crushed tablets

Duration:
• Total study participation: approximately 5 weeks, including 5 visits to the study clinic.
• Three of these visits require overnight stays in the hospital.

Hudson Biomarker LP study

Characterization of Biomarker Levels in Blood and CSF from Amyotrophic Lateral Sclerosis (ALS) Patients, ALS- Frontotemporal Dementia (FTD) patients and Demographically-Matched Control Participants

Sometimes, when someone has a disease, their blood and spinal fluid contain substances that are different from those found in people who do not have the disease. We are trying to identify these substances in the blood and spinal fluid of ALS and ALS-FTD patients. We would like to know whether these substances could be linked with different stages of the disease(s). We would also like to know whether ALS and ALS-FTD patients have some of these substances in common.

For more information, please contact Natalie Saunders

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.

PATIENTS: for more information about upcoming or recruiting trials, contact Natalie Saunders

UPCOMING TRIALS

ANX005-ALS-01 (ANX005)
Phase 2a Safety, Tolerability, Pharmacokinetic, and Pharmacodynamic Study of Intravenous ANX005 in Subjects with Amyotrophic Lateral Sclerosis

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.

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APL2-ALS-206 (Pegcetacoplan)
A Phase 2, randomized, double-blind, placebo-controlled, multicenter study to evaluate the efficacy and safety of pegcetacoplan in adults with Amyotrophic Lateral Sclerosis (ALS)

The purpose of this study is to see if the investigational drug, pegcetacoplan (also known as APL-2), can slow the progression of ALS. Investigational means that pegcetacoplan is still being tested for safety and effectiveness but is not yet approved by any regulatory authorities (such as the United States Food and Drug Administration, Health Canada, or the European Medicines Agency) for general use. The study will also evaluate how the body processes pegcetacoplan and how well it is tolerated.

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AT-1501-A201 (AT-1501)
A Phase 2a Open-Label, Multi-Center Study to Evaluate the Safety and Tolerability of Multiple Doses of AT-1501 in Adults with ALS

The purpose of this study is to assess the safety and tolerability of AT-1501.

For this study, 54 male and female participants will be recruited at 10-13 sites. The participants will be ages 18 years and older.

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CY5031 Reldesemtiv Phase 3 (reldesemtiv)
A Phase 3, Multi-Center, Double-Blind, Randomized, Placebo-Controlled Trial to Evaluate the Efficacy and Safety of Reldesemtiv in Patients with Amyotrophic Lateral Sclerosis (ALS)

The purpose of this study is to evaluate the efficacy and safety of reldesemtiv.

By efficacy, we mean the effect of taking reldesemtiv and your ability to perform certain functional assessments. By safety, we mean the collection of information about whether there are any negative symptoms and/or side effects that may result from taking reldesemtiv.

Additionally, this research study will evaluate the pharmacokinetics (PK) of reldesemtiv (how much reldesemtiv gets into the blood stream and how long it takes the body to get rid of it).

For this study, approximately 555 male and female participants will be recruited at 80 sites globally. The participants will be aged between 18 and 80.

ION363-CS1 (ION363)
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).

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Machine Learning in ALS
Machine learning in the detection of upper and lower motor neuron features in speech of patients with 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).

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.

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MT-1186-A03 (A01 extension study) (oral edaravone)
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.

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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.

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ACTIVE (NOT RECRUITING) TRIALS

Alexion (ravulizumab)
Safety Study of Oral Edaravone Administered in Subjects With ALS

From clinicaltrials.gov:

The purpose of the study is to assess the efficacy and safety of ravulizumab for the treatment of adult participants with ALS.

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MT-1186 (oral edaravone)
Safety Study of Oral Edaravone Administered in Subjects With ALS

From clinicaltrials.gov:

The objective of this study is to evaluate the long-term safety and tolerability of oral edaravone in subjects with Amyotrophic Lateral Sclerosis (ALS) over 24 and 48 weeks.

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ORARIALS-01 (arimoclomol)

Arimoclomol in Amyotropic Lateral Sclerosis

From clinicaltrials.gov:

The purpose of this study is to investigate the efficacy, the safety and tolerability of the study drug in patients with ALS. Specifically, the study will look to find out if arimoclomol improves the ability for one to breathe on their own, improves survival, functional health and safety.

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