Updated October 17, 2018

The opportunity to be involved in clinical trials for experimental ALS treatments is something that is often on the radar for people who are living with ALS. Did you know that there are three clinical trials in Canada that recently started recruiting volunteers or will open soon?

On Wednesday, October 17, 2018, Dr. David Taylor, VP Research at ALS Canada hosted speakers from Canada’s world-class ALS research community as well as international researchers and experts to share their updates with you.

Our speaker line-up included clinical trial experts from three pharmaceutical companies who shared the latest updates on these new studies:

Drug iconPhase 3 study of arimoclomol
Orphazyme

A common feature of ALS is the accumulation of misfolded proteins in motor neurons that make it difficult for them to function correctly. In lab research, the drug arimoclomol demonstrated an ability to reduce the buildup of misfolded proteins by increasing the heat shock response. (Learn more about the heat shock response.)

Clinical research among a small group of human volunteers with ALS found that arimoclomol administered in an oral capsule format was safe and well-tolerated. But to further confirm the drug’s safety and to find out whether it can slow disease progression and/or improve survival, larger numbers of study participants are needed. (Learn more about clinical trial phases in our Clinical Trial FAQs or in this blog post from last year’s clinical trials webinar.)

The drug manufacturer Orphazyme recently launched a Phase 3 clinical trial. The first study site opened in Miami, Florida at the end of July 2018. Multiple locations in Canada, the United States and Europe will open soon.

Headshot of Richard BennettRichard Bennett is a senior clinical project manager with Orphazyme A/S, a biopharmaceutical company based in Copenhagen, Denmark. During the Virtual Research Forum, he provided an update on the arimoclomol study and shared details about timing for Canadian study sites. He also discussed what he believes matters most to people who are living with ALS and how Orphazyme is including patient-centric approaches in the study.

Drug iconPhase 3 study of levosimendan (ODM-109)
Orion

ALS is characterized by a progressive inability to use muscles involved in movement, speech, swallowing and breathing. Eventually, most people with ALS need breathing support and adaptive aids to assist mobility. Levosimendan (ODM-109) is an oral medication that helps muscles contract more easily and exert a stronger force, possibly slowing down their loss of function.

An intravenous form of levosimendan called Simdax is already marketed in more than 60 countries around the world, (but not in Canada), for the treatment of acute heart failure. A Phase 2 study of an oral form of the drug for people with ALS was conducted in Europe and the United Kingdom in 2017. The results for a small number of study volunteers were promising, but not conclusive.

Orion, the maker of levosimendan, launched a Phase 3 clinical trial in April 2018 to determine whether an oral capsule format of the drug can improve respiratory function in people with ALS. The trial is called REFALS (Respiratory Function in Patients with ALS).
Six Canadian trial sites will open for recruiting soon in Fredericton, Moncton, Montreal (Neuro and CHUM), Calgary and Edmonton.

Paula Rytilä, MD, PhD is Chief Medical Officer at Orion, a pharmaceutical company based in Finland. During the Virtual Research Forum, Dr. Rytilä presented the latest update on the REFALS clinical trial for oral levosimendan (ODM-109).

 

 

Drug iconPhase 2 study of H.P. Acthar Gel
Mallinckrodt Pharmaceuticals

One of the hallmarks of ALS disease progression is the over-activation of immune cells in the central nervous system. H.P. Acthar Gel is a medication that is injected beneath the skin. It contains a synthetic version of a hormone normally secreted by the pituitary gland called adrenocorticotrophic hormone (ACTH) that plays a role in regulating inflammation.

The drug is already approved in the United States for the treatment of flares associated with inflammatory diseases including lupus and multiple sclerosis.

Results from a Phase 1 study among a small group of people with ALS suggested that H.P. Acthar Gel might help reduce inflammation. Based on those results, Mallinckrodt Pharmaceuticals has launched a Phase 2 trial to examine whether the drug can slow functional decline in adults living with ALS. The trial is already underway and open for recruiting. Researchers are looking for 213 volunteers across 43 study sites located in Canada, the United States and Colombia. Currently, there are three locations recruiting in Canada — Edmonton, Longueuil and Montreal.

Susan VanMeter, MD, is Senior Director of Clinical Research at Mallinckrodt Pharmaceuticals, a company based in the United Kingdom. She shared more details about the clinical trial’s objectives at the Virtual Research Forum, how it is designed and how Canadians with ALS can participate.

Clinical research is essential towards realizing a future without ALS. Participating in clinical trials gives Canadians living with ALS an opportunity to try new therapies earlier and helps others who may one day be diagnosed with the disease.

Posted in: Research