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What’s New in Research

Clinical Trials

Every year, hundreds of new clinical trials are initiated to test new “experimental” drugs, or to test new uses of already approved drugs on humans, in a wide range of disease areas. These trials are conducted by researchers in countries all around the world, including Canada, in locations like hospitals, universities, doctors’ offices, and community clinics. Through voluntary participation in clinical trials, patients get a chance to take part in research that could improve their health and help them access a drug, prior to its approval. Like all drugs, the ones used in clinical trials have potential benefits as well as risks, and since they are still being studied, there is usually limited information about safety and efficacy. Before deciding to take part in a clinical trial, discuss the potential risks and benefits with your health care provider, so that you can make an informed decision about your health.

There is an unprecedented number of clinical trials currently underway in PNH, including:

Achillion Pharmaceuticals

ACH-0144471 – factor D inhibitors

  • Phase 2; recruiting (New Zealand)

Akari Therapeutics

Coversin – for PNH patients with resistance to eculizumab due to complement C5 polymorphisms

ALXN1210 – evaluating the safety, tolerability and efficacy

  • Phase 2; ongoing, not recruiting, including three trial sites in Canada (Alberta, Ontario and Quebec)

ALXN1210 – versus eculizumab-treatment naive adult patients

  • Phase 3; recruiting, including one site in Canada (Ontario)

ALXN1210 – open label dose escalation study

  • Phase 1/2; ongoing, not recruiting (Australia, Republic of Korea)

ALXN1210 – adult patients currently treated with eculizumab

  • Phase 3; not yet open for recruitment

Alnylam Pharmaceuticals


  • Phase 1/2; ongoing, not recruiting (Europe)


Apellis Pharmaceuticals

APL-2 – for patients who have not received treatment with eculizumab in the past

  • Phase 1; recruiting (New Zealand)

APL-2 – add-on to standard care in subjects with PNH

  • Phase 1; ongoing, not recruiting (United States)

Fred Hutchinson Cancer Research Center

Donor peripheral stem cell transplantation

  • Phase 2; ongoing, not recruiting (United States)

Novartis Pharmaceuticals - partnered with MorphoSys


  • Phase 2; recruiting (Europe, Asia)

Ra Pharma

RA101495 – for PNH patients

  • Phase 2; recruiting (United Kingdom)

RA101495 – for patients with inadequate response to eculizumab

  • Phase 2; recruiting (United States)


RO7112689 – to assess safety, efficacy, pharmacokinetics, and pharmacodynamics in healthy volunteers and participants with PNH

  • Phase 1/2; ongoing, recruiting (Germany, Hungary, Japan, Korea)

Donor peripheral stem cell transplantation

  • Phase 2; ongoing, not recruiting (United States)

Mismatched family member donor stem cell transplantation

  • Phase 2; ongoing, not recruiting (United States)

If you’re looking for a simple snapshot of the drug approval and funding process in Canada, we’ve got the solution for you. Please click here for an infographic that will help you navigate a drug’s journey – from “bench to bedside.”