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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)
    • Learn more here

Akari Therapeutics

  • Coversin – for PNH patients with resistance to eculizumab due to complement C5 polymorphisms
    • Phase 2; recruiting (Netherlands)
    • Learn more here

Alexion Pharmaceuticals

  • ALXN1210 – evaluating the safety, tolerability and efficacy
    • Phase 2; ongoing, not recruiting, including three trial sites in Canada (Alberta, Ontario and Quebec)
    • Learn more here
  • ALXN1210 – versus eculizumab-treatment naive adult patients
    • Phase 3; recruiting, including one site in Canada (Ontario)
    • Learn more here
  • ALXN1210 – open label dose escalation study
    • Phase 1 / 2; ongoing, not recruiting (Australia, Republic of Korea)
    • Learn more here
  • ALXN1210 – adult patients currently treated with eculizumab
    • Phase 3; not yet open for recruitment
    • Learn more here

Alnylam Pharmaceuticals

  • A5LN-CC
    • Phase 1 / 2; ongoing, not recruiting (Europe)
    • Learn more here
  • Amy-101
    • Phase 1
    • Learn more here

Apellis Pharmaceuticals

  • APL-2 – for patients who have not received treatment with eculizumab in the past
    • Phase 1; recruiting (New Zealand)
    • Learn more here
  • APL-2 – add-on to standard care in subjects with PNH
    • Phase 1, ongoing, not recruiting (United States)
    • Learn more here

Fred Hutchinson Cancer Research Center

  • Donor peripheral stem cell transplantation
    • Phase 2; ongoing, not recruiting (United States)
    • Learn more here

Novartis Pharmaceuticals - partnered with MorphoSys

  • LFG316
    • Phase 2; recruiting (Europe, Asia)
    • Learn more here

Ra Pharma

  • RA101495 – for patients with inadequate response to eculizumab
    • Phase 2; recruiting (United States)
    • Learn more here
  • RA101495 – for patients with inadequate response to eculizumab
    • Phase 2; recruiting (United Kingdom)
    • Learn more here
  • Donor peripheral stem cell transplantation
    • Phase 2; ongoing, not recruiting (United States)
    • Learn more here
  • Mismatched family member donor stem cell transplantation
    • Phase 2; ongoing, not recruiting (United States)
    • Learn more here