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International Alliance of ALS/MND Associations

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  • Support for PALS & CALS
    • Fundamental Rights of People with ALS/MND and Caregivers
    • Research
      • Voice Preservation
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      • Improving Regulatory Pathways
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      • Open Label Extension
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    • Drugs in Development
      • AB Science – Masitinib
      • Amylyx – AMX0035
      • Biogen – Tofersen
      • BrainStorm Cell Therapeutics – NurOwn
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      • Collaborative Medicinal Development – CuATSM
      • Cytokinetics – Reldesemtiv
      • Kadimastem – AstroRx
      • Mitsubishi Tanabe Pharma America – Oral Edaravone
      • Orphazyme – Arimoclomol
      • T Regulatory Cell Therapies
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Open Label Extension

When clinical trials end, the participants typically stop taking the study drug until it receives approval from their country’s regulatory agency. But if patients are benefitting from the drug and have no other treatment options without it, they are sometimes allowed to continue taking the drug as part of an open-label extension (OLE). People are only allowed to participate in an OLE if they’ve completed the initial portion of the study.

OLEs often follow phase 3 trials, the last phase before regulatory approval. But it is not uncommon for them to follow phase 1 or phase 2 trials for ALS/MND treatments. Many organizations are strongly advocating for the inclusion of some form of OLE in all industry-supported clinical trials.

OLEs are an act of compassion for people with serious illnesses like ALS/MND who participate in clinical trials. ALS/MND is not only severe, but clinical trial participants are making a sacrifice by willing to volunteer knowing there is a chance they may end up on placebo. In these cases, everyone should have access to an OLE that could maximize the potential benefit of the treatment, should it ultimately prove effective.

Scientific Gain

Besides benefitting trial participants directly, a well-designed OLE can also provide useful data about the therapy that could benefit more people. OLEs often extend a trial for months, allowing researchers to collect more information on the long-term efficacy, tolerability, and safety of the drug. However, the data’s validity is limited by the fact that participants know they are receiving the treatment. This could create a placebo effect that makes the drug’s benefit appear greater only because participants believe it should work.

The Alliance believes that open label extension studies must be incorporated into all clinical trial design and protocols to ensure people living with ALS/MND can continue to access experimental therapies once their participate in a clinical trial has ended.

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Advocacy

  • Advocacy Toolkit

  • Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

    Antonio Ventriglia, ALS Liga Belgium, Diagnosed 2013, Belgium

  • Charlie “Hark” Dourney, Diagnosed 2007 - Hark ALS, USA

    Charlie “Hark” Dourney, Diagnosed 2007 – Hark ALS, USA

  • Alejandro Aquino, Diagnosed 2011 - Asociación ELA Argentina

    Alejandro Aquino, Diagnosed 2011 – Asociación ELA Argentina

  • Zabun Nassar, MND Association, Diagnosed 2016, England

    Zabun Nassar, MND Association, Diagnosed 2016, England

  • Marcel R. Wernard, Diagnosed 2016 - ALS Patients Connected, The Netherlands

    Marcel R. Wernard, Diagnosed 2016 – ALS Patients Connected, The Netherlands

  • Cath Muir

    Cath Muir
    Cath

  • Kirsten Harley, Diagnosed 2013 - Australia

    Kirsten Harley, Diagnosed 2013 – Australia

  • David Watson, MND Scotland, Diagnosed 2018, Scotland

    David Watson, MND Scotland, Diagnosed 2018, Scotland

  • David Bishop

    David Bishop

  • Osiel Mendoza, Diagnosed 2016 - ALS Therapy Development Institute, USA

    Osiel Mendoza, Diagnosed 2016 – ALS Therapy Development Institute, USA

  • Joanne Pratt, Diagnosed 2011 - MND Australia

    Joanne Pratt, Diagnosed 2011 – MND Australia

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Angie Bordaen, Diagnosed 2014 - ALS Liga België, Belgium

    Angie Bordaen, Diagnosed 2014 – ALS Liga België, Belgium

  • Alberto Baez Murillo, Colombia

    Alberto Baez Murillo, Colombia

  • Jeff Sutherland

    Jeff Sutherland
    jspic

  • Phil Rossall, MND-Association, UK

    Phil Rossall, MND-Association, UK

  • Carlos Alberto Arango, Colombia

    Carlos Alberto Arango, Colombia

  • Jack Buzby, USA

    Jack Buzby, USA

  • Timothy Holman, Switzerland

    Timothy Holman, Switzerland

  • Mike Rannie, ALS Canada, Diagnosed 2017, Canada

    Mike Rannie, ALS Canada, Diagnosed 2017, Canada

  • Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014, Canada

    Norm MacIsaac, ALS Society of Canada, ALS Society of Quebec, Diagnosed 2014, Canada

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Chris McCauley, Diagnosed 2015 - ALS Canada

    Chris McCauley, Diagnosed 2015 – ALS Canada

  • Orlando Ruiz, Diagnosed 2001 - ACELA, Colombia

    Orlando Ruiz, Diagnosed 2001 – ACELA, Colombia

  • Maurice LeClerc, ALS Canada

    Maurice LeClerc, ALS Canada

  • Ann Nicol

    Ann Nicol

  • Margreth Burger-Saile, Diagnosed 2011 - ALS Schweiz, Switzerland

    Margreth Burger-Saile, Diagnosed 2011 – ALS Schweiz, Switzerland

  • Roy

    Roy
    roy

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Denis Blais, Diagnosed 2015 - ALS Canada

    Denis Blais, Diagnosed 2015 – ALS Canada

  • Mark Miller

    Mark Miller

  • Brian Lovell, Diagnosed 2011 - MND Australia

    Brian Lovell, Diagnosed 2011 – MND Australia

  • Willi Klein

    Willi Klein

  • Daniel Hare

    Daniel Hare

  • Ailsa Malcolm-Hutton, Diagnosed 2013 - MND Association of England, Wales and N Ireland

    Ailsa Malcolm-Hutton, Diagnosed 2013 – MND Association of England, Wales and N Ireland

  • Sally Pauls, Diagnosed 2006 - Les Turner ALS Foundation

    Sally Pauls, Diagnosed 2006 – Les Turner ALS Foundation

  • Jon Newsome, Les Turner ALS Foundation, USA

    Jon Newsome, Les Turner ALS Foundation, USA

  • Fabrice Kamp, Germany

    Fabrice Kamp, Germany

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

  • Karl Hughes, Diagnosed 2010 - IMNDA, Ireland

    Karl Hughes, Diagnosed 2010 – IMNDA, Ireland

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • John and Loretta Russo, USA

    John and Loretta Russo, USA
    final3878

  • Chun Ju Xiao, China

    Chun Ju Xiao, China

  • Mary Thomas, Diagnosed 2013 - MND Australia

    Mary Thomas, Diagnosed 2013 – MND Australia

  • Fernando Ocampo Cardona, Colombia

    Fernando Ocampo Cardona, Colombia

  • Claire Garry, USA

    Claire Garry, USA
    20200117_214643

  • Danny Reviers, Diagnosed 1979 - ALS Liga België, Belgium

    Danny Reviers, Diagnosed 1979 – ALS Liga België, Belgium

  • Ian Roberts

    Ian Roberts

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