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International Alliance of ALS/MND Associations

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  • What is ALS/MND
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  • Support for PALS & CALS
    • Fundamental Rights for People with ALS/MND and Caregivers
    • Research
      • Voice Preservation
      • Open Science
      • Expanded Access
      • Understanding ALS/MND Research
      • Improving Regulatory Pathways
      • Right to Try
      • US FDA Orphan Drug Designation
      • Unproven (Off-Label) Treatments
      • Open Label Extension
    • Advocacy
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    • Drugs in Development
      • AB Science – Masitinib
      • BrainStorm Cell Therapeutics – NurOwn
      • Clene Nanomedicine – CNM-Au8
      • Collaborative Medicinal Development – CuATSM
      • ILB – Tikomed
      • Kadimastem – AstroRx
      • Mitsubishi Tanabe Pharma America – Oral Edaravone
      • Neuronata-R/Lenzumestrocel
      • NeuroSense – PrimeC
      • Neuvivo – NP001
      • Prilenia Therapeutics – Pridopidine
      • SOD1 Therapies & Trials
      • T Regulatory Cell Therapies
      • Ulefnersen – Ionis Pharmaceuticals
    • Approved Drugs
      • Nuedexta
      • Radicava/Edaravone
      • Riluzole/Tiglutik
      • Rozebalamin/Methylcobalamin
      • Tofersen/Qalsody
    • Drugs No Longer in Development
      • Amylyx – AMX0035
      • Collaborative Medicinal Development – CuATSM
      • Cytokinetics – Reldesemtiv
      • Orphazyme – Arimoclomol
      • TUDCA Trial
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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

  • Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

    Carlos Gomez Matallanas, Diagnosed 2014 , FUNDELA, Spain

  • Natalya Rybakova, Russian Charity ALS Foundation

    Natalya Rybakova, Russian Charity ALS Foundation

  • Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

    Jose Rivero Muñoz, Diagnosed 2015, FYADENMAC, Mexico

  • Bob Simonds and Drew O'Neill , Les Turner ALS Foundation, USA

    Bob Simonds and Drew O’Neill , Les Turner ALS Foundation, USA

  • Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

    Teddy Hanono Annie, Apoyo Integral Gila A.C., Diagnosed 2018, Mexico

  • 393647_2252248542053_984912751_n

    393647_2252248542053_984912751_n

  • IMG_2658

    IMG_2658

  • Juvenal Bayona Romero

    Juvenal Bayona Romero

  • 83

    83

  • Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

    Xian-Zhang Niu, Diagnosed 2006 , Shaanxi ALS Association, China

  • Olga Cosentino, Diagnosed 2013,  Asociación ELA Argentina

    Olga Cosentino, Diagnosed 2013, Asociación ELA Argentina

  • Claudia Gotti, Brazil

    Claudia Gotti, Brazil

  • Mary Thomas, Diagnosed 2013 , MND Australia

    Mary Thomas, Diagnosed 2013 , MND Australia

  • Jose Espinosa, Argentina

    Jose Espinosa, Argentina

  • Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

    Rolf Mauch, Association ALS Switzerland, Diagnosed 2015

  • Paul Launer, USA

    Paul Launer, USA

  • Joyce Rusinak, Forbes Norris ALS Center, USA

    Joyce Rusinak, Forbes Norris ALS Center, USA

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

    Glen Elison, ALS Hope Foundation, Diagnosed 2019, USA

  • Jan Zuring, Diagnosed 2010 , The Netherlands

    Jan Zuring, Diagnosed 2010 , The Netherlands

  • Hollister

    Hollister
    hollister

  • Andrietta

    Andrietta

  • Christian Bär, Germany

    Christian Bär, Germany

  • Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

    Anthony (Tony) Lynch, MND New South Wales, Diagnosed 2016, Australia

  • Jo Knowlton and her dog, Scotland

    Jo Knowlton and her dog, Scotland

  • Andrea Zicchieri, Associazione conSLAncio Onlus, Italy

    Andrea Zicchieri, Associazione conSLAncio Onlus, Italy
    AndreaZicchieri_conSLAncioItaly

  • Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

    Margarita Pizarro, Asociacion ELA Argentina, Diagnosed 2017, Argentina

  • Frank "Papa" Taylor

    Frank “Papa” Taylor

  • Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

    Marcelo Farinelli, Diagnosed 2006, ABrELA, Brazil

  • Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

    Wiebke Braach, Deutsche Gesellschaft für Muskelkranke, Germany

  • Imelda Arenas, ACELA, Colombia

    Imelda Arenas, ACELA, Colombia

  • Steven Spencer, Diagnosed 2014 , MND New Zealand

    Steven Spencer, Diagnosed 2014 , MND New Zealand

  • Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

    Gudjon Sigurdsson, Diagnosed 2004 , MND Association of Iceland

  • Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

    Sanjay Kumar Srivastava, Asha Ek Hope Foundation for ALS/MND, Diagnosed 2018, India

  • Colm Francis Davis, Ireland

    Colm Francis Davis, Ireland

  • Wilfried Leusing

    Wilfried Leusing

  • Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

    Elkin Ramiro Gaviria Muñoz, Diagnosed December 2018

  • Mauril Bélanger, Diagnosed 2015 , ALS Canada

    Mauril Bélanger, Diagnosed 2015 , ALS Canada

  • Lombana, Spain

    Lombana, Spain

  • Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

    Feng Gin Sun, Diagnosed 2014 , Shaanxi ALS Association, China

  • Ian and Teresa Roberts

    Ian and Teresa Roberts

  • Mauril Belanger

    Mauril Belanger

  • Cath Muir

    Cath Muir
    Cath

  • Josée Kolijn-de Man, Diagnosed 2015 , ALS Patients Connected, The Netherlands

    Josée Kolijn-de Man, Diagnosed 2015 , ALS Patients Connected, The Netherlands

  • Catherine Pearce, Australia

    Catherine Pearce, Australia

  • Willi Klein

    Willi Klein

  • Oliver Juenke, DGM, Germany

    Oliver Juenke, DGM, Germany

  • Valdomiro Xavier Honório, Brazil

    Valdomiro Xavier Honório, Brazil

  • Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

    Jette Odgaard Villemoes, Muskelsvindfonden, Denmark

  • Natalya Rybakova, Russia

    Natalya Rybakova, Russia

  • John Dinon, MND Australia

    John Dinon, MND Australia

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