Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

for the Healey ALS Platform Trial Study Group; HEALEY ALS Platform Trial Study Group; Biostatisticians; Investigators; Clinical Operations Team; Medical Monitors

doi:10.1002/ana.26285

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

for the Healey ALS Platform Trial Study Group, HEALEY ALS Platform Trial Study Group, Biostatisticians, Investigators, Clinical Operations Team, Medical Monitors

Research output: Contribution to journal › Article › peer-review

57 Scopus citations

Abstract

Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints. ANN NEUROL 2022;91:165–175.

Original language	English
Pages (from-to)	165-175
Number of pages	11
Journal	Annals of neurology
Volume	91
Issue number	2
DOIs	https://doi.org/10.1002/ana.26285
State	Published - Feb 1 2022

Access to Document

10.1002/ana.26285

Cite this

@article{18327c65808645dcb50caf285354e5f8,

title = "Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development",

abstract = "Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints. ANN NEUROL 2022;91:165–175.",

author = "{for the Healey ALS Platform Trial Study Group} and {HEALEY ALS Platform Trial Study Group} and Biostatisticians and Investigators and {Clinical Operations Team} and {Medical Monitors} and Sabrina Paganoni and Berry, {James D.} and Melanie Quintana and Eric Macklin and Saville, {Benjamin R.} and Detry, {Michelle A.} and Marianne Chase and Sherman, {Alexander V.} and Hong Yu and Kristin Drake and Jinsy Andrews and Jeremy Shefner and Chibnik, {Lori B.} and Matteo Vestrucci and Cudkowicz, {Merit E.} and Eric Macklin and Melanie Quintana and Ben Saville and Detry, {Michelle A.} and Lori Chibnik and Bind, {Marie Abele} and James Chan and Matteo Vestrucci and Eduardo Locatelli and Gustavo Alameda and Sabrina Paganoni and Merit Cudkowicz and Alexandra McCaffrey and James Berry and Suma Babu and Katie Nicholson and Jennifer Scalia and Zachary Simmons and James Grogan and Xiaowei Su and Mansoureh Mamarabadi and Daragh Heitzman and Nasri, {Mohamad Asaad} and Jonathan Katz and Liberty Jenkins and Kevin Felice and Charles Whitaker and Senda Ajroud-Driss and Adam Quick and Stephen Kolb and {Sarah Heintzman}, {N. P.} and Appel, {Stanley H.} and Ericka Greene and Jason Thonhoff and Sheetal Shroff and Bing Liao and Goutman, {Stephen A.} and Feldman, {Eva L.} and Timothy Miller and {Amber Malcolm}, {N. P.} and Newman, {Daniel S.} and Ximena Arcila-Londono and Kara Steijlen and Owegi, {Margaret Ayo} and Brown, {Robert H.} and Mehdi Ghasemi and Fernandes, {Joseph Americo M.} and Ezequiel Piccione and Pariwat Thaisetthawatkul and Weiss, {Michael D.} and Nassim Rad and Hristelina Ilieva and Piera Pasinelli and {Judith Guarnieri}, {N. P.} and Shafeeq Ladha and Bill Jacobsen and Robert Bowser and Jeremy Shefner and Laura Foster and Carlayne Jackson and Jeffrey Rosenfeld and Karthikeyan Bhuvaneswaran and Lauren Elman and Colin Quinn and Jinsy Andrews and {Julia Yasek}, {N. P.} and Dominic Fee and Terry Heiman-Patterson and Anahita Deboo and Andrea Swenson and Christopher Nance and Tuan Vu and Niraja Suresh and Jerrica Farias and Jim Caress and Michael Cartwright and Kourosh Rezania and Matthew Elliott and Seward Rutkove and Courtney McIlduff and Cohen, {Jeffrey A.} and Elijah Stommel and Jonathan Glass and Christina Fournier and Eufrosina Young and Wymer, {James P.} and Volkan Granit and Michael Benatar and Richard Lewis and Matthew Burford and Frank Diaz and Maragakis, {Nicholas J.} and Lora Clawson and Alpa Uchil and Kristen Riley and Betsy Mosmiller and Paul Twydell and Goyal, {Namita A.} and Omar Jawdat and Duaa Jabari and Jeffrey Statland and Kasarskis, {Edward J.} and Russell, {James W.} and Peter Jin and David Walk and Sam Maiser and Jaimin Shah and Bjorn Oskarsson and Richard Bedlack and Xiaoyan Li and Chen, {I. Hweii Amy} and Nicholas Olney and Tracy Bazan and Ghazala Hayat and Pattee, {Gary L.} and Donofrio, {Peter D.} and Peltier, {Amanda C.} and Jennifer Martinez-Thompson and Nathan and Kristin Johnson and Raghav Govindarajan and Vovanti Jones and Tulio Bertorini and Tourenne, {Clotilde Lagier} and Vakili, {Ghazaleh Sadri} and Marianne Chase and Alex Sherman and Hong Yu and Derek Dagostino and Scott Bocchio and Matthew Eydinov and Kenneth Faulconer and Olga Kharakozova and Alexander Korin and Ilya Novak and Ervin Sinani and Natalia Tarasenko and Prasha Vigneswaran and Igor Katsovskiy and Isaac Whitworth and Yusra Wahab and Catherine Gladden and Aileen Shaughnessy and Eric Tustison and Katie Jentoft and Joe Ostrow and Sangwon Lim and Genevive Changkuon and Mattos, {Annette De} and Janae Patterson and Robin Scott and Precious Figueroa-Szostek and Lindsay Pothier and Brittney Harkey and Sofia DiStefano and Erin Kennedy and Abbey Bailey and Nicole DiOrio and Jacob Feulner and Matt Davis and Jassira Gomes and Marisa DiMarzio and Jennifer Henrique and Molly Benson and Stacey Grabert and Tessa Garozzo and Meghan Hall and Rebecca Randall and Mariah Connolly and {De Santiago}, Diana and Courtney Lavery and Gale Kittle and Jenny Hamilton and Patrick Bolger and Ahmed Fetouh and Cornelia Kamp and Julie Kennedy and Catherine Small and Allison Bulat and Kristin Drake and Andrew McGarry and Margherita Torti",

note = "Publisher Copyright: {\textcopyright} 2021 American Neurological Association.",

year = "2022",

month = feb,

day = "1",

doi = "10.1002/ana.26285",

language = "English",

volume = "91",

pages = "165--175",

journal = "Annals of neurology",

issn = "0364-5134",

number = "2",

}

TY - JOUR

T1 - Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

AU - for the Healey ALS Platform Trial Study Group

AU - HEALEY ALS Platform Trial Study Group

AU - Biostatisticians

AU - Investigators

AU - Clinical Operations Team

AU - Medical Monitors

AU - Paganoni, Sabrina

AU - Berry, James D.

AU - Quintana, Melanie

AU - Macklin, Eric

AU - Saville, Benjamin R.

AU - Detry, Michelle A.

AU - Chase, Marianne

AU - Sherman, Alexander V.

AU - Yu, Hong

AU - Drake, Kristin

AU - Andrews, Jinsy

AU - Shefner, Jeremy

AU - Chibnik, Lori B.

AU - Vestrucci, Matteo

AU - Cudkowicz, Merit E.

AU - Macklin, Eric

AU - Quintana, Melanie

AU - Saville, Ben

AU - Detry, Michelle A.

AU - Chibnik, Lori

AU - Bind, Marie Abele

AU - Chan, James

AU - Vestrucci, Matteo

AU - Locatelli, Eduardo

AU - Alameda, Gustavo

AU - Paganoni, Sabrina

AU - Cudkowicz, Merit

AU - McCaffrey, Alexandra

AU - Berry, James

AU - Babu, Suma

AU - Nicholson, Katie

AU - Scalia, Jennifer

AU - Simmons, Zachary

AU - Grogan, James

AU - Su, Xiaowei

AU - Mamarabadi, Mansoureh

AU - Heitzman, Daragh

AU - Nasri, Mohamad Asaad

AU - Katz, Jonathan

AU - Jenkins, Liberty

AU - Felice, Kevin

AU - Whitaker, Charles

AU - Ajroud-Driss, Senda

AU - Quick, Adam

AU - Kolb, Stephen

AU - Sarah Heintzman, N. P.

AU - Appel, Stanley H.

AU - Greene, Ericka

AU - Thonhoff, Jason

AU - Shroff, Sheetal

AU - Liao, Bing

AU - Goutman, Stephen A.

AU - Feldman, Eva L.

AU - Miller, Timothy

AU - Amber Malcolm, N. P.

AU - Newman, Daniel S.

AU - Arcila-Londono, Ximena

AU - Steijlen, Kara

AU - Owegi, Margaret Ayo

AU - Brown, Robert H.

AU - Ghasemi, Mehdi

AU - Fernandes, Joseph Americo M.

AU - Piccione, Ezequiel

AU - Thaisetthawatkul, Pariwat

AU - Weiss, Michael D.

AU - Rad, Nassim

AU - Ilieva, Hristelina

AU - Pasinelli, Piera

AU - Judith Guarnieri, N. P.

AU - Ladha, Shafeeq

AU - Jacobsen, Bill

AU - Bowser, Robert

AU - Shefner, Jeremy

AU - Foster, Laura

AU - Jackson, Carlayne

AU - Rosenfeld, Jeffrey

AU - Bhuvaneswaran, Karthikeyan

AU - Elman, Lauren

AU - Quinn, Colin

AU - Andrews, Jinsy

AU - Julia Yasek, N. P.

AU - Fee, Dominic

AU - Heiman-Patterson, Terry

AU - Deboo, Anahita

AU - Swenson, Andrea

AU - Nance, Christopher

AU - Vu, Tuan

AU - Suresh, Niraja

AU - Farias, Jerrica

AU - Caress, Jim

AU - Cartwright, Michael

AU - Rezania, Kourosh

AU - Elliott, Matthew

AU - Rutkove, Seward

AU - McIlduff, Courtney

AU - Cohen, Jeffrey A.

AU - Stommel, Elijah

AU - Glass, Jonathan

AU - Fournier, Christina

AU - Young, Eufrosina

AU - Wymer, James P.

AU - Granit, Volkan

AU - Benatar, Michael

AU - Lewis, Richard

AU - Burford, Matthew

AU - Diaz, Frank

AU - Maragakis, Nicholas J.

AU - Clawson, Lora

AU - Uchil, Alpa

AU - Riley, Kristen

AU - Mosmiller, Betsy

AU - Twydell, Paul

AU - Goyal, Namita A.

AU - Jawdat, Omar

AU - Jabari, Duaa

AU - Statland, Jeffrey

AU - Kasarskis, Edward J.

AU - Russell, James W.

AU - Jin, Peter

AU - Walk, David

AU - Maiser, Sam

AU - Shah, Jaimin

AU - Oskarsson, Bjorn

AU - Bedlack, Richard

AU - Li, Xiaoyan

AU - Chen, I. Hweii Amy

AU - Olney, Nicholas

AU - Bazan, Tracy

AU - Hayat, Ghazala

AU - Pattee, Gary L.

AU - Donofrio, Peter D.

AU - Peltier, Amanda C.

AU - Martinez-Thompson, Jennifer

AU - Nathan,

AU - Johnson, Kristin

AU - Govindarajan, Raghav

AU - Jones, Vovanti

AU - Bertorini, Tulio

AU - Tourenne, Clotilde Lagier

AU - Vakili, Ghazaleh Sadri

AU - Chase, Marianne

AU - Sherman, Alex

AU - Yu, Hong

AU - Dagostino, Derek

AU - Bocchio, Scott

AU - Eydinov, Matthew

AU - Faulconer, Kenneth

AU - Kharakozova, Olga

AU - Korin, Alexander

AU - Novak, Ilya

AU - Sinani, Ervin

AU - Tarasenko, Natalia

AU - Vigneswaran, Prasha

AU - Katsovskiy, Igor

AU - Whitworth, Isaac

AU - Wahab, Yusra

AU - Gladden, Catherine

AU - Shaughnessy, Aileen

AU - Tustison, Eric

AU - Jentoft, Katie

AU - Ostrow, Joe

AU - Lim, Sangwon

AU - Changkuon, Genevive

AU - Mattos, Annette De

AU - Patterson, Janae

AU - Scott, Robin

AU - Figueroa-Szostek, Precious

AU - Pothier, Lindsay

AU - Harkey, Brittney

AU - DiStefano, Sofia

AU - Kennedy, Erin

AU - Bailey, Abbey

AU - DiOrio, Nicole

AU - Feulner, Jacob

AU - Davis, Matt

AU - Gomes, Jassira

AU - DiMarzio, Marisa

AU - Henrique, Jennifer

AU - Benson, Molly

AU - Grabert, Stacey

AU - Garozzo, Tessa

AU - Hall, Meghan

AU - Randall, Rebecca

AU - Connolly, Mariah

AU - De Santiago, Diana

AU - Lavery, Courtney

AU - Kittle, Gale

AU - Hamilton, Jenny

AU - Bolger, Patrick

AU - Fetouh, Ahmed

AU - Kamp, Cornelia

AU - Kennedy, Julie

AU - Small, Catherine

AU - Bulat, Allison

AU - Drake, Kristin

AU - McGarry, Andrew

AU - Torti, Margherita

PY - 2022/2/1

Y1 - 2022/2/1

N2 - Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints. ANN NEUROL 2022;91:165–175.

AB - Current therapeutic development in amyotrophic lateral sclerosis (ALS) relies on individual randomized clinical trials to test a specific investigational product in a single patient population. This approach has intrinsic limitations, including cost, time, and lack of flexibility. Adaptive platform trials represent a novel approach to investigate several interventions for a single disease in a continuous manner. Already in use in oncology, this approach is now being employed more often in neurology. Here, we describe a newly launched platform trial for ALS. The Healey ALS Platform Trial is testing multiple investigational products concurrently in people with ALS, with the goal of rapidly identifying novel treatments, biomarkers, and trial endpoints. ANN NEUROL 2022;91:165–175.

UR - http://www.scopus.com/inward/record.url?scp=85122780992&partnerID=8YFLogxK

U2 - 10.1002/ana.26285

DO - 10.1002/ana.26285

M3 - Article

C2 - 34935174

AN - SCOPUS:85122780992

SN - 0364-5134

VL - 91

SP - 165

EP - 175

JO - Annals of neurology

JF - Annals of neurology

IS - 2

ER -

Adaptive Platform Trials to Transform Amyotrophic Lateral Sclerosis Therapy Development

Abstract

Access to Document

Other files and links

Fingerprint

Cite this