Suitability of external controls for drug evaluation in Duchenne muscular dystrophy.

Child Drug Evaluation / methods Humans Male Muscular Dystrophy, Duchenne / drug therapy Pragmatic Clinical Trials as Topic / methods Research Design Walk Test

Journal

Neurology
ISSN: 1526-632X
Titre abrégé: Neurology
Pays: United States
ID NLM: 0401060

Informations de publication

Date de publication:
08 09 2020
Historique:
received: 10 10 2019
accepted: 06 03 2020
pubmed: 3 7 2020
medline: 23 10 2020
entrez: 3 7 2020
Statut: ppublish

Résumé

To evaluate the suitability of real-world data (RWD) and natural history data (NHD) for use as external controls in drug evaluations for ambulatory Duchenne muscular dystrophy (DMD). The consistency of changes in the 6-minute walk distance (Δ6MWD) was assessed across multiple clinical trial placebo arms and sources of NHD/RWD. Six placebo arms reporting 48-week Δ6MWD were identified via literature review and represented 4 sets of inclusion/exclusion criteria (n = 383 patients in total). Five sources of RWD/NHD were contributed by Universitaire Ziekenhuizen Leuven, DMD Italian Group, The Cooperative International Neuromuscular Research Group, ImagingDMD, and the PRO-DMD-01 study (n = 430 patients, in total). Mean Δ6MWD was compared between each placebo arm and RWD/NHD source after subjecting the latter to the inclusion/exclusion criteria of the trial for baseline age, ambulatory function, and steroid use. Baseline covariate adjustment was investigated in a subset of patients with available data. Analyses included ∼1,200 patient-years of follow-up. Differences in mean Δ6MWD between trial placebo arms and RWD/NHD cohorts ranged from -19.4 m (i.e., better outcomes in RWD/NHD) to 19.5 m (i.e., worse outcomes in RWD/NHD) and were not statistically significant before or after covariate adjustment. We found that Δ6MWD was consistent between placebo arms and RWD/NHD subjected to equivalent inclusion/exclusion criteria. No evidence for systematic bias was detected. These findings are encouraging for the use of RWD/NHD to augment, or possibly replace, placebo controls in DMD trials. Multi-institution collaboration through the Collaborative Trajectory Analysis Project rendered this study feasible.

Identifiants

DOI: 10.1212/WNL.0000000000010170 PMID: 32611643 PMC: PMC7538219
pubmed: 32611643
pii: WNL.0000000000010170
doi: 10.1212/WNL.0000000000010170
pmc: PMC7538219
doi:

Types de publication

Journal Article Research Support, N.I.H., Extramural Research Support, Non-U.S. Gov't Research Support, U.S. Gov't, Non-P.H.S.

Langues

eng

Sous-ensembles de citation

IM

Pagination

e1381-e1391

Subventions

Organisme : NIAMS NIH HHS
ID : R01 AR056973
Pays : United States
Organisme : NIAMS NIH HHS
ID : R01 AR061875
Pays : United States

Investigateurs

Nicolas Deconinck (N)
Mar Tulinius (M)
Erik H Niks (EH)
Imelda De Groot (I)
Kevin Flanigan (K)
Erik Henricson (E)
Brenda Wong (B)
Maria Bernadete Dutra de Resende (MBD)
Gian Luca Vita (GL)
Ulrike Schara (U)
JanBerd Kirschner (J)
Eugenio Mercuri (E)
Haluk Topaloglu (H)
Soledad Monges (S)
Claude Cances (C)
V Vishwanathan (V)
S Chidambaranathan (S)
W Douglas Biggar (W)
Laura C. McAdam (LC)
Jean K Mah (JK)
Mar Tulinius (M)
Avital Cnaan (A)
Lauren P Morgenroth (LP)
Robert Leshner (R)
Carolina Tesi-Rocha (C)
Mathula Thangarajh (M)
Tina Duong (T)
Andrew Kornberg (A)
Monique M. Ryan (MM)
Yoram Nevo (Y)
Alberto Dubrovsky (A)
Paula R Clemens (PR)
Hoda Abdel-Hamid (H)
Anne Connolly (A)
Alan Pestronk (A)
Jean Teasley (J)
Tulio Bertorini (T)
Richard Webster (R)
Hanna Kolski (H)
Nancy L Kuntz (NL)
Sherilyn Driscoll (S)
John Bodensteiner (J)
Jose Carlo (J)
Ksenija Gornia (K)
Timothy Lotze (T)
John Day (J)
Peter Karachunski (P)
E Henricson (E)
R. Ted Abresch (RT)
Nannette Joyce (N)
Harneet Arora (H)
Rebecca J Willcocks (RJ)
Donovan J Lott (DJ)
Ann T Harrington (AT)
Claudia R Senesac (CR)
Kirsten L Zilke (KL)
Michael J Daniels (MJ)
Dandan Xu (D)
Gihan I Tennekoon (GI)
Erika L Finanger (EL)
Barry S Russman (BS)
Richard S Finkel (RS)
William T Triplett (WT)
Barry J Byrne (BJ)
Glenn A Walter (GA)
H L Sweeney (HL)
Marika Pane (M)
Elena Mazzone (E)
Sonia Messina (S)
GianLuca Vita (G)
Maria Pia Sormani (MP)
Adele D'Amico (A)
Enrico Bertini (E)
Angela Berardinelli (A)
Francesca Magri (F)
Giacomo Pietro Comi (GP)
Giovanni Baranello (G)
Tiziana Mongini (T)
Antonella Pini (A)
Roberta Battini (R)
Elena Pegoraro (E)
Claudio Bruno (C)
Luisa Politano (L)
Stefano Previtali (S)

Informations de copyright

Copyright © 2020 The Author(s). Published by Wolters Kluwer Health, Inc. on behalf of the American Academy of Neurology.

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Auteurs

Nathalie Goemans (N)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

James Signorovitch (J)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

Gautam Sajeev (G)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

Zhiwen Yao (Z)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

Heather Gordish-Dressman (H)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

Craig M McDonald (CM)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

Krista Vandenborne (K)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

Debra Miller (D)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

Susan J Ward (SJ)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy.

Eugenio Mercuri (E)

From the University Hospitals Leuven (N.G.), Child Neurology, Belgium; Analysis Group, Inc (J.S., G.S., Z.Y.), Boston; Collaborative Trajectory Analysis Project (J.S., S.J.W.), Cambridge, MA; Children's National Medical Center (H.G.-D.), Research Center for Genetic Medicine, Washington, DC; Department of Physical Medicine and Rehabilitation and Pediatrics (C.M.M.), University of California, Davis, Sacramento; Department of Physical Therapy (K.V.), University of Florida, Gainesville; CureDuchenne (D.M.), Newport Beach, CA; and Department of Pediatric Neurology (E.M.), Fondazione Policlinico Gemelli IRCCS, Catholic University, Rome, Italy. eugeniomaria.mercuri@unicatt.it.

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Classifications MeSH

questionsmedicales.fr › Personnes › Tranches d'âge › Enfant › Suitability of external controls for drug...
questionsmedicales.fr › Techniques d'investigation › Évaluation de médicament › Suitability of external controls for drug...
questionsmedicales.fr › Eucaryotes › Animaux › Chordés › Vertébrés › Mammifères › Eutheria › Primates › Haplorhini › Catarrhini › Hominidae › Humains › Suitability of external controls for drug...
questionsmedicales.fr › Malformations et maladies congénitales, héréditaires et néonatales › Maladies génétiques congénitales › Dystrophies musculaires › Myopathie de Duchenne › Suitability of external controls for drug...
questionsmedicales.fr › Environnement et santé publique › Santé publique › Méthodes épidémiologiques › Caractéristiques des études épidémiologiques › Études cliniques comme sujet › Essais cliniques comme sujet › Essais cliniques contrôlés comme sujet › Essais contrôlés randomisés comme sujet › Essais cliniques pragmatiques comme sujet › Suitability of external controls for drug...
questionsmedicales.fr › Disciplines des sciences naturelles › Science › Recherche › Plan de recherche › Suitability of external controls for drug...
questionsmedicales.fr › Diagnostic › Techniques et procédures diagnostiques › Techniques de diagnostic cardiovasculaire › Tests de la fonction cardiaque › Test de marche › Suitability of external controls for drug...