Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson’s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The ‘Exenatide-PD3’ study

Nirosen Vijiaratnam; Christine Girges; Grace Auld; Marisa Chau; Kate Maclagan; Alexa King; Simon Skene; Kashfia Chowdhury; Steve Hibbert; Huw Morris; Patricia Limousin; Dilan Athauda; Camille B. Carroll; Michele T. Hu; Monty Silverdale; Gordon W. Duncan; Ray Chaudhuri; Christine Lo; Din S Del; Alison J. Yarnall; Lynn Rochester; Rachel Gibson; John Dickson; Rachael Hunter; Vincenzo Libri; Thomas Foltynie

doi:10.1136/bmjopen-2020-047993

Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson’s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The ‘Exenatide-PD3’ study

Nirosen Vijiaratnam, Christine Girges, Grace Auld, Marisa Chau, Kate Maclagan, Alexa King, Simon Skene, Kashfia Chowdhury, Steve Hibbert, Huw Morris, Patricia Limousin, Dilan Athauda, Camille B. Carroll, Michele T. Hu, Monty Silverdale, Gordon W. Duncan, Ray Chaudhuri, Christine Lo, Din S Del, Alison J. YarnallLynn Rochester, Rachel Gibson, John Dickson, Rachael Hunter, Vincenzo Libri, Thomas Foltynie

Peninsula Medical School

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Abstract

IntroductionParkinson’s disease (PD) is a common neurodegenerative disorder with substantial morbidity. No disease-modifying treatments currently exist. The glucagon like peptide-1 receptor agonist exenatide has been associated in single-centre studies with reduced motor deterioration over 1 year. The aim of this multicentre UK trial is to confirm whether these previous positive results are maintained in a larger number of participants over 2 years and if effects accumulate with prolonged drug exposure.Methods and analysisThis is a phase 3, multicentre, double-blind, randomised, placebo-controlled trial of exenatide at a dose of 2 mg weekly in 200 participants with mild to moderate PD. Treatment duration is 96 weeks. Randomisation is 1:1, drug to placebo. Assessments are performed at baseline, week 12, 24, 36, 48, 60, 72, 84 and 96 weeks.The primary outcome is the comparison of Movement Disorders Society Unified Parkinson’s Disease Rating Scale part 3 motor subscore in the practically defined OFF medication state at 96 weeks between participants according to treatment allocation. Secondary outcomes will compare the change between groups among other motor, non-motor and cognitive scores. The primary outcome will be reported using descriptive statistics and comparisons between treatment groups using a mixed model, adjusting for baseline scores. Secondary outcomes will be summarised between treatment groups using summary statistics and appropriate statistical tests to assess for significant differences.Ethics and disseminationThis trial has been approved by the South Central-Berkshire Research Ethics Committee and the Health Research Authority. Results will be disseminated in peer-reviewed journals, presented at scientific meetings and to patients in lay-summary format.Trial registration numbersNCT04232969, ISRCTN14552789.

Original language	English
Pages (from-to)	e047993-e047993
Number of pages	0
Journal	BMJ Open
Volume	11
Issue number	5
DOIs	https://doi.org/10.1136/bmjopen-2020-047993
Publication status	Published - May 2021

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Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinsons disease protocol for a multicen

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Vijiaratnam, N., Girges, C., Auld, G., Chau, M., Maclagan, K., King, A., Skene, S., Chowdhury, K., Hibbert, S., Morris, H., Limousin, P., Athauda, D., Carroll, C. B., Hu, M. T., Silverdale, M., Duncan, G. W., Chaudhuri, R., Lo, C., Del, D. S., ... Foltynie, T. (2021). Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson’s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The ‘Exenatide-PD3’ study. BMJ Open, 11(5), e047993-e047993. https://doi.org/10.1136/bmjopen-2020-047993

@article{e9d96a6d0b854638a8d9e1865d6464fe,

title = "Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson{\textquoteright}s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The {\textquoteleft}Exenatide-PD3{\textquoteright} study",

abstract = "IntroductionParkinson{\textquoteright}s disease (PD) is a common neurodegenerative disorder with substantial morbidity. No disease-modifying treatments currently exist. The glucagon like peptide-1 receptor agonist exenatide has been associated in single-centre studies with reduced motor deterioration over 1 year. The aim of this multicentre UK trial is to confirm whether these previous positive results are maintained in a larger number of participants over 2 years and if effects accumulate with prolonged drug exposure.Methods and analysisThis is a phase 3, multicentre, double-blind, randomised, placebo-controlled trial of exenatide at a dose of 2 mg weekly in 200 participants with mild to moderate PD. Treatment duration is 96 weeks. Randomisation is 1:1, drug to placebo. Assessments are performed at baseline, week 12, 24, 36, 48, 60, 72, 84 and 96 weeks.The primary outcome is the comparison of Movement Disorders Society Unified Parkinson{\textquoteright}s Disease Rating Scale part 3 motor subscore in the practically defined OFF medication state at 96 weeks between participants according to treatment allocation. Secondary outcomes will compare the change between groups among other motor, non-motor and cognitive scores. The primary outcome will be reported using descriptive statistics and comparisons between treatment groups using a mixed model, adjusting for baseline scores. Secondary outcomes will be summarised between treatment groups using summary statistics and appropriate statistical tests to assess for significant differences.Ethics and disseminationThis trial has been approved by the South Central-Berkshire Research Ethics Committee and the Health Research Authority. Results will be disseminated in peer-reviewed journals, presented at scientific meetings and to patients in lay-summary format.Trial registration numbersNCT04232969, ISRCTN14552789.",

author = "Nirosen Vijiaratnam and Christine Girges and Grace Auld and Marisa Chau and Kate Maclagan and Alexa King and Simon Skene and Kashfia Chowdhury and Steve Hibbert and Huw Morris and Patricia Limousin and Dilan Athauda and Carroll, {Camille B.} and Hu, {Michele T.} and Monty Silverdale and Duncan, {Gordon W.} and Ray Chaudhuri and Christine Lo and Del, {Din S} and Yarnall, {Alison J.} and Lynn Rochester and Rachel Gibson and John Dickson and Rachael Hunter and Vincenzo Libri and Thomas Foltynie",

year = "2021",

month = may,

doi = "10.1136/bmjopen-2020-047993",

language = "English",

volume = "11",

pages = "e047993--e047993",

journal = "BMJ Open",

issn = "2044-6055",

publisher = "BMJ Publishing Group",

number = "5",

}

Vijiaratnam, N, Girges, C, Auld, G, Chau, M, Maclagan, K, King, A, Skene, S, Chowdhury, K, Hibbert, S, Morris, H, Limousin, P, Athauda, D, Carroll, CB, Hu, MT, Silverdale, M, Duncan, GW, Chaudhuri, R, Lo, C, Del, DS, Yarnall, AJ, Rochester, L, Gibson, R, Dickson, J, Hunter, R, Libri, V & Foltynie, T 2021, 'Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson’s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The ‘Exenatide-PD3’ study', BMJ Open, vol. 11, no. 5, pp. e047993-e047993. https://doi.org/10.1136/bmjopen-2020-047993

Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson’s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The ‘Exenatide-PD3’ study. / Vijiaratnam, Nirosen; Girges, Christine; Auld, Grace et al.
In: BMJ Open, Vol. 11, No. 5, 05.2021, p. e047993-e047993.

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

TY - JOUR

T1 - Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson’s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The ‘Exenatide-PD3’ study

AU - Vijiaratnam, Nirosen

AU - Girges, Christine

AU - Auld, Grace

AU - Chau, Marisa

AU - Maclagan, Kate

AU - King, Alexa

AU - Skene, Simon

AU - Chowdhury, Kashfia

AU - Hibbert, Steve

AU - Morris, Huw

AU - Limousin, Patricia

AU - Athauda, Dilan

AU - Carroll, Camille B.

AU - Hu, Michele T.

AU - Silverdale, Monty

AU - Duncan, Gordon W.

AU - Chaudhuri, Ray

AU - Lo, Christine

AU - Del, Din S

AU - Yarnall, Alison J.

AU - Rochester, Lynn

AU - Gibson, Rachel

AU - Dickson, John

AU - Hunter, Rachael

AU - Libri, Vincenzo

AU - Foltynie, Thomas

PY - 2021/5

Y1 - 2021/5

N2 - IntroductionParkinson’s disease (PD) is a common neurodegenerative disorder with substantial morbidity. No disease-modifying treatments currently exist. The glucagon like peptide-1 receptor agonist exenatide has been associated in single-centre studies with reduced motor deterioration over 1 year. The aim of this multicentre UK trial is to confirm whether these previous positive results are maintained in a larger number of participants over 2 years and if effects accumulate with prolonged drug exposure.Methods and analysisThis is a phase 3, multicentre, double-blind, randomised, placebo-controlled trial of exenatide at a dose of 2 mg weekly in 200 participants with mild to moderate PD. Treatment duration is 96 weeks. Randomisation is 1:1, drug to placebo. Assessments are performed at baseline, week 12, 24, 36, 48, 60, 72, 84 and 96 weeks.The primary outcome is the comparison of Movement Disorders Society Unified Parkinson’s Disease Rating Scale part 3 motor subscore in the practically defined OFF medication state at 96 weeks between participants according to treatment allocation. Secondary outcomes will compare the change between groups among other motor, non-motor and cognitive scores. The primary outcome will be reported using descriptive statistics and comparisons between treatment groups using a mixed model, adjusting for baseline scores. Secondary outcomes will be summarised between treatment groups using summary statistics and appropriate statistical tests to assess for significant differences.Ethics and disseminationThis trial has been approved by the South Central-Berkshire Research Ethics Committee and the Health Research Authority. Results will be disseminated in peer-reviewed journals, presented at scientific meetings and to patients in lay-summary format.Trial registration numbersNCT04232969, ISRCTN14552789.

AB - IntroductionParkinson’s disease (PD) is a common neurodegenerative disorder with substantial morbidity. No disease-modifying treatments currently exist. The glucagon like peptide-1 receptor agonist exenatide has been associated in single-centre studies with reduced motor deterioration over 1 year. The aim of this multicentre UK trial is to confirm whether these previous positive results are maintained in a larger number of participants over 2 years and if effects accumulate with prolonged drug exposure.Methods and analysisThis is a phase 3, multicentre, double-blind, randomised, placebo-controlled trial of exenatide at a dose of 2 mg weekly in 200 participants with mild to moderate PD. Treatment duration is 96 weeks. Randomisation is 1:1, drug to placebo. Assessments are performed at baseline, week 12, 24, 36, 48, 60, 72, 84 and 96 weeks.The primary outcome is the comparison of Movement Disorders Society Unified Parkinson’s Disease Rating Scale part 3 motor subscore in the practically defined OFF medication state at 96 weeks between participants according to treatment allocation. Secondary outcomes will compare the change between groups among other motor, non-motor and cognitive scores. The primary outcome will be reported using descriptive statistics and comparisons between treatment groups using a mixed model, adjusting for baseline scores. Secondary outcomes will be summarised between treatment groups using summary statistics and appropriate statistical tests to assess for significant differences.Ethics and disseminationThis trial has been approved by the South Central-Berkshire Research Ethics Committee and the Health Research Authority. Results will be disseminated in peer-reviewed journals, presented at scientific meetings and to patients in lay-summary format.Trial registration numbersNCT04232969, ISRCTN14552789.

U2 - 10.1136/bmjopen-2020-047993

DO - 10.1136/bmjopen-2020-047993

M3 - Article

SN - 2044-6055

VL - 11

SP - e047993-e047993

JO - BMJ Open

JF - BMJ Open

IS - 5

ER -

Vijiaratnam N, Girges C, Auld G, Chau M, Maclagan K, King A et al. Exenatide once weekly over 2 years as a potential disease-modifying treatment for Parkinson’s disease: protocol for a multicentre, randomised, double blind, parallel group, placebo controlled, phase 3 trial: The ‘Exenatide-PD3’ study. BMJ Open. 2021 May;11(5):e047993-e047993. doi: 10.1136/bmjopen-2020-047993