parallel randomized trial

MSA

An Open Label, Single Site, 48 Week, Randomised Controlled Trial Evaluating the Safety and Efficacy of Exenatide Once-weekly in the Treatment of Patients With Multiple System Atrophy

Other conditions NCT04431713S000204

Design

Randomized: 50
Start year: 2020
Treatment: 48 weeks, follow-up 96 weeks
Countries: United Kingdom (1 sites)
Sponsor: University College London (mixed)
Eligibility: age 30 to 80; possible or probable MSA within 5 years; ambulant; HbA1c below 48 mmol/mol; BMI 18.5 or more

Conduct and analysis

Blinding: open label
Sequence generation: computer
Allocation concealment: central
Outcome adjudication: not reported
Primary estimand: not stated
Analysis population: modified itt
Missing data: mixed model; MAR and NMAR imputation sensitivity

Arms

Arm	Treatment	Background	Randomized	Analyzed	Completed treatment
A1	Standard care or no added treatment Standard of care (control)	standard of care for MSA; lifestyle: none	25	24
A2	Exenatide extended release 2 mg subcutaneous once weekly Exenatide once weekly 2 mg	standard of care for MSA; lifestyle: none	25	24

Baseline characteristics

As printed in the trial reports or registry, per arm or for the whole trial.

Characteristic	Statistic	Standard care or no added treatment A1	Exenatide extended release 2 mg subcutaneous once weekly A2
age	mean (years)	62.4	63.3
age	sd (years)	7.3	8.4
female	n	13	13
female	percent	52	52
hispanic	n	0	0
other msa p subtype	n	14	14
other msa p subtype	percent	56	56
other race chinese	n	1	0
other race chinese	percent	4	0
other race indian	n	0	3
other race indian	percent	0	12
other race other	n	1	1
other race other	percent	4	4
other race pakistani	n	1	0
other race pakistani	percent	4	0
other race white british	n	19	17
other race white british	percent	76	68
other race white irish	n	0	1
other race white irish	percent	0	4
other race white other	n	3	3
other race white other	percent	12	12
other umsars 1 2	mean (points)	42.6	46.0
other umsars 1 2	sd (points)	8.2	11.1
other years since diagnosis	mean (years)	0.9	1.2
other years since diagnosis	sd (years)	0.6	1.0
weight	mean (kg)	76.7	74.2
weight	sd (kg)	15.0	15.4

Results

17 adjudicated result lines, grouped by outcome. Open an outcome to see its values.

Physical functioning (any instrument) (7 lines; O24B)

Week	Arm	Statistic	Estimate	SE or SD	95% CI	Events / N	Detail
48	A1	change	10.4 points	15.7			not stated; other; R0011797:Supplement Table S1
48	A1	final	53.7 points	23.7		23	not stated; completers; registry:outcomes
48	A2	change	9.5 points	14.5			not stated; other; R0011797:Supplement Table S1
48	A2	final	53.1 points	19.8		21	not stated; completers; registry:outcomes
48	A2 vs A1	md	-0.1 points		-9.2 to 9.1		not stated; modified itt; R0011797:Supplement Table S1
96	A1	change	24.4 points	13.9			not stated; other; R0011797:Supplement Table S1
96	A2	change	22.3 points	19.9			not stated; other; R0011797:Supplement Table S1

Serious adverse events (2 lines; O25)

Week	Arm	Statistic	Estimate	SE or SD	95% CI	Events / N	Detail
	A1	events				10 / 25	not stated; randomized; registry:adverse_events
	A2	events				10 / 25	not stated; randomized; registry:adverse_events

Discontinuation due to adverse events (1 lines; O26)

Week	Arm	Statistic	Estimate	SE or SD	95% CI	Events / N	Detail
	A2	events				2 / 25	not stated; randomized; R0011797:Results

Nausea (2 lines; O27A)

Week	Arm	Statistic	Estimate	SE or SD	95% CI	Events / N	Detail
	A1	events				1 / 25	not stated; randomized; registry:adverse_events
	A2	events				6 / 25	not stated; randomized; registry:adverse_events

Diarrhea (2 lines; O27C)

Week	Arm	Statistic	Estimate	SE or SD	95% CI	Events / N	Detail
	A1	events				0 / 25	not stated; randomized; registry:adverse_events
	A2	events				1 / 25	not stated; randomized; registry:adverse_events

Constipation (2 lines; O27D)

Week	Arm	Statistic	Estimate	SE or SD	95% CI	Events / N	Detail
	A1	events				0 / 25	not stated; randomized; registry:adverse_events
	A2	events				1 / 25	not stated; randomized; registry:adverse_events

Discontinuation due to gastrointestinal adverse events (1 lines; O27E)

Week	Arm	Statistic	Estimate	SE or SD	95% CI	Events / N	Detail
	A2	events				1 / 25	not stated; randomized; R0011797:Results