Phase 3 Study of Ampreloxetine for Treatment of Symptomatic nOH for Multiple System Atrophy
Research type
Research Study
Full title
A Phase 3, Multi-center, Randomized Withdrawal and Long-Term Extension Study of Ampreloxetine for the Treatment of Symptomatic Neurogenic Orthostatic Hypotension in Participants with Multiple System Atrophy
IRAS ID
1007382
Contact name
Ross Vickery
Contact email
Sponsor organisation
Theravance Biopharma Ireland Limited
Eudract number
2022-003903-14
Clinicaltrials.gov Identifier
Research summary
Multiple System Atrophy (MSA) is a rare disease that causes failure of the involuntary nervous system affecting blood pressure regulation, movement and balance. Failure of the nervous system can also result in a condition called neurogenic orthostatic hypotension (nOH) that occurs following a change from a reclining or seated position to suddenly standing or walking. nOH is present in approximately 80% of patients with MSA and can cause symptoms like dizziness, light-headedness, blurred vision and neck/back pain, weakness and fatigue. These symptoms have a substantial impact on day-to-day functioning. The goal of treatment in nOH is to support blood pressure when standing or walking above critical limits necessary to alleviate these symptoms. Currently, no drug has demonstrated sustained effectiveness in treating nOH in patients with MSA.
Ampreloxetine is a potent selective norepinephrine (NE) reuptake inhibitor with selectivity for uptake over other neurotransmitters.
The current study is a phase 3 study to demonstrate the effectiveness and durability of ampreloxetine in reducing symptomatic nOH in patients diagnosed with MSA.
Approximately 102 participants will be enrolled into an Open-Label period where they will receive ampreloxetine for 12 weeks. Following this, at least 72 participants will be randomised into a double blind period (randomised withdrawal (RW)) where they will receive either ampreloxetine or placebo for 8 weeks. Participants who complete the RW will move onto a long-term evaluation period and will receive ampreloxetine for up to 104 weeks. Study follow-up will be undertaken 30 days after the last dose of ampreloxetine.
Overall, patients will participate in the study for up to 126 weeks.
This study is funded by Theravance Biopharma Ireland Limited and will take place at approximately 4 study centres in the UK.
REC name
North West - Greater Manchester Central Research Ethics Committee
REC reference
23/NW/0158
Date of REC Opinion
20 Sep 2023
REC opinion
Further Information Favourable Opinion