Interventional, randomized, double-blind, placebo-controlled, optional open-label extension trial of Lu AF82422 in participants with Multiple System Atrophy
Study of an Investigational Medication for Multiple System Atrophy
Alex Barboi
Primary Investigator
Brief description of study
The main goal of this trial is to evaluate the efficacy and safety of Lu AF82422 for the treatment of participants with Multiple System Atrophy (MSA).
PRIMARY OBJECTIVES:
- To evaluate the efficacy of Lu AF82422 on clinical progression in participants with MSA
SECONDARY OBJECTIVES:
- To evaluate the efficacy of Lu AF82422 on: global clinical impression, severity of illness global disability functionality disease milestones health-related quality of life overall survival
- To evaluate clinical meaningfulness of Lu AF82422
- To evaluate the efficacy of Lu AF82422 on disease progression, as measured by brain MRI
- To determine the population pharmacokinetic parameters of Lu AF82422
- To evaluate the safety and tolerability of Lu AF82422 in participants with MSA during the placebo-controlled period and OLE period
- To evaluate the immunogenicity of Lu AF82422
THIS STUDY IS ENROLLING BY INVITATION ONLY
Detailed description of study
This study will consist of a 3-6-week screening period, a 72-week placebo-controlled period (PCP), and will include a 72-week optional dose-blinded open-label treatment extension (OLE) period. Participants in the PCP will be randomized to Lu AF82422 high dose, Lu AF82422 low dose or placebo (1:1:1). All participants entering the OLE will receive Lu AF82422 during the OLE. Participants will receive intravenous infusions approximately every 4 weeks during both the PCP and OLE.
Eligibility of study
You may be eligible for this study if you meet the following criteria:
- Conditions: Multiple System Atrophy, MSA
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Age: 40 years - 75 years
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Gender: All
Key Inclusion Criteria:
- The participant has a diagnosis of clinically established multiple system atrophy parkinsonian type (MSA-P) or multiple system atrophy cerebellar type (MSA-C), or clinically probable MSA-P or MSA-C, according to the 2022 Movement Disorders Society (MDS) criteria for the diagnosis of MSA at the Screening Visit.
- The participant had onset of motor MSA symptoms (i.e., parkinsonian and/or cerebellar) within 5 years prior to the Screening Visit in the judgement of the investigator.
- The participant has an anticipated survival of >3 years, in the opinion of the investigator, at the Screening Visit.
- The participant has suitable peripheral venous access for investigational medicinal product (IMP) administration and blood sampling.
- The participant has an UMSARS Part I score ≤16 (omitting item 11 on sexual function) at the Screening Visit.
Exclusion Criteria:
- The participant has previously been dosed with Lu AF82422.
- The participant has taken any IMP <3 months or <5 half lives of that product, whichever is longer, prior to the first dose of IMP.
- The participant has 2 or more first degree relatives with a history of MSA.
- The participant, if of MSA-P subtype, has unexplained anosmia (not explained by other common causes such as allergic rhinitis or smoking, nasal structural lesions, or nasal surgery) on olfactory testing at the Screening Visit.
- The participant has evidence (clinically or on magnetic resonance imaging (MRI)) and/or history of any clinically significant disease or condition other than MSA, that is, in the investigator's opinion, likely to affect CNS functioning, e.g., serious neurological disorder, other intracranial or systemic disease.
- The participant has a current diagnosis of movement disorders that could mimic MSA, e.g., Parkinson' disease, dementia with Lewy bodies, essential tremor, progressive supranuclear palsy, spinocerebellar ataxia, spastic paraparesis, corticobasal degeneration, or vascular, pharmacological, or post-encephalitic parkinsonism, per investigator discretion. Participants who have previously been incorrectly diagnosed with Parkinson's disease will not be excluded.
This study investigates the effects of an investigational medication on individuals with Multiple System Atrophy (MSA). MSA is a rare condition that affects the nervous system, causing symptoms similar to Parkinson's disease. The purpose of this study is to evaluate the medication's impact on disease progression, overall survival, and quality of life for those with MSA.
Participants will be randomly assigned to receive either a high dose, a low dose of the investigational medication, or a placebo. The study involves intravenous infusions approximately every 4 weeks. Safety, tolerability, and the medication's effects on brain MRI will also be assessed. A placebo is an inactive substance that looks like the investigational medication but does not contain any medicine.
- Who can participate: Adults diagnosed with Multiple System Atrophy, either parkinsonian or cerebellar type, who have had symptom onset within the last 5 years, may be eligible. Participants need to have suitable venous access for treatment and meet other health criteria. Those who have already received the investigational medication or have certain other conditions are not eligible.
- Study details: In the open-label extension, all participants will receive the investigational medication.
- Study timelines: The study will last 72 weeks.
