Adults 40 to 80, any sex, with Multiple System Atrophy. Patients with the condition only — healthy volunteers not accepted.
Results — posted to ClinicalTrials.gov
Per-arm endpoint measurements with 95% confidence intervals where reported. Source: trial results section.
Change From Baseline in the Modified UMSARS Score at Week 48Primary· Baseline and Week 48
UMSARS - clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination), Part IV (Global Disability Scale). Modified UMSARS is composed of subset of 9 items from original UMSARS Part I and Part II. Responses are measured on 4-point scale ranged from 0-3, where 0= no/mild impairment, 1= moderate impairment, 2= severe impairment, 3=complete impairment. Total modified UMSARS score is sum of these 9 items, score range from 0 to 27. Higher scores indicate greater impairment.
Group
Value
95% CI
Verdiperstat - Randomization Phase
5.20
4.52 – 5.89
Placebo - Randomization Phase
4.85
4.19 – 5.51
Number of Participants With Treatment-Emergent Adverse Events (TEAEs) and Serious TEAEsPrimary· Up to 100 weeks
An adverse event (AE) is defined as any new untoward medical occurrence or worsening of a pre-existing medical condition in participants or clinical investigation participants administered an investigational (medicinal) product and that does not necessarily have a causal relationship with this treatment. A serious AE (SAE) is defined as any event that met any of the following criteria at any dose: death; life-threatening; inpatient hospitalization or prolongation of existing hospitalization; persistent or significant disability/incapacity; congenital anomaly/birth defect in the offspring of a
TEAE
Group
Value
95% CI
Verdiperstat - Randomization Phase
160
Placebo - Randomization Phase
156
Verdiperstat - Randomization Phase/ Verdiperstat - OLE Phase
95
Placebo - Randomization Phase/ Verdiperstat - OLE Phase
112
Serious TEAE
Group
Value
95% CI
Verdiperstat - Randomization Phase
44
Placebo - Randomization Phase
29
Verdiperstat - Randomization Phase/ Verdiperstat - OLE Phase
30
Placebo - Randomization Phase/ Verdiperstat - OLE Phase
27
Clinical Global Impression of Improvement (CGI-I) Score at Week 48Secondary· Week 48
The CGI-I is a clinician-rated scale measuring the change in the participant's clinical status from a specific point in time. It is scored on a 7- point scale, ranging from 1 (very much improved) to 7 (very much worse), with a score of 4 indicating no change. Higher scores indicate greater impairment.
Group
Value
95% CI
Verdiperstat - Randomization Phase
5.07
4.92 – 5.21
Placebo - Randomization Phase
5.14
5.00 – 5.27
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Motor Subscale at Week 48Secondary· Baseline and Week 48
The MSA-QoL is a participant-rated scale that was designed to measure health-related quality of life specifically in MSA. It assesses activities of daily living and has subscales for motor, nonmotor, and emotional/social domains. The MSA-QoL motor subscale includes 14 items. The response to each question ranges from 0= no problem, to 4= extreme problem, with a total scale ranging from 0-56. Higher scores indicates higher impact of the disease on the aspect measured by each subscale.
Group
Value
95% CI
Verdiperstat - Randomization Phase
13.83
10.97 – 16.69
Placebo - Randomization Phase
13.45
10.72 – 16.18
Change From Baseline in Multiple System Atrophy Quality of Life (MSA-QoL) Non-motor Subscale at Week 48Secondary· Baseline and Week 48
The MSA-QoL is a participant-rated scale that was designed to measure health-related quality of life specifically in MSA. It assesses activities of daily living and has subscales for motor, nonmotor, and emotional/social domains. The MSA-QoL nonmotor subscale includes 12 items. The response to each question ranges from 0= no problem, to 4= extreme problem, with a total scale ranging from 0-48. Higher scores indicates higher impact of the disease on the aspect measured by each subscale.
Group
Value
95% CI
Verdiperstat - Randomization Phase
7.52
4.93 – 10.11
Placebo - Randomization Phase
5.56
3.07 – 8.04
Change From Baseline in UMSARS Part I and Part II Total Score at Week 48Secondary· Baseline and Week 48
The UMSARS is a clinician-rated scale comprised of 4 parts: Part I (Historical Review with 12 questions), Part II (Motor Examination with 14 questions), Part III (Autonomic Examination), and Part IV (Global Disability Scale). UMSARS Part I and Part II responses are measured on a 5-point scale ranging from 0 to 4, where 0= no impairment, 1= mild impairment, 2= moderate impairment, 3= severe impairment, 4=complete impairment. Each subscale score is a sum of its items and total score is sum of all 26 items, score range from 0 to 104. Higher scores indicates greater impairment.
Group
Value
95% CI
Verdiperstat - Randomization Phase
12.00
10.37 – 13.63
Placebo - Randomization Phase
11.34
9.77 – 12.90
Change From Baseline in Patient Global Impression of Severity (PGI-S) at Week 48Secondary· Baseline and Week 48
The PGI-S is a participant-rated scale that measures how participants perceive the severity of their illness. The PGI-S is a 1-item questionnaire on a 4-point scale ranging from 1 to 4, where, 1 = normal, 2 = mild, 3 = moderate, 4 = severe. Higher scores indicate greater impairment.
Group
Value
95% CI
Verdiperstat - Randomization Phase
0.33
0.23 – 0.42
Placebo - Randomization Phase
0.27
0.18 – 0.36
Change From Baseline in Clinical Global Impression of Severity (CGI-S) at Week 48Secondary· Baseline and Week 48
The CGI-S is a clinician-rated scale measuring the severity of the participant's illness. It is scored on a 7- point scale ranging from 1 (normal, not ill at all) to 7 (among the most extremely ill patients). Higher scores indicate greater impairment.
Group
Value
95% CI
Verdiperstat - Randomization Phase
0.79
0.66 – 0.92
Placebo - Randomization Phase
0.78
0.66 – 0.90
Change From Baseline in UMSARS Part III at Week 48 (Blood Pressure (BP) Only)Secondary· Baseline and Week 48
The UMSARS is a clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination) and Part IV (Global Disability Scale). Only Part III of UMSARS was analyzed. Part III of the UMSARS is an Autonomic Exam, consisting of Supine and Standing Systolic and Diastolic Blood Pressure, Heart rate and presence Orthostatic Symptoms. Only change in Orthostatic Blood Pressure reported.
Change in Orthostatic Systolic BP
Group
Value
95% CI
Verdiperstat - Randomization Phase
-2.18
-5.90 – 1.55
Placebo - Randomization Phase
-3.09
-6.63 – 0.44
Change in Orthostatic Diastolic BP
Group
Value
95% CI
Verdiperstat - Randomization Phase
-2.87
-5.13 – -0.61
Placebo - Randomization Phase
-2.54
-4.70 – -0.38
Change From Baseline in UMSARS Part III at Week 48 (Heart Rate (HR) Only)Secondary· Baseline and Week 48
The UMSARS is a clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination) and Part IV (Global Disability Scale). Only Part III of UMSARS was analyzed. Part III of the UMSARS is an Autonomic Exam, consisting of Supine and Standing Systolic and Diastolic Blood Pressure, Heart rate and presence Orthostatic Symptoms. Only change in Orthostatic Heart Rate reported.
Group
Value
95% CI
Verdiperstat - Randomization Phase
0.20
-1.48 – 1.87
Placebo - Randomization Phase
-0.68
-2.27 – 0.91
Change From Baseline in UMSARS Part IV at Week 48Secondary· Baseline and Week 48
The UMSARS is a clinician-rated scale comprised of 4 parts: Part I (Historical Review), Part II (Motor Examination), Part III (Autonomic Examination) and Part IV (Global Disability Scale). Only Part IV of UMSARS was analyzed. Part IV was measured on a 5-point scale ranging from 1= Completely independent. Able to do all chores with minimal difficulty or impairment. Essentially normal. Unaware of any difficulty, to 5= Totally dependent and helpless. Bedridden. Higher scores indicate greater impairment.
Group
Value
95% CI
Verdiperstat - Randomization Phase
0.82
0.69 – 0.96
Placebo - Randomization Phase
0.85
0.72 – 0.99
Adverse events — posted to ClinicalTrials.gov
Time frame: TEAEs were reported from first dose of study drug and prior to 30 days after the last dose of study drug, maximum of 100 weeks..
Reporting threshold: 5%.
Adverse-event reports describe events observed during the trial — not all are caused by the drug.
Verdiperstat - Randomization Phase
Serious: 44/168 (26%)
Deaths: 11/168
Placebo - Randomization Phase
Serious: 29/168 (17%)
Deaths: 5/168
Verdiperstat - Randomization Phase/Verdiperstat - OLE Phase
Serious: 30/123 (24%)
Deaths: 12/123
Placebo - Randomization Phase/Verdiperstat - OLE Phase
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Biohaven Pharmaceuticals, Inc.
Last refreshed: 29 September 2023
Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03952806.