Multiple System Atrophy
Conditions
Keywords
Multiple System Atrophy (MSA)
Brief summary
The primary objective of the study is to evaluate the efficacy of TEV-56286 administered orally for the treatment of adult participants with Multiple System Atrophy (MSA). A secondary objective of the study is to evaluate specific efficacy parameters of TEV-56286. Another secondary objective is to evaluate the safety and tolerability of TEV-56286. The planned study period per participant is 56 weeks including a screening period (up to 4 weeks), a 48-week double-blind treatment period, and a follow-up visit (approximately 4 weeks after the end of the double-blind treatment period). The study duration will be approximately 27 months.
Detailed description
We plan to open locations in the following countries: US, Israel, Italy, Spain, Germany, France, Japan, and Serbia.
Interventions
DRUGTEV-56286
TEV-56286 capsules administered orally
DRUGPlacebo
Matching placebo administered orally
Sponsors
Teva Branded Pharmaceutical Products R&D LLC
Study design
Allocation
RANDOMIZED
Intervention model
PARALLEL
Primary purpose
TREATMENT
Masking
QUADRUPLE (Subject, Caregiver, Investigator, Outcomes Assessor)
Eligibility
Sex/Gender
ALL
Age
30 Years to 75 Years
Healthy volunteers
No
Inclusion criteria
* is considered to be "clinically possible" or "clinically probable" MSA as determined by the Gilman criteria * is medically and psychiatrically stable, as indicated by medical and psychiatric history, as well as physical and neurological examination * Females of child bearing potential (CBP) may be included only if they have a negative pregnancy test at the screening and baseline visits * Females of CBP whose male partners are potentially fertile (ie, no vasectomy) must use highly effective birth control methods * Males who are potentially fertile/reproductively competent (not surgically \[eg, vasectomy\] or congenitally sterile) and their female partners who are of CBP must use, together with their female partners, highly effective birth control methods * Additional criteria apply; please contact the investigator for more information
Exclusion criteria
* has 2 or more relatives with history of MSA, suggestive of an alternative diagnosis other than MSA * has participated in another clinical study involving administration of an IMP within 3 months or 5 half-lives (whichever is longer) of this IMP prior to screening * has a history of, or acknowledges, alcohol or other substance abuse in the 12 months before screening * is a female participant who is pregnant or breastfeeding, or plans to become pregnant during the study * has a known hypersensitivity to any components of the IMP * is of a vulnerable population (eg, people kept in detention or jail) * participant is using or consuming any prohibited concomitant medications within the specified exclusionary windows of this study * Additional criteria apply; please contact the investigator for more information
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| For non-EU: Change From Baseline in the Modified Unified Multiple System Atrophy Rating Scale (UMSARS) Part I Score (excluding item 11) | Baseline to Week 48 | The UMSARS is a multidimensional, validated scale for semi-quantitative clinical assessments of MSA participants. Modified UMSARS part I includes all items with the exclusion of item 11. Item scoring is scaled 0-3 using a range of 0 (no impairment) to 3 (severe impairment). |
| For EU: Change From Baseline in the Total UMSARS Score Part I and Part II Combined | Baseline to Week 48 | The UMSARS is comprised of 4 parts: part I, historical review of disease-related impairments, 12 items and part II, motor examination, 14 items. As UMSARS is a unified scale, each item in parts I and II achieves a single score using a range of 0 (no impairment) to 4 (severe impairment). |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| For non-EU: Change From Baseline in the Total UMSARS Score (Part I and Part II combined) | Baseline to Week 48 | — |
| For EU: Change From Baseline in the Modified UMSARS part I score (excluding item 11, item scoring rescaled 0-3) | Baseline to Week 48 | — |
| Change From Baseline in the UMSARS Part 1 Score | Baseline to Week 48 | — |
| Change From Baseline in Lateral Ventricle Volume Measured by MRI | Baseline to Week 48 | — |
| Change From Baseline in the Clinical Global Impression - Severity scale (CGI-S) | Baseline to Week 48 | The CGI-S scale permits a global evaluation of the participant's current severity of illness on a Likert type scale ranging from 1 to 7, where 1=normal/not at all ill, 2=borderline ill, 3=mildly ill, 4=moderately ill, 5=markedly ill, 6=severely ill, and 7=among the most extremely ill participants |
| Change From Baseline in the Neurofilament Light Chain (NfL) Concentrations in Cerebrospinal Fluid (CSF) | Baseline to Week 48 | — |
| Change From Baseline in the Patient Global Impression-Severity Scale (PGI-S) | Baseline to Week 48 | The PGI-S scale permits an evaluation of the participant's MSA severity, according to the participant. The PGI-S scale rates the participant's MSA severity on a 5-point Likert type scale ranging from 0 (not severe) to 4 (very severe) |
| Change From Baseline in the Pons volume measured by MRI | Baseline to Week 48 | — |
| Change From Baseline in the Cerebellar volume measured by MRI | Baseline to Week 48 | — |
| Change From Baseline in the UMSARS part II score | Baseline to Week 48 | — |
| Change From Baseline in the UMSARS part IV score | Baseline to Week 48 | — |
| Change From Baseline in the Two-minute walk test as part of gait assessment | Baseline to Week 48 | — |
| Change From Baseline in the Multiple System Atrophy - Quality of Life (MSA-QoL) Score | Baseline to Week 48 | The 40-item MSA-QoL questionnaire is self-administered and each item is rated on a Likert type scale (0: No problem) to (4: Extreme problem). It is comprised of 3 subscales relevant to MSA: motor (14 items), non-motor (12 items), and emotional/social (14 items). The MSA-QoL total score is the sum of all the items and lower scores indicate better QoL. |
| Number of Participants With At Least One Treatment-Emergent Adverse Event (TEAEs) | Up to Week 48 | — |
| Number of Participants Who Withdraw From the Study Due to an Adverse Event | Up to Week 48 | — |
| Number of Participants Who Withdraw From Treatment Due to an Adverse Event | Up to Week 48 | — |
| Number of Participants With At Least One Potentially Clinically Significant Abnormal Vital Sign Value | Up to Week 48 | — |
| Number of Participants With At Least One Potentially Clinically Significant Laboratory Test Value | Up to Week 48 | — |
| Number of Participants with at Least One Potentially Clinically Significant Change in 12-lead Electrocardiogram (ECG) Findings | Up to Week 48 | — |
Countries
France, Germany, Israel, Italy, Japan, Serbia, Spain, United States
Contacts
CONTACTTeva U.S. Medical Information
STUDY_DIRECTORTev Medical Expert, Study Director
Teva Branded Pharmaceutical Products R&D LLC
Outcome results
None listed