A study to assess whether a drug called BHV-3241 works and is safe to use in people with Multiple System Atrophy (M-STAR Study)
- Conditions
- Multiple System AtrophyMedDRA version: 21.1Level: PTClassification code 10064060Term: Multiple system atrophySystem Organ Class: 10029205 - Nervous system disordersTherapeutic area: Diseases [C] - Nervous System Diseases [C10]
- Registration Number
- EUCTR2019-001100-38-DE
- Lead Sponsor
- Biohaven Pharmaceuticals, Inc
- Brief Summary
Not available
- Detailed Description
Not available
Recruitment & Eligibility
- Status
- Authorised-recruitment may be ongoing or finished
- Sex
- All
- Target Recruitment
- 422
Informed Consent
a. Subjects must provide a written signed and dated informed consent
form/forms (IRB/EC specific)in accordance with regulatory and
institutional guidelines prior to the initiation of any protocol required
procedures. Only patients with the capacity to understand the nature ,
significance, and scope of the clinical trial interventions and to express
their wishes accordingly may provide consent to participate in the study.
b. Caregivers must be willing to sign and date an IRB/EC-approved
written informed consent form that outlines the caregiver expectations
and responsibilities in this study in accordance with regulatory and
institutional guidelines, as appropriate.
Age and Sex
a. Male and female subjects between the ages of > or = 40 to = or <80
years at time of Screening.
Target Population
a. Diagnosis of probable or possible MSA according to consensus clinical
criteria including subjects with MSA of either subtype (MSA-P or MSA-
C).
b. Able to ambulate without the assistance of another person, defined as
the ability to take at least 10 steps. Use of assistive devices is allowed.
c. Anticipated survival of at least 3 years at the time of Screening, as
judged by the Investigator.
d. A brain MRI scan (conducted within the 14 days prior to Baseline/Day
1, approximately) that does not rule out a diagnosis of MSA.
e. Able to tolerate MRI.
f. Body mass index (BMI) = or < 40 kg/m2at Screening.
g. Able to swallow tablets whole and anticipated to be able to do so
throughout the duration of the study.
h. Willing and able to adhere to the study drug regimen.
i. Willing and able to perform all protocol-specified assessments and
comply with the study visit schedule.
j. Able to read, understand, and speak local language fluently to ensure
comprehension of informed consent and protocol-specified assessments.
k. Must have reliable caregiver to accompany subject to study visits.
With the same Caregiver giving caregiver completing assessments at
Baseline, Week 24 and Week 48/ Early discontinuation, when possible.
Caregiver must be able to read, understand, and speak local language
fluently to ensure comprehension of informed consent and protocol-
specified assessments. Caregiver must also have frequent contact with
subject (at least 3 hours per week at one time or different times) and be
willing to monitor the subject's health and concomitant medications
throughout the study.
l. If subject is receiving treatment for MSA, the doses must have been
stable for at least 30 days prior to Baseline/Randomization and
medication present at Baseline. expected to remain relatively stable during the study period. This may include medications commonly used
for Parkinson's disease or those for autonomic dysfunction.
m. Stable on other chronic medications and supplements for at least 30
days prior to Baseline/Randomization and expected to remain relatively
stable during the study period..
n. Women of child bearing potential (WOCBP) and fertile men (including
those vasectomized for less than 6 months) with female partners who
are WOCBP (not surgically sterile and not post-menopausal) must agree
to use highly effective birth control, including two methods of
contraception, for the duration of the study (beginning 30 days prior to
Baseline/Randomization and extending to 30 days for women and 90
days for men after the last dose of study drug). The two methods of
contraception should include:
- one barrier metho
Target Disease Exceptions
a. Subjects having advanced disease as defined in the protocol.
b. Subjects having significant cognitive impairment, defined by a score of
less than or equal to 20 on the MoCA
Medical History Exclusions
a. Any condition that would interfere with the subject's ability to comply
with
study instructions, place the subject at unacceptable risk, and/or
confound the
interpretation of safety or efficacy data from the study, as judged by the
Investigator.
b. Diagnosis of neurological disorders, other than MSA as defined in the
protocol
c. History of or Screening brain MRI scan indicative of significant
abnormality.
d. Contraindication to MRI examination for any reason.
e. For optional CSF sub-study: contraindication to undergoing an LP.
f. Presence of clinically significant thyroid disease with abnormal free T4
levels and TSH >10mIU/L (despite treatment) at Screening , confirmed
by repeat.
g. Within 1 year prior to Screening or between Screening and Baseline
(Day -1), any of the following: myocardial infarction; hospitalization for
congestive heart failure; hospitalization for, or symptoms of, unstable
angina; or syncope not related to MSA.
h. Diagnosis of clinically significant psychiatric disorder as defined in the
protocol.
i. History of substance use disorder (drug or alcohol) in the last 12
months, with
the exception of nicotine, as defined by DSM-V criteria.
j. History or presence of gastrointestinal or other disease known to
interfere with absorption, distribution, metabolism, or excretion of
drugs, or a history of surgery known to interfere with absorption or
excretion of drugs (i.e. gastric bypass).
k. History of any other clinically significant disease that, based on the
judgment of the Investigator, is clinically unstable, is likely to
deteriorate during the course of the study, could put the patient at risk
because of participation in the study, could affect the subject's ability to
complete the study, or could influence the study results.
l. History of human immunodeficiency virus infection.
m. Hematologic or solid malignancy diagnosis within 5 years prior to
Screening.
n. Any major surgery within 4 weeks of Screening.
o. Blood transfusion within 4 weeks of Screening.
p. History of brain surgery for Parkinsonism.
q. History of stem-cell treatment.
r. Women who are pregnant or breastfeeding.
s. Subjects or prisoners who are involuntarily detained or incarcerated
for treatment of either a psychiatric or physical illness must not be
enrolled into the study.
t. Any medical condition, based on the judgement of the Investigator,
that would confound the ability to adequately assess safety and efficacy
outcome measures
Physical and Laboratory Test Findings
a. Evidence of organ dysfunction or any clinically significant deviation
from normal in physical examination, vital signs, ECG, or clinical
laboratory determinations beyond what is consistent with the target
population.
b. Clinically significant abnormality on 12-lead ECG prior to study drug
administration beyond what is consistent with the target population,
confirmed by repeat.
c. QTcF (Fridericia) interval = 470 msec during the Screening/Baseline
period or uncontrolled arrhythmia or frequent premature ventricular
contraction (PVCs) (> 5/minute) or Mobitz Type II second or third
degree atrioventricular (AV) block or left bundle branch block, or right
bundle branch block with a QRS
duration = 150 msec or intraventricula
Study & Design
- Study Type
- Interventional clinical trial of medicinal product
- Study Design
- Not specified
- Primary Outcome Measures
Name Time Method
- Secondary Outcome Measures
Name Time Method