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-FR
- 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
- 336
Informed Consent
a. Subjects must provide a written signed and dated informed consent form/forms prior to the initiation of any protocol required procedures.
b. Caregivers must be willing to sign and date an IRB/EC-approved written informed consent form in accordance with regulatory and institutional guidelines.
Age and Sex
a. Male and female subjects between the ages of >40 to <75 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 14 days of Baseline/Day 1) that does not rule out a diagnosis of MSA.
e. Able to tolerate MRI.
f. Body mass index (BMI) < 35 kg/m2 at 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 all study visits. 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 60 days prior to Screening and 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 Screening.
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 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 method (e.g. diaphragm with spermicide, condom with spermicidal gel, intrauterine contraceptive devices [placed at least 4 weeks prior to sexual intercourse], cervical cap);
- and one other method that could include hormonal contraceptives (e.g. oral contraceptives, injectable contraceptives, contraceptive implant, patch) used for at least 4 weeks prior to sexual intercourse or another barrier method.
o. WOCBP must have a negative serum pregnancy test at Screening and a negative urine pregnancy test within approximately 24 hours prior to dosing at Baseline.
Are the trial subjects under 18? no
Number of subjects for this age range:
F.1.2 Adults (18-64 years) yes
F.1.2.1 Number of subject
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 23 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. History or presence of clinically significant thyroid disease
g. Within 1 year prior to screen or between screen 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.
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 intraventricular conduction defect with a QRS duration = 150 msec or evidence of acute or sub-acute myocardial infarction or ischem
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