Open-label Study to Evaluate Safety, Tolerability and PK of BHV-0223 in ALS

• Register: ClinicalTrials.gov
• Last refreshed on: 17 October 2022
• Main ID: NCT03520517
• Date of registration: 17/04/2018
• Prospective Registration: No
• Primary sponsor: Biohaven Pharmaceuticals, Inc.
• Public title: Open-label Study to Evaluate Safety, Tolerability and PK of BHV-0223 in ALS
• Scientific title: Open-Label Study to Evaluate Safety, Tolerability and Pharmacokinetics of Multiple Doses of BHV-0223 in Subjects With Amyotrophic Lateral Sclerosis
• Date of first enrolment: February 2, 2018
• Target sample size: 22
• Recruitment status: Completed
• URL: https://clinicaltrials.gov/show/NCT03520517
• Study type: Interventional
• Study design: Allocation: N/A. Intervention model: Single Group Assignment. Primary purpose: Treatment. Masking: None (Open Label).
• Phase: Phase 1

Countries of recruitment

United States

Key inclusion & exclusion criteria

Inclusion Criteria:

1. Subjects with diagnosed ALS by the revised El Escorial diagnostic criteria, including
laboratory supported probable, probable, or definite ALS;

2. Subjects who have never taken riluzole tablets, OR Subjects who previously took
riluzole tablets but discontinued at least 1 month prior to the screening visit.
Subjects must not have had ALT or AST > 5 x ULN while taking riluzole tablets, or any
other clinically significant tolerability issues (e.g., hypersensitivity reactions) in
the judgement of the investigator;

3. Subjects determined by the investigator to be medically stable;

4. Subjects determined by the investigator to be willing and physically able to complete
the study as designed, with or without caregiver assistance.

Exclusion Criteria:

1. Target Disease Exceptions

2. Medical History Exceptions

1. Subject is known to have a current diagnosis of acute or chronic viral hepatitis;

2. Subject is known to have any other acute or chronic liver disease that is
clinically significant in the investigator's judgment;

3. Subject has a history of a clinically significant medical condition that would
interfere with the subject's ability to comply with study instructions, would
place the subject at increased risk, or might confound the interpretation of the
study results in the investigator's judgment;

4. Any other sound medical, psychiatric and/or social reason in the investigator's
judgment;

3. Physical and Laboratory Test Findings

1. Positive urine pregnancy test in WOCBP at screening;

2. Subject has evidence of organ dysfunction or any clinically significant deviation
from normal in physical examination, vital signs, or other determinations beyond
what is consistent with the target population, in the investigator's judgment;

3. Subject has liver function testing abnormalities (ALT, AST , or total bilirubin)
that are > 1 x ULN;

4. Other Exclusions a. Subjects who are unable to be compliant with the visit schedule or
protocol procedures.



Age minimum: 18 Years
Age maximum: N/A
Gender: All

Health Condition(s) or Problem(s) studied

Motor Neuron Disease, Amyotrophic Lateral Sclerosis

Amyotrophic Lateral Sclerosis

ALS

Lou-Gehrigs Disease

Lou Gehrig Disease

Lou Gehrig's Disease

Intervention(s)

Drug: BHV-0223

Primary Outcome(s)

Incidence of Treatment Emergent Adverse Events (Safety and Tolerability) [Time Frame: Through Week 8 (Day 57)]

Secondary Outcome(s)

BHV-0223 Concentrations in Blood at Days 1, 29 and 57 [Time Frame: Through Week 8 (Day 57)]

Secondary ID(s)

BHV0223-103

Source(s) of Monetary Support

Please refer to primary and secondary sponsors

Secondary Sponsor(s)

Cognitive Research Corporation