Open-Label Study to Evaluate Safety, Tolerability and Pharmacokinetics of Multiple Doses of BHV-0223 in Subjects With Amyotrophic Lateral Sclerosis

Study Purpose:

Phase 1, open-label study of BHV-0223 in ALS.

Study Status:

Not recruiting


Amyotrophic Lateral Sclerosis , ALS , Lou Gehrig Disease , Lou Gehrig's Disease , Lou-Gehrigs Disease , Motor Neuron Disease, Amyotrophic Lateral Sclerosis

Study Type:


Type of Intervention:


Intervention Name:





Phase 1

Study Chair(s)/Principal Investigator(s):



Neals Affiliated?


Coordinating Center Contact Information

United States

Full Study Summary:

This is a phase 1, open-label, single arm study to evaluate the safety, tolerability and pharmacokinetics of multiple doses of BHV-0223 in subjects with ALS.

Study Sponsor:

Biohaven Pharmaceuticals, Inc.

Estimated Enrollment:


Estimated Study Start Date:

02 / 02 / 2018

Estimated Study Completion Date:

10 / 08 / 2018

Posting Last Modified Date:

10 / 06 / 2022

Date Study Added to

05 / 09 / 2018

Minimum Age:

18 Years

Maximum Age:


Can participants use Riluzole?


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.

Holy Cross Neuroscience Research Institute

Fort Lauderdale, Florida 33334
United States

Somnos/Neurology Associates Clinical Research

Lincoln, Nebraska 68506
United States

Neurosciences Institute, Neurology - Charlotte

Charlotte, North Carolina 28207
United States

Wesley Neurology Clinic

Cordova, Tennessee 38018
United States

Texas Neurology

Dallas, Texas 75214
United States