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

Amyotrophic Lateral Sclerosis Clinical Trial

Official title:

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

Clinical Trial Details — Status: Completed

Administrative data

• NCT number: NCT03520517
• Other study ID #: BHV0223-103
• Status: Completed
• Phase: Phase 1
• Start date: February 2, 2018
• Est. completion date: October 8, 2018

Study information

• Verified date: February 2022
• Source: Biohaven Pharmaceuticals, Inc.
• Contact: n/a
• Is FDA regulated: No
• Study type: Interventional

Clinical Trial Summary

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

Description:

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.

Recruitment information / eligibility

• Status: Completed
• Enrollment: 22
• Est. completion date: October 8, 2018
• Est. primary completion date: October 8, 2018
• Accepts healthy volunteers: No
• Gender: All
• Age group: 18 Years and older

Eligibility

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.

Related Conditions & MeSH terms

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

Intervention

Drug:
• BHV-0223
BHV-0223, 40 mg BID

Locations

Country	Name	City	State
United States	Neurosciences Institute, Neurology - Charlotte	Charlotte	North Carolina
United States	Wesley Neurology Clinic	Cordova	Tennessee
United States	Texas Neurology	Dallas	Texas
United States	Holy Cross Neuroscience Research Institute	Fort Lauderdale	Florida
United States	Somnos/Neurology Associates Clinical Research	Lincoln	Nebraska

Sponsors (2)

Lead Sponsor	Collaborator
Biohaven Pharmaceuticals, Inc.	Cognitive Research Corporation

Country where clinical trial is conducted

United States, 

Outcome

Type	Measure	Description	Time frame	Safety issue
Primary	Incidence of Treatment Emergent Adverse Events (Safety and Tolerability)	Incidence of treatment emergent adverse events (safety and tolerability) as measured through laboratory tests and physical exam findings.	Through Week 8 (Day 57)
Secondary	BHV-0223 Concentrations in Blood at Days 1, 29 and 57	BHV-0223 concentrations at days 1, 29 and 57 through blood collections at days 1, 29 and 57.	Through Week 8 (Day 57)