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

Phase 1

Completed

• Conditions: Lou Gehrig's Disease; Amyotrophic Lateral Sclerosis; ALS; Lou Gehrig Disease; Lou-Gehrigs Disease; Motor Neuron Disease, Amyotrophic Lateral Sclerosis
• Interventions: Drug: BHV-0223

• Registration Number: NCT03520517
• Lead Sponsor: Biohaven Pharmaceuticals, Inc.

Brief Summary

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

Detailed 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.

Study Timeline

• Study Start: 2018-02-02
• Study First Submitted: 2018-04-17
• Study First Submitted QC: 2018-05-08
• Study First Posted: 2018-05-09
• Primary Completion: 2018-10-08
• Study Completion: 2018-10-08
• Status Verified: 2022-02
• Last Update Submitted: 2022-10-04
• Last Update Posted: 2022-10-06

Recruitment & Eligibility

• Status: COMPLETED
• Sex: All
• Target Recruitment: 22

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.

Study & Design

• Study Type: INTERVENTIONAL
• Study Design: SINGLE_GROUP

Arm && Interventions

Group	Intervention	Description
BHV-0223	BHV-0223	riluzole 40 mg sublingual tablet

Primary Outcome Measures

Name	Time	Method
Incidence of Treatment Emergent Adverse Events (Safety and Tolerability)	Through Week 8 (Day 57)	Incidence of treatment emergent adverse events (safety and tolerability) as measured through laboratory tests and physical exam findings.

Secondary Outcome Measures

Name	Time	Method
BHV-0223 Concentrations in Blood at Days 1, 29 and 57	Through Week 8 (Day 57)	BHV-0223 concentrations at days 1, 29 and 57 through blood collections at days 1, 29 and 57.

Trial Locations

Locations (5)

Wesley Neurology Clinic; 🇺🇸 Cordova, Tennessee, United States

Holy Cross Neuroscience Research Institute; 🇺🇸 Fort Lauderdale, Florida, United States

Texas Neurology; 🇺🇸 Dallas, Texas, United States

Somnos/Neurology Associates Clinical Research; 🇺🇸 Lincoln, Nebraska, United States

Neurosciences Institute, Neurology - Charlotte; 🇺🇸 Charlotte, North Carolina, United States