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

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

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

Study Overview

• Status: Completed
• Conditions: Amyotrophic Lateral Sclerosis; ALS; Lou Gehrig Disease; Lou Gehrig's Disease; Lou-Gehrigs Disease; Motor Neuron Disease, Amyotrophic Lateral Sclerosis
• Intervention / Treatment: Drug: BHV-0223

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 Type: Interventional
• Enrollment (Actual): 22
• Phase: Phase 1

Contacts and Locations

Study Locations

• United States
  • Florida
    • Fort Lauderdale, Florida, United States, 33334
      • Holy Cross Neuroscience Research Institute
  • Nebraska
    • Lincoln, Nebraska, United States, 68506
      • Somnos/Neurology Associates Clinical Research
  • North Carolina
    • Charlotte, North Carolina, United States, 28207
      • Neurosciences Institute, Neurology - Charlotte
  • Tennessee
    • Cordova, Tennessee, United States, 38018
      • Wesley Neurology Clinic
  • Texas
    • Dallas, Texas, United States, 75214
      • Texas Neurology

Participation Criteria

Eligibility Criteria

• Ages Eligible for Study: 18 years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Genders Eligible for Study: All

Description

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 Plan

How is the study designed?

Design Details

• Primary Purpose: Treatment
• Allocation: N/A
• Interventional Model: Single Group Assignment
• Masking: None (Open Label)

Number of Arms

1

Arms and Interventions

Participant Group / Arm	Intervention / Treatment
Experimental: BHV-0223; riluzole 40 mg sublingual tablet	Drug: BHV-0223; BHV-0223, 40 mg BID

What is the study measuring?

Primary Outcome Measures

Outcome Measure	Measure Description	Time Frame
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 Outcome Measures

Outcome Measure	Measure Description	Time Frame
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)

Collaborators and Investigators

• Sponsor: Biohaven Pharmaceuticals, Inc.
• Collaborators: Cognitive Research Corporation

Study record dates

Study Major Dates

• Study Start (Actual): February 2, 2018
• Primary Completion (Actual): October 8, 2018
• Study Completion (Actual): October 8, 2018

Study Registration Dates

• First Submitted: April 17, 2018
• First Submitted That Met QC Criteria: May 8, 2018
• First Posted (Actual): May 9, 2018

Study Record Updates

• Last Update Posted (Actual): October 6, 2022
• Last Update Submitted That Met QC Criteria: October 4, 2022
• Last Verified: February 1, 2022

More Information

Terms related to this study

• Keywords: Amyotrophic Lateral Sclerosis; ALS; Riluzole; Administration, Oral; Lou Gehrig's Disease; Biological Availability; Biohaven; Motor Neuron Disease, Amyotrophic Lateral Sclerosis; Lou-Gehrigs Disease; Lou Gehrig Disease; Rilutek®; Neuroprotective Agents; Administration, Sublingual
• Additional Relevant MeSH Terms: Pathologic Processes; Metabolic Diseases; Central Nervous System Diseases; Nervous System Diseases; Neuromuscular Diseases; Neurodegenerative Diseases; Spinal Cord Diseases; TDP-43 Proteinopathies; Proteostasis Deficiencies; Sclerosis; Motor Neuron Disease; Amyotrophic Lateral Sclerosis
• Other Study ID Numbers: BHV0223-103

Plan for Individual participant data (IPD)

• Plan to Share Individual Participant Data (IPD)?: No

Drug and device information, study documents

• Studies a U.S. FDA-regulated drug product: Yes
• Studies a U.S. FDA-regulated device product: No