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

NCT03520517 | Completed Phase 1 FDA Drug

• 1 other identifier: BHV0223-103
• Study Type: interventional
• Target: 22
• Locations: 1 country
• Sites: 5

Brief Summary

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

Conditions

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

Keywords

Amyotrophic Lateral Sclerosis; ALS; Biohaven; Motor Neuron Disease, Amyotrophic Lateral Sclerosis; Lou Gehrig's Disease; Lou-Gehrigs Disease; Lou Gehrig Disease; Riluzole; Rilutek®; Neuroprotective Agents; Administration, Sublingual; Administration, Oral; Biological Availability

Outcome Measures

Primary Outcomes (1)

• 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 Outcomes (1)

• BHV-0223 Concentrations in Blood at Days 1, 29 and 57

  Through Week 8 (Day 57)

Study Arms (1)

BHV-0223

EXPERIMENTAL

riluzole 40 mg sublingual tablet

Drug: BHV-0223

Interventions

BHV-0223 DRUG

BHV-0223, 40 mg BID

BHV-0223

Eligibility Criteria

• Age: 18 Years+
• Sex: all
• Healthy Volunteers: No
• Age Groups: Adult (18-64), Older Adult (65+)

You may qualify if:

• Subjects with diagnosed ALS by the revised El Escorial diagnostic criteria, including laboratory supported probable, probable, or definite ALS;
• 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;
• Subjects determined by the investigator to be medically stable;
• Subjects determined by the investigator to be willing and physically able to complete the study as designed, with or without caregiver assistance.

You may not qualify if:

• Target Disease Exceptions
• Medical History Exceptions
• Subject is known to have a current diagnosis of acute or chronic viral hepatitis;
• Subject is known to have any other acute or chronic liver disease that is clinically significant in the investigator's judgment;
• 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;
• Any other sound medical, psychiatric and/or social reason in the investigator's judgment;
• Physical and Laboratory Test Findings
• Positive urine pregnancy test in WOCBP at screening;
• 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;
• Subject has liver function testing abnormalities (ALT, AST , or total bilirubin) that are \> 1 x ULN;

Sponsors & Collaborators

• Biohaven Pharmaceuticals, Inc.: lead
• Cognitive Research Corporation: collaborator

Study Sites (5)

Holy Cross Neuroscience Research Institute

Fort Lauderdale, Florida, 33334, United States

Location

Somnos/Neurology Associates Clinical Research

Lincoln, Nebraska, 68506, United States

Location

Neurosciences Institute, Neurology - Charlotte

Charlotte, North Carolina, 28207, United States

Location

Wesley Neurology Clinic

Cordova, Tennessee, 38018, United States

Location

Texas Neurology

Dallas, Texas, 75214, United States

Location

MeSH Terms

Conditions

Amyotrophic Lateral Sclerosis

Condition Hierarchy (Ancestors)

Spinal Cord Diseases; Central Nervous System Diseases; Nervous System Diseases; Motor Neuron Disease; Neurodegenerative Diseases; TDP-43 Proteinopathies; Neuromuscular Diseases; Proteostasis Deficiencies; Metabolic Diseases; Nutritional and Metabolic Diseases

Study Design

• Study Type: interventional
• Phase: phase 1
• Allocation: NA
• Masking: NONE
• Purpose: TREATMENT
• Intervention Model: SINGLE GROUP
• Sponsor Type: INDUSTRY
• Responsible Party: SPONSOR

Study Record Dates

• First Submitted: April 17, 2018
• First Posted: May 9, 2018
• Study Start: February 2, 2018
• Primary Completion: October 8, 2018
• Study Completion: October 8, 2018
• Last Updated: October 6, 2022

Record last verified: 2022-02

Data Sharing

• IPD Sharing: Will not share

Locations

US (5)