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

• ClinicalTrials.gov Identifier: NCT03520517
• Recruitment Status: Completed
• First Posted: May 9, 2018
• Last Update Posted: October 6, 2022
• Sponsor: Biohaven Pharmaceuticals, Inc.
• Collaborator: Cognitive Research Corporation
• Information provided by (Responsible Party): Biohaven Pharmaceuticals, Inc.

Tracking Information

• First Submitted Date: April 17, 2018
• First Posted Date: May 9, 2018
• Last Update Posted Date: October 6, 2022
• Actual Study Start Date: February 2, 2018
• Actual Primary Completion Date: October 8, 2018 (Final data collection date for primary outcome measure)

Current Primary Outcome Measures (submitted: May 8, 2018)

Incidence of Treatment Emergent Adverse Events (Safety and Tolerability) [ Time Frame: Through Week 8 (Day 57) ]

Incidence of treatment emergent adverse events (safety and tolerability) as measured through laboratory tests and physical exam findings.

• Original Primary Outcome Measures: Same as current

Current Secondary Outcome Measures (submitted: May 8, 2018)

BHV-0223 Concentrations in Blood at Days 1, 29 and 57 [ Time Frame: Through Week 8 (Day 57) ]

BHV-0223 concentrations at days 1, 29 and 57 through blood collections at days 1, 29 and 57.

• Original Secondary Outcome Measures: Same as current
• Current Other Pre-specified Outcome Measures: Not Provided
• Original Other Pre-specified Outcome Measures: Not Provided

Descriptive Information

• Brief Title: Open-label Study to Evaluate Safety, Tolerability and PK of BHV-0223 in ALS
• Official Title: Open-Label Study to Evaluate Safety, Tolerability and Pharmacokinetics of Multiple Doses of BHV-0223 in Subjects With Amyotrophic Lateral Sclerosis
• 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 Type: Interventional
• Study Phase: Phase 1
• Study Design: Allocation: N/A; Intervention Model: Single Group Assignment; Masking: None (Open Label); Primary Purpose: Treatment

Condition

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

Intervention

Drug: BHV-0223

BHV-0223, 40 mg BID

Study Arms

Experimental: BHV-0223

riluzole 40 mg sublingual tablet

Intervention: Drug: BHV-0223

• Publications *: Not Provided

Recruitment Information

• Recruitment Status: Completed
• Actual Enrollment (submitted: August 3, 2018): 22
• Original Estimated Enrollment (submitted: May 8, 2018): 20
• Actual Study Completion Date: October 8, 2018
• Actual Primary Completion Date: October 8, 2018 (Final data collection date for primary outcome measure)

Eligibility Criteria

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.

Sex/Gender

• Sexes Eligible for Study: All

• Ages: 18 Years and older (Adult, Older Adult)
• Accepts Healthy Volunteers: No
• Contacts: Contact information is only displayed when the study is recruiting subjects
• Listed Location Countries: United States

Administrative Information

• NCT Number: NCT03520517
• Other Study ID Numbers: BHV0223-103
• Has Data Monitoring Committee: No

U.S. FDA-regulated Product

• Studies a U.S. FDA-regulated Drug Product: Yes
• Studies a U.S. FDA-regulated Device Product: No

IPD Sharing Statement

• Plan to Share IPD: No

• Current Responsible Party: Biohaven Pharmaceuticals, Inc.
• Original Responsible Party: Biohaven Pharmaceutical Holding Company Ltd.
• Current Study Sponsor: Biohaven Pharmaceuticals, Inc.
• Original Study Sponsor: Biohaven Pharmaceutical Holding Company Ltd.
• Collaborators: Cognitive Research Corporation
• Investigators: Not Provided
• PRS Account: Biohaven Pharmaceuticals, Inc.
• Verification Date: February 2022