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

The safety and scientific validity of this study is the responsibility of the study sponsor and investigators. Listing a study does not mean it has been evaluated by the U.S. Federal Government. Read our disclaimer for details.
ClinicalTrials.gov Identifier: NCT03520517
Recruitment Status : Completed
First Posted : May 9, 2018
Last Update Posted : October 6, 2022
Cognitive Research Corporation
Information provided by (Responsible Party):
Biohaven Pharmaceuticals, Inc.

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

Condition or disease Intervention/treatment Phase
Amyotrophic Lateral Sclerosis ALS Lou Gehrig Disease Lou Gehrig's Disease Lou-Gehrigs Disease Motor Neuron Disease, Amyotrophic Lateral Sclerosis Drug: BHV-0223 Phase 1

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.

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Study Type : Interventional  (Clinical Trial)
Actual Enrollment : 22 participants
Allocation: N/A
Intervention Model: Single Group Assignment
Masking: None (Open Label)
Primary Purpose: Treatment
Official Title: Open-Label Study to Evaluate Safety, Tolerability and Pharmacokinetics of Multiple Doses of BHV-0223 in Subjects With Amyotrophic Lateral Sclerosis
Actual Study Start Date : February 2, 2018
Actual Primary Completion Date : October 8, 2018
Actual Study Completion Date : October 8, 2018

Arm Intervention/treatment
Experimental: BHV-0223
riluzole 40 mg sublingual tablet
Drug: BHV-0223
BHV-0223, 40 mg BID

Primary Outcome Measures :
  1. 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.

Secondary Outcome Measures :
  1. 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.

Information from the National Library of Medicine

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Ages Eligible for Study:   18 Years and older   (Adult, Older Adult)
Sexes Eligible for Study:   All
Accepts Healthy Volunteers:   No

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.

Information from the National Library of Medicine

To learn more about this study, you or your doctor may contact the study research staff using the contact information provided by the sponsor.

Please refer to this study by its ClinicalTrials.gov identifier (NCT number): NCT03520517

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United States, Florida
Holy Cross Neuroscience Research Institute
Fort Lauderdale, Florida, United States, 33334
United States, Nebraska
Somnos/Neurology Associates Clinical Research
Lincoln, Nebraska, United States, 68506
United States, North Carolina
Neurosciences Institute, Neurology - Charlotte
Charlotte, North Carolina, United States, 28207
United States, Tennessee
Wesley Neurology Clinic
Cordova, Tennessee, United States, 38018
United States, Texas
Texas Neurology
Dallas, Texas, United States, 75214
Sponsors and Collaborators
Biohaven Pharmaceuticals, Inc.
Cognitive Research Corporation
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Responsible Party: Biohaven Pharmaceuticals, Inc.
ClinicalTrials.gov Identifier: NCT03520517    
Other Study ID Numbers: BHV0223-103
First Posted: May 9, 2018    Key Record Dates
Last Update Posted: October 6, 2022
Last Verified: February 2022
Individual Participant Data (IPD) Sharing Statement:
Plan to Share IPD: No

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Studies a U.S. FDA-regulated Drug Product: Yes
Studies a U.S. FDA-regulated Device Product: No
Keywords provided by Biohaven Pharmaceuticals, Inc.:
Amyotrophic Lateral Sclerosis
Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Lou Gehrig's Disease
Lou-Gehrigs Disease
Lou Gehrig Disease
Neuroprotective Agents
Administration, Sublingual
Administration, Oral
Biological Availability
Additional relevant MeSH terms:
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Motor Neuron Disease
Amyotrophic Lateral Sclerosis
Pathologic Processes
Neurodegenerative Diseases
Nervous System Diseases
Neuromuscular Diseases
Spinal Cord Diseases
Central Nervous System Diseases
TDP-43 Proteinopathies
Proteostasis Deficiencies
Metabolic Diseases