Clinical trial NCT03520517: 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
Study type: INTERVENTIONAL
Overall status: COMPLETED
Phase: PHASE1
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
Lead sponsor: Biohaven Pharmaceuticals, Inc.
Sponsor class: INDUSTRY
Collaborators: name: Cognitive Research Corporation
First posted: 2018-05-09
Enrollment: 22
Sex: ALL
Minimum age (years): 18
Standard ages: ADULT
OLDER_ADULT
Accepts healthy volunteers: no
Allocation: NA
Intervention model: SINGLE_GROUP
Primary purpose: TREATMENT

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.

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.

Primary outcomes:
measure: Incidence of Treatment Emergent Adverse Events (Safety and Tolerability)
timeFrame: Through Week 8 (Day 57)
Secondary outcomes:
measure: BHV-0223 Concentrations in Blood at Days 1, 29 and 57
timeFrame: Through Week 8 (Day 57)
Other outcomes:
Locations:
city: Fort Lauderdale
country: United States
facility: Holy Cross Neuroscience Research Institute
state: Florida
city: Lincoln
country: United States
facility: Somnos/Neurology Associates Clinical Research
state: Nebraska
city: Charlotte
country: United States
facility: Neurosciences Institute, Neurology - Charlotte
state: North Carolina
city: Cordova
country: United States
facility: Wesley Neurology Clinic
state: Tennessee
city: Dallas
country: United States
facility: Texas Neurology
state: Texas
Study officials:
References: