ALS, Amyotrophic Lateral Sclerosis, Lou Gehrig Disease, Lou Gehrig's Disease, Lou-Gehrigs Disease, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
Conditions
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
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.
Interventions
DRUGBHV-0223
BHV-0223, 40 mg BID
Sponsors
Cognitive Research Corporation
Biohaven Pharmaceuticals, Inc.
Study design
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT
Masking
NONE
Eligibility
Sex/Gender
ALL
Age
18 Years to No maximum
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.
Design outcomes
Primary
| Measure | Time frame | Description |
|---|---|---|
| Incidence of Treatment Emergent Adverse Events (Safety and Tolerability) | Through Week 8 (Day 57) | Incidence of treatment emergent adverse events (safety and tolerability) as measured through laboratory tests and physical exam findings. |
Secondary
| Measure | Time frame | Description |
|---|---|---|
| BHV-0223 Concentrations in Blood at Days 1, 29 and 57 | Through Week 8 (Day 57) | BHV-0223 concentrations at days 1, 29 and 57 through blood collections at days 1, 29 and 57. |
Countries
United States
Outcome results
None listed