Amyotrophic Lateral SclerosisPhase 1NCT03520517
Open-label Study to Evaluate Safety, Tolerability and PK of BHV-0223 in ALS
A Phase 1 study testing BHV-0223 in people with Amyotrophic Lateral Sclerosis and ALS, run by Biohaven Pharmaceuticals, Inc. at 5 sites in United States.
Trial summary
Phase 1, open-label study of BHV-0223 in ALS. Read more about this study
- Who is it for?
- Ages 18 years and older
- Conditions the study is for
- Amyotrophic Lateral Sclerosis, ALS, Lou Gehrig Disease
- What is being tested?
- BHV-0223
- Type of treatment
- Pill or oral medicine
- Could I receive a placebo?
- No, everyone receives an active treatment
- How many people
- 22 participants
- Where
- 5 sitesUnited States
- Study dates
- 2 Feb 2018to 8 Oct 2018
- Phase
- Phase 1
- Are there results?
- None posted in the registry
Key requirements
Who can take part, as listed by the sponsor. Only the study team can confirm whether you are eligible, so take this list to your doctor.
- Ages 18 years and older
- All sexes
- No healthy volunteers
- Adult, Older adult
Who may be able to join
- 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.
Who cannot join
- 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;
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- 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;
- Other Exclusions a. Subjects who are unable to be compliant with the visit schedule or protocol procedures.
Trial locations and contacts
5 sites in 1 country.
5 sites in 1 country
United States5 sites
Neurosciences Institute, Neurology - Charlotte
Charlotte, North Carolina, 28207
Wesley Neurology Clinic
Cordova, Tennessee, 38018
Texas Neurology
Dallas, Texas, 75214
Holy Cross Neuroscience Research Institute
Fort Lauderdale, Florida, 33334
Somnos/Neurology Associates Clinical Research
Lincoln, Nebraska, 68506
About this study
Official title: Open-Label Study to Evaluate Safety, Tolerability and Pharmacokinetics of Multiple Doses of BHV-0223 in Subjects With Amyotrophic Lateral Sclerosis
Phase 1, open-label study of BHV-0223 in ALS.
Read the 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.
- Conditions
- Amyotrophic Lateral Sclerosis, ALS, Lou Gehrig Disease, Lou Gehrig's Disease, Lou-Gehrigs Disease, Motor Neuron Disease, Amyotrophic Lateral Sclerosis
- Being tested
- BHV-0223
- 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
- Medical subject headings
- Amyotrophic Lateral Sclerosis
What participants receive
What participants receive. In a randomised study each person is assigned to one group by chance.
BHV-0223Drug
BHV-0223, 40 mg BID
| Study group | Role | What they receive |
|---|---|---|
| BHV-0223 | Experimental | riluzole 40 mg sublingual tablet Receives: Drug: BHV-0223 |
What is being measured
The primary outcome is the main question the study is designed to answer.
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.
Measured: Through Week 8 (Day 57)
Secondary outcomes (1)
BHV-0223 Concentrations in Blood at Days 1, 29 and 57
BHV-0223 concentrations at days 1, 29 and 57 through blood collections at days 1, 29 and 57.
Measured: Through Week 8 (Day 57)
Timeline
From the start of the study to its completion; the mark is today. Estimated dates are the sponsor’s plan.
- First posted9 May 2018
- Study start2 Feb 2018
- Primary completion8 Oct 2018
- Study completion8 Oct 2018
Resources
- Official record on ClinicalTrials.govThe registry entry this page is built from, with its full change history.
- Other trials for Amyotrophic Lateral SclerosisStudies of the same condition, open and under way.
- What the trial phases meanHow a treatment moves from first tests in people to approval.
More details
For doctors and researchers: how the study is designed, who runs it, and its registry IDs.
Study design
- Study type
- Interventional
- Phase
- Phase 1
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Enrolment
- 22 participants (actual)
Sponsor and investigators
- Lead sponsor
- Biohaven Pharmaceuticals, Inc.
- Sponsor type
- Industry (pharma, biotech, devices)
- Collaborators
- Cognitive Research Corporation
- Responsible party
- Sponsor
IDs, dates and publications
- ClinicalTrials.gov ID
- NCT03520517
- Sponsor's protocol ID
- BHV0223-103
- Status last verified
- Feb 2022
- First submitted
- 17 Apr 2018
- Last updated
- 6 Oct 2022
- Results posted
- No
- Data monitoring committee
- No
- FDA-regulated drug
- Yes
- FDA-regulated device
- No
- Individual data sharing
- No
Study at a glance
From the registry record, last updated by the sponsor on 6 Oct 2022. Recruitment status is as the sponsor reported it: confirm with the study team.
- Official title
- Open-Label Study to Evaluate Safety, Tolerability and Pharmacokinetics of Multiple Doses of BHV-0223 in Subjects With Amyotrophic Lateral Sclerosis
- ClinicalTrials.gov ID
- NCT03520517
- Acronym / sponsor ID
- BHV0223-103
- 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
- Phase
- Phase 1
- Study type
- Interventional
- Recruitment status
- Completed
- Enrolment
- 22 participants (actual)
- Lead sponsor
- Biohaven Pharmaceuticals, Inc.Industry (pharma, biotech, devices)
- Collaborators
- Cognitive Research Corporation
- Study start
- 2 Feb 2018 (actual)
- Primary completion
- 8 Oct 2018 (actual)
- Study completion
- 8 Oct 2018 (actual)
- First posted
- 9 May 2018 (actual)
- Results
- Not posted in the source record
Clinical summary
The research question as the sponsor registered it. This describes what the study plans to test, not what it found.
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 design
- Primary purpose
- Treatment
- Allocation
- Not applicable
- Intervention model
- Single group
- Masking
- None (open label)
- Number of arms
- 1
| Arm | Type | Receives |
|---|---|---|
| BHV-0223 | Experimental | Drug: BHV-0223riluzole 40 mg sublingual tablet |
Who may qualify
- Ages
- 18 years and older
- Sex
- All
- Healthy volunteers
- Not accepted
- Age groups
- Adult, Older adult
Key inclusion criteria (4 listed)
- 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.
Key exclusion criteria (11 listed)
- Target Disease Exceptions
- Medical History Exceptions
- Physical and Laboratory Test Findings
- Other Exclusions a. Subjects who are unable to be compliant with the visit schedule or protocol procedures.
Eligibility is decided by the study team after screening; this overview does not establish that a patient qualifies. Full inclusion and exclusion criteria.
Interventions
| Intervention | Type | Arms |
|---|---|---|
| BHV-0223BHV-0223, 40 mg BID | Drug | BHV-0223 |
Primary outcome
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.
1 secondary outcome
BHV-0223 Concentrations in Blood at Days 1, 29 and 57
Time frame: Through Week 8 (Day 57)
Results and safety
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
Research links
- Official record on ClinicalTrials.govLast updated by the sponsor 6 Oct 2022.
- Individual participant dataSharing planned: No
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Referral and contact
AzTrials does not enrol participants or take referrals. Contact the study team or a site listed below; they decide who can take part. This study is not recruiting at the moment.
The record lists no contact. Use the official record to reach the sponsor.
5 sites in 1 country: see every site and its status.
Questions about this trial
What is this clinical trial studying?
A Phase 1 study testing BHV-0223 in people with Amyotrophic Lateral Sclerosis and ALS, run by Biohaven Pharmaceuticals, Inc. at 5 sites in United States.
The full registered description is in the About this study section and the official record.
Who may be eligible to take part?
The sponsor lists 4 inclusion and 11 exclusion criteria. Eligibility is decided by the study team after screening, so read the full criteria and contact a listed site.
Is this clinical trial currently recruiting?
The record says: finished, as of the sponsor's last update on 6 Oct 2022. Status can change between updates, so confirm with the study contact before making plans.
Where is the study taking place?
The record lists 5 sites in United States. Whether a site is taking part right now should be confirmed with that site. See every site.
What are the possible benefits and risks?
They depend on the study and the treatment, and no study can promise a benefit. The study team explains the known risks during informed consent, before anyone joins; the record itself does not list them.
What does this phase mean?
Phase 1 studies test a treatment in a small group of people for the first time or in a new way, mainly to check safety and find the right dose.
Not every study follows the same path, and some studies (observational studies, device studies) have no phase.
Are results available for this study?
The study is completed, but results are not available in the source record. That does not mean the treatment did or did not work.
How can a doctor refer a patient or contact the research team?
The record lists no contact. The official record names the sponsor, who can be asked directly. AzTrials does not enrol participants or take referrals.
What does the primary outcome measure?
Incidence of Treatment Emergent Adverse Events (Safety and Tolerability) (time frame: Through Week 8 (Day 57)). It is what the study was designed to measure, not a finding.
Where can I read the original study information?
Does a listing mean the treatment is approved or proven to work?
No. A listing means the study is registered. It does not show that the treatment is safe, works, or is approved by a regulator.
How often is this information updated?
AzTrials copies changed records from ClinicalTrials.gov every night and reloads the whole registry every week. The sponsor last updated this record on 6 Oct 2022.