- ICH GCP
- US Clinical Trials Registry
- Clinical Trial NCT05873062
Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK (AUT022201)
February 13, 2024 updated by: Autifony Therapeutics Limited
A Randomized, Double-blind, Placebo-controlled, Crossover Study of the Effects of Single Doses of AUT00201 in Patients With Myoclonus Epilepsy and Ataxia Due to Potassium (K+) Channel Mutation (MEAK)
A randomized, double-blind, placebo-controlled, crossover study to assess the safety, tolerability, and pharmacokinetics of single doses of AUT00201 at 100 mg or matching placebo in patients with myoclonus epilepsy and ataxia due to potassium channel mutation (MEAK).
Study Overview
Status
Recruiting
Conditions
Intervention / Treatment
Detailed Description
6 to 10 patients aged 18 years and older, diagnosed with MEAK will be enrolled in the study.
Patients will be administered a single dose of AUT00201 and matching placebo in a crossover design.
The study is comprised of an outpatient screening and procedure orientation followed by approximately 5 days of an inpatient stay at a clinical research unit.
After screening/orientation (Visit 1), and baseline assessments (Visit 2), patients will be administered a single dose of 100 mg of AUT00201 or matching placebo the morning of Visit 3. PK assessments will be done at Visits 3, 4, 5, and 6 from predose and up to 27 hours postdose.
Visit 4 will be a washout day for patients.
At Visit 5 patients will be administered the crossover treatment.
At Visit 6 patients will be discharged from the unit.
Safety and tolerability assessments will be conducted throughout.
PD parameters will also be assessed.
Patients will be followed up by telephone 14 days after discharge.
Study Type
Interventional
Enrollment (Estimated)
10
Phase
- Phase 1
Contacts and Locations
This section provides the contact details for those conducting the study, and information on where this study is being conducted.
Study Locations
-
-
Pennsylvania
-
Philadelphia, Pennsylvania, United States, 19104
- Recruiting
- University of Pennsylvania, Penn Epilepsy Center
-
Contact:
- Melissa Johnston Esparza
- Phone Number: 215-614-0520
- Email: melissa.johnstonesparza@pennmedicine.upenn.edu
-
-
Participation Criteria
Researchers look for people who fit a certain description, called eligibility criteria. Some examples of these criteria are a person's general health condition or prior treatments.
Eligibility Criteria
Ages Eligible for Study
- Adult
- Older Adult
Accepts Healthy Volunteers
No
Description
Inclusion Criteria:
- Male or female patients aged 18 years or older at time of consenting.
- Diagnosed with MEAK, based on documented genetic evidence of the presence of the KCNC1 (c.959G>A; p.Arg320His) variant.
- If take anticonvulsants, must be on a stable anticonvulsant regiment for at least 30 days prior to Visit 1 and anticipated to remain stable throughout the study or if not on an anticonvulsant regimen, must be stable in regards to seizures for at least 30 days prior to Visit 1 and anticipated to remain stable throughout the study.
- Must be able to participate and willing to give written informed consent. If patient is unable to provide written informed consent, a legally authorized representative can sign on their behalf.
- Must be willing to perform study assessments and comply with the study protocol.
- If the patient is dependent on a caregiver and/or will need assistance either travelling to the site, whilst attending clinic visits and/or helping to document study assessment responses provided by the patient (eg, questionnaires administered on a tablet device), they must have an identified caregiver, considered reliable by the Investigator, to provide support to the patient for the duration of the study. The caregiver must be willing and able to provide support to the patient and, if required, stay for the duration of the study.
- Medically stable based on Investigator's judgement for at least 90 days prior to Visit 1.
- Women of childbearing potential must have a negative urine pregnancy test on Visit 2.
- If a vagal nerve stimulator is used, it must be implanted at least 150 days before Day. -1, and parameters must be stable for at least 30 days before Visit 1 and expected to remain stable throughout the study.
- If a ketogenic diet is followed, it must be stable for at least 30 days before Visit 1 and expected to remain stable throughout the study.
- Willing to comply with contraceptive requirements.
- Able to speak, read and understand English at a fluent level
Exclusion Criteria:
- Known pathogenic mutation in another gene that causes epilepsy or a different mutation in the KCNC1 gene than the c.959G>A variant.
- Clinically significant metabolic, hepatic, hematological, pulmonary, cardiovascular, gastrointestinal, or urological disorder.
- Clinically significant abnormal vital signs or laboratory test results.
- Hypersensitivity to AUT00201 or any of the excipients.
- Any medical condition or other factors, as judged by the Investigator, which may interfere with the patient's participation in this study and/or compromise the patient's ability to safely complete the study.
- Known to abuse drugs or those who test positive on urine screen for drugs of abuse will be excluded based on Investigator's judgement.
- Positive hepatitis B surface antigen or hepatitis C antibody.
- Clinically significant abnormality on the 12-lead electrocardiogram.
- Having received an investigational product 90 days prior to Visit 1.
- Currently using felbamate <1 year prior to Visit 1, or any evidence of ongoing hepatic or bone marrow dysfunction associated with current/prior felbamate treatment. Patients who are currently using felbamate for >1 year prior to Visit 1 and have no evidence of ongoing hepatic or bone marrow dysfunction associated with felbamate treatment are allowed.
- Currently using vigabatrin and having received vigabatrin for <2 years prior to Visit 1.
- Suicidal ideation with some intent to act within 6 months prior to Visit 1 based upon response in the Columbia-Suicide Severity Rating Scale (positive response to questions 4 or 5 of the suicidal ideation section) and as judged by the Investigator as having a significant impact on trial participation or patient safety. History of suicidal behavior within 1 year prior to Visit 1.
Study Plan
This section provides details of the study plan, including how the study is designed and what the study is measuring.
How is the study designed?
Design Details
- Primary Purpose: Basic Science
- Allocation: Randomized
- Interventional Model: Crossover Assignment
- Masking: Triple
Arms and Interventions
Participant Group / Arm |
Intervention / Treatment |
---|---|
Experimental: Experimental: AUT00201
Single dose (oral, capsule) of AUT00201
|
Single oral dose
|
Placebo Comparator: Experimental: Placebo
Single dose matching placebo oral capsules
|
Single oral dose
|
What is the study measuring?
Primary Outcome Measures
Outcome Measure |
Time Frame |
---|---|
Number of Participants With Treatment-Related Adverse Events after Single Dose Treatment of AUT00201 compared to Placebo
Time Frame: 19 Days
|
19 Days
|
Secondary Outcome Measures
Outcome Measure |
Measure Description |
Time Frame |
---|---|---|
Pharmacokinetics: Maximum Plasma Concentrations (Cmax) of AUT00201
Time Frame: 4 Days
|
4 Days
|
|
Pharmacokinetics: Area under the plasma concentration-time curve (AUC) of AUT00201
Time Frame: 4 Days
|
4 Days
|
|
Change from baseline in cortical inhibition
Time Frame: 5 Days
|
Short interval cortical inhibition as measured by paired-pulse TMS EMG
|
5 Days
|
Collaborators and Investigators
This is where you will find people and organizations involved with this study.
Sponsor
Investigators
- Principal Investigator: Michael Gelfand, MD, Penn Epilepsy Center, Department of Neurology
Study record dates
These dates track the progress of study record and summary results submissions to ClinicalTrials.gov. Study records and reported results are reviewed by the National Library of Medicine (NLM) to make sure they meet specific quality control standards before being posted on the public website.
Study Major Dates
Study Start (Actual)
May 12, 2023
Primary Completion (Estimated)
March 1, 2024
Study Completion (Estimated)
March 1, 2024
Study Registration Dates
First Submitted
April 18, 2023
First Submitted That Met QC Criteria
May 15, 2023
First Posted (Actual)
May 24, 2023
Study Record Updates
Last Update Posted (Estimated)
February 14, 2024
Last Update Submitted That Met QC Criteria
February 13, 2024
Last Verified
February 1, 2024
More Information
Terms related to this study
Additional Relevant MeSH Terms
Other Study ID Numbers
- AUT022201
Plan for Individual participant data (IPD)
Plan to Share Individual Participant Data (IPD)?
NO
Drug and device information, study documents
Studies a U.S. FDA-regulated drug product
Yes
Studies a U.S. FDA-regulated device product
No
This information was retrieved directly from the website clinicaltrials.gov without any changes. If you have any requests to change, remove or update your study details, please contact register@clinicaltrials.gov. As soon as a change is implemented on clinicaltrials.gov, this will be updated automatically on our website as well.
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