AMT-260 Gene Therapy Study in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy
Part of paid clinical trials in Birmingham, Alabama.
- Sponsor
- UniQure Biopharma B.V.
- Study ID
- NCT06063850
- Phase
- PHASE1/PHASE2
- Status
- Recruiting
Conditions
- Mesial Temporal Lobe Epilepsy
Eligibility Criteria
- Sex
- ALL
- Age
- 18 Years - 75 Years
- Healthy Volunteers
- Not accepted
Interventions
- AAV9-hSyn1-miGRIK2 — GENETICAMT-260 is an AAV9 gene therapy product that locally delivers miRNA silencing technology to target the GRIK2 gene and suppress aberrantly expressed GluK2 containing kainate receptors. Intervention will be a one-time intracerebral administration of AMT-260.
Study Details
The main goals of this clinical study are to learn if AMT-260 is safe and tolerable and works to reduce the frequency of seizures in adults with unilateral mesial temporal lobe epilepsy (MTLE).
Key Dates
- Start date
- Jun 12, 2024
- Status verified
- May 2026
- Primary completion
- Nov 30, 2026
- Completion
- Dec 31, 2031
Study Design
- Enrollment
- 12 participants (estimated)
- Allocation
- NA
- Intervention model
- SEQUENTIAL
- Primary purpose
- TREATMENT
Arms
- Experimental: AMT-260Cohort 1: AMT-260 starting dose (1.0x 10E12 gc/mL). Cohort 2: AMT-260 adapted dose (6.0x 10E11 gc/mL or 3.0x 10E12 gc/mL).
Primary Outcome Measure
To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE. [ Time Frame: 1 year ]
Central Contacts
- uniQure1-866-520-1257
Locations (18)
| Facility | City | State | ZIP | Site coordinators |
|---|---|---|---|---|
| University of Alabama at Birmingham | Birmingham | Alabama | 35294-0021 | Jerzy P Szaflarski, MD, PhD (PRINCIPAL_INVESTIGATOR) |
| Mayo Clinic Arizona | Phoenix | Arizona | 85054 | Jonathon Parker, MD (PRINCIPAL_INVESTIGATOR) |
| Stanford University | Palo Alto | California | 94304 | Yi Li, MD, PhD (PRINCIPAL_INVESTIGATOR) |
| Mayo Clinic Florida | Jacksonville | Florida | 32224 | Brin Freund, MD (PRINCIPAL_INVESTIGATOR) |
| Kansas University Medical Center | Kansas City | Kansas | 66160 | P Landazuri, MD (PRINCIPAL_INVESTIGATOR) |
| Johns Hopkins School of Medicine | Baltimore | Maryland | 21287 | Joon Kang, MD (PRINCIPAL_INVESTIGATOR) |
| Midatlantic Epilepsy and Sleep Center | Bethesda | Maryland | 20817 | Pavel Klein, M.D. (PRINCIPAL_INVESTIGATOR) |
| Massachusetts General Hospital | Boston | Massachusetts | 02114 | Mark Richardson, MD, PhD (PRINCIPAL_INVESTIGATOR) |
| Corewell Health | Grand Rapids | Michigan | 49503 | David Burdette, MD (PRINCIPAL_INVESTIGATOR) |
| Dartmouth Hitchcock Medical Center | Lebanon | New Hampshire | 03756 | Barbara Jobst, MD (PRINCIPAL_INVESTIGATOR) |
| Northeast Regional Epilepsy Group | Hackensack | New Jersey | 07601 | Asfi Rafiuddin (PRINCIPAL_INVESTIGATOR) |
| Robert Wood Johnson Hospital | New Brunswick | New Jersey | 08901 | Robert E Gross, MD, PhD (PRINCIPAL_INVESTIGATOR) |
| Cleveland Clinic | Cleveland | Ohio | 44195 | Imad Najm, MD (PRINCIPAL_INVESTIGATOR) |
| Ohio State University | Columbus | Ohio | 43210 | James Elder, MD (PRINCIPAL_INVESTIGATOR) |
| Hospital of the University of Pennsylvania | Philadelphia | Pennsylvania | 19104 | Michael Gelfand, MD, PhD (PRINCIPAL_INVESTIGATOR) |
| Allegheny Health Network | Pittsburgh | Pennsylvania | 15212 | Alexander Whiting, MD (PRINCIPAL_INVESTIGATOR) |
| Baylor Scott & White Medical Center | Austin | Texas | 78735 | Victor H Gonzalez Montoya, MD, FAES (PRINCIPAL_INVESTIGATOR) |
| Medical College of Wisconsin | Milwaukee | Wisconsin | 53226 | Kunal Gupta, MD, PhD (PRINCIPAL_INVESTIGATOR) |
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