NCT06063850: GenTLE

A Multi-center, Phase 1/2a, First-in-human (FIH) Study Investigating the Safety, Tolerability, and Efficacy of AMT-260 in Adults With Unilateral Refractory Mesial Temporal Lobe Epilepsy (MTLE) Administered Via Magnetic Resonance Imaging (MRI)-Guided Convection-enhanced Delivery (CED)

Recruiting now Phase 1/Phase 2 Last updated 28 May 2026

What this trial tests

Phase 1/Phase 2 trial testing AAV9-hSyn1-miGRIK2 in Mesial Temporal Lobe Epilepsy in 12 participants. Currently enrolling.

Timeline

12 June 2024

Primary endpoint
30 November 2026

1 December 2031

Quick facts

Lead sponsor	UniQure Biopharma B.V.
Phase	Phase 1/Phase 2
Status	Recruiting now
Study type	INTERVENTIONAL
Allocation	na
Design	sequential
Masking	none
Primary purpose	treatment
Enrollment	12
Start date	12 June 2024
Primary completion	30 November 2026
Estimated completion	1 December 2031
Sites	18 locations across United States

Drugs / interventions tested

• AAV9-hSyn1-miGRIK2

Conditions studied

• Mesial Temporal Lobe Epilepsy — all drugs for Mesial Temporal Lobe Epilepsy →

Sponsor

UniQure Biopharma B.V. — full company profile →

Who can join

Adults 18 to 75, any sex, with Mesial Temporal Lobe Epilepsy. Patients with the condition only — healthy volunteers not accepted.

What's being measured

Primary outcomes are the specific endpoints the trial is designed to prove or disprove.

• To evaluate the safety and tolerability of AMT-260 in adults with unilateral refractory MTLE.
  Time frame: 1 year
  Occurrence of Adverse Events during the period of 1 year after AMT-260 administration, including seriousness, severity, and causal relationship to AMT-260.

Sponsor's own description

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).

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

1. Recombinant Adeno-Associated Virus Vectors for Gene Therapy of the Central Nervous System: Delivery Routes and Clinical Aspects.
   Słyk Ż, Stachowiak N, Małecki M. · · 2024 · cited 14× · PMID 39062095 · DOI 10.3390/biomedicines12071523
2. Synthetic Promoters in Gene Therapy: Design Approaches, Features and Applications.
   Artemyev V, Gubaeva A, Paremskaia AI, Dzhioeva AA, et al · · 2024 · cited 13× · PMID 39682712 · DOI 10.3390/cells13231963
3. Neurosurgical gene therapy for central nervous system diseases.
   Patel RV, Nanda P, Richardson RM. · · 2024 · cited 10× · PMID 39191071 · DOI 10.1016/j.neurot.2024.e00434
4. Sudden Unexpected Death in Epilepsy: Central Respiratory Chemoreception.
   Dereli AS, Apaire A, El Tahry R. · · 2025 · cited 4× · PMID 40004062 · DOI 10.3390/ijms26041598
5. The potential of gene delivery for the treatment of traumatic brain injury.
   Dooley J, Hughes JG, Needham EJ, Palios KA, et al · · 2024 · cited 3× · PMID 39069631 · DOI 10.1186/s12974-024-03156-x
6. Tumor Suppressor MicroRNAs in Clinical and Preclinical Trials for Neurological Disorders.
   Lui A, Do T, Alzayat O, Yu N, et al · · 2024 · cited 3× · PMID 38675388 · DOI 10.3390/ph17040426
7. Gene therapy for epilepsy: An emerging, promising approach for a serious neurological disorder.
   Ledri M, Kokaia M. · · 2026 · cited 1× · PMID 41366856 · DOI 10.1111/joim.70059
8. miRNAs as neuro-oncologic therapeutics: A narrative review
   Das N, Bhat S, Fugo P, Dhawan A. · · 2024