3 active not recruiting, 2 completed, 1 recruiting. 6 Phase 1, 5 Phase 2.
| Phase | Trials | Status |
|---|---|---|
| Phase 1 | 6 | Active not recruiting 3, Completed 2, Recruiting 1 |
| Phase 2 | 5 | Active not recruiting 3, Completed 1, Recruiting 1 |
Follow Myoclonic Epilepsies, Progressive: new trials by email Literature Review of Myoclonic Epilepsies, Progressive trials Research Report on Myoclonic Epilepsies, Progressive
Tick up to 5 trials to compare them side by side (eligibility, arms, endpoints, adverse events) in a Literature Matrix.
| Trial | Phase | Status | Sponsor | Enrollment | Start | Updated |
|---|---|---|---|---|---|---|
| Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation nL-ATN1-001 - Dentatorubral-Pallidoluysian Atrophy | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2025-11-06 | 2026-04-07 |
| A Safety and Efficacy of Intrathecally Administered ION283 in Patients With Lafora Disease ION283 - Lafora Disease | Phase 1 / Phase 2 | Recruiting | Berge Minassian | 10 | 2024-12-03 | 2025-12-18 |
| Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation nL-ATN1-002 - Dentatorubral-Pallidoluysian Atrophy | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2024-10-24 | 2026-07-13 |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation nL-ATN1-002 - Dentatorubral-Pallidoluysian Atrophy | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2024-02-21 | 2026-04-09 |
| Promoting Resilience in Stress Management (PRISM) and Clinical-focused Narrative (CFN) Pilot in Adults With Primary Mitochondrial Disease (PMD). Promoting Resilience in Stress Management (PRISM), Clinical-focused narrative (CFN) - Mitochondrial Diseases, Mitochondrial Myopathies | Phase 1 / Phase 2 | Completed | Children's Hospital of Philadelphia | 16 | 2023-09-22 | 2024-11-29 |
| Safety, Blood Levels and Effects of AUT00201 in Patients With MEAK AUT00201, Placebo - Myoclonus Epilepsies, Progressive | Phase 1 | Completed | Autifony Therapeutics Limited | 6 | 2023-05-12 | 2025-03-25 |
Lafora Disease (1) · MELAS Syndrome (1) · MERRF Syndrome (1) · Mitochondrial Diseases (1) · Mitochondrial Encephalomyopathies (1) · Mitochondrial Myopathies (1)
Brain Diseases (6) · Central Nervous System Diseases (6) · Epilepsies, Myoclonic (6) · Epilepsy (6) · Epilepsy, Generalized (6) · Epileptic Syndromes (6) · Nervous System Diseases (6) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (2) · Genetic Diseases, Inborn (2) · Brain Diseases, Metabolic (1) · Brain Diseases, Metabolic, Inborn (1) · Cardiovascular Diseases (1) · Cerebral Small Vessel Diseases (1) · Cerebrovascular Disorders (1)
n-Lorem Foundation (3) · Autifony Therapeutics Limited (1) · Berge Minassian (1) · Children's Hospital of Philadelphia (1)
United States (6)
Berge Minassian, MD (1) · Elizabeth M McCormick, MS (1) · Michael Gelfand, MD (1)
U54NS078059 (1)