3 active not recruiting, 2 completed, 1 recruiting, 1 terminated. 8 Phase 2, 5 Phase 1, 1 Phase 3.
| Phase | Trials | Status |
|---|---|---|
| Phase 2 | 8 | Active not recruiting 3, Completed 2, Recruiting 1, Terminated 1, Unknown 1 |
| Phase 1 | 5 | Active not recruiting 3, Completed 1, Recruiting 1 |
| Phase 3 | 1 | Terminated 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 |
| OMT-28 in Patients With Primary Mitochondrial Disease (PMD) (PMD-OPTION) OMT-28 - Primary Mitochondrial Disease | Phase 2 | Completed | Omeicos Therapeutics GmbH | 28 | 2023-05-22 | 2026-01-22 |
| A Study to Evaluate Efficacy and Safety of Vatiquinone for Treating Mitochondrial Disease in Participants With Refractory Epilepsy Vatiquinone, Placebo - Mitochondrial Diseases, Drug Resistant Epilepsy | Phase 2 / Phase 3 | Terminated | PTC Therapeutics | 68 | 2020-09-28 | 2026-03-31 |
| Effect of Ropinirole Hydrochloride in Progressive Myoclonic Epilepsy of Unverricht-Lundborg Type Ropinirole - Unverricht-Lundborg Syndrome | Phase 2 | Unknown | University of Turku | 16 | 2007-08-01 | 2008-03-19 |
MELAS Syndrome (3) · MERRF Syndrome (3) · Mitochondrial Diseases (2) · Mitochondrial encephalopathy (2) · Acidosis, Lactic (1) · Cardiomyopathies (1) · Deafness (1) · Diffuse Cerebral Sclerosis of Schilder (1) · Drug Resistant Epilepsy (1) · Lafora Disease (1) · Leigh Disease (1) · Mitochondrial Encephalomyopathies (1) · Mitochondrial Myopathies (1) · Muscular Diseases (1)
Brain Diseases (8) · Central Nervous System Diseases (8) · Epilepsies, Myoclonic (8) · Epilepsy (8) · Epilepsy, Generalized (8) · Epileptic Syndromes (8) · Nervous System Diseases (8) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (5) · Genetic Diseases, Inborn (5) · Brain Diseases, Metabolic (3) · Brain Diseases, Metabolic, Inborn (3) · Cardiovascular Diseases (3) · Cerebral Small Vessel Diseases (3) · Cerebrovascular Disorders (3)
alpha-tocotrienol quinone (1) · ropinirole (1)
n-Lorem Foundation (3) · Berge Minassian (1) · Children's Hospital of Philadelphia (1) · Omeicos Therapeutics GmbH (1) · PTC Therapeutics (1) · University of Turku (1)
United States (6) · Italy (2) · Canada (1) · Finland (1) · France (1) · Germany (1) · Japan (1) · Netherlands (1) · Poland (1) · Spain (1) · Sweden (1) · United Kingdom (1)
Berge Minassian, MD (1) · Elizabeth M McCormick, MS (1) · Robert Fischer, MD (1) · Vinay Penematsa, MD (1)
U54NS078059 (1)