9 completed, 4 recruiting, 3 active not recruiting, 3 unknown. 8 Phase 2, 6 Phase 1, 5 Phase 3.
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 |
| Registry and Natural History Study for Progressive Myoclonus Epilepsy Type 1 (EPM1) Progressive Myoclonus Epilepsy Type 1, EPM1 | Recruiting | Boston Children's Hospital | 200 | 2024-10-10 | 2026-03-18 | |
| 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 |
| 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 |
| A Safety Study for Previously Treated Vatiquinone (PTC743) Participants With Inherited Mitochondrial Disease Vatiquinone - Inherited Mitochondrial Disease | Phase 3 | Completed | PTC Therapeutics | 101 | 2022-06-22 | 2025-12-22 |
| Dentatorubral-pallidoluysian Atrophy Natural History and Biomarkers Study Positive genetic test for pathological expansion in ATN1 - Dentatorubral-Pallidoluysian Atrophy | Unknown | University College, London | 225 | 2022-05-01 | 2024-05-10 | |
| 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 |
| Natural History and Functional Status Study of Patients With Lafora Disease Lafora Disease | Completed | Ionis Pharmaceuticals, Inc. | 33 | 2019-01-09 | 2022-09-28 | |
| Intravenous Immunoglobulin for Unverricht-Lundborg Disease. Intravenous immunoglobulin - Unverricht-Lundborg Disease | Phase 3 | Unknown | Azienda Socio Sanitaria Territoriale di Mantova | 1 | 2015-12-09 | 2017-11-27 |
| North American Mitochondrial Disease Consortium Patient Registry and Biorepository (NAMDC) Mitochondrial Disorders, Mitochondrial Genetic Disorders | Recruiting | Columbia University | 1000 | 2011-01-31 | 2026-02-04 | |
| Global Registry and Natural History Study for Mitochondrial Disorders Mitochondrial Diseases, Kearns-Sayre Syndrome | Recruiting | LMU Klinikum | 6000 | 2009-02-01 | 2026-08-07 | |
| 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 |
| Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease in Adolescents and Adults Brivaracetam 25 mg, Brivaracetam 50 mg, Placebo - Unverricht-Lundborg Disease | Phase 3 | Completed | UCB Pharma SA | 50 | 2006-11-01 | 2015-05-18 |
| Brivaracetam as add-on Treatment of Unverricht-Lundborg Disease (ULD) in Adolescents and Adults Placebo, BRV 2.5 mg, BRV 25 mg - Unverricht-Lundborg Disease | Phase 3 | Completed | UCB Pharma | 56 | 2006-11-01 | 2023-05-31 |
| Ketogenic Diet in Lafora Disease Lafora Disease | Completed | National Institute of Neurological Disorders and Stroke (NINDS) | 15 | 2000-12-01 | 2008-03-04 | |
| Clinical and Genetic Studies of Familial Presenile Dementia With Neuronal Inclusion Bodies Familial Dementia With Neuroserpin Inclusion Bodies, Nervous System Heredodegenerative Disorder | Completed | National Human Genome Research Institute (NHGRI) | 100 | 2000-08-10 | 2017-07-02 |
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MELAS Syndrome (6) · MERRF Syndrome (6) · Unverricht-Lundborg Syndrome (5) · Lafora Disease (4) · Leigh Disease (4) · Mitochondrial Diseases (4) · Myoclonus (4) · Mitochondrial Myopathies (3) · Acidosis, Lactic (2) · Barth Syndrome (2) · Deafness (2) · Dementia (2) · Diffuse Cerebral Sclerosis of Schilder (2) · Drug Resistant Epilepsy (2)
Central Nervous System Diseases (21) · Epilepsy, Generalized (21) · Epileptic Syndromes (21) · Nervous System Diseases (21) · Brain Diseases (20) · Epilepsies, Myoclonic (20) · Epilepsy (19) · Congenital, Hereditary, and Neonatal Diseases and Abnormalities (16) · Genetic Diseases, Inborn (16) · Neurodegenerative Diseases (11) · Heredodegenerative Disorders, Nervous System (10) · Neurologic Manifestations (10) · Pathological Conditions, Signs and Symptoms (10) · Signs and Symptoms (10)
alpha-tocotrienol quinone (2) · brivaracetam (2) · Immunoglobulins, Intravenous (1) · ropinirole (1)
n-Lorem Foundation (3) · PTC Therapeutics (2) · Autifony Therapeutics Limited (1) · Azienda Socio Sanitaria Territoriale di Mantova (1) · Berge Minassian (1) · Boston Children's Hospital (1) · Children's Hospital of Philadelphia (1) · Columbia University (1) · Ionis Pharmaceuticals, Inc. (1) · LMU Klinikum (1) · National Human Genome Research Institute (NHGRI) (1) · National Institute of Neurological Disorders and Stroke (NINDS) (1) · Omeicos Therapeutics GmbH (1) · Parasail, LLC (1) · UCB Pharma (1)
United States (15) · Italy (6) · France (4) · Canada (3) · Finland (3) · Spain (3) · United Kingdom (3) · Germany (2) · Japan (2) · Netherlands (2) · Poland (2) · Sweden (2) · Tunisia (2) · Austria (1) · Israel (1)
Vinay Penematsa, MD (2) · Berge Minassian, MD (1) · Elizabeth M McCormick, MS (1) · Michael Gelfand, MD (1) · Michelangelo Mancuso, Prof. Dr. (1) · Michio Hirano, MD (1) · Paola Giunti (1) · Robert Fischer, MD (1) · Thomas Klopstock, Prof. Dr. (1)
U54NS078059 (2), 2017 (1)