n-Lorem Foundation
n-Lorem Foundation
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Clinical trials
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| Trial | Conditions | Status | Phase | Updated | More info | NCT ID |
|---|---|---|---|---|---|---|
| Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS | Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorder | Enrolling by invitation | Phase 1 / Phase 2 | 2026-08-03 | NCT07743268 | |
| Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS | Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorder | Enrolling by invitation | Phase 1 / Phase 2 | 2026-07-14 | NCT07703462 | |
| Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS | Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorder | Enrolling by invitation | Phase 1 / Phase 2 | 2026-07-13 | NCT07095686 | |
| Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation | Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, dentatorubral-pallidoluysian atrophy, epilepsy, nervous system disorder, progressive myoclonus epilepsy | Active, not recruiting | Phase 1 / Phase 2 | 2026-07-13 | NCT07084311 | |
| Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS | Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorder | Active, not recruiting | Phase 1 / Phase 2 | 2026-07-13 | NCT06977451 | |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD) | Autosomal Dominant Leukodystrophy | Active, not recruiting | Phase 1 / Phase 2 | 2026-07-13 | NCT06816498 | |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS | Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorder | Active, not recruiting | Phase 1 / Phase 2 | 2026-07-13 | NCT06392126 | |
| Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation | Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA) | Active, not recruiting | Phase 1 / Phase 2 | 2026-05-15 | NCT07588581 | |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation | Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, dentatorubral-pallidoluysian atrophy, epilepsy, nervous system disorder, progressive myoclonus epilepsy | Active, not recruiting | Phase 1 / Phase 2 | 2026-04-09 | NCT06706388 | |
| Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation | Epilepsies, Myoclonic, Epilepsy, Generalized, Epileptic Syndromes, brain disorder, central nervous system disorder, dentatorubral-pallidoluysian atrophy, epilepsy, nervous system disorder, progressive myoclonus epilepsy | Active, not recruiting | Phase 1 / Phase 2 | 2026-04-07 | NCT07221760 | |
| Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS) | Schuurs-Hoeijmakers syndrome | Not yet recruiting | Phase 1 / Phase 2 | 2026-03-16 | NCT07474298 | |
| Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, hereditary disease | Enrolling by invitation | Phase 1 / Phase 2 | 2026-03-02 | NCT06369974 | |
| Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576) | Nutritional and Metabolic Diseases, TDP-43 proteinopathy, amyotrophic lateral sclerosis, central nervous system disorder, metabolic disease, motor neuron disorder, nervous system disorder, neurodegenerative disease, neuromuscular disease, proteostasis deficiencies, spinal cord disorder | Not yet recruiting | Phase 1 / Phase 2 | 2026-02-20 | NCT07423494 | |
| Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D) | Charcot-Marie-Tooth disease type 2D | Enrolling by invitation | Phase 1 / Phase 2 | 2025-11-10 | NCT07226297 | |
| Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA) | Neurodevelopmental Disorder With or Without Variable Brain Abnormalities | Active, not recruiting | Phase 1 / Phase 2 | 2025-09-29 | NCT07197294 | |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation | Bainbridge-Ropers syndrome | Active, not recruiting | Phase 1 / Phase 2 | 2025-09-29 | NCT07197268 | |
| Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Retinal Dystrophy | eye disorder, inherited retinal dystrophy, retinal degeneration, retinal disorder | Active, not recruiting | Phase 1 / Phase 2 | 2025-09-16 | NCT07177196 | |
| Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A | Congenital, Hereditary, and Neonatal Diseases and Abnormalities, hereditary disease | Active, not recruiting | Phase 1 / Phase 2 | 2025-04-01 | NCT06314490 |