11 active not recruiting, 5 enrolling by invitation, 2 not yet recruiting, 1 completed. 19 Phase 1, 19 Phase 2.
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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 Therapy for a Participant With TARDBP ALS nL-TARDB-002 - Amyotrophic Lateral Sclerosis (ALS) | Phase 1 / Phase 2 | Enrolling by invitation | n-Lorem Foundation | 1 | 2026-12-01 | 2026-07-14 |
| Personalized Antisense Oligonucleotide for A Single Participant With PACS1 Gene Mutation Associated With Schuurs-Hoeijmakers Syndrome (SHMS) nL-PACS1-001 - Schuurs-Hoeijmakers Syndrome | Phase 1 / Phase 2 | Not yet recruiting | n-Lorem Foundation | 1 | 2026-04-01 | 2026-03-16 |
| Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576) nL-CHCHD-001 - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Not yet recruiting | n-Lorem Foundation | 1 | 2026-03-01 | 2026-02-20 |
| Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS nL-TARDB-006 - Amyotrophic Lateral Sclerosis (ALS) | Phase 1 / Phase 2 | Enrolling by invitation | n-Lorem Foundation | 6 | 2026-02-19 | 2026-08-03 |
| 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 |
| Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D) nL-GARS1-001 - Charcot-Marie-Tooth Disease Type 2D | Phase 1 / Phase 2 | Enrolling by invitation | n-Lorem Foundation | 1 | 2025-10-27 | 2025-11-10 |
| Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Retinal Dystrophy nL-PRPH2-001 - Retinal Dystrophy | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2025-08-28 | 2025-09-16 |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation nL-ASXL3-001 - Bainbridge-Ropers Syndrome | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2025-05-19 | 2025-09-29 |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD) nL-LMNB1-001 - Autosomal Dominant Leukodystrophy | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2025-03-17 | 2026-07-13 |
| Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA) nL-MAPK8-001 - Neurodevelopmental Disorder With or Without Variable Brain Abnormalities | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2025-02-24 | 2025-09-29 |
| Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation nL-UBTF-001 - Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA) | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2025-02-05 | 2026-05-15 |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS nL-TARD-001 - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Completed | n-Lorem Foundation | 1 | 2024-11-25 | 2026-04-09 |
| 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 for Participants With CHCHD10 ALS nL-CHCHD-001 - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Enrolling by invitation | n-Lorem Foundation | 9 | 2024-10-02 | 2026-07-13 |
| Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy Antisense oligonucleotide treatment (ASO) - Genetic Disease | Phase 1 / Phase 2 | Enrolling by invitation | Massachusetts General Hospital | 1 | 2024-09-18 | 2026-03-02 |
| Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS nL-CHCHD-001 - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2024-06-24 | 2026-07-13 |
| Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS nL-CHCHD-001 - Amyotrophic Lateral Sclerosis | Phase 1 / Phase 2 | Active not recruiting | n-Lorem Foundation | 1 | 2024-04-16 | 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 |
| Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A nL-SCN2A-002 - Genetic Disease | Phase 1 / Phase 2 | Active not recruiting | University of California, San Diego | 1 | 2024-02-16 | 2025-04-01 |
Amyotrophic Lateral Sclerosis (7) · Myoclonic Epilepsies, Progressive (3) · Genetic Diseases, Inborn (2) · Charcot-Marie-Tooth disease, Type 2D (1) · Retinal Dystrophies (1)
United States (18) · Canada (1)
Florian Eichler, MD (1) · Olivia Kim-McManus, M.D. (1)