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ClinicalTrials.gov public records Last synced Sep 25, 2026, 1:29 AM EDT

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Showing 1–18 of 18 matching trials from the live ClinicalTrials.gov search.
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Filters: Sponsor: n-Lorem Foundation Clear all
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ASXL3 Gene Mutation

NCT07197268
View directory record Official record
Conditions
Bainbridge-Ropers Syndrome
Interventions
nL-ASXL3-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
4 Years to 4 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Chapel Hill, North Carolina
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Sep 25, 2026, 1:29 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for Participants With CHCHD10 ALS

NCT07095686
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
18 Years and older
Enrollment
9 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With UBTF Gene Mutation

NCT07588581
View directory record Official record
Conditions
Childhood-Onset Neurodegeneration With Brain Atrophy (CONDBA)
Interventions
nL-UBTF-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated May 14, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Single Participant Study of an Experimental ASO Treatment for TUBB4A-related Leukodystrophy

NCT06369974
View directory record Official record
Conditions
Genetic Disease
Interventions
Antisense oligonucleotide treatment (ASO)
Drug
Lead sponsor
Massachusetts General Hospital
Other
Eligibility
4 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Boston, Massachusetts
Source: ClinicalTrials.gov public record
Updated Mar 1, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Not yet recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Patient With CHCHD10 ALS (nL18576)

NCT07423494
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
18 Years and older
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Feb 19, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for a Single Participant With CHCHD10 ALS

NCT06977451
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
63 Years to 63 Years
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS

NCT07703462
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
nL-TARDB-002
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
1
States / cities
Philadelphia, Pennsylvania
Source: ClinicalTrials.gov public record
Updated Jul 13, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for Participants With TARDBP ALS

NCT07743268
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis (ALS)
Interventions
nL-TARDB-006
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Not listed
Enrollment
6 participants
Healthy volunteers
Healthy volunteers not accepted
Timeline
2026 – 2028
U.S. locations
2
States / cities
St Louis, Missouri • New York, New York
Source: ClinicalTrials.gov public record
Updated Aug 2, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With ATN1 Gene Mutation

NCT07084311
View directory record Official record
Conditions
Dentatorubral-Pallidoluysian Atrophy
Interventions
nL-ATN1-002
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
29 Years to 29 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
Honolulu, Hawaii
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Enrolling by invitation Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant With GARS1 Gene Mutation Associated With Charcot-Marie-Tooth Disease Type 2D (CMT2D)

NCT07226297
View directory record Official record
Conditions
Charcot-Marie-Tooth Disease Type 2D
Interventions
nL-GARS1-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
13 Years and older · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Houston, Texas
Source: ClinicalTrials.gov public record
Updated Nov 9, 2025 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With LMNB1 Mutation Associated Autosomal Dominant Leukodystrophy (ADLD)

NCT06816498
View directory record Official record
Conditions
Autosomal Dominant Leukodystrophy
Interventions
nL-LMNB1-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
51 Years to 51 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Rochester, Minnesota
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Completed Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With TARDBP ALS

NCT07095712
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-TARD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
49 Years to 49 Years · Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2025
U.S. locations
2
States / cities
New York, New York • Houston, Texas
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for Rare Pediatric Genetic Disease: SCN2A

NCT06314490
View directory record Official record
Conditions
Genetic Disease
Interventions
nL-SCN2A-002
Drug
Lead sponsor
University of California, San Diego
Other
Eligibility
Not listed
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2026
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Mar 31, 2025 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for A Single Participant (nL62541) With ATN1 Gene Mutation

NCT07221760
View directory record Official record
Conditions
Dentatorubral-Pallidoluysian Atrophy
Interventions
nL-ATN1-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
22 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
Austin, Texas
Source: ClinicalTrials.gov public record
Updated Apr 6, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With ATN1 Gene Mutation

NCT06706388
View directory record Official record
Conditions
Dentatorubral-Pallidoluysian Atrophy
Interventions
nL-ATN1-002
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
17 Years to 17 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Apr 8, 2026 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide for a Single Participant With MAPK8IP3 Neurodevelopmental Disorder With or Without Variable Brain Abnormalities (NEDBA)

NCT07197294
View directory record Official record
Conditions
Neurodevelopmental Disorder With or Without Variable Brain Abnormalities
Interventions
nL-MAPK8-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
5 Years to 5 Years · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
New York, New York
Source: ClinicalTrials.gov public record
Updated Sep 28, 2025 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for a Single Participant With PRPH2 Mutation Associated With Retinal Dystrophy

NCT07177196
View directory record Official record
Conditions
Retinal Dystrophy
Interventions
nL-PRPH2-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
Female only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2025 – 2027
U.S. locations
1
States / cities
San Diego, California
Source: ClinicalTrials.gov public record
Updated Sep 15, 2025 · Synced Sep 25, 2026, 1:29 AM EDT
Active, not recruiting Phase 1Phase 2 Interventional

Personalized Antisense Oligonucleotide Therapy for A Single Participant With CHCHD10 ALS

NCT06392126
View directory record Official record
Conditions
Amyotrophic Lateral Sclerosis
Interventions
nL-CHCHD-001
Drug
Lead sponsor
n-Lorem Foundation
Other
Eligibility
48 Years and older · Male only
Enrollment
1 participant
Healthy volunteers
Healthy volunteers not accepted
Timeline
2024 – 2027
U.S. locations
1
States / cities
Jacksonville, Florida
Source: ClinicalTrials.gov public record
Updated Jul 12, 2026 · Synced Sep 25, 2026, 1:29 AM EDT