Personalized Antisense Oligonucleotide Therapy for a Participant With TARDBP ALS

Part of paid clinical trials in Philadelphia, Pennsylvania.

Sponsor
n-Lorem Foundation
Study ID
NCT07703462
Phase
PHASE1/PHASE2
Status
Enrolling By Invitation

Conditions

  • Amyotrophic Lateral Sclerosis (ALS)

Eligibility Criteria

Sex
ALL
Age
N/A - N/A
Healthy Volunteers
Not accepted

Interventions

  • nL-TARDB-002 — DRUG
    Personalized antisense oligonucleotide

Study Details

This research project entails delivery of a personalized antisense oligonucleotide (ASO) drug designed for a single participant with amyotrophic lateral sclerosis (ALS) due to a pathogenic variant in TARDBP.

Key Dates

First listed
Jul 14, 2026
Start date
Dec 31, 2026
Status verified
Jul 2026
Primary completion
Dec 31, 2028
Completion
Dec 31, 2028

Study Design

Enrollment
1 participants (estimated)
Allocation
NA
Intervention model
SINGLE_GROUP
Primary purpose
TREATMENT

Arms

  • Experimental: Open Label

Primary Outcome Measure

Clinical Functioning [ Time Frame: Baseline to 12 months ]

Locations (1)

FacilityCityStateZIPSite coordinators
Jefferson HealthPhiladelphiaPennsylvania19107-

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By research site
Jefferson Health· Philadelphia, PA

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