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Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Evaluate Safety, Tolerability, PK and PD of Antisense Oligonucleotide AMX0114 Administered to Adult Participants With Amyotrophic Lateral Sclerosis

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Overview

This study is a placebo-controlled Phase I study to evaluate the safety, tolerability, pharmacokinetics, and pharmacodynamics of the antisense oligonucleotide (ASO) AMX0114 in adult participants with amyotrophic lateral sclerosis (ALS).

Clinical Trial Information

Protocol

IRB#: 32265

Phase

1

Condition(s)

ALS

Investigator

Drs. Terry Heiman-Patterson and Anahita Deboo

Coordinator

Kathleen Hatala, RN, BSN, Christine Barr, RN
215-707-7845

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Contact

For more information about this trial or to inquire about eligibility, call 215-707-7845 or email [email protected].

Full Title

Phase 1, Randomized, Double-blind, Placebo-controlled, Multiple Ascending Dose Study to Evaluate Safety, Tolerability, PK and PD of Antisense Oligonucleotide AMX0114 Administered to Adult Participants With Amyotrophic Lateral Sclerosis