A Study to Evaluate Safety, Tolerability, and Distribution of a Microdose of Radiolabeled BIIB067 (99mTc-MAG3-BIIB067) Co-administered With BIIB067 (Tofersen) to Healthy Adults

Phase 1 Completed 8 enrolled Biogen

Non-randomizedSequentialOpen-labelBasic science

Summary

The primary objective of the study is to evaluate the distribution in the central nervous system (CNS) of a microdose 99mTc-MAG3-BIIB067 co-administered with unlabeled BIIB067 (Tofersen). The secondary objective of the study is to assess the safety and tolerability of unlabeled BIIB067 co-administered with a microdose of 99mTc-MAG3-BIIB067 to healthy participants.

Timeline

Start: 2018-12-20
Primary completion: 2021-06-10
Completion: 2021-07-10

Publications

Cook BE, McLaren DG, Sullivan JM, El Fakhri G, Yokell DL, Freeman MW, Currier N, Oestergaard ME, Dobson H, Hesterman J, Salem N, Nestorov I, Monine M, Martarello L, Evans KC, Fradette S, Ferguson TA, Graham D, Passamonti L. Central Nervous System Biodistribution and Pharmacokinetics of Radiolabeled Tofersen in Rodents, Nonhuman Primates, and Humans. J Nucl Med. 2026 Jan 2;67(1):139-144. doi: 10.2967/jnumed.125.270731.

Drugs

Evaluation	Drug	Modality	Dose	Route
Subject	99mTc-MAG3-BIIB067	Unknown	129.5 mbq	Intrathecal
Subject	Tofersen	Antisense oligonucleotide	—	Intrathecal

Indications

No indication recorded.