NCT03764488 is a Phase 1 clinical trial of Tofersen in Healthy Volunteers, sponsored by Biogen.

Who is sponsoring NCT03764488?

NCT03764488 is sponsored by Biogen.

What drugs are being tested in NCT03764488?

Interventions in NCT03764488: Tofersen, 99mTc-MAG3-BIIB067.

What condition does NCT03764488 study?

NCT03764488 studies Healthy Volunteers.

Is NCT03764488 still recruiting?

NCT03764488 is currently completed. Last updated 22 June 2023.

Last reviewed 2023-06-22 · How we verify

NCT03764488

Name: Drug Landscape Pharmaceutical Database
Creator: Drug Landscape
Published: 2026-01-01
License: https://creativecommons.org/licenses/by/4.0/

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

Completed Phase 1 Last updated 22 June 2023

What this trial tests

Phase 1 trial testing Tofersen in Healthy Volunteers in 8 participants. Completed in 10 July 2021.

Timeline

20 December 2018

Primary endpoint
10 June 2021

10 July 2021

Quick facts

Lead sponsor	Biogen
Phase	Phase 1
Status	Completed
Study type	INTERVENTIONAL
Allocation	non randomized
Design	sequential
Masking	none
Primary purpose	basic science
Enrollment	8
Start date	20 December 2018
Primary completion	10 June 2021
Estimated completion	10 July 2021
Sites	2 locations across United States

Drugs / interventions tested

Tofersen (TOFERSEN) — full drug profile →
99mTc-MAG3-BIIB067 — full drug profile →

Conditions studied

Healthy Volunteers — all drugs for Healthy Volunteers →

Sponsor

Biogen — full company profile →

Who can join

Adults 18 to 65, any sex, with Healthy Volunteers. Patients with the condition only — healthy volunteers not accepted.

Sponsor's own description

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.

Publications & conference data

8 peer-reviewed publications reference this trial (live from Europe PMC):

Gene-based therapies for neurodegenerative diseases.
Sun J, Roy S. · · 2021 · cited 126× · PMID 33526943 · DOI 10.1038/s41593-020-00778-1
Landscape of small nucleic acid therapeutics: moving from the bench to the clinic as next-generation medicines.
Liu M, Wang Y, Zhang Y, Hu D, et al · · 2025 · cited 62× · PMID 40059188 · DOI 10.1038/s41392-024-02112-8
Breaking barriers with tofersen: Enhancing therapeutic opportunities in amyotrophic lateral sclerosis.
Saini A, Chawla PA. · · 2024 · cited 36× · PMID 37975798 · DOI 10.1111/ene.16140
Translational Approaches for Brain Delivery of Biologics via Cerebrospinal Fluid.
Sadekar SS, Bowen M, Cai H, Jamalian S, et al · · 2022 · cited 27× · PMID 35064573 · DOI 10.1002/cpt.2531
ASO drug Qalsody (tofersen) targets amyotrophic lateral sclerosis.
Jin J, Zhong XB. · · 2023 · cited 24× · PMID 37709589 · DOI 10.1016/j.tips.2023.08.008
Central Nervous System Biodistribution and Pharmacokinetics of Radiolabeled Tofersen in Rodents, Nonhuman Primates, and Humans.
Cook BE, McLaren DG, Sullivan JM, El Fakhri G, et al · · 2026 · cited 1× · PMID 41233143 · DOI 10.2967/jnumed.125.270731
Significance of gene therapy in neurodegenerative diseases.
Wang L, Ma L, Gao Z, Wang Y, et al · · 2025 · cited 1× · PMID 40406043 · DOI 10.3389/fnins.2025.1515255
Gene Targeted Therapies for Neurodegenerative Disorders: Strategies and Implications in ALS and SMA.
Yesbek Kaymaz A, Bora-Akoğlu G, Erdem Yurter H, Grunseich C. · · 2026 · PMID 42074537 · DOI 10.3390/genes17040419

Verify or expand the search:

Other trials of Tofersen

Trials testing the same drug.

NCT07259980 — A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Participants With Superoxide Dismutase 1 (SOD- · recruiting
NCT07223723 — A Study to Learn More About the Long-Term Safety of Tofersen (Qalsody) in Chinese Participants With SOD-1 Amyotrophic La · Phase 4 · recruiting
NCT04856982 — A Study of BIIB067 (Tofersen) Initiated in Clinically Presymptomatic Adults With a Confirmed Superoxide Dismutase 1 Muta · Phase 3 · active not recruiting
NCT03070119 — Long-Term Evaluation of BIIB067 (Tofersen) · Phase 3 · completed
NCT02623699 — An Efficacy, Safety, Tolerability, Pharmacokinetics and Pharmacodynamics Study of BIIB067 (Tofersen) in Adults With Inhe · Phase 3 · completed

Other recruiting trials for Healthy Volunteers

Currently open trials in the same condition.

NCT07504003 — Influence of Training Session Duration on Improvements in Physiological Resilience to Exercise · NA · recruiting
NCT07446400 — A Trial to Examine the Interaction of Repinatrabit With Ethinyl Estradiol/Norethindrone, Metformin,Carbamazepine, Rosuva · Phase 1 · recruiting
NCT07495813 — A Study to See How RO7763505 Works and How Safe it is When Given to Healthy People and People With Stable Heart Disease · Phase 1 · recruiting
NCT07499050 — A Study of Multiple Doses of Orally Administered RO7795081 in Otherwise Healthy Chinese Adult Participants With Obesity · Phase 1 · recruiting
NCT07342114 — A Dose-Escalation Study of RO7875913 in Healthy Participants · Phase 1 · recruiting

Other Biogen trials

Trials by the same sponsor.

NCT07483632 — A Study to Learn About the Safety of Diroximel Fumarate (DRF) and Dimethyl Fumarate (DMF) and Their Effects on Relapses · Phase 3 · not yet recruiting
NCT06628687 — A Study to Learn How BIIB141 (Omaveloxolone) Affects the Health of Participants With Friedrich's Ataxia Who Took it Duri · recruiting
NCT07444450 — A Study to Learn About the Safety and Effects of Salanersen (BIIB115) When Given to Babies With Spinal Muscular Atrophy · Phase 3 · not yet recruiting
NCT07444489 — A Study to Learn More About the Long-Term Safety and Effects of Felzartamab Infusions in Adults With Kidney Transplants · Phase 3 · not yet recruiting
NCT07444476 — A Study to Learn About Salanersen's (BIIB115) Effects on Movement and Its Safety in Participants Aged 15 to 60 Years Wit · Phase 3 · not yet recruiting

Verify against primary sources

ClinicalTrials.gov — authoritative US registry record
WHO ICTRP — international registry index
EU Clinical Trials Register
Sponsor press releases (Google)

Data sources for this page

Trial protocol + status: ClinicalTrials.gov NCT03764488 (US National Library of Medicine, public domain)
Publications: Europe PMC API search by NCT ID, retrieved 10 June 2026
Drug + disease cross-links: matched in real time against Drug Landscape's normalised drug + company + condition tables
Sponsor: as reported to ClinicalTrials.gov by Biogen
Last refreshed: 22 June 2023

Drug Landscape aggregates and links these public records for informational use only. Always verify against the primary source before clinical or regulatory decisions. Canonical URL: https://druglandscape.com/trial/NCT03764488.

Primary sources · FDA · ClinicalTrials.gov · EMA · SEC EDGAR · ChEMBL · Wikidata · full sourcing